Cardiol Therapeutics Inc. (TSX: CRDL) (OTCQX: CRTPF), a leader in the production of pharmaceutical cannabidiol (CBD) products and in the development of innovative cannabidiol medicines for heart disease, is pleased to announce the formation of the Clinical Steering Committee (CSC) for a Phase 2 international trial in acute myocarditis using the Companys CardiolRx100 cannabidiol formulation.

The CSC, which comprises key opinion leaders in acute myocarditis from North America and Europe, recently met during the American Heart Associations Scientific Sessions in Philadelphia held November 16th to 18th. The role of the CSC is to advise on the trial design, provide overall supervision of the trial, and ensure that it is being conducted in accordance with the principles of Good Clinical Practice. The CSC has oversight of the protocol, any protocol amendments, and provides advice to the investigators on all aspects of the trial.

Acute myocarditis is characterized by inflammation of the heart muscle (myocardium). The most common cause is viral infection of the heart tissue which is initially responsible for the inflammation. In a significant number of cases, perhaps due to an autoimmune process, the inflammation persists with ongoing myocardial damage and depressed heart function. Although the symptoms are often mild, myocarditis remains an important cause of acute and fulminant heart failure and is the most common cause of sudden cardiac death in people less than 35 years old. In addition, some patients proceed to develop chronic dilated cardiomyopathy which continues to be the leading indication for cardiac transplantation. Symptoms include chest pain, fatigue, shortness of breath, and arrhythmias. Because of the progressive damage to heart cells, heart failure develops (defined as the inability of the heart to pump sufficient blood to meet the needs of the body). The study will use left ventricular ejection fraction (LVEF) as one measure of heart function.

CardiolRx100 is Cardiol Therapeutics pure pharmaceutically (cGMP) produced high concentration cannabidiol formulation that is THC free (<10ppm). Based on the large body of experimental evidence of the anti-inflammatory and cardioprotective properties of cannabidiol in models of cardiovascular disease, Cardiol believes there is an opportunity to develop a potential breakthrough therapy for acute myocarditis that would be eligible for designation as an orphan drug. In the United States, an orphan drug designation is granted for pharmaceuticals being developed to treat medical conditions affecting fewer than 200,000 people. These conditions are referred to as orphan diseases. In the U.S. and the European Union, orphan drugs are eligible for accelerated marketing approvals and companies developing orphan drugs typically receive other incentives, including a prolonged period of market exclusivity that can extend over seven years, during which the drug developer has sole rights to market the drug.

Cardiol has assembled eight highly distinguished thought leaders in cardiology from North America and Europe to oversee and guide our acute myocarditis trial that is being planned at world leading heart institutes, including the Cleveland Clinic, the Mayo Clinic, the Houston Methodist DeBakey Heart and Vascular Center, the University of Ottawa Heart Institute, and Charit University Medicine Berlin, stated David Elsley, President and CEO of Cardiol Therapeutics. The U.S. orphan drug program was successfully utilized to accelerate the first FDA approval of cannabidiol for the treatment of two pediatric epilepsy orphan diseases. We see a similar opportunity with our international trial in acute myocarditis to fast track the development of our CardiolRx formulation for a serious cardiovascular orphan disease for which there is currently no accepted standard of care.

Members of Cardiols Acute Myocarditis CSC include:

Dennis M. McNamara, MD (Chair)

Dr. Dennis McNamara is a Professor of Medicine at the University of Pittsburgh. He is also the Director of the Heart Failure/Transplantation Program at the University of Pittsburgh Medical Center. Dr. McNamara received his undergraduate/graduate education at Yale University, New Haven, Connecticut, and Harvard Medical School, Boston, Massachusetts, respectively. He completed his internship, residency, and cardiology fellowship at Massachusetts General Hospital in Boston. McNamaras current research interests include etiology and pathogenesis of dilated cardiomyopathies; inflammatory syndromes of cardiovascular disease; myocardial recovery in recent onset non-ischemic primary cardiomyopathy; etiology and management of peripartum cardiomyopathy; and genetic modulation of outcomes in cardiovascular disease.

Leslie T. Cooper, Jr., MD (Co-Chair)

Dr. Leslie T. Cooper, Jr., is a general cardiologist and the chair of the Mayo Clinic Enterprise Department of Cardiovascular Medicine, as well as chair of the Department of Cardiovascular Medicine at the Mayo Clinic in Florida. Dr. Coopers clinical interests and research focus on clinical and translational studies of rare and undiagnosed cardiomyopathies, myocarditis, and inflammatory cardiac and vascular diseases, such as giant cell myocarditis, cardiac sarcoidosis, eosinophilic myocarditis, and Takayasus arteritis. He has published over 130 original peer-reviewed papers, as well as contributing to and editing books on myocarditis. In addition to his clinical and research work, Dr. Cooper is a fellow of the American College of Cardiology, the American Heart Association, the European Society of Cardiology Heart Failure Association, the International Society for Heart and Lung Transplantation, and the Society for Vascular Medicine and Biology. He is also the founder and former president of the Myocarditis Foundation and continues to serve on its Board of Directors.

Arvind Bhimaraj, MD

Dr. Arvind Bhimaraj is a specialist in Heart Failure and Transplantation Cardiology and is Assistant Professor of Cardiology, Institute for Academic Medicine, at Houston Methodist and at Weill Cornell Medical College, NYC. He has been Co-Director of the Heart Failure Research Laboratory at Houston Methodist since 2016. His area of focus is anti-fibrotic mechanisms and how to promote recovery of a damaged heart. Dr. Bhimaraj was a Heart Failure Fellow at the Cleveland Clinic from July 2010 to September 2011. Dr. Bhimaraj also specializes in Interventional Cardiology, is board certified in Cardiovascular Disease, and the author of numerous cardiovascular publications.

Matthias Friedrich, MD

Dr. Matthias Friedrich is Full Professor with the Departments of Medicine and Diagnostic Radiology at the McGill University in Montreal and Chief, Cardiovascular Imaging at the McGill University Health Centre. He is also Professor of Medicine at Heidelberg University in Germany. Dr. Friedrich earned his MD at the Friedrich-Alexander-University Erlangen-Nrnberg, Germany. He completed his training as an internist and cardiologist at the Charit University Medicine Center, Humboldt University in Berlin. Dr. Friedrich founded one of the first large Cardiovascular Magnetic Resonance centres in Germany at the Charit University Hospital in Berlin. After his move to Canada, from 2004 to 2011, he was Director of the Stephenson Cardiovascular MR Centre at the Libin Cardiovascular Institute of Alberta and Professor of Medicine within the Departments of Cardiac Sciences and Radiology at the University of Calgary, Canada. From 2011 to 2015, he directed the Philippa and Marvin Carsley Cardiovascular MR Centre at the Montreal Heart Institute and was Michel and Renata Hornstein Chair in Cardiac Imaging at the Universit de Montral.

Peter Liu, MD

Dr. Peter Liu is the Chief Scientific Officer and Vice President, Research, of the University of Ottawa Heart Institute, and Professor of Medicine and Physiology at the University of Toronto and University of Ottawa. He was the former Scientific Director of the Institute of Circulatory and Respiratory Health at the Canadian Institutes of Health Research, the major federal funding agency for health research in Canada. Prior to that role, he was the inaugural Director of the Heart & Stroke/Lewar Centre of Excellence in Cardiovascular Research at University of Toronto. Dr. Liu received his MD from the University of Toronto, and postgraduate training at Harvard University. His laboratory investigates the causes and treatments of heart failure, the role of inflammation, and the identification of novel biomarkers and interventions in cardiovascular disease. Dr. Liu has published over 300 peer-reviewed articles in high impact journals and received numerous awards in recognition of his research and scientific accomplishments.

Wai Hong Wilson Tang, MD

Dr. Wai Hong Wilson Tang is the Advanced Heart Failure and Transplant Cardiology specialist at the Cleveland Clinic in Cleveland, Ohio. Dr. Tang is also the Director of the Cleveland Clinics Center for Clinical Genomics; Research Director, and staff cardiologist in the Section of Heart Failure and Cardiac Transplantation Medicine in the Sydell and Arnold Miller Family Heart & Vascular Institute at the Cleveland Clinic. He attended and graduated from Harvard Medical School in 1996, having over 23 years of diverse experience, especially in Advanced Heart Failure and Transplant Cardiology. Dr. Tang is affiliated with many hospitals including the Cleveland Clinic and cooperates with other doctors and physicians in medical groups including The Cleveland Clinic Foundation.

Barry Trachtenberg, MD

Dr. Barry H. Trachtenberg is a cardiologist specializing in heart failure and cardiac transplantation. He is also the director of the Michael DeBakey Cardiology Associates Cardio-Oncology program, an evolving field devoted to prevention and management of cardiovascular complications of cancer therapies such as chemotherapy and radiation. His clinical experience includes heart failure and heart transplantation, mechanical support pumps, and cardio-oncology. He has contributed to multiple publications related to advanced heart failure, cardiac transplantation, regenerative therapies, and ventricular assist devices. Dr. Trachtenberg is a member of the American Heart Association, the International Society for Heart and Lung Transplantation, the Heart Failure Society of America, and the International CardiOncology Society of North America.

Carsten Tschpe, MD

Dr. Carsten Tschpe is Professor of Medicine and Cardiology and Vice Director of the Department of Internal Medicine and Cardiology, Charit University Medicine Berlin. He received his doctorate in medicine in 1993 and has over 140 peer-reviewed publications, including overview and book articles, and 120 international original articles. His research interests include inflammatory cardiomyopathy, diabetic cardiopathy, and ischemic cardiopathy. He also includes diastolic dysfunction, endothelial dysfunction, peptide systems, and experimental and clinical studies in cardiology and stem cells in his research studies. For his outstanding research work, Dr. Tschpe was awarded the prestigious Arthur Weber Prize by the German Cardiac Society Cardiovascular Research.

About Cardiol Therapeutics

Cardiol Therapeutics Inc. (TSX: CRDL)(OTCQX: CRTPF) is focused on producing pharmaceutical cannabidiol (CBD) products and developing innovative therapies for heart disease, including acute myocarditis and other causes of heart failure. The Companys lead product, CardiolRx, is designed to be one of the safest and most consistent CBD formulations on the market. CardiolRx is pharmaceutically produced, cGMP certified, and is THC free. The Company plans to commercialize CardiolRx in the billion-dollar market for medicinal cannabinoids in Canada and is also pursuing distribution opportunities in Europe and Latin America.

In heart failure, Cardiol is planning an international clinical study of CardiolRx in acute myocarditis, a condition caused by inflammation in heart tissue, which remains the most common cause of sudden cardiac death in people less than 35 years of age. The Company is also developing proprietary nanotechnology to uniquely deliver pharmaceutical CBD and other anti-inflammatory drugs directly to sites of inflammation in the heart that are associated with heart failure. Heart failure is the leading cause of death and hospitalization in North America with associated healthcare costs in the U.S. alone exceeding $30 billion. For further information about Cardiol Therapeutics, please visitwww.cardiolrx.com.

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Breaking News: Cardiol Therapeutics Announces Clinical Steering Committee for Phase 2 International Trial in Acute Myocarditis Using CardiolRx(TM) 100...

Cardiac Stem Cells Comments Off on Breaking News: Cardiol Therapeutics Announces Clinical Steering Committee for Phase 2 International Trial in Acute Myocarditis Using CardiolRx(TM) 100… | November 20th, 2019

Stem Cell Therapy Market: Snapshot

Of late, there has been an increasing awareness regarding the therapeutic potential of stem cells for management of diseases which is boosting the growth of the stem cell therapy market. The development of advanced genome based cell analysis techniques, identification of new stem cell lines, increasing investments in research and development as well as infrastructure development for the processing and banking of stem cell are encouraging the growth of the global stem cell therapy market.

One of the key factors boosting the growth of this market is the limitations of traditional organ transplantation such as the risk of infection, rejection, and immunosuppression risk. Another drawback of conventional organ transplantation is that doctors have to depend on organ donors completely. All these issues can be eliminated, by the application of stem cell therapy. Another factor which is helping the growth in this market is the growing pipeline and development of drugs for emerging applications. Increased research studies aiming to widen the scope of stem cell will also fuel the growth of the market. Scientists are constantly engaged in trying to find out novel methods for creating human stem cells in response to the growing demand for stem cell production to be used for disease management.

It is estimated that the dermatology application will contribute significantly the growth of the global stem cell therapy market. This is because stem cell therapy can help decrease the after effects of general treatments for burns such as infections, scars, and adhesion. The increasing number of patients suffering from diabetes and growing cases of trauma surgery will fuel the adoption of stem cell therapy in the dermatology segment.

Global Stem Cell Therapy Market: Overview

Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.

Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.

Global Stem Cell Therapy Market: Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

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Stem Cell Therapy Market to Surge at a Robust Pace in Terms of Revenue Over 2025 - The Denton Chronicle

Cardiac Stem Cells Comments Off on Stem Cell Therapy Market to Surge at a Robust Pace in Terms of Revenue Over 2025 – The Denton Chronicle | November 20th, 2019

This disease is especially debilitating since it can be highly visible and affect a patients appearance and their daily functions, such as eating and speaking, said Professor Kevin Harrington, investigator for KEYNOTE-048, professor of biological cancer therapies at The Institute of Cancer Research, London, and consultant clinical oncologist at The Royal Marsden NHS Foundation Trust. Considering the great need for new treatment options, we are encouraged by todays KEYTRUDA approval in Europe, which will allow certain patients to be treated with immunotherapy earlier in the course of their treatment.

This approval allows marketing of the KEYTRUDA monotherapy and combination regimen in all 28 EU member states plus Iceland, Lichtenstein and Norway.

KEYTRUDA is now the first anti-PD-1 treatment option in the first-line setting for metastatic or unresectable recurrent head and neck cancer, a disease that has been treated the same way in the EU for more than a decade, said Dr. Jonathan Cheng, vice president, clinical research, Merck Research Laboratories. The European Commission approval underscores our commitment to transforming the way cancer is treated around the world.

Data Supporting the European Approval

This approval is based on data from the Phase 3 KEYNOTE-048 trial, a multi-center, randomized, open-label, active-controlled trial conducted in 882 patients with histologically confirmed metastatic or recurrent HNSCC of the oral cavity, pharynx or larynx, who had not previously received systemic therapy for recurrent or metastatic disease and who were considered incurable by local therapies. Randomization was stratified by tumor PD-L1 expression (Tumor Proportion Score [TPS] 50% or <50%), HPV status (positive or negative), and ECOG Performance Status (PS) (0 vs. 1). The dual primary endpoints were OS and progression-free survival (PFS). Patients were randomized 1:1:1 to one of the following treatment arms:

Treatment with KEYTRUDA continued until RECIST v1.1-defined progression of disease as determined by the investigator, unacceptable toxicity or a maximum of 24 months.

Efficacy Results for KEYTRUDA as Monotherapy in KEYNOTE-048 with PD-L1 Expression(CPS 1)

Endpoint

KEYTRUDA

n=257

Standard

Treatment*

n=255

OS

Number (%) of patients with event

197 (77%)

229 (90%)

Median in months (95% CI)

12.3 (10.8, 14.3)

10.3 (9.0, 11.5)

Hazard ratio (95% CI)

0.74 (0.61, 0.90)

p-Value

0.00133

PFS

Number (%) of patients with event

228 (89%)

237 (93%)

Median in months (95% CI)

3.2 (2.2, 3.4)

5.0 (4.8, 6.0)

Hazard ratio (95% CI)

1.13 (0.94, 1.36)

p-Value

0.89580

ORR

Objective response rate (95% CI)

19.1% (14.5, 24.4)

35% (29.1, 41.1)

Complete response

5%

3%

Partial response

14%

32%

p-Value

1.0000

Duration of Response

Median in months (range)

23.4 (1.5+, 43.0+)

4.5 (1.2+, 38.7+)

% with duration 6 months

81%

36%

*

Cetuximab, platinum, and 5-FU

Based on the stratified Cox proportional hazard model

Based on stratified log-rank test

Response: Best objective response as confirmed complete response or partial response

Based on Miettinen and Nurminen method stratified by ECOG (0 vs. 1), HPV status (positive vs. negative) and PD-L1 status (strongly positive vs. not strongly positive)

Efficacy Results for KEYTRUDA plus Chemotherapy in KEYNOTE-048 with PD-L1 Expression(CPS 1)

Endpoint

KEYTRUDA +

Platinum Chemotherapy +

5-FU

n=242

Standard

Treatment*

n=235

OS

Number (%) of patients with event

177 (73%)

213 (91%)

Median in months (95% CI)

13.6 (10.7, 15.5)

10.4 (9.1, 11.7)

Hazard ratio (95% CI)

0.65 (0.53, 0.80)

p-Value

0.00002

PFS

Number (%) of patients with event

212 (88%)

221 (94%)

Median in months (95% CI)

5.1 (4.7, 6.2)

5.0 (4.8, 6.0)

Hazard ratio (95% CI)

0.84 (0.69, 1.02)

p-Value

0.03697

ORR

Objective response rate (95% CI)

36% (30.3, 42.8)

36% (29.6, 42.2)

Complete response

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European Commission Approves Two New Regimens of Merck's KEYTRUDA (pembrolizumab) as First-Line Treatment for Metastatic or Unresectable Recurrent...

