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Cord Blood Banking Services Market projected to expand at a CAGR of 10.9% from 2019 to 2027 KSU | The Sentinel Newspaper – KSU | The Sentinel…

By daniellenierenberg

Transparency Market Research (TMR) has published a new report titled, Cord Blood Banking Services Market Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 20192027. According to the report, the globalcord blood banking services marketwas valued atUS$ 25.8 Mnin2018and is projected to expand at a CAGR of10.9%from2019to2027.

Overview

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High Incidence of genetic disorders and rise in hematopoietic stem cell transplantation rates to Drive Market

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Private Cord Blood Bank Segment to Dominate Market

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North America to Dominate Global Market

Competitive Landscape

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Jasper Therapeutics Announces Launch of New Clinical Trial with National Heart, Lung, and Blood Institute to Evaluate JSP191 in Sickle Cell Disease -…

By daniellenierenberg

REDWOOD CITY, Calif.--(BUSINESS WIRE)--Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, today announced the launch of a Phase 1/2 clinical trial to evaluate JSP191, Jaspers first-in-class anti-CD117 monoclonal antibody, as a targeted, non-toxic conditioning regimen prior to allogeneic transplant for sickle cell disease (SCD). Jasper Therapeutics and the National Heart, Lung, and Blood Institute (NHLBI) have entered into a clinical trial agreement in which NHLBI will serve as the Investigational New Drug (IND) sponsor for this study.

SCD is a lifelong inherited blood disorder that affects hemoglobin, a protein in red blood cells that delivers oxygen to tissues and organs throughout the body. Approximately 300,000 infants are born with SCD annually worldwide, and the number of cases is expected to significantly increase. Currently, hematopoietic stem cell transplantation (HSCT) is the only cure available for SCD.

"This clinical trial agreement with the NHLBI expands the development of JSP191 for transplant conditioning and could bring curative transplants to more patients in need," said Kevin N. Heller, M.D., Executive Vice President, Research and Development, of Jasper Therapeutics. "We look forward to collaborating with the NHLBI and learning more about the potential for JSP191 in patients living with sickle cell disease."

About JSP191

JSP191 (formerly AMG 191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from the bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals in stem cells leading to cell death. This creates space in the bone marrow for engraftment of donor or gene-corrected transplanted stem cells.

Preclinical studies have shown that JSP191, as a single agent, safely depletes normal and diseased hematopoietic stem cells, including in animal models of severe combined immunodeficiency (SCID), myelodysplastic syndromes (MDS), and sickle cell disease (SCD). Treatment with JSP191 creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients.

JSP191 is currently being evaluated in two separate Jasper Therapeutics-sponsored clinical studies in hematopoietic cell transplant. The first clinical study is evaluating JSP191 as a sole conditioning agent in a Phase 1/2 dose-escalation and expansion trial to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplant for SCID. Blood stem cell transplantation offers the only potentially curative therapy for SCID. JSP191 is also being evaluated in combination with another conditioning regimen in a Phase 1 study in patients with MDS or acute myeloid leukemia (AML) who are receiving hematopoietic cell transplant. For more information about the design of these clinical trials, visit http://www.clinicaltrials.gov (NCT02963064 and NCT04429191).

Additional studies are planned to advance JSP191 as a conditioning agent for patients with other rare and ultra-rare monogenic disorders and autoimmune diseases.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The companys lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplant. This first-in-class conditioning antibody is designed to enable safer and more effective curative hematopoietic cell transplants and gene therapies. For more information, please visit us at jaspertherapeutics.com.

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Novartis, Gates Foundation pursue a simpler gene therapy for sickle cell – STAT

By daniellenierenberg

Novartis and the Bill and Melinda Gates Foundation are joining forces to discover and develop a gene therapy to cure sickle cell disease with a one-step, one-time treatment that is affordable and simple enough to treat patients anywhere in the world, especially in sub-Saharan Africa where resources may be scarce but disease prevalence is high.

The three-year collaboration, announced Wednesday, has initial funding of $7.28 million.

