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Imago Receives Fast Track Designation from U.S. FDA for Bomedemstat for Treatment of Essential Thrombocythemia – Yahoo Finance

By daniellenierenberg

Imago BioSciences, Inc., a clinical-stage biotechnology company developing innovative treatments for myeloid diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the development of bomedemstat (IMG-7289) for the treatment of essential thrombocythemia (ET), a bone marrow disease associated with high platelet counts and potentially catastrophic vascular complications. Bomedemstat inhibits the enzyme LSD1 (lysine-specific demethylase 1), thus preventing excess platelet and neutrophil production.

"The Fast Track designation by the FDA recognizes the need for novel therapeutics for myeloid diseases and mirrors our own urgency in addressing these devastating conditions," said Hugh Young Rienhoff, Jr. M.D., CEO, Imago Biosciences. "ET is a quiet bone marrow cancer than can linger for years. In a subset of patients, the excess of platelets leads to bleeding and clotting including strokes and infractions, each having a significant impact on these patients. With only one FDA approved therapy, one that does not increase overall survival, patients are in desperate need of new options. Based on its mechanism and safety data obtained to date, we believe bomedemstat has the promise to be that new treatment."

The FDA grants Fast Track designation to facilitate development and expedite the review of therapies with the potential to treat a serious condition where there is an unmet medical need. A therapeutic that receives Fast Track designation can benefit from early and frequent communication with the agency, in addition to a rolling submission of the marketing application, with the objective of getting important new therapies to patients more quickly.

About Bomedemstat (IMG-7289)

Bomedemstat is a small molecule discovered by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme essential for production and normal function of megakaryocytes and for self-renewal of malignant hematopoietic stem or progenitor cells. Megakaryocytes are the primary producer of platelets and cytokines that drive essential thrombocythemia pathogenesis.

In non-clinical studies, bomedemstat demonstrated robust in vivo efficacy as a single agent, and in combination with other therapeutics across a range of myeloid malignancy models including the myeloproliferative neoplasms encompassing myelofibrosis, essential thrombocythemia and polycythemia vera.

The FDA has also granted Fast Track designation to bomedemstat for the treatment of myelofibrosis, which is currently being studied in an international Phase 2b study. In this study IMG-7289 was effective in reducing spleen volumes and substantially improved symptom scores in a majority of evaluable patients. For more information visit http://www.clinicaltrials.gov (NCT03136185). Additional clinical studies in hematologic disorders will begin in 2020.

About Imago BioSciences

Imago BioSciences is a clinical-stage, venture-backed pharmaceutical company whose investors include a fund managed by Blackstone Life Sciences, Frazier Healthcare Partners, Omega Funds, Amgen Ventures, MRL Ventures Fund, HighLight Capital, Pharmaron, Greenspring Associates and Xeraya Capital, as well as other corporate and venture investors.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200113005164/en/

Contacts

Ian StoneCanale Communicationsian@canalecomm.com (619) 849-5388

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Stem Cell Assay Market is estimated to witness the highest growth during the forecast period 2017 2025 – Pro News Time

By daniellenierenberg

Stem Cell Assay Market: Snapshot

Stem cell assay refers to the procedure of measuring the potency of antineoplastic drugs, on the basis of their capability of retarding the growth of human tumor cells. The assay consists of qualitative or quantitative analysis or testing of affected tissues and tumors, wherein their toxicity, impurity, and other aspects are studied.

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With the growing number of successful stem cell therapy treatment cases, the global market for stem cell assays will gain substantial momentum. A number of research and development projects are lending a hand to the growth of the market. For instance, the University of Washingtons Institute for Stem Cell and Regenerative Medicine (ISCRM) has attempted to manipulate stem cells to heal eye, kidney, and heart injuries. A number of diseases such as Alzheimers, spinal cord injury, Parkinsons, diabetes, stroke, retinal disease, cancer, rheumatoid arthritis, and neurological diseases can be successfully treated via stem cell therapy. Therefore, stem cell assays will exhibit growing demand.

Another key development in the stem cell assay market is the development of innovative stem cell therapies. In April 2017, for instance, the first participant in an innovative clinical trial at the University of Wisconsin School of Medicine and Public Health was successfully treated with stem cell therapy. CardiAMP, the investigational therapy, has been designed to direct a large dose of the patients own bone-marrow cells to the point of cardiac injury, stimulating the natural healing response of the body.

Newer areas of application in medicine are being explored constantly. Consequently, stem cell assays are likely to play a key role in the formulation of treatments of a number of diseases.