Cardiac Stem Cells Comments Off on European Commission Approves Two New Regimens of Merck’s KEYTRUDA (pembrolizumab) as First-Line Treatment for Metastatic or Unresectable Recurrent… | November 20th, 2019

The following are the top rated Healthcare stocks according to Validea's Small-Cap Growth Investor model based on the published strategy of Motley Fool. This strategy looks for small cap growth stocks with solid fundamentals and strong price performance.

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Validea's Top Five Healthcare Stocks Based On Motley Fool - 11/17/2019 - Nasdaq

Cardiac Stem Cells Comments Off on Validea’s Top Five Healthcare Stocks Based On Motley Fool – 11/17/2019 – Nasdaq | November 18th, 2019

As per a recent study, Human heart muscle cells show some changes in the way they function in space, although they operate normally within 10 to 12 days after returning to the Earth, as per the new study. The research examined the cell-level cardiac function and gene expression in human heart cells cultured aboard the International Space Station (ISS) for 5.5 weeks. Coverage to microgravity altered the expression of thousands of genes, but often standard patterns of gene expression reappeared within ten days after returning to the Earth, the researchers said. Our study is novel because it is the first to use human induced pluripotent stem cells to study the effects of spaceflight on human heart function, as stated by Joseph C Wu of Stanford University School of Medicine in the US.

Past studies have revealed that spaceflight induces physiological changes in cardiac function, which includes lowered arterial pressure, reduced heart rate, and increased cardiac output. On the other hand, to date, most cardiovascular microgravity physiology research has been conducted either in non-human models or at tissue, organ, or systemic levels. Comparatively, little is known to date about the role of microgravity in influencing human cardiac function at the cellular level. Wu and his collaborators examined human-induced pluripotent stem cell-derived cardiomyocytes (hiPSC-CMs). They removed hiPSC lines from three individuals by reprogramming blood cells and then differentiated them into hiPSC-CMs. Upon returning to Earth, space-flown hiPSC-CMs showed normal morphology and structure. However, they did adapt through modification of their beating pattern and calcium recycling pattern, the researchers explained. They also conducted RNA sequencing of hiPSC-CMs harvested at around 4 weeks aboard the ISS, and 10 to 12 days after returning to Earth.

These results showed that 2,647 genes were differentially expressed among flight, post-flight & ground control samples, the researchers stated. Gene pathways associated with mitochondrial function were expressed more in space-flown hiPSC-CMs, they further said. Comparing the samples revealed that hiPSC-CMs implement a unique gene expression pattern during spaceflight, which reverts to one that is very similar to ground side controls upon return to regular gravity, as per the researchers. These studies may offer insight into cellular mechanisms that could improve astronaut health during long-duration spaceflight, or potentially lay the foundation for latest insights into enhancing heart health on Earth, he further added.

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Human heart cells behave differently in space - SellRegular

Cardiac Stem Cells Comments Off on Human heart cells behave differently in space – SellRegular | November 18th, 2019

ANI | Updated: Nov 16, 2019 17:52 IST

Washington D.C. [USA], Nov 16 (ANI): Researchers have developed a breakthrough cell therapy to improve memory and prevent seizures in mice following traumatic brain injury.The study -- 'Transplanted interneurons improve memory precision after traumatic brain injury' -- was published in the journal of 'Nature Communications.'Traumatic brain injuries (TBI) affect two million Americans each year and cause cell death and inflammation in the brain. People, who experience a head injury often, suffer from lifelong memory loss and can develop epilepsy.In the study, the team transplanted embryonic progenitor cells capable of generating inhibitory interneurons, a specific type of nerve cell that controls the activity of brain circuits, into the brains of mice with traumatic brain injury. They targeted the hippocampus, a brain region responsible for learning and memory.The researchers have discovered that the transplanted neurons migrated into the injury where they formed new connections with the injured brain cells and thrived long term.Within a month after treatment, the mice showed signs of memory improvement such as being able to tell the difference between a box where they had an unpleasant experience from one where they did not.They were able to do this just as well as mice that never had a brain injury. The cell transplants also prevented the mice from developing epilepsy, which affected more than half of the mice who were not treated with new interneurons."Inhibitory neurons are critically involved in many aspects of memory, and they are extremely vulnerable to dying after a brain injury," said Robert Hunt, PhD, assistant professor of anatomy and neurobiology at UCI School of Medicine, who led the study."While we cannot stop interneurons from dying, it was exciting to find that we can replace them and rebuild their circuits," added Hunt.To further test their observations, Hunt and his team silenced the transplanted neurons with a drug, which caused the memory problems to return."It was exciting to see the animals' memory problems come back after we silenced the transplanted cells because it showed that the new neurons really were the reason for the memory improvement," said Bingyao Zhu, a junior specialist and first author of the study.Currently, there are no treatments for people who experience a head injury. If the results in mice can be replicated in humans, it could have a tremendous impact on patients. The next step is to create interneurons from human stem cells."So far, nobody has been able to convincingly create the same types of interneurons from human pluripotent stem cells," Hunt said. "But I think we're close to being able to do this." (ANI)

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New cell therapy improves memory and stops seizures following TBI: Study - ANI News

Cardiac Stem Cells Comments Off on New cell therapy improves memory and stops seizures following TBI: Study – ANI News | November 17th, 2019

SUMMIT, N.J.--(BUSINESS WIRE)--Celgene Corporation (NASDAQ:CELG) today announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion, recommending the approval of REVLIMID (lenalidomide) in combination with rituximab (anti-CD20 antibody) (R) for the treatment of adult patients with previously treated follicular lymphoma (FL) (Grade 1-3a). If approved by the European Commission (EC), R2 will be the first combination treatment regimen for patients with FL that does not include chemotherapy.

Since its initial approval in 2007, REVLIMID has continued to demonstrate its benefits across a range of serious blood disorders in Europe and a CHMP positive opinion for this combination with rituximab is very good news for patients with follicular lymphoma. We look forward to the European Commission decision, said Tuomo Ptsi, President of Hematology/Oncology for Celgene Worldwide Markets.

In FL, a subtype of indolent NHL, the immune system is not functioning optimally.1,2 When this dysfunction occurs, the immune system either fails to detect or attack cancerous cells.1,2 Rituximab is a monoclonal antibody that targets the CD 20 antigen on the surface of pre-B and mature B-lymphocytes. Upon binding to CD20, rituximab causes B-cell lysis. Lenalidomide is an immunomodulator that increases the number and activation of T and natural killer (NK) cells, resulting in the lysis of tumor cells. The R2 combination regimen acts by complementary mechanisms to help the patients immune system to find and destroy the cancer cells.3

Given the incurable nature of FL2, a high unmet medical need exists for the development of novel treatment options with new mechanisms of action and a tolerable safety profile to help improve progression-free survival (PFS) especially in the setting of previously treated FL.

The estimated incidence of NHL in Europe was 100,055 cases in 2018; FL accounts for approximately 25% of all NHL cases and is the most common form of indolent NHL.3,4,5

Chemotherapy is a standard of care for indolent forms of NHL, but most patients will relapse or become refractory to their current treatment, said Prof. John Gribben, President of EHA and Centre for Haemato-Oncology, Barts Cancer Institute, in England The combination of REVLIMID and rituximab could represent a new, chemotherapy-free treatment option for patients with previously treated follicular lymphoma.

The CHMP positive opinion is based primarily on results from the randomized, multi-center, double-blind, Phase 3 AUGMENT study, which evaluated the efficacy and safety of the R combination versus rituximab plus placebo in patients with previously treated FL (n=295).6,7 Additionally, findings from the MAGNIFY study were included as support for the safety and the efficacy of lenalidomide plus rituximab in patients with relapsed or refractory FL, including rituximab refractory FL patients.8

The CHMP reviews applications for all member states of the European Union (EU), as well as Norway, Liechtenstein, and Iceland. The European Commission, which generally follows the recommendation of the CHMP, is expected to make its final decision in approximately two months. If approval is granted, detailed conditions for the use of this product will be described in the REVLIMID Summary of Product Characteristics (SmPC), which will be published in the revised European Public Assessment Report (EPAR).

About Follicular Lymphoma

Lymphoma is a blood cancer that develops in lymphocytes, a type of white blood cell in the immune system that helps protect the body from infection.9 There are two classes of lymphoma Hodgkins lymphoma and non-Hodgkins lymphoma (NHL) each with specific subtypes that determine how the cancer behaves, spreads and should be treated.3,10,11 Other differentiating factors of lymphomas are what type of lymphocyte is affected (T cell or B cell) and how mature the cells are when they become cancerous.11

Follicular lymphoma is the most common indolent (slow-growing) form of NHL, accounting for approximately 25% of all Non-Hodgkin lymphoma (NHL) patients.5,12 Most patients present with advanced disease usually when lymphoma-related symptoms appear (e.g., nodal disease, B symptoms, cytopenia) and receive systemic chemoimmunotherapy.5 While follicular lymphoma patients are generally responsive to initial treatment, the disease course is characterized by recurrent relapses over time with shorter remission periods.13

About AUGMENT

AUGMENT is a Phase 3, randomized, double-blind clinical trial evaluating the efficacy and safety of REVLIMID (lenalidomide) in combination with rituximab (R) versus rituximab plus placebo in patients with previously treated follicular lymphoma (FL). AUGMENT included patients diagnosed with Grade 1, 2 or 3a FL, who were previously treated with at least 1 prior systemic therapy and two previous doses of rituximab. Patients were documented relapsed, refractory or progressive disease following systemic therapy, but were not rituximab-refractory.6,7

The primary endpoint was progression-free survival, defined as the time from date of randomization to the first observation of disease progression or death due to any cause. Secondary and exploratory endpoints included overall response rate, durable complete response rate, complete response rate, duration of response, duration of complete response, overall survival, event-free survival and time to next anti-lymphoma therapy.6,7

About REVLIMID

REVLIMID is approved in Europe and the United States as monotherapy, indicated for the maintenance treatment of adult patients with newly diagnosed multiple myeloma (MM) who have undergone autologous stem cell transplantation. REVLIMID as combination therapy is approved in Europe, in the United States, in Japan and in around 25 other countries for the treatment of adult patients with previously untreated MM who are not eligible for transplant. REVLIMID is also approved in combination with dexamethasone for the treatment of patients with MM who have received at least one prior therapy in nearly 70 countries, encompassing Europe, the Americas, the Middle-East and Asia, and in combination with dexamethasone for the treatment of patients whose disease has progressed after one therapy in Australia and New Zealand.

REVLIMID is also approved in the United States, Canada, Switzerland, Australia, New Zealand and several Latin American countries, as well as Malaysia and Israel, for transfusion-dependent anaemia due to low- or intermediate-1-risk myelodysplastic syndromes (MDS) associated with a deletion 5q cytogenetic abnormality with or without additional cytogenetic abnormalities and in Europe for the treatment of patients with transfusion-dependent anemia due to low- or intermediate-1-risk MDS associated with an isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate.

In addition, REVLIMID is approved in Europe for the treatment of patients with mantle cell lymphoma (MCL) and in the United States for the treatment of patients with MCL whose disease has relapsed or progressed after two prior therapies, one of which included bortezomib. In Switzerland, REVLIMID is indicated for the treatment of patients with relapsed or refractory MCL after prior therapy that included bortezomib and chemotherapy/rituximab.

REVLIMID is not indicated and is not recommended for the treatment of patients with chronic lymphocytic leukemia (CLL) outside of controlled clinical trials.

Important Safety Information

WARNING: EMBRYO-FETAL TOXICITY, HEMATOLOGIC TOXICITY, and VENOUS and ARTERIAL THROMBOEMBOLISM

Embryo-Fetal Toxicity

Do not use REVLIMID during pregnancy. Lenalidomide, a thalidomide analogue, caused limb abnormalities in a developmental monkey study. Thalidomide is a known human teratogen that causes severe life-threatening human birth defects. If lenalidomide is used during pregnancy, it may cause birth defects or embryo-fetal death. In females of reproductive potential, obtain 2 negative pregnancy tests before starting REVLIMID treatment. Females of reproductive potential must use 2 forms of contraception or continuously abstain from heterosexual sex during and for 4 weeks after REVLIMID treatment. To avoid embryo-fetal exposure to lenalidomide, REVLIMID is only available through a restricted distribution program, the REVLIMID REMS program.

Information about the REVLIMID REMS program is available at http://www.celgeneriskmanagement.com or by calling the manufacturers toll-free number 1-888-423-5436.

Hematologic Toxicity (Neutropenia and Thrombocytopenia)

REVLIMID can cause significant neutropenia and thrombocytopenia. Eighty percent of patients with del 5q MDS had to have a dose delay/reduction during the major study. Thirty-four percent of patients had to have a second dose delay/reduction. Grade 3 or 4 hematologic toxicity was seen in 80% of patients enrolled in the study. Patients on therapy for del 5q MDS should have their complete blood counts monitored weekly for the first 8 weeks of therapy and at least monthly thereafter. Patients may require dose interruption and/or reduction. Patients may require use of blood product support and/or growth factors.

Venous and Arterial Thromboembolism

REVLIMID has demonstrated a significantly increased risk of deep vein thrombosis (DVT) and pulmonary embolism (PE), as well as risk of myocardial infarction and stroke in patients with MM who were treated with REVLIMID and dexamethasone therapy. Monitor for and advise patients about signs and symptoms of thromboembolism. Advise patients to seek immediate medical care if they develop symptoms such as shortness of breath, chest pain, or arm or leg swelling. Thromboprophylaxis is recommended and the choice of regimen should be based on an assessment of the patients underlying risks.

CONTRAINDICATIONS

Pregnancy: REVLIMID can cause fetal harm when administered to a pregnant female and is contraindicated in females who are pregnant. If this drug is used during pregnancy or if the patient becomes pregnant while taking this drug, the patient should be apprised of the potential risk to the fetus

Severe Hypersensitivity Reactions: REVLIMID is contraindicated in patients who have demonstrated severe hypersensitivity (e.g., angioedema, Stevens-Johnson syndrome, toxic epidermal necrolysis) to lenalidomide

WARNINGS AND PRECAUTIONS

Embryo-Fetal Toxicity: See Boxed WARNINGS

REVLIMID REMS Program: See Boxed WARNINGS: Prescribers and pharmacies must be certified with the REVLIMID REMS program by enrolling and complying with the REMS requirements; pharmacies must only dispense to patients who are authorized to receive REVLIMID. Patients must sign a Patient-Physician Agreement Form and comply with REMS requirements; female patients of reproductive potential who are not pregnant must comply with the pregnancy testing and contraception requirements and males must comply with contraception requirements

Hematologic Toxicity: REVLIMID can cause significant neutropenia and thrombocytopenia. Monitor patients with neutropenia for signs of infection. Advise patients to observe for bleeding or bruising, especially with use of concomitant medications that may increase risk of bleeding. MM: Patients taking REVLIMID/dex or REVLIMID as maintenance therapy should have their complete blood counts (CBC) assessed every 7 days for the first 2 cycles, on days 1 and 15 of cycle 3, and every 28 days thereafter. MDS: Patients on therapy for del 5q MDS should have their complete blood counts monitored weekly for the first 8 weeks of therapy and at least monthly thereafter. Patients may require dose interruption and/or dose reduction. Please see the Black Box WARNINGS for further information. MCL: Patients taking REVLIMID for MCL should have their CBCs monitored weekly for the first cycle (28 days), every 2 weeks during cycles 2-4, and then monthly thereafter. Patients may require dose interruption and/or dose reduction

Venous and Arterial Thromboembolism: See Boxed WARNINGS: Venous thromboembolic events (DVT and PE) and arterial thromboses (MI and CVA) are increased in patients treated with REVLIMID. Patients with known risk factors, including prior thrombosis, may be at greater risk and actions should be taken to try to minimize all modifiable factors (e.g., hyperlipidemia, hypertension, smoking). Thromboprophylaxis is recommended and the regimen should be based on patients underlying risks. ESAs and estrogens may further increase the risk of thrombosis and their use should be based on a benefit-risk decision

Increased Mortality in Patients with CLL: In a clinical trial in the first-line treatment of patients with CLL, single agent REVLIMID therapy increased the risk of death as compared to single agent chlorambucil. Serious adverse cardiovascular reactions, including atrial fibrillation, myocardial infarction, and cardiac failure, occurred more frequently in the REVLIMID arm. REVLIMID is not indicated and not recommended for use in CLL outside of controlled clinical trials

Second Primary Malignancies (SPM): In clinical trials in patients with MM receiving REVLIMID, an increase of hematologic plus solid tumor SPM, notably AML and MDS, have been observed. Monitor patients for the development of SPM. Take into account both the potential benefit of REVLIMID and risk of SPM when considering treatment

Increased Mortality with Pembrolizumab: In clinical trials in patients with multiple myeloma, the addition of pembrolizumab to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of patients with multiple myeloma with a PD-1 or PD-L1 blocking antibody in combination with a thalidomide analogue plus dexamethasone is not recommended outside of controlled clinical trials

Hepatotoxicity: Hepatic failure, including fatal cases, has occurred in patients treated with REVLIMID/dex. Pre-existing viral liver disease, elevated baseline liver enzymes, and concomitant medications may be risk factors. Monitor liver enzymes periodically. Stop REVLIMID upon elevation of liver enzymes. After return to baseline values, treatment at a lower dose may be considered

Severe Cutaneous Reactions: Severe cutaneous reactions including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN), and drug reaction with eosinophilia and systemic symptoms (DRESS) have been reported. These events can be fatal. Patients with a prior history of Grade 4 rash associated with thalidomide treatment should not receive REVLIMID. Consider REVLIMID interruption or discontinuation for Grade 2-3 skin rash. Permanently discontinue REVLIMID for Grade 4 rash, exfoliative or bullous rash, or for other severe cutaneous reactions such as SJS, TEN, or DRESS.