Current gene therapy approaches being developed for sickle cell disease are complex, enormously expensive, and bespoke, crafting treatments for individual patients one at a time. The collaboration aims to instead create an off-the-shelf treatment that bypasses many of the steps of current approaches, in which cells are removed and processed outside the body before being returned to patients.

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Sickle cells cause is understood. The people it affects are known. But its cure has been elusive, Jay Bradner, president of the Novartis Institutes for BioMedical Research, told STAT.

We understand perfectly the disease pathway and the patient, but we dont know what it would take to have a single-administration, in vivo gene therapy for sickle cell disease that you could deploy in a low-resource setting with the requisite safety and data to support its use, he said. Im a hematologist and can assure you that in my experience in the clinic, it was extremely frustrating to understand a disease so perfectly but have so little to offer.

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Sickle cell disease is a life-threatening inherited blood disorder that affects millions around the world, with about 80% of affected people in sub-Saharan Africa and more than 100,000 in the U.S. The mutation that causes the disease emerged in Africa, where it protects against malaria. While most patients with sickle cell share African ancestry, those with ancestry from South America, Central America, and India, as well as Italy and Turkey, can also have the hereditary disease.

The genetic mutation does its damage by changing the structure of hemoglobin, hampering the ability of red blood cells to carry oxygen and damaging blood vessels when the misshapen cells get stuck and block blood flow. Patients frequently suffer painful crises that can be fatal if not promptly treated with fluids, medication, and oxygen. Longer term, organs starved of oxygen eventually give out. In the U.S., that pain and suffering is amplified when systemic and individual instances of racism deny Black people the care they need.

Delivering gene therapy for other diseases has been costly and difficult even in the best financed, most sophisticated medical settings. Challenges include removing patients cells so they can be altered in a lab, manufacturing the new cells in high volume, reinfusing them, and managing sometimes severe responses to the corrected cells. Patients also are given chemotherapy to clear space in their bone marrow for the new cells.

Ideally, many of those steps could be skipped if there were an off-the-shelf gene therapy. That means, among other challenges, inventing a way to eliminate the step where each patients cells are manipulated outside the body and given back the in vivo part of the plan to correct the genetic mutation.

Thats not the only obstacle. For a sickle cell therapy to be successful, Bradner said, it must be delivered only to its targets, which are blood stem cells. The genetic material carrying corrected DNA must be safely transferred so it does not become randomly inserted into the genome and create the risk of cancer, a possibility that halted a Bluebird Bio clinical trial on Tuesday. The payload itself mustnt cause such problems as the cytokine storm of immune overreaction. And the intended response has to be both durable and corrective.

In a way, the gene delivery is the easy part because we know that expressing a normal hemoglobin, correcting the mutated hemoglobin, or reengineering the switches that once turned off normal fetal hemoglobin to turn it back on, all can work, Bradner said. The payload is less a concern to me than the safe, specific, and durable delivery of that payload.

For each of these four challenges delivery, gene transfer, tolerability, durability there could be a bespoke technical solution, Bradner said. The goal is to create an ensemble form of gene therapy.

Novartis has an existing sickle-cell project using CRISPR with the genome-editing company Intellia, now in early human trials, whose lessons may inform this new project. CRISPR may not be the method used; all choices are still on the table, Bradner said.

Vertex Pharmaceuticals has seen encouraging early signs with its candidate therapy developed with CRISPR Therapeutics. Other companies, including Beam Therapeutics, have also embarked on gene therapy development.

The Novartis-Gates collaboration is different in its ambition to create a cure that does not rely on an expensive, complicated framework. Novartis has worked with the Gates Foundation on making malaria treatment accessible in Africa. And in October 2019, the Gates Foundation and the National Institutes of Health said together they would invest at least $200 million over the next four years to develop gene-based cures for sickle cell disease and HIV that would be affordable and available in the resource-poor countries hit hardest by the two diseases, particularly in Africa.

Gene therapies might help end the threat of diseases like sickle cell, but only if we can make them far more affordable and practical for low-resource settings, Trevor Mundel, president of global health at the Gates Foundation, said in a statement about the Novartis collaboration. Its about treating the needs of people in lower-income countries as a driver of scientific and medical progress, not an afterthought.