Global Stem Cell Assay Market: Overview

The increasing investment in research and development of novel therapeutics owing to the rising incidence of chronic diseases has led to immense growth in the global stem cell assay market. In the next couple of years, the market is expected to spawn into a multi-billion dollar industry as healthcare sector and governments around the world increase their research spending.

The report analyzes the prevalent opportunities for the markets growth and those that companies should capitalize in the near future to strengthen their position in the market. It presents insights into the growth drivers and lists down the major restraints. Additionally, the report gauges the effect of Porters five forces on the overall stem cell assay market.

Global Stem Cell Assay Market: Key Market Segments

For the purpose of the study, the report segments the global stem cell assay market based on various parameters. For instance, in terms of assay type, the market can be segmented into isolation and purification, viability, cell identification, differentiation, proliferation, apoptosis, and function. By kit, the market can be bifurcated into human embryonic stem cell kits and adult stem cell kits. Based on instruments, flow cytometer, cell imaging systems, automated cell counter, and micro electrode arrays could be the key market segments.

In terms of application, the market can be segmented into drug discovery and development, clinical research, and regenerative medicine and therapy. The growth witnessed across the aforementioned application segments will be influenced by the increasing incidence of chronic ailments which will translate into the rising demand for regenerative medicines. Finally, based on end users, research institutes and industry research constitute the key market segments.

The report includes a detailed assessment of the various factors influencing the markets expansion across its key segments. The ones holding the most lucrative prospects are analyzed, and the factors restraining its trajectory across key segments are also discussed at length.

Global Stem Cell Assay Market: Regional Analysis

Regionally, the market is expected to witness heightened demand in the developed countries across Europe and North America. The increasing incidence of chronic ailments and the subsequently expanding patient population are the chief drivers of the stem cell assay market in North America. Besides this, the market is also expected to witness lucrative opportunities in Asia Pacific and Rest of the World.

Global Stem Cell Assay Market: Vendor Landscape

A major inclusion in the report is the detailed assessment of the markets vendor landscape. For the purpose of the study the report therefore profiles some of the leading players having influence on the overall market dynamics. It also conducts SWOT analysis to study the strengths and weaknesses of the companies profiled and identify threats and opportunities that these enterprises are forecast to witness over the course of the reports forecast period.

Some of the most prominent enterprises operating in the global stem cell assay market are Bio-Rad Laboratories, Inc (U.S.), Thermo Fisher Scientific Inc. (U.S.), GE Healthcare (U.K.), Hemogenix Inc. (U.S.), Promega Corporation (U.S.), Bio-Techne Corporation (U.S.), Merck KGaA (Germany), STEMCELL Technologies Inc. (CA), Cell Biolabs, Inc. (U.S.), and Cellular Dynamics International, Inc. (U.S.).

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TMR Research is a premier provider of customized market research and consulting services to business entities keen on succeeding in todays supercharged economic climate. Armed with an experienced, dedicated, and dynamic team of analysts, we are redefining the way our clients conduct business by providing them with authoritative and trusted research studies in tune with the latest methodologies and market trends.

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Canine Stem Cell Therapy Market Will Make a Huge Impact in Near Future – Expert Recorder

By daniellenierenberg

A synopsis of the global canine stem cell therapy market with reference to the global healthcare pharmaceutical industry

Despite the economic and political uncertainty in the recent past, the global healthcare industry has been receiving positive nudges from reformative and technological disruptions in medical devices, pharmaceuticals and biotech, in-vitro diagnostics, and medical imaging. Key markets across the world are facing a massive rise in demand for critical care services that are pushing global healthcare spending levels to unimaginable limits.

A rapidly multiplying geriatric population; increasing prevalence of chronic ailments such as cancer and cardiac disease; growing awareness among patients; and heavy investments in clinical innovation are just some of the factors that are impacting the performance of the global healthcare industry. Proactive measures such as healthcare cost containment, primary care delivery, innovation in medical procedures (3-D printing, blockchain, and robotic surgery to name a few), safe and effective drug delivery, and well-defined healthcare regulatory compliance models are targeted at placing the sector on a high growth trajectory across key regional markets.