Tumor Lysis Syndrome (TLS): Fatal instances of TLS have been reported during treatment with lenalidomide. The patients at risk of TLS are those with high tumor burden prior to treatment. These patients should be monitored closely and appropriate precautions taken

Tumor Flare Reaction (TFR): TFR has occurred during investigational use of lenalidomide for CLL and lymphoma. Monitoring and evaluation for TFR is recommended in patients with MCL. Tumor flare may mimic the progression of disease (PD). In patients with Grade 3 or 4 TFR, it is recommended to withhold treatment with REVLIMID until TFR resolves to Grade 1. REVLIMID may be continued in patients with Grade 1 and 2 TFR without interruption or modification, at the physicians discretion

Impaired Stem Cell Mobilization: A decrease in the number of CD34+ cells collected after treatment (>4 cycles) with REVLIMID has been reported. Consider early referral to transplant center to optimize timing of the stem cell collection

Thyroid Disorders: Both hypothyroidism and hyperthyroidism have been reported. Measure thyroid function before start of REVLIMID treatment and during therapy

Early Mortality in Patients with MCL: In another MCL study, there was an increase in early deaths (within 20 weeks), 12.9% in the REVLIMID arm versus 7.1% in the control arm. Risk factors for early deaths include high tumor burden, MIPI score at diagnosis, and high WBC at baseline (10 x 109/L)

Hypersensitivity: Hypersensitivity, including angioedema, anaphylaxis, and anaphylactic reactions to REVLIMID has been reported. Permanently discontinue REVLIMID for angioedema and anaphylaxis.

ADVERSE REACTIONS

Multiple Myeloma

Myelodysplastic Syndromes

Mantle Cell Lymphoma

DRUG INTERACTIONS

Periodic monitoring of digoxin plasma levels is recommended due to increased Cmax and AUC with concomitant REVLIMID therapy. Patients taking concomitant therapies such as erythropoietin stimulating agents or estrogen containing therapies may have an increased risk of thrombosis. It is not known whether there is an interaction between dex and warfarin. Close monitoring of PT and INR is recommended in patients with MM taking concomitant warfarin

USE IN SPECIFIC POPULATIONS

Please see full Prescribing Information, including Boxed WARNINGS.

Please see full SmPC for further information.

About Celgene

Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through next-generation solutions in protein homeostasis, immuno-oncology, epigenetics, immunology and neuro-inflammation. For more information, please visit http://www.celgene.com. Follow Celgene on Social Media: @Celgene, Pinterest, LinkedIn, Facebook and YouTube.

Forward-Looking Statements

This press release contains forward-looking statements, which are generally statements that are not historical facts. Forward-looking statements can be identified by the words "expects," "anticipates," "believes," "intends," "estimates," "plans," "will," "outlook" and similar expressions. Forward-looking statements are based on management's current plans, estimates, assumptions and projections, and speak only as of the date they are made. Celgene undertakes no obligation to update any forward-looking statement in light of new information or future events, except as otherwise required by law. Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict and are generally beyond each company's control. Actual results or outcomes may differ materially from those implied by the forward-looking statements as a result of the impact of a number of factors, many of which are discussed in more detail in the Annual Report on Form 10-K and other reports of each company filed with the Securities and Exchange Commission, including factors related to the proposed transaction between Bristol-Myers Squibb and Celgene, such as, but not limited to, the risks that: managements time and attention is diverted on transaction related issues; disruption from the transaction make it more difficult to maintain business, contractual and operational relationships; legal proceedings are instituted against Bristol-Myers Squibb, Celgene or the combined company could delay or prevent the proposed transaction; and Bristol-Myers Squibb, Celgene or the combined company is unable to retain key personnel.

1 Scott DW, Gascoyne RD. The tumour microenvironment in B cell lymphomas. Nat Rev Cancer. 2014;14(8):517-534.2 Kridel R, Sehn LH, Gascoyne RD. Pathogenesis of follicular lymphoma. J Clin Invest. 2012;122(10):3424-3431.3 Chiu H, Trisal P, Bjorklund C, et al. Combination lenalidomide-rituximab immunotherapy activates anti-tumour immunity and induces tumour cell death by complementary mechanisms of action in follicular lymphoma. Br J Haematol. 2019;185(2):240-253.4 European Cancer Information System. Estimates of cancer incidence and mortality in 2018, for all countries. Available at: https://ecis.jrc.ec.europa.eu/explorer.php. Accessed August 2019.5 European Society for Medical Oncology. Follicular Lymphoma: A Guide for Patients. 2014. Available at: https://www.esmo.org/content/download/52236/963497/file/EN-Follicular-Lymphoma-Guide-for-Patients.pdf . Accessed September 2019.6 Leonard JP, Trneny M, Izutsu K, et al. AUGMENT: A Phase III Study of Lenalidomide Plus Rituximab Versus Placebo Plus Rituximab in Relapsed or Refractory Indolent Lymphoma. J Clin Oncol. 2019;10;37(14):1188-1199.7 ClinicalTrials.gov Rituximab Plus Lenalidomide for Patients With Relapsed / Refractory Indolent Non-Hodgkin's Lymphoma (Follicular Lymphoma and Marginal Zone Lymphoma) (AUGMENT). Available at: https://clinicaltrials.gov/ct2/show/NCT01938001 Accessed September 2019.8 ClinicalTrials.gov Lenalidomide Plus Rituximab Followed by Lenalidomide Versus Rituximab Maintenance for Relapsed/Refractory Follicular, Marginal Zone or Mantle Cell Lymphoma (MAGNIFY). Available at: https://clinicaltrials.gov/ct2/show/NCT01996865 Accessed August 2019.9 American Cancer Society. Lymphoma. Available at: https://www.cancer.org/cancer/lymphoma.html. Accessed August 2019.10 American Cancer Society. What is Hodgkin Lymphoma? Available at: https://www.cancer.org/cancer/hodgkin-lymphoma/about/what-is-hodgkin-disease.html. Accessed August 2019.11 American Cancer Society. What is Non-Hodgkin Lymphoma? Available at: https://www.cancer.org/cancer/non-hodgkin-lymphoma/about/what-is-non-hodgkin-lymphoma.html. Accessed August 2019.12 Lymphoma Action. Follicular lymphoma. Available at: https://lymphoma-action.org.uk/types-lymphoma-non-hodgkin-lymphoma/follicular-lymphoma. Accessed November 2019.13 Montoto S, Lopez-Guillermo A, Ferrer A, et al. Survival after progression in patients with follicular lymphoma: analysis of prognostic factors. Ann Oncol. 2002;13(4):523-30.

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Celgene Receives CHMP Positive Opinion for REVLIMID (lenalidomide) in Combination With Rituximab for the Treatment of Adult Patients With Previously...

Cardiac Stem Cells Comments Off on Celgene Receives CHMP Positive Opinion for REVLIMID (lenalidomide) in Combination With Rituximab for the Treatment of Adult Patients With Previously… | November 15th, 2019

Heart failure (HF) is the most frequent cardiovascular diagnosis and exacts significant health and financial costs around the globe. It is estimated that at least 26 million people worldwide are living with HF, including nearly 6 million in the United States.1, 2 One in nine U.S. deaths in 2009 included heart failure as a contributing cause and about 50 percent of people in the U.S. with HF die within five years of diagnosis.2 The annual cost of HF-related healthcare services, medication and missed days of work is estimated at $40 billion in the United States and $108 billion globally.3, 4 Quality of life in HF patients is frequently worse than many other chronic diseases and comorbidities are common.5-7 The challenges of HF are expected to grow, as it is estimated that more than 8 million people in the United States alone will have HF by 2030.2 Current therapies improve quality of life in the short-term and have improved long-term survival but a significant number of patients have Class 3 HF despite optimal medical and device therapy. These patients have limited treatment options beyond heart transplant and left ventricular assist devices (LVAD). New therapeutic approaches that address the underlying causes of HF are needed to improve patient outcomes.

Heart failure is a complex disease process and multiple pathways contribute to its development and progression. Myocardial ischemia is frequently an issue in both ischemic and non-ischemic cardiomyopathy as well as HF with preserved and/or reduced ejection fraction. Myocardial ischemia results in insufficient oxygen and nutrients and leads to hypoxia, cardiomyocyte and fibrosis, which all contribute to the progression of heart failure. More effective angiogenesis may prevent this progression. Cell homing also plays a critical role, as injured cardiac tissue secretes factors that lead to the recruitment, proliferation, migration and differentiation of progenitor cells that can help repair tissue damage. Stromal cell-derived factor (SDF)-1 has been shown to play an important role in cardiac repair by mediating cell homing.10 Mitochondrial energy generation is also impaired in HF, leading to decreased contractility and adverse changes to cardiac architecture.11 Scar tissue formed in response to cardiomyocyte injury or death can compromise the hearts mechanical strength or electrical signaling results in myocardial infarction. Inflammatory responses to cardiac tissue damage can promote inappropriate and chronic inflammation and the expression of pro-inflammatory molecules that lead to pathologic changes to cardiac architecture.12, 13

These pathways offer a variety of potential new targets for therapeutic intervention to prevent the development and progression of HF. This opens the door to the development of novel therapies that address the underlying molecular and cellular causes of disease rather than treating HF symptoms alone.

After decades of development, gene-based therapies are now validated therapeutic modalities for the treatment of inherited retinal disorders and cancer and are undergoing clinical evaluation in a variety of inherited, acute and chronic diseases. Nearly two dozen single gene-based therapies for HF have been evaluated in clinical trials.14 Genes evaluated as monogenic gene therapy for HF in clinical trials include vascular endothelial growth factor (VEGF) and fibroblast growth factor type 4 (FGF4) to promote angiogenesis; adenylyl cyclase type 6 (AC6) and sarco/endoplasmic reticulum Ca2+-ATPase type 2 (SERCA2) to improve cardiac calcium homeostasis, which plays a critical role in the contraction and relaxation of heart muscle; and stromal cell-derived factor-1 (SDF-1) to improve cell homing and promote cardiac tissue repair. Late-stage trials of single gene therapies have yielded conflicting results, raising the question as to whether positively impacting a single pathway can be sufficient to overcome detrimental activity of other pathways that contribute to the development and progression of HF. Other potential limitations to HF therapies evaluated in clinical trials to date include the method of delivery, dose and the potency of vectors and gene products.

Given the multiple molecular and cellular pathways active in HF, a multi-gene approach to HF gene therapy may be needed. Simultaneously delivering multiple genes that target diverse HF-related pathways has the potential to improve cardiac biology and function. A triple gene therapy approach (INXN-4001, Triple-Gene LLC, a majority-owned subsidiary of Intrexon Corporation) is currently in clinical development, with each of the genes targeting a specific HF-related pathway. The investigational drug candidate INXN4001 vector expresses: the S100A1 gene product, which regulates calcium-controlled networks and modulates contractility, excitability, maintenance of cellular metabolism and survival; SDF-1a which recruits stem cells, inhibits apoptosis and supports new blood vessel formation; and VEGF-165 which initiates new vessel formation, endothelial cell migration/activation, stem cell recruitment and tissue regeneration. The hypothesis is that the simultaneous delivery of multiple genes in a single vector would more effectively improve multiple aspects of cardiac function compared with single gene therapy. It is delivered by retrograde coronary sinus infusion of a triple effector plasmid designed with a self-cleaving linker to constitutively express human S100A1, SDF-1a and VEGF 165. This route is designed to allow for delivery of a dose to the ventricle which may help achieve improved therapeutic effect.

Several preclinical studies have set the foundation on which to advance a triple gene therapy for HF into the clinic.15-17 Using in vitro studies, transfecting cells derived from patients with dilated cardiomyopathy with a triple gene combination demonstrated improvement in contraction rate and duration, to the levels demonstrated by the control cells and did not result in increased cell death compared to controls.15 Studies in an Adriamycin-induced cardiomyopathy rodent model demonstrated triple gene therapy increased fractional shortening and myocardial wall thickness compared to controls.16 In addition, retrograde coronary sinus infusion of INXN-4001 in a porcine model of ischemic HF resulted in a cardiac-specific biodistribution profile.17

A Phase 1 clinical study has been initiated to evaluate the safety of a single dose of triple gene therapy in stable patients implanted with a LVAD for mechanical support of end-stage HF. An independent Data and Safety Monitoring Board agreed to proceeding to the second cohort following review of the data from the first cohort in the multi-site study.18 The study is ongoing and final results will help to inform our understanding of the potential that multi-gene therapy may play in the treatment of HF.

The recent FDA approvals of gene therapies for an inherited retinal disease and cancer are evidence that gene therapy is a valid therapeutic strategy. Realizing the potential of gene therapy in HF will require appropriately designed clinical trials, but several interesting approaches currently in development may prove to be effective. The results of the initial investigational drug INXN-4001 Phase 1 trial should provide insight into the safety of combining S100A1, SDF-1a and VEGF-165. Evaluation of additional multi-gene combinations will also be important for understanding which targeted pathways yield the greatest effects with respect to relevant clinical endpoints. Continued refinement and optimization of vector design and delivery methods will also be important for advancing further HF gene therapies from bench to bedside.

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The Heart of the Matter: Leveraging Advances in Cardiac Biology to Innovate Gene-Based Therapies for Heart Failure - Physician's Weekly

Cardiac Stem Cells Comments Off on The Heart of the Matter: Leveraging Advances in Cardiac Biology to Innovate Gene-Based Therapies for Heart Failure – Physician’s Weekly | November 14th, 2019

Representational image

Heart cells are altered in space, but return to normal within 10 days on Earth, say researchers who examined cell-level cardiac function and gene expression in human heart cells cultured aboard the International Space Station (ISS) for 5.5 weeks.

Exposure to microgravity altered the expression of thousands of genes, but largely normal patterns of gene expression reappeared within 10 days after returning to Earth, according to the study published in the journal Stem Cell Reports.

"We're surprised about how quickly human heart muscle cells are able to adapt to the environment in which they are placed, including microgravity," said senior study author Joseph C. Wu from Stanford University.

These studies may not only provide insight into cellular mechanisms that could benefit astronaut health during long-duration spaceflight, but also potentially lay the foundation for new insights into improving heart health on Earth.

Past studies have shown that spaceflight induces physiological changes in cardiac function, including reduced heart rate, lowered arterial pressure, and increased cardiac output.

But to date, most cardiovascular microgravity physiology studies have been conducted either in non-human models or at tissue, organ, or systemic levels.

Relatively little is known about the role of microgravity in influencing human cardiac function at the cellular level.

To address this question, the research team studied human induced pluripotent stem cell-derived cardiomyocytes (hiPSC-CMs). They generated hiPSC lines from three individuals by reprogramming blood cells, and then differentiated them into heart cells.

Beating heart cells were then sent to the ISS aboard a SpaceX spacecraft as part of a commercial resupply service mission. Simultaneously, ground control heart cells were cultured on Earth for comparison purposes.

Upon return to Earth, space-flown heart cells showed normal structure and morphology. However, they did adapt by modifying their beating pattern and calcium recycling patterns.

In addition, the researchers performed RNA sequencing of heart cells harvested at 4.5 weeks aboard the ISS, and 10 days after returning to Earth.

These results showed that 2,635 genes were differentially expressed among flight, post-flight, and ground control samples.

Most notably, gene pathways related to mitochondrial function were expressed more in space-flown heart cells.

A comparison of the samples revealed that heart cells adopt a unique gene expression pattern during spaceflight, which reverts to one that is similar to ground-side controls upon return to normal gravity, the study noted.

According to Wu, limitations of the study include its short duration and the use of 2D cell culture.

In future studies, the researchers plan to examine the effects of spaceflight and microgravity using more physiologically relevant hiPSC-derived 3D heart tissues with various cell types, including blood vessel cells.

"We also plan to test different treatments on the human heart cells to determine if we can prevent some of the changes the heart cells undergo during spaceflight," Wu said.

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Human Heart Cells Transform in Space; Return to Normal on Earth: Study - The Weather Channel

Cardiac Stem Cells Comments Off on Human Heart Cells Transform in Space; Return to Normal on Earth: Study – The Weather Channel | November 14th, 2019

BioRestorative Therapies (OTCMKTS:BRTX) and Livongo Health (NASDAQ:LVGO) are both medical companies, but which is the better stock? We will compare the two companies based on the strength of their risk, institutional ownership, profitability, earnings, valuation, analyst recommendations and dividends.