Asked which is the harder problem to solve: one-time, in vivo gene therapy, or making it accessible around the world, David Williams, chief of hematology/oncology at Boston Childrens Hospital, said: Both are going to be difficult to solve. The first will likely occur before the therapy is practically accessible to the large number of patients suffering the disease around the world.

Williams is also working with the Gates Foundation, as well as the Koch Institute for Integrative Cancer Research at MIT, Dana-Farber Cancer Institute, and Massachusetts General Hospital, on another approach in which a single injection of a reagent changes the DNA of blood stem cells. But there are obstacles to overcome there, too, that may be solved by advances in both the technology to modify genes and the biological understanding of blood cells.

Bradner expects further funding to come to reach patients around the world, once the science progresses more.

There is no plug-and-play solution for this project in the way that mRNA vaccines were perfectly set up for SARS-CoV-2. We have no such technology to immediately redeploy here, he said. Were going to have to reimagine what it means to be a gene therapy for this project.

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After 13 years of trials and tribulations RTP firm G1 wins first FDA approval for cancer drug – WRAL Tech Wire

By daniellenierenberg

RESEARCH TRIANGLE PARK After 13 years as a clinical-stage oncology company,G1 Therapeuticsof Research Triangle Park transformed into a commercial-stage company overnight upon the approval of its first drug by the U.S. Food and Drug Administration.

The FDA on Feb. 12 approved G1s trilaciclib, to be marketed as Cosela, for protecting bone marrow from chemotherapy damage in adult patients with extensive-stage small cell lung cancer (ES-SCLC).

Cosela will help change the chemotherapy experience for people who are battling ES-SCLC, said Jack Bailey, the companys chief executive officer. G1 is proud to deliver Cosela to patients and their families as the first and only therapy to help protect against chemotherapy-induced myelosuppression.

Myelosuppression, or damage to the bone marrow, is the most serious and life-threatening side effect of chemotherapy. Chemotherapy-induced myelosuppression reduces the bodys essential supply of white blood cells, red blood cells and platelets, and can lead to increased risks of infection, severe anemia and bleeding.

RTP drug firm G1 secures FDA approval for treatment to prevent chemo damage to bone marrow

These complications impact patients quality of life and may also result in chemotherapy dose reductions and delays, said Jeffrey Crawford,M.D., Geller Professorfor Research in Cancer in theDepartment of MedicineandDuke Cancer Institute. In clinical trials, the addition of trilaciclib to extensive-stage small cell lung cancer chemotherapy treatment regimens reduced myelosuppression and improved clinical outcomes.The good news is that these benefits of trilaciclib will now be available for our patients in clinical practice.

Cosela is expected to be commercially available through G1s specialty distributor partner network in early March, the company said.

G1 is committed to helping patients with in theU.S.gain access to treatment with Cosela through access and affordability programs. Patients and healthcare can call the companys support center at 833-418-6663 for information.

Cosela is intended to be given as a 30-minute infusion four hours prior to chemotherapy treatments containing platinum/etoposide or topotecan. About 90 percent of all patients with ES-SCLC receive at least one of these chemotherapy regimens during their treatment, according to G1.

The approval of Cosela is based on data from three randomized, placebo-controlled trials. Data showed that patients receiving Cosela before the start of chemotherapy had less neutropenia, an abnormally low number of neutrophils, white blood cells that fight bacterial and fungal infection.

Data also showed a positive impact on red blood cell transfusions and other myeloprotective measures.

Chemotherapy is the most effective and widely used approach to treating people diagnosed with extensive-stage small cell lung cancer, Bailey said. However, standard-of-care chemotherapy regimens are highly myelosuppressive and can lead to costly hospitalizations and rescue interventions.

To date, oncologists have relied on rescue therapy, a mix of growth factor agents, antibiotics and red blood cell transfusions, to restore bone marrow after it has been damaged by chemotherapy.

By contrast, trilaciclib provides the first proactive approach to myelosuppression through a unique mechanism of action that helps protect the bone marrow from damage by chemotherapy, Crawford said.