Institutional and Insider Ownership

0.1% of Livongo Health shares are owned by institutional investors. 17.9% of BioRestorative Therapies shares are owned by company insiders. Strong institutional ownership is an indication that hedge funds, endowments and large money managers believe a stock will outperform the market over the long term.

Analyst Recommendations

This is a breakdown of recent ratings and recommmendations for BioRestorative Therapies and Livongo Health, as reported by MarketBeat.

Livongo Health has a consensus target price of $44.30, indicating a potential upside of 71.17%. Given Livongo Healths higher probable upside, analysts plainly believe Livongo Health is more favorable than BioRestorative Therapies.

Profitability

This table compares BioRestorative Therapies and Livongo Healths net margins, return on equity and return on assets.

Valuation & Earnings

This table compares BioRestorative Therapies and Livongo Healths top-line revenue, earnings per share (EPS) and valuation.

BioRestorative Therapies has higher earnings, but lower revenue than Livongo Health.

Summary

Livongo Health beats BioRestorative Therapies on 7 of the 9 factors compared between the two stocks.

BioRestorative Therapies Company Profile

BioRestorative Therapies, Inc. develops therapeutic products and medical therapies using cell and tissue protocols, primarily involving adult stem cells for the treatment of disc/spine disease and metabolic disorders. The company's lead cell therapy candidate is the BRTX-100, which focuses on providing non-surgical treatment for protruding and bulging lumbar discs in patients suffering from chronic lumbar disc disease. It also develops the ThermoStem program, a pre-clinical program for the treatment of metabolic diseases, such as type 2 diabetes, obesity, hypertension, and other metabolic disorders, as well as cardiac deficiencies. In addition, the company provides curved needle device, a needle system with a curved inner cannula that allows access to difficult-to-locate regions for the delivery or removal of fluids and other substances. Further, it offers skin care products under the Stem Pearls brand name. BioRestorative Therapies, Inc. has a research and development agreement with Rohto Pharmaceutical Co., Ltd.; and a research agreement with Pfizer, Inc. and the University of Pennsylvania. The company was formerly known as Stem Cell Assurance, Inc. and changed its name to BioRestorative Therapies, Inc. in August 2011. BioRestorative Therapies, Inc. was incorporated in 1997 and is headquartered in Melville, New York.

Livongo Health Company Profile

Livongo Health, Inc. provides an integrated suite of solutions for the healthcare industry in North America. It solutions promote health behavior change based on real-time data capture supported by intuitive devices and insights driven by data science. The company offers a platform that provides cellular-connected devices, supplies, informed coaching, data science-enabled insights, and facilitates access to medications. Its products include Livongo for Diabetes, Livongo for Hypertension, Livongo for Prediabetes and Weight Management, and Livongo for Behavioral Health by myStrength. The company was formerly known as EosHealth, Inc. and changed its name to Livongo Health, Inc. in 2014. Livongo Health, Inc. was incorporated in 2008 and is headquartered in Mountain View, California.

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Financial Contrast: BioRestorative Therapies (OTCMKTS:BRTX) and Livongo Health (OTCMKTS:LVGO) - DFS Caller

Cardiac Stem Cells Comments Off on Financial Contrast: BioRestorative Therapies (OTCMKTS:BRTX) and Livongo Health (OTCMKTS:LVGO) – DFS Caller | November 12th, 2019

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Global Stem Cell Therapy Market: Overview

Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.

Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.

Know the Growth Opportunities in Emerging Markets

Global Stem Cell Therapy Market: Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

The regional analysis covers:

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Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

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Stem Cell Therapy Market Poised to Expand at a Robust Pace Over 2025 - Tech Admirers

Cardiac Stem Cells Comments Off on Stem Cell Therapy Market Poised to Expand at a Robust Pace Over 2025 – Tech Admirers | November 11th, 2019

US Stem Cell (OTCMKTS:USRM) and Auxly Cannabis Group (OTCMKTS:CBWTF) are both small-cap medical companies, but which is the superior business? We will contrast the two businesses based on the strength of their risk, earnings, analyst recommendations, valuation, profitability, dividends and institutional ownership.

Valuation & Earnings

This table compares US Stem Cell and Auxly Cannabis Groups gross revenue, earnings per share (EPS) and valuation.

US Stem Cell has higher revenue and earnings than Auxly Cannabis Group.

Risk & Volatility

US Stem Cell has a beta of 5.05, suggesting that its stock price is 405% more volatile than the S&P 500. Comparatively, Auxly Cannabis Group has a beta of 0.62, suggesting that its stock price is 38% less volatile than the S&P 500.

Analyst Ratings

This is a summary of current ratings for US Stem Cell and Auxly Cannabis Group, as provided by MarketBeat.com.

Institutional and Insider Ownership

0.0% of Auxly Cannabis Group shares are held by institutional investors. 16.7% of US Stem Cell shares are held by company insiders. Strong institutional ownership is an indication that large money managers, endowments and hedge funds believe a company will outperform the market over the long term.

Profitability

This table compares US Stem Cell and Auxly Cannabis Groups net margins, return on equity and return on assets.

Summary

US Stem Cell beats Auxly Cannabis Group on 6 of the 9 factors compared between the two stocks.

US Stem Cell Company Profile

U.S. Stem Cell, Inc., a biotechnology company, focuses on the discovery, development, and commercialization of autologous cellular therapies for the treatment of chronic and acute heart damage, and vascular and autoimmune diseases in the United States and internationally. Its lead product candidates include MyoCell, a clinical therapy designed to populate regions of scar tissue within a patient's heart with autologous muscle cells or cells from a patient's body for enhancing cardiac function in chronic heart failure patients; and AdipoCell, a patient-derived cell therapy for the treatment of acute myocardial infarction, chronic heart ischemia, and lower limb ischemia. The company's product development pipeline includes MyoCell SDF-1, an autologous muscle-derived cellular therapy for improving cardiac function in chronic heart failure patients. It is also developing MyoCath, a deflecting tip needle injection catheter that is used to inject cells into cardiac tissue in therapeutic procedures to treat chronic heart ischemia and congestive heart failure. In addition, the company provides physician and patient based regenerative medicine/cell therapy training, cell collection, and cell storage services; and cell collection and treatment kits for humans and animals, as well operates a cell therapy clinic. The company was formerly known as Bioheart, Inc. and changed its name to U.S. Stem Cell, Inc. in October 2015. U.S. Stem Cell, Inc. was founded in 1999 and is headquartered in Sunrise, Florida.

Auxly Cannabis Group Company Profile

Auxly Cannabis Group Inc. operates as a cannabis streaming company. It provides funding for cannabis production; and holds contractual rights and minority equity interest relating to the operation of cannabis facilities. The company was formerly known as Cannabis Wheaton Income Corp. and changed its name to Auxly Cannabis Group Inc. in June 2018. Auxly Cannabis Group Inc. was incorporated in 1987 and is headquartered in Vancouver, Canada.

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Reviewing US Stem Cell (OTCMKTS:USRM) and Auxly Cannabis Group (OTCMKTS:CBWTF) - Riverton Roll

Cardiac Stem Cells Comments Off on Reviewing US Stem Cell (OTCMKTS:USRM) and Auxly Cannabis Group (OTCMKTS:CBWTF) – Riverton Roll | November 11th, 2019

Scientists from Dystrogen Therapeutics Corp. published data supporting cardioprotective effects of the Companys therapy for muscular dystrophy disorders. Cardiomyopathy is the most devastating cause of morbidity and mortality in Duchenne Muscular Dystrophy (DMD) patients and affects 30% of patients by 14years of age and 50% of patients by 18years of age. Heart failure in these patients is the result of cardiac myocyte death and fibrosis, leading to both diastolic and systolic dysfunction.

Dystrogen Therapeutics Corp has developed an engineered chimeric cell therapy which has been previously shown to restore muscle function in pre-clinical studies. For Duchennes muscular dystrophy, the company has developed dystrophin expressing chimeras DECs. Using the companys proprietary technology, DECs are created by an ex vivo fusion of allogeneic human myoblast from a healthy donor with autologous human myoblast received from DMD patient. DECs have been shown to maintain the ability to express normal dystrophin protein in previously published pre-clinical studies.

The new study published in theOctober 15th, 2019online edition of the journalStem Cell Reports and Reviewsconfirmed the protective effect of DEC on cardiac function after intraosseous delivery shown by increased values of both ejection fraction and fractional shortening, which at 90days revealed a rebound effect when compared to the vehicle injected controls and mice receiving not-chimeric cell therapy. Moreover, these functional improvements correlated with restoration of dystrophin expression in cardiac muscle at 90days post-DEC treatment.

These findings are potentially significant for the treatment of DMD, said Dr. Maria Siemionow, MD, PhD Dystrogen Therapeutics Corp chief scientific officer and the therapys inventor. This study establishes DEC as a promising new option for cardiac protection and potential amelioration of DMD related cardiac pathology.

These data add to the growing body of literature supporting the potential of our chimeric cell platform to restore systemic muscle function, with less potential side effects then gene therapy-based approaches, said Dr. Kris Siemionow, MD, PhD Dystrogen Therapeutics Corp CEO. We are very pleased to have these data published in a highly relevant journal for the field and look forward to further exploring this opportunity.

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Dystrogen Therapeutics Announces That Treatment With DEC Cells Improves Cardiac Function Cardiology2.0 - Cardiology2.0

Cardiac Stem Cells Comments Off on Dystrogen Therapeutics Announces That Treatment With DEC Cells Improves Cardiac Function Cardiology2.0 – Cardiology2.0 | November 11th, 2019

Home News Going into Space Changes the Human Heart Cells, but What Happens When They Get Back on Earth?

Commonly, astronauts stay in space for a more extended period of time, and NASA is planning longer missions to the Moon and Mars. Researchers say that we need to understand better the effects that microgravity has on the heart.

Studies have shown that spaceflight can reduce heart rate and the lower arterial pressure, and can also increase cardiac output. However, new research shows how microgravity zero gravity has an impact on the human heart when it comes to the cellular level.

Scientists have been able to check the health of astronauts while they were in space, which was a great way to understand the molecular cell changes. This comes from Joseph C. Wu, from Stanford Universitys School of Medicine. He is the author of the study.

The health of humans can be sustained for about a year in space, says NASA. When trying to answer this, researchers from Stanford University have taken a look at the cardiac function and at the gene expression in the human heart cells from three people. The cells did not come from biopsies, but they were made by reprogramming a sample of blood into the human stem cells. Then, the heart cells were cultured abroad the International Space Station for around 5 weeks. This is the first study of this kind.

Scientists found that the exposure to microgravity changed the expression of 2.635 genes, which was a temporary change in the RNA, that is made from DNA. Most of them returned to the normal patterns of gene expression in about 10 days after coming back to Earth. RNA is a temporary and handwritten copy of the DNA. So the gene expression was temporarily changed by the environment microgravity. The changes were subtle, but they were still significant.

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Going into Space Changes the Human Heart Cells, but What Happens When They Get Back on Earth? - Henri Le Chat Noir

Cardiac Stem Cells Comments Off on Going into Space Changes the Human Heart Cells, but What Happens When They Get Back on Earth? – Henri Le Chat Noir | November 10th, 2019

Science Foundation Ireland 2019 Science Awards recognise key leaders in the Irish Research Community

Associate Professor Dr Eilish McLoughlin has been honoured with a prestigious 2019 Science Foundation (SFI) award announced today at the annual SFI Science Summit in Athlone attended by over 300 leading members of Irelands research community who gathered to celebrate the significant contributions made over the past year to Science, Technology, Engineering and Maths (STEM) in Ireland.

Dr McLoughlin was presented with the SFI Outstanding Contribution to STEM Communication in recognition of her incredible contribution to the popularisation of science and her sterling efforts in raising public awareness of the value of science to human progress.

Dr McLoughlin is Director of the Research Centre for the Advancement of STEM Teaching and learning (CASTeL) at DCU. She obtained her BSc in Applied Physics and PhD in Surface Physics from DCU.

A firm believer in the mantra that science is for all, she has led several large-scale national initiatives to widen participation in STEM including Physics Busking, Science on Stage, Improving Gender Balance (all 3 have been funded by SFI) and the STEM Teacher Internship.

Her significant contributions to STEM engagement have resulted in many awards, especially the prestigious Institute of Physics Lise Meitner Medal in 2018 and the DCU President's Award for Engagement in 2017.

She has led several EU collaborations in STEM Education including coordinator of ESTABLISH, Co-Coordinator SAILS and is currently National Coordinator of the H2020 Open Schools for Open Societies project.

Speaking about her award Dr McLoughlin said:

I would like to thank Science Foundation Ireland for presenting me with this award.

I really appreciate their on-going support for STEM education and public engagement activities that allow me to engage with members of the public and teachers and students in schools across Ireland and share my passion for physics.

I hope that my interaction with young people and their parents will encourage more students to choose physics and follow a career in STEM.

Young girls need role models to encourage them to follow their interests and achieve their potential in physics. I hope by winning this award, more young people will realise that physics is a rewarding pathway to follow.

Dr McLoughlin was among ten award winners including a new award for Mentorship which was introduced to celebrate the important role mentors play in providing guidance, motivation and emotional support in our research system.

Acknowledging the award winners, Minister for Training, Skills, Innovation and Research and Development, John Halligan TD, said:

The Science Foundation Ireland Awards recognise the breadth and depth that research encompasses from industry collaborations to public engagement and the innovative breakthroughs that are leading research globally in the areas of Immunology, Biomaterials, Cancer research and much more.

I would like to congratulate each awardee on their achievements, which illustrate the invaluable knowledge and resource that Irelands research community offers.

I am also pleased to see mentorship amongst the awards this year, highlighting the importance of supporting the next generation of researchers and enriching our growing research community.

Professor Mark Ferguson, Director General of Science Foundation Ireland and Chief Scientific Adviser to the Government of Ireland, also congratulated the award winners, saying:

On behalf of Science Foundation Ireland, I would like to congratulate the award winners on their success and recognise their dedication in realising their ambitions and in doing so, building Irelands reputation as a global research leader.

We are very proud of the excellent quality of research that our funding enables, and the SFI Awards are an important acknowledgement of the collective achievements of the Irish research community, which continue to be impactful, inspirational and world-leading.

The 2019 Recipients are as follows:

SFI Researcher of the Year 2019

The SFI Researcher of the Year Award recognises the accomplishments of an SFI funded researcher who has contributed significantly to the Irish research community in the year of the award and/or throughout their career.

The successful researcher has achieved exceptional scientific and engineering research outputs combined with a clear demonstration of the ability to communicate their research.

Recipient: Professor Kevin OConnor, Director of the BEACON SFI Bioeconomy Research Centre, University College Dublin

Professor Kevin OConnor received his BSc degree and PhD from University College Cork.

He is a Professor of Microbial biotechnology in the School of Biomolecular and Biomedical Science at UCD and an investigator in the UCD Earth Institute.

As Director of the BEACON SFI Bioeconomy Research Centre, Professor OConnor is leading blue skies and industry focused research to build and support the development of Irelands bioeconomy.

He is shaping the European Bioeconomy Strategy through his chairmanship of the Scientific Committee for the Bio-based Industries Joint Undertaking (BBIJU), a 3.7 billion Public-Private Partnership.

His research work is seminal in the area of circular economy (plastics to biodegradable plastics), circular bioeconomy (dairy processing by-product to value-added chemical) and biotechnology (hydroxytyrosol production by a biocatalyst).

Collaborating with industry, Professor OConnor developed technology to convert a dairy by-product into an organic acid, which was patented and licensed to industry.

It is now being scaled and implemented in a world first second generation dairy biorefinery, which has received over 30 million in EU funding.

He has published extensively and patented technologies on the conversion of waste plastics to biodegradable plastic and the biotechnological production of hydroxytyrosol (a health promoting molecule) and founded two spin-out companies Bioplastech and Nova Mentis.

Commenting on receiving the Award Professor Kevin OConnor stated:

I am delighted and honoured to receive this prestigious SFI award.

It is a recognition of the dedication of the many researchers and industry partners with whom I work and collaborate with, across multiple scientific fields and sectors, at UCD, across Ireland and internationally.

Through these collaborations we are creating knowledge and translating this knowledge into innovative technological solutions to address global and societal bioeconomy challenges.

I would especially like to acknowledge and thank SFI for their funding, and UCD, BEACON centre members and my wife and family for all their support.

SFI Early Career Researcher of the Year

The SFI Early Career Researcher Award recognises outstanding early career research talent and in recognition of the high calibre of nominations in 2019, there are two individual recipients of the Early Career Researcher of the Year Award:

Recipient: Associate Professor Lydia Lynch, Trinity College Dublin

An Associate Professor at Trinity College Dublin (TCD), in the School of Biochemistry and Immunology, Dr Lydia Lynch established and runs the Lynch Laboratory.

She graduated from University College Dublin with a BSc in Cell Biology and Genetics and a PhD in Immunology and went onto receive a Newman Fellowship for her early post-doctoral studies in St. Vincents University Hospital, where she helped establish the Immunology and Obesity Lab.

Here she discovered adipose iNKT cells and demonstrated that their activation could help manage obesity and metabolic disease.

Dr Lynch is also the recipient of the prestigious LOreal-UNESCO International Women in Science Award and a Marie Curie International Fellowship, which allowed her to move to Harvard Medical School in 2013 and continue studying immunometabolism.

Whilst at Harvard, she was a recipient of the inaugural Innovation Evergreen Fund award. She is also the holder of an American Diabetes Association Award and a Cancer Research Institute Award as well as a European Research Council (ERC) Starting grant and SFI President of Ireland Future Research Leader Award and currently leads an international team in immunometabolism at TCD.

Recipient: Dr Orla OSullivan, APC Microbiome Ireland SFI Research Centre and Vistamilk SFI Research Centre, Teagasc

Dr Orla OSullivan completed her degree in Biochemistry and PhD in Bioinformatics in UCC. She went on to complete a postdoctoral fellowship at the Conway Institute UCD and then joined Teagasc, where she focuses on profiling the microbiome and where she has worked on the ELDERMET project amongst many others.

Dr OSullivan is a funded investigator within the APC Microbiome Ireland SFI Research Centre and Vistamilk SFI Research Centre.

In 2014, Dr OSullivan was awarded an SFI Starting Investigator Research Grant to allow her to establish herself as an independent scientist.

In the same year she was awarded the APC Junior Scientist of the Year.

She is committed to communicating science to all and actively participates in a number of outreach programmes such as BIG STEM communicators, BT Young Scientist, Fota Mad Scientist and World Microbiome Day.

Her research focuses on the microbiome and her studies have established that healthy and protein-rich athlete diets result in a more diverse gut microbiota than standard diets.

She aims to utilise outputs from this research to holistically manage chronic illnesses associated with the gut microbiome, thereby addressing a number of critical societal health challenges.

In 2018, Dr OSullivan was named by Clarivate Analytics as a Highly Cited Researcher placing her in the top 1% of researchers worldwide.

SFI Industry Partnership Award

The SFI Industry Partnership Award celebrates a collaboration between an SFI-funded academic research group and industry.

Recipient: Professor Danny Kelly, AMBER SFI Research Centre for Advanced Materials and BioEngineering Research, Trinity College Dublin, for collaboration with Johnson & Johnson Services, Inc.

Professor Danny Kelly is a Professor of Biomedical Engineering and is Director of the Trinity Centre for Biomedical Engineering where he leads a large multidisciplinary orthopaedic tissue engineering group.

He holds the Chair of Tissue Engineering at TCD and has received three prestigious European Research Council (ERC) awards. Professor Kelly is at the forefront of tissue regeneration using 3D bioprinting strategies.

Through his position at AMBER he has led the Johnson & Johnson partnership on the TRANSITION programme, funded under SFIs Spokes programme to develop a new class of 3D-printed biological implants that will regenerate, rather than replace, diseased joints.

TRANSITION is a shared vision and expands upon AMBERs long-standing collaboration with DePuy Ireland Unlimited Company.

TRANSITION, led by Professor Danny Kelly, brings together Principal Investigators and researchers from four AMBER partners (DCU, RCSI, TCD & UCD) and scientists and engineers from Johnson & Johnsons 3D Printing Centre of Excellence and DePuy Synthes.

A significant milestone was realised earlier this year with the establishment of the Collaborative Bioprinting Laboratory in TCDs Trinity Biomedical Sciences Institute, which co-locates researchers from both sides of the partnership.

SFI Best International Engagement Award

This award recognises the accomplishments of a Science Foundation Ireland-funded researcher/group specifically in the context of their international activities.

Recipient: Professor Abhay Pandit, Scientific Director, CRAM SFI Research Centre for Medical Devices, NUI Galway

Professor Abhay Pandit is Professor of Biomaterials at NUI Galway and Scientific Director of CRAM SFI Research Centre for Medical Devices.

Professor Pandit has been an elected member on the Council for both the Tissue Engineering and Regenerative Medicine International Society and European Society for Biomaterials Society.

He was the first Irish academic to be inducted as an International Fellow in Biomaterials Science and Engineering by the International Union of Societies for Biomaterials Science and Engineering and elected as a Fellow of the Tissue Engineering and Regenerative International Society.

He was also elected to the American Institute of Medical and Biological Engineering (AIMBE) College of Fellows.

Professor Pandit has published more than 250 papers in peer-reviewed journals, filed numerous patent applications and has licensed four technologies to medical device companies.

He has coordinated four EU grants to date and has generated research contracts from industry and government funding agencies totalling 90 million.

Throughout his career, his work has been outward facing, from engaging in international collaborations and hosting international conferences, to supporting trade missions and championing residency programs for leaders in the community (artists, filmmakers, teachers) to empower them with the STEM message.

SFI Entrepreneurship Award

The SFI Entrepreneurship Award celebrates an entrepreneurial achievement by SFI supported researchers.

Recipient: Professor William Gallagher, University College Dublin

Professor William Gallagher is Director of the UCD Conway Institute of Biomolecular and Biomedical Research and Professor of Cancer Biology in the UCD School of Biomolecular & Biomedical Science at University College Dublin.

He was also the Director of the first Irish Cancer Society Collaborative Cancer Research Centre, BREAST-PREDICT, which completed its ground-breaking six year programme in September 2019. Professor Gallagher co-founded the molecular diagnostics company OncoMark in 2007 and is currently its Chief Scientific Officer.

OncoMark focuses on the development and application of biomarker panels which address critical unmet needs for cancer patients.

A major focus of Professor Gallaghers research work is the identification and validation of candidate biomarkers of breast and other cancers, particularly those which guide treatment decision making.

He has received a number of awards to date, including the BACR/AstraZeneca Young Scientist Frank Rose Award in 2004, the St. Luke's Silver Medal Award in 2008, the NovaUCD Innovation Award in 2011 and the inaugural IACR Award for 'Outstanding Contribution to Cancer Medicine and Research' in 2017.

Professor Gallagher has led multiple EU networks under EU programmes, he has had many collaborations with a variety of industrial partners throughout his research, and has filed multiple patents.

SFI Outstanding Contribution to STEM Communication (There are two recipients of this award, including Dr McLoughlin)

Recipient: Dr Muriel Grenon, NUI Galway

Dr Muriel Grenon is a lecturer in Biochemistry, School of Natural Sciences, NUI Galway and the founding Director of the Cell EXPLORERS science outreach programme.

Dr Grenon started out the programme in 2012 with a team of 10 undergraduate science students in NUI Galway and has built Cell EXPLORERS into a national network comprising 13 partner teams with members from 15 Higher Education Institutions in Ireland.

Between 2012 and 2018 Cell EXPLORERS involved 1,187 team members, visited 471 classrooms in 280 schools and reached 32,000 members of the public.

Cell EXPLORERS has also successfully integrated science outreach projects into the final year of the Biochemistry undergraduate course at NUI Galway allowing the creation of potential novel science outreach resources each semester.

Dr Grenon is also involved in driving science communication internationally: Cell EXPLORERS is part of Scientix, the community for Science Education in Europe.

The programme has also started a collaboration with the University of Kwatzulu-Natal in South Africa, where a team is currently piloting the Fantastic DNA school visits.

Dr Grenons contribution and dedication to the popularisation of STEM has been recognised by the Outstanding Contribution to STEM award at the 2013 Galway Science and Technology Festival, the 2017 NUI Galway President Award for Societal Impact and being made Knight of the Order of the Palmes Acadmiques by the French Ministry of Education in 2019.

SFI Mentorship Award

This inaugural award recognises outstanding mentorship provided by a researcher funded by Science Foundation Ireland.

Recipient: Dr Fatima Gunning, IPIC SFI Research Centre and Tyndall National Institute

Dr Fatima Gunning completed her BSc in Physics and PhD in Optoelectronics from Pontifcia Universidade Catlica do Rio de Janeiro (PUC-Rio), Brazil before joining IPIC SFI Research Centre, hosted by Tyndall National Institute after a brief two year period at Corning.

Currently serving as Head of Graduate Studies at Tyndall National Institute and a PI at IPIC SFI Research Centre, she is looking at novel photonics technologies for the Internet of the future.

She has also led many diversity and inclusion programmes that are directly targeted at improving the deficit of diverse talent and gender balance in the field including Empowering Women@Tyndall and being a key advocate for Tyndall to apply for Athena SWAN by 2020.

Dr Gunning has been selected to become Vice President of Membership and Outreach of the IEEE Photonics Society starting January 2020 to expand the diversity, inclusion and mentorship efforts to an international scale.

Dr Gunning believes that all students are different, are driven by different motivations and develop their research in different ways.

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Dr Eilish McLoughlin honoured by SFI for Outstanding Contribution to STEM Communication - Dublin City University

Cardiac Stem Cells Comments Off on Dr Eilish McLoughlin honoured by SFI for Outstanding Contribution to STEM Communication – Dublin City University | November 9th, 2019

The thought of spaceflight may make the heart skip a beat, but actually travelling beyond Earth could alter the organs cells.

With extended stays aboard the International Space Station (ISS) commonplace, and the likelihood of humans spending longer periods in space increasing, there is a need to better understand the effects of micro-gravity on cardiac function.

New research suggests heart muscle cells derived from stem cells have a remarkable ability to adapt to their environment during and after spaceflight.

Scientists examined cell-level cardiac function and gene expression in human heart cells cultured aboard the International Space Station for five-and-a-half weeks.They found that exposure to micro-gravity changed the expression of thousands of genes, but largely normal patterns reappeared within 10 days after returning to Earth.

Read more about the body in space:

Senior study author, Joseph Wu, of Stanford University School of Medicine, said: Our study is novel because it is the first to use human induced pluripotent stem cells to study the effects of spaceflight on human heart function.

Micro-gravity is an environment that is not very well understood, in terms of its overall effect on the human body, and studies like this could help shed light on how the cells of the body behave in space, especially as the world embarks on more and longer space missions such as going to the Moon and Mars.

Until now, most studies on how the heart reacts to micro-gravity have been conducted in either non-human models or at tissue, organ or systemic level.To address this, the beating cells were launched to the ISS aboard a SpaceX spacecraft as part of a commercial resupply service mission.Simultaneously, they were also cultured on Earth for comparison purposes.

When they returned to the planet, the cells showed normal structure and morphology.However, they did adapt by modifying their beating pattern and calcium recycling patterns.

Immunofluorescence imaging of the cells grown in micro-gravity aboard the International Space Station Joseph Wu lab, Stanford University School of Medicine/PA

Researchers sequenced the cells harvested at four-and-a-half weeks aboard the ISS, and 10 days after returning to Earth.Results showed that 2,635 genes were differentially expressed among flight, post-flight, and ground control samples.

Most notably, gene pathways related to mitochondrial function were expressed more in the space-flown cells, according to the research published in the Stem Cells Reports journal.

A comparison of the samples revealed the space cells adopted a unique gene expression pattern during spaceflight, which reverted to one that is similar to ground-side controls upon return to normal gravity.

Dr Wu added: Were surprised about how quickly human heart muscle cells are able to adapt to the environment in which they are placed, including micro-gravity.

These studies may provide insight into cellular mechanisms that could benefit astronaut health during long-duration spaceflight, or potentially lay the foundation for new insights into improving heart health on Earth.

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Space travel affects heart cells, but only temporarily - BBC Focus Magazine

Cardiac Stem Cells Comments Off on Space travel affects heart cells, but only temporarily – BBC Focus Magazine | November 9th, 2019

JERUSALEM & PARSIPPANY, N.J. & INCHEON, South Korea--(BUSINESS WIRE)--Teva Pharmaceuticals USA, Inc., a U.S. affiliate of Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA), Celltrion, Inc., (KRX KRX:068270) and Celltrion Healthcare, Co., Ltd. (KRX KOSDAQ:091990), today announced that TRUXIMA (rituximab-abbs) injection is the first biosimilar to the reference product Rituxan1 (rituximab) now available in the United States with a full oncology label. TRUXIMA is currently indicated for the treatment of adult patients with non-Hodgkins Lymphoma (NHL) and Chronic Lymphocytic Leukemia (CLL):

We are excited about the first FDA-approved biosimilar to rituximab in the U.S., stated Brendan OGrady, Executive Vice President and Head of North America Commercial at Teva. Tevas commitment to biosimilars is focused on the potential to create lower healthcare costs and increased price competition. This focus is consistent with Tevas mission of making accessible medications to help improve the lives of patients.

TRUXIMA was approved by the U.S. Food and Drug Administration (FDA) as the first rituximab biosimilar. The approval was based on a review of a comprehensive data package inclusive of foundational and extensive analytical characterization, nonclinical data, clinical pharmacology, immunogenicity, clinical efficacy, and safety data. In May 2019, the FDA approved TRUXIMA to match all of the reference products oncology indications for NHL and CLL. In light of a patent settlement with Genentech, Celltrion and Teva have a pending FDA submission for rheumatoid arthritis (RA), granulomatosis with polyangiitis (GPA), and microscopic polyangiitis (MPA), and a license from Genentech to expand the TRUXIMA label to include these indications in Q2 2020.

We are pleased to announce the launch of the first rituximab biosimilar, TRUXIMA, with our marketing partner Teva in the U.S. said Mr. Hyoung-Ki Kim, Vice Chairman at Celltrion Healthcare. We believe that the introduction of TRUXIMA into the U.S. market will contribute to addressing unmet needs of U.S. patients as well.

The Wholesale Acquisition Cost (WAC or list price) for TRUXIMA will be 10 percent lower than the reference product. TRUXIMA is being made available through primary wholesalers at a WAC of $845.55 for 100mg vial and $4227.75 for 500mg vial. Actual costs to individual patients and providers for TRUXIMA are anticipated to be lower than WAC because WAC does not account for additional rebates and discounts that may apply. Savings on out-of-pocket costs may vary depending on the patients insurance payer and eligibility for participation in the assistance program.

Dedicated patient support services are also available from Teva through the Comprehensive Oncology Reimbursement Expertise (CORE) program. CORE is available to help eligible patients, caregivers and healthcare professionals navigate the reimbursement process. CORE offers a range of services, including benefits verification and coverage determination, support for precertification and prior authorization, assistance with coverage guidelines and claims investigation, and support through the claims and appeals process. A savings program is also available for eligible commercially insured patients. To learn more, please visit TevaCORE.com. For healthcare professionals seeking additional information, there is also a dedicated site at TRUXIMAhcp.com.

Celltrion and Teva Pharmaceutical Industries Ltd. entered into an exclusive partnership in October 2016 to commercialize TRUXIMA in the U.S. and Canada.

Please see the Important Safety Information below including the Boxed Warning regarding fatal infusion-related reactions, severe mucocutaneous reactions, hepatitis B virus reactivation and progressive multifocal leukoencephalopathy. For more information, please visit the full prescribing information.

Important Safety Information

WARNING: FATAL INFUSION-RELATED REACTIONS, SEVERE MUCOCUTANEOUS REACTIONS, HEPATITIS B VIRUS REACTIVATION and PROGRESSIVE MULTIFOCAL LEUKOENCEPHALOPATHY

Infusion-Related Reactions - Administration of rituximab products, including TRUXIMA, can result in serious, including fatal, infusion-related reactions. Deaths within 24 hours of rituximab infusion have occurred. Approximately 80% of fatal infusion-related reactions occurred in association with the first infusion. Monitor patients closely. Discontinue TRUXIMA infusion for severe reactions and provide medical treatment for Grade 3 or 4 infusion-related reactions

Severe Mucocutaneous Reactions - Severe, including fatal, mucocutaneous reactions can occur in patients receiving rituximab products

Hepatitis B Virus (HBV) Reactivation - HBV reactivation can occur in patients treated with rituximab products, in some cases resulting in fulminant hepatitis, hepatic failure, and death. Screen all patients for HBV infection before treatment initiation, and monitor patients during and after treatment with TRUXIMA. Discontinue TRUXIMA and concomitant medications in the event of HBV reactivation

Progressive Multifocal Leukoencephalopathy (PML), including fatal PML, can occur in patients receiving rituximab products

Warnings and Precautions

Infusion-Related Reactions - Rituximab products can cause severe, including fatal, infusion-related reactions. Severe reactions typically occurred during the first infusion with time to onset of 30-120 minutes. Rituximab product-induced infusion-related reactions and sequelae include urticaria, hypotension, angioedema, hypoxia, bronchospasm, pulmonary infiltrates, acute respiratory distress syndrome, myocardial infarction, ventricular fibrillation, cardiogenic shock, anaphylactoid events, or death.