Cosela helps protect bone marrow cells from chemotherapy damage by inhibiting cyclin- dependent kinase 4 and 6, two enzymes involved in cancer cell growth. Inhibiting these enzymes temporarily stops hematopoietic stem cells and progenitor cells in the bone marrow from dividing, making them resistant to damage from chemotherapy drugs that target dividing cells.

Bonnie J. Addario, lung cancer survivor, co-founder and board chair of theGo2 Foundation for Lung Cancer, said many people with extensive-stage small cell lung cancerrely on chemotherapy to extend their lives and alleviate their symptoms.

Unfortunately, the vast majority will experience chemotherapy-induced side effects, resulting in dose delays and reductions, and increased utilization of healthcare services, she said.

G1 shares our organizations goal to improve the quality of life of those diagnosed with lung cancer and to transform survivorship among people living with this insidious disease. We are thrilled to see new advancements that can help improve the lives of those living with small cell lung cancer.

About 30,000 small cell lung cancer patients are treated inthe United Statesannually. SCLC, one of the two main types of lung cancer, accounts for about 10 to 15 percent of all lung cancers but is the more aggressive disease, tending to grow and spread faster than the other type, non-small cell lung cancer.

InJune 2020, G1 announced a three-yearco-promotion agreementwithBoehringer Ingelheimfor Cosela in small cell lung cancer in theU.S.andPuerto Rico. G1 will lead marketing, market access and medical engagement initiatives for Cosela whileBoehringer Ingelheimsoncology commercial team will lead sales force engagement initiatives.

G1 will book revenue and retain development and commercialization rights to Cosela and payBoehringer Ingelheima promotional fee based on net sales.

The three-year agreement does not extend to additional indications that G1 is evaluating for trilaciclib: breast, colorectal, bladder and non-small cell lung cancers.

G1 is a 2008 spin-out of the University of North Carolina at Chapel Hill.

The company raised $108 million in an initial public offering of stock in 2017 after receiving more than $95 million in three rounds of venture capital funding. The North Carolina Biotechnology Center provided two early-stage loans totaling $500,000.

G1s stock is traded on the Nasdaq Global Select Market under the ticker symbol GTHX.

(C) N.C. Biotech Center

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Research Associate in Stem Cells and Regenerative Medicine job with KINGS COLLEGE LONDON | 246711 – Times Higher Education (THE)

By daniellenierenberg

Job descriptionThe Centre for Stem Cells & Regenerative Medicine is located in Guys Hospital.It is internationally recognized for research on adult and pluripotent stem cells and is a focus for cutting-edge stem cell research currently taking place across the College and its partner NHS trusts, as part of Kings Health Partners. Through the Centre, Kings aims to drive collaboration between scientists and clinicians to translate the potential of stem cells into clinical reality for patients.Applications are invited for a postdoctoral researcher funded as part of the PIs Wellcome Clinical Fellowship, and will work with a dynamic group of scientists focussed on reproductive biology, early embryonic development and the causes of infertility. The post holder will contribute to the regenerative medicine theme and will be involved in the generation and processing of single cell experiments using a variety of techniques.This is an exciting opportunity following our recent work (Sangrithi et al. 2017, Dev Cell & Lau et al. 2020, Dev Cell). The project aims to discover the function of genes on the X-chromosome in male germline stem cells (spermatogonia) and their role in idiopathic and sex chromosome aneuploidy associated infertility. We aim to understand physiological gene regulatory networks functional in spermatogonial stem cells using a combination of single-cell methods, to explain how perturbation in X-gene dosage in SSCs may cause infertility. The postholder will also identify and validate candidate disease bio-markers.This post will be offered on an a fixed-term contract until 05/04/2026This is a full-time post - 100% full time equivalent

Key responsibilities Carry out world class research. Are adept at working in a wet lab setting with experience in designing and executing experiments. Familiarity in single cell work nucleic acid manipulation is desirable Communicate results effectively in writing and orally Contribute to publications arising from the research projects Keep clear and up-to-date records of work Attend and present at seminars, journal clubs and conferences Contribute to collaborative atmosphere of the department Share skills by training others Comply with all relevant safety legislation to ensure a safe working environment Take part in public engagement activities To support grant writing, for maintaining the continual research in this domain, e.g. Fellowships Post holder will be expected to plan and prioritise their own workload, with competing and shifting priorities under pressure of deadlinesThe above list of responsibilities may not be exhaustive, and the post holder will be required to undertake such tasks and responsibilities as may reasonably be expected within the scope and grading of the post.

Skills, knowledge, and experience

Essential criteria PhD awarded in the biological sciences Excellent general knowledge of molecular biology Knowledge of cell biology Knowledge of flow cytometry Relevant postdoctoral experience Experience in a molecular biology research lab Excellent record keeping / attention to detail Organized and systematic approach to research Pro-active, enthusiastic, positive attitude Self-motivated, with the ability to work under pressure & to meet deadlines Keen interest in infertility and regenerative medicine Ability to think strategically

Desirable criteria Understanding of the biology of germ cells and embryo development Previous experience in working with the laboratory mouse ES cell culture experience General knowledge of computational tools for single cell RNAseq Ability to make collaborative and independent decisions*Please note that this is a PhD level role but candidates who have submitted their thesis and are awaiting award of their PhDs will be considered. In these circumstances the appointment will be made at Grade 5, spine point 30 with the title of Research Assistant. Upon confirmation of the award of the PhD, the job title will become Research Associate and the salary will increase to Grade 6.Further informationABOUT THE SCHOOLThe School of Basic & Medical Biosciences is led by Professor Mathias Gautel and comprises five departments with a wide range of expertise and interests. Using a bench to bedside approach, the School aims to answer fundamental questions about biology in health and disease and apply this to the development of new and innovative clinical practise, alongside providing a rigorous academic programme for students.DepartmentsThe Centre for Human & Applied Physiological Sciences (CHAPS) uses an integrative and translational research approach focusing on fundamental questions about human physiological function in health and disease to explore 3 research themes: skeletal muscle & aging, sensory-motor control & pain and aerospace & extreme environment adaptation.The Centre for Stem Cells & Regenerative Medicine focuses on cutting-edge stem cell research, how stem cells interact with their local environment and how these interactions are important for developing effective cell therapies in the clinic.The Department of Medical & Molecular Genetics uses cutting-edge technologies and analysis techniques to explore the mechanistic basis of disease, improve diagnostics and understand the epigenetic mechanisms of gene regulation and RNA processing, working from whole population level to complex and rare disease genomesThe Randall Centre of Cell & Molecular Biophysics takes a multi-disciplinary approach at the interface of Biological and Physical Sciences to explore the underlying mechanisms behind common diseases.St Johns Institute of Dermatology seeks to improve the diagnosis and management of severe skin diseases, through a better understanding of the basic pathogenetic mechanisms that cause and sustain these conditions focussing on cutaneous oncology, genetic skin disorders, inflammatory & autoimmune skin disorders, and photomedicine.About the Department of Centre for Stem Cells & Regenerative MedicineThe Centre for Stem Cells & Regenerative Medicine is led by Professor Fiona Watt, whos laboratory comprises approximately 30 research staff and visiting scientists and is internationally recognised for research on adult and pluripotent stem cells. Along with Professor Watts group there are nine other research groups operating at the Centre, bringing the total number of staff to approximately 80 people.Research at the Centre is focused on how stem cells interact with their local environment, or niche. We believe that an understanding of these interactions is important for developing effective cell therapies in the clinic. Located on the Guys Hospital campus, the Centre acts as a focus for cutting-edge stem cell research taking place across the College and its partner NHS Trusts, as part of Kings Health Partners. To facilitate collaborations within Kings and with external partners, we have opened a Stem Cell Hotel where researchers can access specialist equipment and technical support to study stem cell behaviour at single cell resolution. We also host an international seminar series and run the Stem Cells @ Lunch seminar series to share ideas and unpublished data. Our researchers are committed to public engagement and take part in diverse outreach events.Detailed information about the Centre for Stem Cells & Regenerative medicine can be found in the link below:http://www.kcl.ac.uk/lsm/research/divisions/gmm/departments/stemcells/index.aspx

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Research Associate in Stem Cells and Regenerative Medicine job with KINGS COLLEGE LONDON | 246711 - Times Higher Education (THE)

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Gergis Explains the Differences Between Acute and Chronic GVHD – Targeted Oncology

By daniellenierenberg

Usama Gergis, MD, MBA Professor of Oncology Director, Bone Marrow Transplant and Immune Cellular Therapy Sidney Kimmel Cancer Center Thomas Jefferson University Hospital Philadelphia, PA, reviewed that difference in acute and chronic graft-versus-host disease (GVHD) and the treatment available for each.