Premedicate patients with an antihistamine and acetaminophen prior to dosing. Institute medical management (e.g. glucocorticoids, epinephrine, bronchodilators, or oxygen) for infusion-related reactions as needed. Depending on the severity of the infusion-related reaction and the required interventions, temporarily or permanently discontinue TRUXIMA. Resume infusion at a minimum 50% reduction in rate after symptoms have resolved. Closely monitor the following patients: those with pre-existing cardiac or pulmonary conditions, those who experienced prior cardiopulmonary adverse reactions, and those with high numbers of circulating malignant cells (>25,000/mm3)

Severe Mucocutaneous Reactions - Mucocutaneous reactions, some with fatal outcome, can occur in patients treated with rituximab products. These reactions include paraneoplastic pemphigus, Stevens-Johnson syndrome, lichenoid dermatitis, vesiculobullous dermatitis, and toxic epidermal necrolysis. The onset of these reactions has been variable and includes reports with onset on the first day of rituximab exposure. Discontinue TRUXIMA in patients who experience a severe mucocutaneous reaction. The safety of re-administration of rituximab products to patients with severe mucocutaneous reactions has not been determined.

Hepatitis B Virus Reactivation - Hepatitis B virus (HBV) reactivation, in some cases resulting in fulminant hepatitis, hepatic failure and death, can occur in patients treated with drugs classified as CD20-directed cytolytic antibodies, including rituximab products. Cases have been reported in patients who are hepatitis B surface antigen (HBsAg) positive and also in patients who are HBsAg negative but are hepatitis B core antibody (anti-HBc) positive. Reactivation also has occurred in patients who appear to have resolved hepatitis B infection (i.e., HBsAg negative, anti-HBc positive and hepatitis B surface antibody [anti-HBs] positive).

HBV reactivation is defined as an abrupt increase in HBV replication manifesting as a rapid increase in serum HBV DNA levels or detection of HBsAg in a person who was previously HBsAg negative and anti-HBc positive. Reactivation of HBV replication is often followed by hepatitis, i.e., increase in transaminase levels. In severe cases increase in bilirubin levels, liver failure, and death can occur.

Screen all patients for HBV infection by measuring HBsAg and anti-HBc before initiating treatment with TRUXIMA. For patients who show evidence of prior hepatitis B infection (HBsAg positive [regardless of antibody status] or HBsAg negative but anti-HBc positive), consult with physicians with expertise in managing hepatitis B regarding monitoring and consideration for HBV antiviral therapy before and/or during TRUXIMA treatment.

Monitor patients with evidence of current or prior HBV infection for clinical and laboratory signs of hepatitis or HBV reactivation during and for several months following TRUXIMA therapy. HBV reactivation has been reported up to 24 months following completion of rituximab therapy.

In patients who develop reactivation of HBV while on TRUXIMA, immediately discontinue TRUXIMA and any concomitant chemotherapy, and institute appropriate treatment. Insufficient data exist regarding the safety of resuming TRUXIMA treatment in patients who develop HBV reactivation. Resumption of TRUXIMA treatment in patients whose HBV reactivation resolves should be discussed with physicians with expertise in managing HBV.

Progressive Multifocal Leukoencephalopathy (PML) - JC virus infection resulting in PML and death can occur in rituximab product-treated patients with hematologic malignancies. The majority of patients with hematologic malignancies diagnosed with PML received rituximab in combination with chemotherapy or as part of a hematopoietic stem cell transplant. Most cases of PML were diagnosed within 12 months of their last infusion of rituximab.

Consider the diagnosis of PML in any patient presenting with new-onset neurologic manifestations. Evaluation of PML includes, but is not limited to, consultation with a neurologist, brain MRI, and lumbar puncture.

Discontinue TRUXIMA and consider discontinuation or reduction of any concomitant chemotherapy or immunosuppressive therapy in patients who develop PML.

Tumor Lysis Syndrome (TLS) - Acute renal failure, hyperkalemia, hypocalcemia, hyperuricemia, or hyperphosphatemia from tumor lysis, sometimes fatal, can occur within 12-24 hours after the first infusion of rituximab products in patients with NHL. A high number of circulating malignant cells (>25,000/mm3) or high tumor burden, confers a greater risk of TLS.

Administer aggressive intravenous hydration and anti-hyperuricemic therapy in patients at high risk for TLS. Correct electrolyte abnormalities, monitor renal function and fluid balance, and administer supportive care, including dialysis as indicated.

Infections - Serious, including fatal, bacterial, fungal, and new or reactivated viral infections can occur during and following the completion of rituximab product-based therapy. Infections have been reported in some patients with prolonged hypogammaglobulinemia (defined as hypogammaglobulinemia >11 months after rituximab exposure). New or reactivated viral infections included cytomegalovirus, herpes simplex virus, parvovirus B19, varicella zoster virus, West Nile virus, and hepatitis B and C. Discontinue TRUXIMA for serious infections and institute appropriate anti-infective therapy. TRUXIMA is not recommended for use in patients with severe, active infections.

Cardiovascular Adverse Reactions - Cardiac adverse reactions, including ventricular fibrillation, myocardial infarction, and cardiogenic shock may occur in patients receiving rituximab products. Discontinue infusions for serious or life-threatening cardiac arrhythmias. Perform cardiac monitoring during and after all infusions of TRUXIMA for patients who develop clinically significant arrhythmias, or who have a history of arrhythmia or angina.

Renal Toxicity - Severe, including fatal, renal toxicity can occur after rituximab product administration in patients with NHL. Renal toxicity has occurred in patients who experience tumor lysis syndrome and in patients with NHL administered concomitant cisplatin therapy during clinical trials. The combination of cisplatin and TRUXIMA is not an approved treatment regimen. Monitor closely for signs of renal failure and discontinue TRUXIMA in patients with a rising serum creatinine or oliguria.

Bowel Obstruction and Perforation - Abdominal pain, bowel obstruction and perforation, in some cases leading to death, can occur in patients receiving rituximab in combination with chemotherapy. In postmarketing reports, the mean time to documented gastrointestinal perforation was 6 (range 1-77) days in patients with NHL. Evaluate if symptoms of obstruction such as abdominal pain or repeated vomiting occur.

Immunization - The safety of immunization with live viral vaccines following rituximab product therapy has not been studied and vaccination with live virus vaccines is not recommended before or during treatment.

Embryo-Fetal Toxicity - Based on human data, rituximab products can cause fetal harm due to B-cell lymphocytopenia in infants exposed to rituximab in-utero. Advise pregnant women of the risk to a fetus. Females of childbearing potential should use effective contraception while receiving TRUXIMA and for 12 months following the last dose of TRUXIMA.

Most common adverse reactions in clinical trials of NHL (>25%) were: infusion-related reactions, fever, lymphopenia, chills, infection, and asthenia

Most common adverse reactions in clinical trials of CLL (>25%) were: infusion-related reactions and neutropenia

Nursing Mothers - There are no data on the presence of rituximab in human milk, the effect on the breastfed child, or the effect on milk production. Since many drugs including antibodies are present in human milk, advise a lactating woman not to breastfeed during treatment and for at least 6 months after the last dose of TRUXIMA due to the potential for serious adverse reactions in breastfed infants.

About TRUXIMA

TRUXIMA (rituximab-abbs) is a U.S. Food and Drug Administration (FDA)-approved biosimilar to RITUXAN (rituximab) for the treatment of adult patients with CD20-positive, B-cell NHL to be used as a single agent or in combination with chemotherapy or CLL in combination with fludarabine and cyclophosphamide (FC).

TRUXIMA has the same mechanism of action as Rituxan and has demonstrated biosimilarity to Rituxan through a totality of evidence.

About Celltrion Healthcare, Co. Ltd.

Celltrion Healthcare conducts the worldwide marketing, sales and distribution of biological medicines developed by Celltrion, Inc. through an extensive global network that spans more than 120 different countries. Celltrion Healthcares products are manufactured at state-of-the-art mammalian cell culture facilities, designed and built to comply with the US Food and Drug Administration (FDA) cGMP guidelines and the EU GMP guidelines.

About Celltrion, Inc.

Headquartered in Incheon, Korea, Celltrion is a leading biopharmaceutical company, specializing in research, development and manufacturing of biosimilar and innovative drugs. Celltrion strives to provide more affordable biosimilar mAbs to patients who previously had limited access to advanced therapeutics. Celltrion received FDA approval for TRUXIMA (rituximab-abbs) and HERZUMA (trastuzumab-pkrb) in 2018.

About Teva

Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA) has been developing and producing medicines to improve peoples lives for more than a century. We are a global leader in generic and specialty medicines with a portfolio consisting of over 3,500 products in nearly every therapeutic area. Around 200 million people around the world take a Teva medicine every day, and are served by one of the largest and most complex supply chains in the pharmaceutical industry. Along with our established presence in generics, we have significant innovative research and operations supporting our growing portfolio of specialty and biopharmaceutical products. Learn more at http://www.tevapharm.com.

Teva's Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 regarding TRUXIMA, which are based on managements current beliefs and expectations and are subject to substantial risks and uncertainties, both known and unknown, that could cause our future results, performance or achievements to differ significantly from that expressed or implied by such forward-looking statements. Important factors that could cause or contribute to such differences include risks relating to:

and other factors discussed in our Quarterly Reports on Form 10-Q for the first and second quarter of 2019 and in our Annual Report on Form 10-K for the year ended December 31, 2018, including in the sections captioned "Risk Factors and Forward Looking Statements. Forward-looking statements speak only as of the date on which they are made, and we assume no obligation to update or revise any forward-looking statements or other information contained herein, whether as a result of new information, future events or otherwise. You are cautioned not to put undue reliance on these forward-looking statements.

1 RITUXAN is a registered trademark of Genentech and Biogen.

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Teva and Celltrion Announce the Availability of TRUXIMA (rituximab-abbs) Injection, the First Biosimilar to Rituxan (rituximab) in the United States -...

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CAMBRIDGE, Mass. & OSAKA, Japan--(BUSINESS WIRE)--Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE:TAK) today announced that it will present a total of 29 company-sponsored abstracts at the 61st American Society of Hematology (ASH) Annual Meeting on December 7-10, 2019 in Orlando, FL, highlighting the companys commitment to advancing the treatment of hematologic cancers and bleeding disorders.

Pursuing Breakthrough, Patient-Centric Innovation in Oncology and Bleeding Disorders

Takeda will present 29 scientific updates on the companys investigational and early-stage therapies, which demonstrates its investment in new compounds to address patient needs, as well as data from Phase 3 trials and real-world evidence findings, in disease states including multiple myeloma, lymphoma and leukemia.

We are presenting notable data on several clinical programs at ASH, highlighting our deep oncology pipeline and our commitment to developing innovative therapies that may address unmet needs for blood cancer patients, said Phil Rowlands, Ph.D., Head, Oncology Therapeutic Area Unit, Takeda. In particular we look forward to sharing data from the Phase 3 clinical trial of ixazomib in amyloidosis patients, data from the US MM-6 study, which evaluates an in-class transition from parenteral bortezomib to oral ixazomib in multiple myeloma, further analyses from the Phase 3 ECHELON-2 trial of ADCETRIS in peripheral T-cell lymphoma, as well as early stage data from several of our pipeline programs.

In hematology, Takeda will present real-world evidence from studies of its portfolio of treatments across bleeding disorders, including hemophilia A, hemophilia B and von Willebrand disease. The company will also present scientific updates related to its hemophilia A and hemophilia B gene therapy programs and adeno-associated virus (AAV) gene therapy platform.

Understanding real-world evidence is critical as Takeda continues to provide patients with innovative therapies for hemophilia A and hemophilia B while broadening our research and development efforts in von Willebrand disease and other bleeding disorders, said Daniel Curran, M.D., Head, Rare Diseases Therapeutic Area Unit, Takeda. Also at ASH, we look forward to providing an update on our gene therapy programs in hemophilia and the optimization of Takedas AAV gene therapy platform, particularly for patients with pre-existing immunity to AAV serotypes.

Accepted oncology abstracts include:

Note: all times listed are in Eastern Standard Time

NINLARO (ixazomib) and Multiple Myeloma

ADCETRIS (brentuximab vedotin) and Lymphoma

ICLUSIG (ponatinib)

Pipeline (multiple myeloma, lymphoma, chronic lymphocytic leukemia, acute myeloid leukemia)

Accepted hematology abstracts include:

Note: all times listed are in Eastern Standard Time

ADYNOVATE (Antihemophilic Factor (Recombinant), PEGylated) and Hemophilia A

FEIBA (Anti-Inhibitor Coagulant Complex)

von Willebrand Disease

Pipeline (hemophilia A, hemophilia B and gene therapies)

About ADCETRISADCETRIS is an antibody-drug conjugate (ADC) comprising an anti-CD30 monoclonal antibody attached by a protease-cleavable linker to a microtubule disrupting agent, monomethyl auristatin E (MMAE), utilizing Seattle Genetics' proprietary technology. The ADC employs a linker system that is designed to be stable in the bloodstream but to release MMAE upon internalization into CD30-positive tumor cells.

ADCETRIS injection for intravenous infusion has received FDA approval for six indications in adult patients with: (1) previously untreated systemic anaplastic large cell lymphoma (sALCL) or other CD30-expressing peripheral T-cell lymphomas (PTCL), including angioimmunoblastic T-cell lymphoma and PTCL not otherwise specified, in combination with cyclophosphamide, doxorubicin, and prednisone, (2) previously untreated Stage III or IV classical Hodgkin lymphoma (cHL), in combination with doxorubicin, vinblastine, and dacarbazine, (3) cHL at high risk of relapse or progression as post-autologous hematopoietic stem cell transplantation (auto-HSCT) consolidation, (4) cHL after failure of auto-HSCT or failure of at least two prior multi-agent chemotherapy regimens in patients who are not auto-HSCT candidates, (5) sALCL after failure of at least one prior multi-agent chemotherapy regimen, and (6) primary cutaneous anaplastic large cell lymphoma (pcALCL) or CD30-expressing mycosis fungoides (MF) who have received prior systemic therapy.

Health Canada granted ADCETRIS approval with conditions for relapsed or refractory Hodgkin lymphoma and sALCL in 2013, and non-conditional approval for post-autologous stem cell transplantation (ASCT) consolidation treatment of Hodgkin lymphoma patients at increased risk of relapse or progression in 2017, adults with pcALCL or CD30-expressing MF who have had prior systemic therapy in 2018, and for previously untreated Stage IV Hodgkin lymphoma in combination with doxorubicin, vinblastine, and dacarbazine in 2019.

ADCETRIS received conditional marketing authorization from the European Commission in October 2012. The approved indications in Europe are: (1) for the treatment of adult patients with relapsed or refractory CD30-positive Hodgkin lymphoma following ASCT, or following at least two prior therapies when ASCT or multi-agent chemotherapy is not a treatment option, (2) for the treatment of adult patients with relapsed or refractory sALCL, (3) for the treatment of adult patients with CD30-positive Hodgkin lymphoma at increased risk of relapse or progression following ASCT, (4) for the treatment of adult patients with CD30-positive cutaneous T-cell lymphoma (CTCL) after at least one prior systemic therapy and (5) for the treatment of adult patients with previously untreated CD30-positive Stage IV Hodgkin lymphoma in combination with AVD.

ADCETRIS has received marketing authorization by regulatory authorities in more than 70 countries for relapsed or refractory Hodgkin lymphoma and sALCL. See important safety information below.

ADCETRIS is being evaluated broadly in more than 70 clinical trials, including a Phase 3 study in first-line Hodgkin lymphoma (ECHELON-1) and another Phase 3 study in first-line CD30-positive peripheral T-cell lymphomas (ECHELON-2), as well as trials in many additional types of CD30-positive malignancies.

Seattle Genetics and Takeda are jointly developing ADCETRIS. Under the terms of the collaboration agreement, Seattle Genetics has U.S. and Canadian commercialization rights and Takeda has rights to commercialize ADCETRIS in the rest of the world. Seattle Genetics and Takeda are funding joint development costs for ADCETRIS on a 50:50 basis, except in Japan where Takeda is solely responsible for development costs.

ADCETRIS (brentuximab vedotin) Important Safety Information (European Union)Please refer to Summary of Product Characteristics (SmPC) before prescribing.

CONTRAINDICATIONS

ADCETRIS is contraindicated for patients with hypersensitivity to brentuximab vedotin and its excipients. In addition, combined use of ADCETRIS with bleomycin causes pulmonary toxicity.

SPECIAL WARNINGS & PRECAUTIONS

Progressive multifocal leukoencephalopathy (PML): John Cunningham virus (JCV) reactivation resulting in progressive multifocal leukoencephalopathy (PML) and death can occur in patients treated with ADCETRIS. PML has been reported in patients who received ADCETRIS after receiving multiple prior chemotherapy regimens. PML is a rare demyelinating disease of the central nervous system that results from reactivation of latent JCV and is often fatal.

Closely monitor patients for new or worsening neurological, cognitive, or behavioral signs or symptoms, which may be suggestive of PML. Suggested evaluation of PML includes neurology consultation, gadolinium-enhanced magnetic resonance imaging of the brain, and cerebrospinal fluid analysis for JCV DNA by polymerase chain reaction or a brain biopsy with evidence of JCV. A negative JCV PCR does not exclude PML. Additional follow up and evaluation may be warranted if no alternative diagnosis can be established Hold dosing for any suspected case of PML and permanently discontinue ADCETRIS if a diagnosis of PML is confirmed.

Be alert to PML symptoms that the patient may not notice (e.g., cognitive, neurological, or psychiatric symptoms).