Targeted OncologyTM: How would you treat a patient with GvHD in the second line?

GERGIS: If you [have a patient with] second-line acute GVHD, your answer should be ruxolitinib [Jakafi] because its the only drug that has been tried in phase 3 trials. If you get a [case of] chronic GVHD, your answer should be ibrutinib [Imbruvica].

What is the efficacy of peripheral blood stem cells (PBSC) versus bone marrow from unrelated donors in patients with acute and chronic GvHD?

[Results from] a phase 3 study of bone marrow versus stem cells for unrelated donors [showed] the acute GVHD population [cumulative incidence] was the same between both.1 For the chronic population, the bone marrow did better [PBSC 53% vs bone marrow 41%; P = .01]. This was published almost 8 years ago, [and it] was reported almost 10 years ago, but we still use stem cells.

This has not changed practices, and the reasons are, number 1, there was more primary graft failure on the bone marrow than the PBSC, and number 2, its pretty involved to do bone marrow harvest, although I have done it for 15 years, at least a few every month.

The benefit of bone marrow versus PBSCand this benefit was only studied in unrelated donors, not in matched related donorswas seen across all organs affected with chronic GVHD except lungs, [gut, and serosa].2 So, there was no real benefit in the lungs.

Can you explain the difference between acute and chronic GvHD?

Chronic GVHD is more complicated and involved than acute GVHD. In acute, you have the skin, gastrointestinal organs, and the liver [that may be affected]. Thats it. In chronic, all the patients other organs can be affected. The patients weight can be affected. [Chronic GVHD is] more debilitating over a long time and [can] go unrecognized for a while. [If a patient is] experiencing acute GVHD, you see them twice a week, whereas if the patient has chronic GVHD, you probably see them once a month. So you can see a very stark change in your patients within that month if they lose 10% of their body weight and they already lost a lot of weight in the period right after [transplantation], so that can be obvious to you.

[In my institution], we have the GVHD clinic where we [grade the patient based on] studying the degree of fibrosis, how many organs are affected, the patients range of motion, and the degrees in range of motion. We do frequent pulmonary function tests and various [other] testing. By looking at all the affected organs, you reach a grade, and that can be mild, moderate, or severe [chronic GVHD].

How do you treat moderate-to-severe chronic GVHD at initial presentation and in the second line?

First-line treatment for chronic GVHD are steroids. For second line, there are many agents [to consider]. Ive tried most of them. I like photopheresis because its not pharmacological, but its pretty involved. Your patient will need a permanent catheter, and they will need to come to the transplant center twice a week, and you see a response after a long time. It takes an average of 50 photopheresis sessions for a response. But the beauty of photopheresis [is that] you could try it with other agents, so its not mutually exclusive. You could use it with ruxolitinib, ibrutinib, or any other agents.

The answer will be ibrutinib [for chronic GVHD], and thats based on the [results of a] phase 2 clinical trial that treated 42 patients with steroid-refractory chronic GVHD, and the efficacy was 69% [best overall response rate], and 31% complete response rate.3

What do you think of these poll results?

Everybody agrees on giving ibrutinib. When I gave this talk a couple months ago, lenalidomide [Revlimid] was not included in the poll. I added it because [recently], a nice study in Blood came out from the National Institutes of Health where they tried lenalidomide at a small dose, 2 mg, in steroid-refractory chronic GVHD. Its a large trial; I think its about 100 patients. Theyve seen responses that are comparable with ibrutinib....I treated a patient for multiple myeloma; he received a transplant for multiple myeloma, and now, 6 months later, he has chronic GVHD and some clonal plasma cells. So for him, I was comforted to know the results of the lenalidomide trial.

How does ruxolitinib play a role in this setting?

Ruxolitinib was reported in the REACH3 trial [NCT03112603] with very good responses in chronic GVHD.4 I think it probably will get approved for that indication. Looking at this study about 2 years ago, nothing was studied well in this indication, and ibrutinib was approved.

REACH3 was a large trial, almost 300 patients, and everybody was randomized to ruxolitinib 10 mg twice a day versus best available treatment. They looked at everybody about 6 months later for response.

What should physicians keep in mind when treating?

Chronic GvHD is pretty involved. Your patients will need a multidisciplinary approach. You need to pay attention to their bones. In the first 100 days post transplant, the average bone aging is 17 years.

So although were trying to treat acute GVHD, viruses, and prevent relapses, [by putting] your patients on some steroids, you are aging your patients bones by 17 years only in the first 100 days. No matter what you do, give your patients vitamin D, calcium, and Fosamax [alendronate sodium].

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Nurix Therapeutics Reports Fourth Quarter and Fiscal Year 2020 Financial Results and Provides a Corporate Update

By Dr. Matthew Watson

First Investigational New Drug (IND) application cleared for NX-2127 in patients with relapsed and refractory B-cell malignancies

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Nurix Therapeutics Reports Fourth Quarter and Fiscal Year 2020 Financial Results and Provides a Corporate Update

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Lumos Pharma Announces Changes to its Board of Directors

By Dr. Matthew Watson

Lumos Pharma appoints new Board member, An van Es-Johansson, M.D., with wealth of experience in rare diseases Lumos Pharma appoints new Board member, An van Es-Johansson, M.D., with wealth of experience in rare diseases

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Lumos Pharma Announces Changes to its Board of Directors

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CRISPR Therapeutics Provides Business Update and Reports Fourth Quarter and Full Year 2020 Financial Results

By Dr. Matthew Watson

- More than 20 patients have been dosed with CTX001™ across CLIMB-Thal-111 and CLIMB-SCD-121 to date; completion of enrollment in both trials is expected in 2021 -

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Study Highlights Potential Clinical Value of ePlex® Blood Culture ID Panels

By Dr. Matthew Watson

Major teaching hospital in Grenoble, France demonstrates that ePlex BCID results could have modified antimicrobial treatment in 45% of patients Major teaching hospital in Grenoble, France demonstrates that ePlex BCID results could have modified antimicrobial treatment in 45% of patients

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Arcutis Announces Fourth Quarter and Full Year 2020 Financial Results and Provides Business Update

By Dr. Matthew Watson

WESTLAKE VILLAGE, Calif., Feb. 16, 2021 (GLOBE NEWSWIRE) -- Arcutis Biotherapeutics, Inc. (Nasdaq: ARQT), a late-stage biopharmaceutical company focused on developing and commercializing treatments for unmet needs in immune-mediated dermatological diseases and conditions, or immuno-dermatology, today reported financial results for the quarter and year ended December 31, 2020, and provided a business update.

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Arcutis Announces Fourth Quarter and Full Year 2020 Financial Results and Provides Business Update

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Morphic Therapeutic to Participate in GI/Microbiome Panel Discussion at the 41st Annual Cowen Health Care Conference

By Dr. Matthew Watson

WALTHAM, Mass., Feb. 16, 2021 (GLOBE NEWSWIRE) -- Morphic Therapeutic (Nasdaq: MORF), a biopharmaceutical company developing a new generation of oral integrin therapies for the treatment of serious chronic diseases, today announced that Praveen Tipirneni, M.D., president and chief executive officer of Morphic Therapeutic, is scheduled to participate in a panel discussion on the gastrointestinal microbiome at the 41st Annual Cowen Health Care Conference. The panel discussion will take place at 11:10 AM ET on Tuesday, March 2, 2021.