Pancreatitis: Acute pancreatitis has been observed in patients treated with ADCETRIS. Fatal outcomes have been reported. Closely monitor patients for new or worsening abdominal pain, which may be suggestive of acute pancreatitis. Patient evaluation may include physical examination, laboratory evaluation for serum amylase and serum lipase, and abdominal imaging, such as ultrasound and other appropriate diagnostic measures. Hold ADCETRIS for any suspected case of acute pancreatitis. ADCETRIS should be discontinued if a diagnosis of acute pancreatitis is confirmed.

Pulmonary Toxicity: Cases of pulmonary toxicity, some with fatal outcomes, including pneumonitis, interstitial lung disease, and acute respiratory distress syndrome (ARDS), have been reported in patients receiving ADCETRIS. Although a causal association with ADCETRIS has not been established, the risk of pulmonary toxicity cannot be ruled out. Promptly evaluate and treat new or worsening pulmonary symptoms (e.g., cough, dyspnoea) appropriately. Consider holding dosing during evaluation and until symptomatic improvement.

Serious infections and opportunistic infections: Serious infections such as pneumonia, staphylococcal bacteremia, sepsis/septic shock (including fatal outcomes), and herpes zoster, and opportunistic infections such as Pneumocystis jiroveci pneumonia and oral candidiasis have been reported in patients treated with ADCETRIS. Carefully monitor patients during treatment for emergence of possible serious and opportunistic infections.

Infusion-related reactions (IRR): Immediate and delayed IRR, as well as anaphylaxis, have been reported with ADCETRIS. Carefully monitor patients during and after an infusion. If anaphylaxis occurs, immediately and permanently discontinue administration of ADCETRIS and administer appropriate medical therapy. If an IRR occurs, interrupt the infusion and institute appropriate medical management. The infusion may be restarted at a slower rate after symptom resolution. Patients who have experienced a prior IRR should be premedicated for subsequent infusions. IRRs are more frequent and more severe in patients with antibodies to ADCETRIS.

Tumor lysis syndrome (TLS): TLS has been reported with ADCETRIS. Patients with rapidly proliferating tumor and high tumor burden are at risk of TLS. Monitor these patients closely and manage according to best medical practice.

Peripheral neuropathy (PN): ADCETRIS treatment may cause PN, both sensory and motor. ADCETRIS-induced PN is typically an effect of cumulative exposure to ADCETRIS and is reversible in most cases. Monitor patients for symptoms of neuropathy, such as hypoesthesia, hyperesthesia, paresthesia, discomfort, a burning sensation, neuropathic pain, or weakness. Patients experiencing new or worsening PN may require a delay and a dose reduction or discontinuation of ADCETRIS.

Hematological toxicities: Grade 3 or Grade 4 anemia, thrombocytopenia, and prolonged (equal to or greater than one week) Grade 3 or Grade 4 neutropenia can occur with ADCETRIS. Monitor complete blood counts prior to administration of each dose.

Febrile neutropenia: Febrile neutropenia has been reported with ADCETRIS. Complete blood counts should be monitored prior to administration of each dose of treatment. Closely monitor patients for fever and manage according to best medical practice if febrile neutropenia develops.

When ADCETRIS is administered in combination with AVD, primary prophylaxis with G-CSF is recommended for all patients beginning with the first dose.

Stevens-Johnson syndrome (SJS): SJS and toxic epidermal necrolysis (TEN) have been reported with ADCETRIS. Fatal outcomes have been reported. Discontinue treatment with ADCETRIS if SJS or TEN occurs and administer appropriate medical therapy.

Gastrointestinal (GI) Complications: GI complications, some with fatal outcomes, including intestinal obstruction, ileus, enterocolitis, neutropenic colitis, erosion, ulcer, perforation and haemorrhage, have been reported with ADCETRIS. Promptly evaluate and treat patients if new or worsening GI symptoms occur.

Hepatotoxicity: Elevations in alanine aminotransferase (ALT) and aspartate aminotransferase (AST) have been reported with ADCETRIS. Serious cases of hepatotoxicity, including fatal outcomes, have also occurred. Pre-existing liver disease, comorbidities, and concomitant medications may also increase the risk. Test liver function prior to treatment initiation and routinely monitor during treatment. Patients experiencing hepatotoxicity may require a delay, dose modification, or discontinuation of ADCETRIS.

Hyperglycemia: Hyperglycemia has been reported during trials in patients with an elevated body mass index (BMI) with or without a history of diabetes mellitus. Closely monitor serum glucose for patients who experiences an event of hyperglycemia. Administer anti-diabetic treatment as appropriate.

Renal and Hepatic Impairment: There is limited experience in patients with renal and hepatic impairment. Available data indicate that MMAE clearance might be affected by severe renal impairment, hepatic impairment, and by low serum albumin concentrations.

CD30+ CTCL: The size of the treatment effect in CD30 + CTCL subtypes other than mycosis fungoides (MF) and primary cutaneous anaplastic large cell lymphoma (pcALCL) is not clear due to lack of high level evidence. In two single arm phase II studies of ADCETRIS, disease activity has been shown in the subtypes Szary syndrome (SS), lymphomatoid papulosis (LyP) and mixed CTCL histology. These data suggest that efficacy and safety can be extrapolated to other CTCL CD30+ subtypes. Carefully consider the benefit-risk per patient and use with caution in other CD30+ CTCL patient types.

Sodium content in excipients: This medicinal product contains 13.2 mg sodium per vial, equivalent to 0.7% of the WHO recommended maximum daily intake of 2 g sodium for an adult.

INTERACTIONSPatients who are receiving a strong CYP3A4 and P-gp inhibitor, concomitantly with ADCETRIS may have an increased risk of neutropenia. If neutropenia develops, refer to dosing recommendations for neutropenia (see SmPC section 4.2). Co-administration of ADCETRIS with a CYP3A4 inducer did not alter the plasma exposure of ADCETRIS, but it appeared to reduce plasma concentrations of MMAE metabolites that could be assayed. ADCETRIS is not expected to alter the exposure to drugs that are metabolized by CYP3A4 enzymes.

PREGNANCY: Advise women of childbearing potential to use two methods of effective contraception during treatment with ADCETRIS and until 6 months after treatment. There are no data from the use of ADCETRIS in pregnant women, although studies in animals have shown reproductive toxicity. Do not use ADCETRIS during pregnancy unless the benefit to the mother outweighs the potential risks to the fetus.

LACTATION (breast-feeding): There are no data as to whether ADCETRIS or its metabolites are excreted in human milk, therefore a risk to the newborn/infant cannot be excluded. With the potential risk, a decision should be made whether to discontinue breast-feeding or discontinue/abstain from therapy with ADCETRIS.

FERTILITY: In nonclinical studies, ADCETRIS treatment has resulted in testicular toxicity, and may alter male fertility. Advise men being treated with ADCETRIS not to father a child during treatment and for up to 6 months following the last dose.

Effects on ability to drive and use machines: ADCETRIS may have a moderate influence on the ability to drive and use machines.

UNDESIRABLE EFFECTS

Monotherapy: The most frequent adverse reactions (10%) were infections, peripheral sensory neuropathy, nausea, fatigue, diarrhoea, pyrexia, upper respiratory tract infection, neutropenia, rash, cough, vomiting, arthralgia, peripheral motor neuropathy, infusion-related reactions, pruritus, constipation, dyspnoea, weight decreased, myalgia and abdominal pain. Serious adverse drug reactions occurred in 12% of patients. The frequency of unique serious adverse drug reactions was 1%. Adverse events led to treatment discontinuation in 24% of patients.

Combination Therapy: In the study of ADCETRIS as combination therapy with AVD in 662 patients with previously untreated advanced HL, the most common adverse reactions ( 10%) were: neutropenia, nausea, constipation, vomiting, fatigue, peripheral sensory neuropathy, diarrhoea, pyrexia, alopecia, peripheral motor neuropathy, decreased weight, abdominal pain, anaemia, stomatitis, febrile neutropenia, bone pain, insomnia, decreased appetite, cough, headache, arthralgia, back pain, dyspnoea, myalgia, upper respiratory tract infection, alanine aminotransferase increased. Serious adverse reactions occurred in 36% of patients. Serious adverse reactions occurring in 3% of patients included febrile neutropenia (17%), pyrexia (6%), and neutropenia (3%). Adverse events led to treatment discontinuation in 13% of patients.

ADCETRIS (brentuximab vedotin) Important Safety Information (U.S.)

BOXED WARNINGPROGRESSIVE MULTIFOCAL LEUKOENCEPHALOPATHY (PML): JC virus infection resulting in PML and death can occur in ADCETRIS-treated patients.

Contraindication

ADCETRIS concomitant with bleomycin due to pulmonary toxicity (e.g., interstitial infiltration and/or inflammation).

Warnings and Precautions

Administer G-CSF primary prophylaxis beginning with Cycle 1 for patients who receive ADCETRIS in combination with chemotherapy for previously untreated Stage III/IV cHL or previously untreated PTCL.

Monitor complete blood counts prior to each ADCETRIS dose. Monitor more frequently for patients with Grade 3 or 4 neutropenia. Monitor patients for fever. If Grade 3 or 4 neutropenia develops, consider dose delays, reductions, discontinuation, or G-CSF prophylaxis with subsequent doses.

Most Common (20% in any study) Adverse ReactionsPeripheral neuropathy, fatigue, nausea, diarrhea, neutropenia, upper respiratory tract infection, pyrexia, constipation, vomiting, alopecia, decreased weight, abdominal pain, anemia, stomatitis, lymphopenia, and mucositis.

Drug InteractionsConcomitant use of strong CYP3A4 inhibitors or inducers has the potential to affect the exposure to monomethyl auristatin E (MMAE).

Use in Specific PopulationsModerate or severe hepatic impairment or severe renal impairment: MMAE exposure and adverse reactions are increased. Avoid use.

Advise males with female sexual partners of reproductive potential to use effective contraception during ADCETRIS treatment and for at least 6 months after the final dose of ADCETRIS.

Advise patients to report pregnancy immediately and avoid breastfeeding while receiving ADCETRIS.

For additional Important Safety Information, including BOXED WARNING, please see the full Prescribing Information for ADCETRIS at http://www.seattlegenetics.com or http://www.ADCETRIS.com.

ADYNOVATE Professional Important Information

ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated] Important Information

Indications and Limitation of UseADYNOVATE is a human antihemophilic factor indicated in children and adults with hemophilia A (congenital factor VIII deficiency) for:

ADYNOVATE is not indicated for the treatment of von Willebrand disease.

DETAILED IMPORTANT RISK INFORMATION

CONTRAINDICATIONSPrior anaphylactic reaction to ADYNOVATE, to the parent molecule (ADVATE [Antihemophilic Factor (Recombinant)]), mouse or hamster protein, or excipients of ADYNOVATE (e.g. Tris, mannitol, trehalose, glutathione, and/or polysorbate 80).

WARNINGS & PRECAUTIONSHypersensitivity ReactionsHypersensitivity reactions are possible with ADYNOVATE. Allergic-type hypersensitivity reactions, including anaphylaxis, have been reported with other recombinant antihemophilic factor VIII products, including the parent molecule, ADVATE. Early signs of hypersensitivity reactions that can progress to anaphylaxis may include angioedema, chest tightness, dyspnea, wheezing, urticaria, and pruritus. Immediately discontinue administration and initiate appropriate treatment if hypersensitivity reactions occur.

Neutralizing AntibodiesFormation of neutralizing antibodies (inhibitors) to factor VIII can occur following administration of ADYNOVATE. Monitor patients regularly for the development of factor VIII inhibitors by appropriate clinical observations and laboratory tests. Perform an assay that measures factor VIII inhibitor concentration if the plasma factor VIII level fails to increase as expected, or if bleeding is not controlled with expected dose.

ADVERSE REACTIONSThe most common adverse reactions (1% of subjects) reported in the clinical studies were headache and nausea.

Click here for Full Prescribing Informationhttps://www.shirecontent.com/PI/PDFs/ADYNOVATE_USA_ENG.pdf

FEIBA [Anti-Inhibitor Coagulant Complex] Indications and Detailed Important Risk Information

Indications for FEIBA

FEIBA is an Anti-Inhibitor Coagulant Complex indicated for use in hemophilia A and B patients with inhibitors for:

FEIBA is not indicated for the treatment of bleeding episodes resulting from coagulation factor deficiencies in the absence of inhibitors to coagulation factor VIII or coagulation factor IX.

Detailed Important Risk Information for FEIBA

WARNING: EMBOLIC AND THROMBOTIC EVENTS

CONTRAINDICATIONS

FEIBA is contraindicated in patients with:

WARNINGS AND PRECAUTIONS

Thromboembolic events (including venous thrombosis, pulmonary embolism, myocardial infarction, and stroke) can occur, particularly following the administration of high doses (>200 units/kg/day) and/or in patients with thrombotic risk factors.

Patients with DIC, advanced atherosclerotic disease, crush injury, septicemia, or concomitant treatment with recombinant factor VIIa have an increased risk of developing thrombotic events due to circulating tissue factor or predisposing coagulopathy. Potential benefit of treatment should be weighed against potential risk of these thromboembolic events.

Infusion should not exceed a single dose of 100 units/kg and daily doses of 200 units/kg. Maximum injection or infusion rate must not exceed 2 units/kg/minute. Monitor patients receiving >100 units/kg for the development of DIC, acute coronary ischemia and signs and symptoms of other thromboembolic events. If clinical signs or symptoms occur, such as chest pain or pressure, shortness of breath, altered consciousness, vision, or speech, limb or abdomen swelling and/or pain, discontinue FEIBA and initiate appropriate diagnostic and therapeutic measures.

Safety and efficacy of FEIBA for breakthrough bleeding in patients receiving emicizumab has not been established. Cases of thrombotic microangiopathy (TMA) were reported in a clinical trial where subjects received FEIBA as part of a treatment regimen for breakthrough bleeding following emicizumab treatment. Consider the benefits and risks with FEIBA if considered required for patients receiving emicizumab prophylaxis. If treatment with FEIBA is required for patients receiving emicizumab, the hemophilia treating physician should closely monitor for signs and symptoms of TMA. In FEIBA clinical studies TMA has not been reported.

Hypersensitivity and allergic reactions, including severe anaphylactoid reactions, can occur. Symptoms include urticaria, angioedema, gastrointestinal manifestations, bronchospasm, and hypotension. Reactions can be severe and systemic (e.g., anaphylaxis with urticaria and angioedema, bronchospasm, and circulatory shock). Other infusion reactions, such as chills, pyrexia, and hypertension have also been reported. If signs and symptoms of severe allergic reactions occur, immediately discontinue FEIBA and provide appropriate supportive care.

Because FEIBA is made from human plasma it may carry a risk of transmitting infectious agents, e.g., viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent.

FEIBA contains blood group isohemagglutinins (anti-A and anti-B). Passive transmission of antibodies to erythrocyte antigens, e.g., A, B, D, may interfere with some serological tests for red cell antibodies, such as antiglobulin test (Coombs test).

ADVERSE REACTIONS

Most frequently reported adverse reactions observed in >5% of subjects in the prophylaxis trial were anemia, diarrhea, hemarthrosis, hepatitis B surface antibody positive, nausea, and vomiting.

Serious adverse reactions seen are hypersensitivity reactions and thromboembolic events, including stroke, pulmonary embolism and deep vein thrombosis.

DRUG INTERACTIONS

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Takeda to Highlight Expanded Portfolio of Products Across Oncology and Hematology at 61st American Society of Hematology (ASH) Annual Meeting -...

Cardiac Stem Cells Comments Off on Takeda to Highlight Expanded Portfolio of Products Across Oncology and Hematology at 61st American Society of Hematology (ASH) Annual Meeting -… | November 8th, 2019

Updated safety and efficacy results from ongoing Phase 1 CRB-402 study of bb21217 in relapsed/refractory multiple myeloma

Updated results from ongoing Phase 1/2 (HGB-206) study of LentiGlobin gene therapy for patients with sickle cell disease

New data from ongoing Phase 3 studies of LentiGlobin gene therapy for -thalassemia in pediatric, adolescent and adult patients

CAMBRIDGE, Mass. bluebird bio, Inc. (Nasdaq: BLUE) announced today that new and updated data from its investigational gene and cell therapy programs for multiple myeloma, sickle cell disease (SCD) and transfusion-dependent -thalassemia (TDT) will be presented at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition in Orlando, Florida, December 7 10.

bluebird bio will present updated safety and efficacy data from the ongoing Phase 1 clinical study (CRB-402) of bb21217. bb21217 is an investigational BCMA-targeted chimeric antigen receptor (CAR) T cell therapy being studied, in partnership with Celgene, in patients with relapsed/refractory multiple myeloma (RRMM).

In addition, data from clinical studies of LentiGlobin gene therapy for -thalassemia, including results up to 61 months from the long-term follow-up study (LTF-303) and updated results from the completed Phase 1/2 Northstar (HGB-204) study, will be presented at ASH. The company will also present new data from the ongoing Phase 3 Northstar-2 (HGB-207) study in pediatric, adolescent and adult patients who do not have a 0/0 genotype and from the ongoing Phase 3 Northstar-3 (HGB-212) study in pediatric, adolescent and adult patients who have 0/0 genotype or an IVS-I-110 mutation at both alleles of the -globin gene.

New data from the companys Phase 1/2 HGB-206 study of LentiGlobin gene therapy for SCD will include additional patients treated in the study and updated data for those previously reported. The company will also present data from exploratory assays designed to assess the relationship between drug product characteristics and red blood cell physiology in patients treated with LentiGlobin for SCD.

Updated Data from Ongoing Phase 1 Clinical Study (CRB-402) of bb21217

Updated Results from an Ongoing Phase 1 Clinical Study of bb21217 Anti-BCMA CAR T Cell Therapy Presenting Author: Jesus G. Berdeja, M.D., Sarah Cannon Center for Blood Cancers, Nashville, Tenn. Date & Time: Oral #927, Monday, December 9, 2019, 6:45 p.m. ET

bb21217, an investigational BCMA-targeted CAR T cell therapy being developed in partnership with Celgene, is one of bluebird bios lead oncology programs. bb21217 uses the idecabtagene vicleucel CAR molecule (formerly referred to as bb2121) and is manufactured with a process intended to increase the in vivo persistence of CAR T cells.

This presentation will include updated data from the Phase 1 CRB-402 study, the first-in-human study of bb21217 in patients with RRMM, designed to assess the primary endpoint of safety as well as other pre-defined endpoints including efficacy and pharmacokinetics measurements. CRB-402 is a two-part (dose escalation and dose expansion), open-label, multi-site Phase 1 study of bb21217 in adults with RRMM with a projected final enrollment of 74 patients.

Data in the abstract include results as of the data cutoff date of April 20, 2019 for 22 patients who have received bb21217 at three dose levels (12 at 150 x 106 CAR+ T cells; six at 300 x 106 CAR+ T cells; and four at 450 x 106 CAR+ T cells). These patients had a median of seven prior lines of therapy (min-max: 4 17 lines), 18 patients had a prior autologous stem cell transplant, 19 patients received daratumumab and 13 patients received prior treatment with bortezomib, lenalidomide, carfilzomib, pomalidomide and daratumumab.

As of the data cutoff, the adverse events observed were consistent with known toxicities of CAR T therapies. Thirteen of 22 patients developed cytokine release syndrome (CRS); five Grade 1, seven Grade 2, and one Grade 3 case. All 13 patients responded to supportive care, tocilizumab and/or corticosteroids. Five of 22 patients developed neurotoxicity; one Grade 1, two Grade 2, one Grade 3 (vertigo/dizziness), and one Grade 4 (encephalopathy, previously reported). For the one patient previously reported with Grade 4 neurotoxicity, Grade 3 CRS was also reported, and both have resolved.

Eighteen patients were evaluable for clinical response with > two months of follow-up or progressive disease within two months. Eighty-three percent (n=15/18) of evaluable patients demonstrated clinical response per the International Myeloma Working Group Uniform Response Criteria for multiple myeloma. As of the data cutoff, with the median follow-up after bb21217 infusion of five months (min-max: <1 18 months), nine patients remained in response, including two patients with ongoing response at 15 and 18 months.

Evidence of myeloma in the bone marrow, known as minimal residual disease, was undetectable by next-generation sequencing at a sensitivity level of 10-5 or better in all responders who had evaluable bone marrow samples (n=10) at Month 1. CAR T cell persistence was observed in six of eight patients evaluable at six months and in two of two patients evaluable at 12 months.

This study is ongoing to evaluate the potential safety and efficacy of treatment with bb21217, and updated results, including early clinical and CAR T cell persistence data, will be shared at the ASH conference.

Multiple Myeloma Presentations at ASH

Markers of Initial and Long-Term Responses to Idecabtagene Vicleucel (Ide-Cel; bb2121) in the CRB-401 Study in Relapsed/Refractory Multiple Myeloma Presenting Author: Ethan G. Thompson, Ph.D., Celgene, Seattle, Wash. Date & Time: Poster #4328, Monday, December 9, 2019, 6:00 8:00 p.m. ET

Updated Results from an Ongoing Phase 1 Clinical Study of bb21217 anti-BCMA CAR T Cell Therapy Presenting Author: Jesus G. Berdeja, M.D., Sarah Cannon Center for Blood Cancers, Nashville, Tenn. Date & Time: Oral #927, Monday, December 9, 2019, 6:45 p.m. ET

SCD Presentations at ASH

The Relationships Between Target Gene Transduction, Engraftment of HSCs and RBC Physiology in Sickle Cell Disease Gene Therapy Presenting Author: Melissa Bonner, Ph.D., bluebird bio, Cambridge, Mass. Date & Time: Oral #206, Saturday, December 7, 2019, 2:15 p.m.

Exploring the Drivers of Clinical Benefit in Initial Patients Treated in the HGB-206 Study of LentiGlobin for Sickle Cell Disease (SCD) Gene Therapy Presenting Author: Mark Walters, M.D., Benioff Childrens Hospital, Oakland, Calif. Date & Time: Poster #2061, Saturday, December 7, 2019, 5:30 7:30 p.m.

Resolution of Sickle Cell Disease Manifestations in Patients Treated with LentiGlobin Gene Therapy: Updated Results from the Phase 1/2 HGB-206 Group C Study Presenting Author: Julie Kanter, M.D., University of Alabama at Birmingham, Birmingham, Ala. Date & Time: Poster #990, Saturday, December 7, 2019, 5:30 7:30 p.m.

TDT Presentations at ASH

Clinical Outcomes after Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Transfusion-Dependent -Thalassemia Treated at the Bambino Ges Childrens Hospital, Rome, Italy Presenting Author: Pietro Merli, M.D., IRCCS Ospedale Pediatrico Bambino Ges, Rome, Italy Date & Time: Poster #969, Saturday, December 7, 2019, 5:30 7:30 p.m.

Northstar-3: Interim Results from a Phase 3 Study Evaluating LentiGlobin Gene Therapy in Patients with Transfusion-Dependent -Thalassemia and Either a 0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene Presenting Author: Ashutosh Lal, M.D., UCSF Benioff Childrens Hospital, Oakland, Calif. Date & Time: Oral #815, Monday, December 9, 2019, 5:30 p.m.

Northstar-2: Updated Safety and Efficacy Analysis of LentiGlobin Gene Therapy in Patients with Transfusion-Dependent -Thalassemia and Non-0/0Genotypes Presenting Author: Alexis Thompson, M.D., MPH, Ann & Robert H. Lurie Childrens Hospital of Chicago, Chicago, Ill. Date & Time: Poster #3543, Monday, December 9, 2019, 6:00 8:00 p.m.

Long-Term Clinical Outcomes of LentiGlobin Gene Therapy for Transfusion-Dependent -Thalassemia in the Northstar (HGB-204) Study Presenting Author: Janet Kwiatkowski, M.D., MSCE, Childrens Hospital of Philadelphia, Philadelphia, Pa. Date & Time: Poster #4628, Monday, December 9, 2019, 6:00 8:00 p.m.

Routine Management, Healthcare Resource Use and Patient/Caregiver-Reported Outcomes of Patients with Transfusion-Dependent -Thalassaemia in the United Kingdom: A Mixed Methods Observational Study Presenting Author: Farrukh Shah, MBBS, FRCP, FRCPath, M.D., Whittington Hospital, London, U.K. Date & Time: Poster #3550, Monday, December 9, 2019, 6:00 8:00 p.m.

SCD and TDT Presentation at ASH

Results from the Completed HGB-205 Trial of LentiGlobin for -Thalassemia and LentiGlobin for Sickle Cell Disease Gene Therapy Presenting Author: Elisa Magrin, Ph.D., Necker Childrens Hospital, Assistance Publique-Hpitaux de Paris, Paris, France Date & Time: Poster #3358, Sunday, December 8, 2019, 6:00 8:00 p.m.

Abstracts outlining bluebird bios accepted data at ASH will be available on the ASH conference website at 9 a.m. EST today.

About ide-cel and bb21217 for Multiple Myeloma

bluebird bios lead oncology programs, idecabtagene vicleucel (ide-cel, formerly referred to as bb2121) and bb21217, are investigational BCMA-targeted chimeric antigen receptor (CAR) T cell therapies being studied in a broad clinical development program for patients with multiple myeloma. ide-cel and bb21217 are being developed in partnership with Celgene.

KarMMa is a registration-enabling, open-label, single-arm, multi-center Phase 2 study evaluating the efficacy and safety of ide-cel in patients with relapsed/refractory multiple myeloma. In November 2018, bluebird bio announced completion of enrollment in the trial. ide-cel was granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration and Priority Medicines (PRIME) eligibility by the European Medicines Agency in November 2017 based on preliminary clinical data from the Phase 1 CRB-401 study.

bluebird bios clinical development program for bb21217 includes the ongoing Phase 1 CRB-402 study. CRB-402 is the first-in-human study of bb21217 in patients with RRMM, designed to assess safety, pharmacokinetics, efficacy and duration of effect. CRB-402 is a two-part (dose escalation and dose expansion), open-label, multi-site Phase 1 study of bb21217 in adults with RRMM with a projected final enrollment of 74 patients. For more information visit: clinicaltrials.gov using identifier NCT03274219.

ide-cel and bb21217 are not approved for any indication in any geography.

About LentiGlobin for Sickle Cell Disease

LentiGlobin for sickle cell disease is an investigational gene therapy being studied as a potential treatment for SCD. bluebird bios clinical development program for LentiGlobin for SCD includes the ongoing Phase 1/2 HGB-206 study.

SCD is a serious, progressive and debilitating genetic disease caused by a mutation in the -globin gene that leads to the production of abnormal sickle hemoglobin (HbS), causing red blood cells (RBCs) to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and painful vaso-occlusive events (VOEs). For adults and children living with SCD, this means unpredictable episodes of excruciating pain due to vaso-occlusion as well as other acute complicationssuch as acute chest syndrome (ACS), stroke, and infections, which can contribute to early mortality in these patients.

LentiGlobin for SCD received Orphan Medicinal Product designation from the European Commission for the treatment of SCD.

The U.S. Food and Drug Administration granted Orphan Drug status and Regenerative Medicine Advanced Therapy designation for LentiGlobin for the treatment of SCD.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for SCD. For more information visit: https://www.bluebirdbio.com/medical-professionals/our-clinical-trials/ or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

About LentiGlobin for -Thalassemia

The European Commission granted conditional marketing authorization for LentiGlobin for TDT, to be marketed as ZYNTEGLO (autologous CD34+ cells encoding A-T87Q-globin gene) gene therapy, for patients 12 years and older with TDT who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

TDT is a severe genetic disease caused by mutations in the -globin gene that result in reduced or absent hemoglobin (Hb). In order to survive, people with TDT maintain Hb levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

LentiGlobin adds functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived-hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

Non-serious adverse events (AEs) observed during clinical studies that were attributed to LentiGlobin for TDT were hot flush, dyspnoea, abdominal pain, pain in extremities and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to LentiGlobin for TDT.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

The conditional marketing authorization for ZYNTEGLO is only valid in the 28 member states of the EU as well as Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

The U.S. Food and Drug Administration granted LentiGlobin for -thalassemia Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT.

LentiGlobin for -thalassemia continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies. For more information about the ongoing clinical studies, visit http://www.northstarclinicalstudies.com or clinicaltrials.gov and use identifier NCT02906202 for Northstar-2 (HGB-207), NCT03207009 for Northstar-3 (HGB-212).

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for TDT. For more information visit: https://www.bluebirdbio.com/medical-professionals/our-clinical-trials/ or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

About bluebird bio, Inc.

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders including cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO, LentiGlobin, and bluebird bio are trademarks of bluebird bio, Inc.

The full common name for ZYNTEGLO: A genetically modified autologous CD34+ cell enriched population that contains hematopoietic stem cells transduced with lentiviral vector encoding the A-T87Q-globin gene.

Forward-Looking Statements

This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Companys views with respect to the potential for LentiGlobin to treat transfusion-dependent -thalassemia and sickle cell disease, the potential for the bb21217 product candidate to treat relapsed/ refractory multiple myeloma. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risks that the preliminary positive efficacy and safety results from our prior and ongoing clinical trials of our product candidates will not continue or be repeated in our ongoing or planned clinical trials or in the commercial context, risks that the current or planned clinical trials of our product candidates will be insufficient to support future regulatory submissions or to support marketing approval in the US and EU, and the risk that any one or more of our product candidates, will not be successfully developed, approved or commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191106005511/en/

Contacts

Investors: Elizabeth Pingpank, 617-914-8736 epingpank@bluebirdbio.com or Media: Catherine Falcetti, 339-499-9436 cfalcetti@bluebirdbio.com

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bluebird bio to Present New Data from Gene and Cell Therapy Programs at 61st American Society of Hematology Annual Meeting and Exposition - Financial...

Cardiac Stem Cells Comments Off on bluebird bio to Present New Data from Gene and Cell Therapy Programs at 61st American Society of Hematology Annual Meeting and Exposition – Financial… | November 6th, 2019

This is therefore a comparing of the institutional ownership, earnings and valuation, profitability, risk, dividends, analyst recommendations in Capricor Therapeutics Inc. (NASDAQ:CAPR) and Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI). The two are both Biotechnology companies that compete with one another.

Earnings and Valuation

Demonstrates Capricor Therapeutics Inc. and Brainstorm Cell Therapeutics Inc. earnings per share, top-line revenue and valuation.

Profitability

Table 2 represents Capricor Therapeutics Inc. (NASDAQ:CAPR) and Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI)s return on assets, return on equity and net margins.

Volatility and Risk

A beta of 1.8 shows that Capricor Therapeutics Inc. is 80.00% more volatile than Standard & Poors 500. Brainstorm Cell Therapeutics Inc. has a 1.19 beta and it is 19.00% more volatile than Standard & Poors 500.

Liquidity

Capricor Therapeutics Inc.s Current Ratio and Quick Ratio are 5.3 and 5.3 respectively. The Current Ratio and Quick Ratio of its competitor Brainstorm Cell Therapeutics Inc. are 1 and 1 respectively. Capricor Therapeutics Inc. therefore has a better chance of paying off short and long-term obligations compared to Brainstorm Cell Therapeutics Inc.

Analyst Ratings

The following table given below contains the ratings and recommendations for Capricor Therapeutics Inc. and Brainstorm Cell Therapeutics Inc.

$11.5 is Capricor Therapeutics Inc.s average target price while its potential upside is 342.31%. Competitively Brainstorm Cell Therapeutics Inc. has a consensus target price of $9, with potential upside of 134.99%. The data from earlier shows that analysts belief suggest that Capricor Therapeutics Inc. seems more appealing than Brainstorm Cell Therapeutics Inc.

Institutional and Insider Ownership

Capricor Therapeutics Inc. and Brainstorm Cell Therapeutics Inc. has shares owned by institutional investors as follows: 5.1% and 11.4%. Insiders owned 32.93% of Capricor Therapeutics Inc. shares. On the other hand, insiders owned about 0.6% of Brainstorm Cell Therapeutics Inc.s shares.

Performance

Here are the Weekly, Monthly, Quarterly, Half Yearly, Yearly and YTD Performance of both pretenders.

For the past year Capricor Therapeutics Inc. has weaker performance than Brainstorm Cell Therapeutics Inc.

Summary

Brainstorm Cell Therapeutics Inc. beats on 7 of the 10 factors Capricor Therapeutics Inc.

Capricor Therapeutics, Inc., a biotechnology company, focuses on the discovery, development, and commercialization of novel therapeutics primarily for the treatment of cardiovascular diseases. The companys development stage drug candidates for cardiovascular diseases include CAP-1002 that is in Phase II clinical trials; and CAP-2003, which is in pre-clinical development for the treatment of certain cardiac and inflammatory conditions. The company was founded in 2005 and is headquartered in Beverly Hills, California.

Brainstorm Cell Therapeutics Inc., a biotechnology company, develops adult stem cell therapies for neurodegenerative disorders that include amyotrophic lateral sclerosis, multiple sclerosis, Parkinsons disease, and others. The company holds rights to develop and commercialize its NurOwn technology through a licensing agreement with Ramot of Tel Aviv University Ltd. Its NurOwn technology is based on a novel differentiation protocol, which induces differentiation of the bone marrow-derived mesenchymal stem cells into neuron-supporting cells and secreting cells that release various neurotrophic factors, including glial-derived neurotrophic factor, brain-derived neurotrophic factor, vascular endothelial growth factor, and hepatocyte growth factor for the growth, survival, and differentiation of developing neurons. The company was formerly known as Golden Hand Resources Inc. and changed its name to Brainstorm Cell Therapeutics Inc. in November 2004 to reflect its new line of business in the development of novel cell therapies for neurodegenerative diseases. Brainstorm Cell Therapeutics Inc. was founded in 2000 and is headquartered in Hackensack, New Jersey.

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Reviewing Capricor Therapeutics Inc. (CAPR)'s and Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI)'s results - FinanceMercury

Cardiac Stem Cells Comments Off on Reviewing Capricor Therapeutics Inc. (CAPR)’s and Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI)’s results – FinanceMercury | November 6th, 2019