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Eton Pharmaceuticals to Participate in 10th Annual SVB Leerink Global Healthcare Conference

By Dr. Matthew Watson

DEER PARK, Ill., Feb. 16, 2021 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc. (Nasdaq: ETON), a specialty pharmaceutical company focused on developing and commercializing innovative treatments for rare pediatric diseases, today announced that company management will be participating in the 10th Annual SVB Leerink Global Healthcare Conference from February 24th through February 26th.

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Myriad Genetics to Announce December 2020 Quarterly Financial Results on February 23, 2021

By Dr. Matthew Watson

SALT LAKE CITY, Feb. 16, 2021 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (NASDAQ: MYGN), a leader in genetic testing and precision medicine, announced that it will hold its quarterly earnings conference call for the period ending December 30, 2020 with investors and analysts at 4:30 p.m. EST on Tuesday, February 23, 2021. During the call, Paul J. Diaz, president and CEO, and R. Bryan Riggsbee, Chief Financial Officer, will provide a financial overview and business update of Myriad’s performance for the quarter.

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MediWound to Report Fourth Quarter and Fiscal 2020 Financial Results and Host a Conference Call and Webcast on February 25, 2021

By Dr. Matthew Watson

YAVNE, Israel, Feb. 16, 2021 (GLOBE NEWSWIRE) -- MediWound Ltd. (Nasdaq: MDWD), a fully-integrated biopharmaceutical company bringing innovative therapies to address unmet needs in severe burn and wound management, today announced that the Company will release its financial results for the fourth quarter and year ended December 31, 2020 at 7:00 am Eastern Time on Thursday, February 25, 2021.

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Aleafia Health Announces $15 Million Bought Deal Offering of Units

By Dr. Matthew Watson

NOT FOR DISTRIBUTION TO UNITED STATES NEWSWIRE SERVICES OR FOR DISSEMINATION IN THE UNITED STATES

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Travere Therapeutics to Report Fourth Quarter and Full Year 2020 Financial Results

By Dr. Matthew Watson

SAN DIEGO, Feb. 16, 2021 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced it will report fourth quarter and full year 2020 financial results on Monday, March 1, 2021 after the close of the U.S. financial markets. The Company will host a conference call and webcast to discuss the financial results and provide a general business update at 4:30 p.m. ET.

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ProQR Announces Expert Perspectives Call on Disease Education and Endpoints in Usher Syndrome

By Dr. Matthew Watson

LEIDEN, Netherlands & CAMBRIDGE, Mass., Feb. 16, 2021 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA therapies for inherited retinal diseases (IRDs), today announced that the Company will host an Expert Perspectives call on February 22, 2021 at 12:00pm EST. The call will feature a discussion between Aniz Girach, MD, Chief Medical Officer of ProQR Therapeutics and Paul Yang, MD, PhD about disease education and endpoints in Usher syndrome and non-syndromic Retinitis Pigmentosa (nsRP).  Areas of focus for the session will include which vision measures are most informative in the context of this disease setting, the role of patient baseline and disease progression, and an overview of the objectives of the Phase 1/2 Stellar trial of QR-421a.

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ProQR Announces Expert Perspectives Call on Disease Education and Endpoints in Usher Syndrome

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Medexus to Present at the Winter Wonderland Conference- Best Ideas from the Buy-Side

By Dr. Matthew Watson

TORONTO, CHICAGO and MONTREAL, Feb. 16, 2021 (GLOBE NEWSWIRE) -- Medexus Pharmaceuticals Inc. (the “Company” or “Medexus”) (TSXV: MDP) (OTCQX: MEDXF) (Frankfurt: P731) announced today that it will be presenting at the Winter Wonderland Conference, hosted by The MicroCap Rodeo, being held virtually on February 16th – February 19th, 2021.

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Wave Life Sciences to Present at the 10th Annual SVB Leerink Global Healthcare Conference  

By Dr. Matthew Watson

CAMBRIDGE, Mass., Feb. 16, 2021 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, announced today that Paul Bolno, MD, MBA, President and Chief Executive Officer, is scheduled to participate in an analyst-led fireside chat at the virtual 10th Annual SVB Leerink Global Healthcare Conference on Thursday, February 25, 2021 at 5:00 p.m. ET.

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