Parent Indicators Healthcare Current expenditure on health, % of gross domestic product Current expenditure on health, per capita, US$ purchasing power parities (current prices, current PPPs) Annual growth rate of current expenditure on health, per capita, in real terms Out-of-pocket expenditure, % of current expenditure on health Out-of-pocket expenditure, per capita, US$ purchasing power parity (current prices, current PPPs) Physicians, Density per 1000 population (head counts) Nurses, Density per 1000 population (head counts) Total hospital beds, per 1000 population Curative (acute) care beds, per 1000 population Medical technology, Magnetic Resonance Imaging units, total, per million population Medical technology, Computed Tomography scanners, total, per million population

Research Methodology

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XploreMR utilizes a triangulation methodology that is primarily based on experimental techniques such as patient-level data, to obtain precise market estimations and insights on Molecule and Drug Classes, API Formulations and preferred modes of administration. Bottom-up approach is always used to obtain insightful data for the specific country/regions. The country specific data is again analysed to derive data at a global level. This methodology ensures high quality and accuracy of information.

Secondary research is used at the initial phase to identify the age specific disease epidemiology, diagnosis rate and treatment pattern, as per disease indications. Each piece of information is eventually analysed during the entire research project which builds a strong base for the primary research information.

Primary research participants include demand-side users such as key opinion leaders, physicians, surgeons, nursing managers, clinical specialists who provide valuable insights on trends and clinical application of the drugs, key treatment patterns, adoption rate, and compliance rate.

Quantitative and qualitative assessment of basic factors driving demand, economic factors/cycles and growth rates and strategies utilized by key players in the market is analysed in detail while forecasting, in order to project Year-on-Year growth rates. These Y-o-Y growth projections are checked and aligned as per industry/product lifecycle and further utilized to develop market numbers at a holistic level.

On the other hand, we also analyse various companies annual reports, investor presentations, SEC filings, 10k reports and press release operating in this market segment to fetch substantial information about the market size, trends, opportunity, drivers, restraints and to analyse key players and their market shares. Key companies are segmented at Tier level based on their revenues, product portfolio and presence.

Please note that these are the partial steps that are being followed while developing the market size. Besides this, forecasting will be done based on our internal proprietary model which also uses different macro-economic factors such as per capita healthcare expenditure, disposable income, industry based demand driving factors impacting the market and its forecast trends apart from disease related factors.

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Standard Report Structure Executive Summary Market Definition Macro-economic analysis Parent Market Analysis Market Overview Forecast Factors Segmental Analysis and Forecast Regional Analysis Competition Analysis

Target Audience Production Companies Suppliers Channel Partners Marketing Authorities Subject Matter Experts Research Institutions Financial Institutions Market Consultants Government Authorities

Market Taxonomy

The global canine stem cell therapy market has been segmented into:

Product Type: Allogeneic Stem Cells Autologous Stem cells

Application: Arthritis Dysplasia Tendonitis Lameness Others

End User: Veterinary Hospitals Veterinary Clinics Veterinary Research Institutes

Region: North America Latin America Europe Asia Pacific Japan Middle East & Africa

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About Us

XploreMR is one of the worlds leading resellers of high-quality market research reports. We feature in-depth reports from some of the worlds most reputed market research companies and international organizations. We serve across a broad spectrum from Fortune 500 to small and medium businesses. Our clients trust us for our unwavering focus onquality and affordability. We believe high price should not be a bottleneck for organizations looking to gain access to quality information.

Contact us:XploreMR111 North Market Street, Suite 300,San Jose, CA 95113, United StatesPh.No: +16692840108E-mail id- [emailprotected]

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Ask the Expert: What are the most common types of brain tumors in children? – The Daily Progress

By daniellenierenberg

What are the most common types of brain tumors in children, and what treatment options are available?

Brain tumors are the most common solid tumors affecting children, with approximately 4,500 new cases each year in the U.S.

As brain tumors expand or block the normal pathways in the brain, the pressures inside the skull expand. As a result, symptoms of brain tumors can include headaches, seizures, lethargy, nausea and vomiting. A child experiencing progressively worsening symptoms like these should be evaluated by a pediatrician or in the emergency room. The doctors evaluation may include a scan of the brain. If the scan shows a tumor, the next step is a consultation with a neurosurgeon.

The majority of pediatric brain tumors occur in the posterior fossa (located near the bottom of the skull and the brain stem). The most common tumors include medulloblastoma, pilocytic astrocytoma, and ependymoma. Other less common tumors can occur in the cerebral hemispheres (the two main portions of the brain) and include astrocytomas, gangliogliomas, craniopharyngiomas, and germ cell tumors.

Surgery is usually the first step in treatment when a brain tumor is discovered. The goals of surgery are to determine whether the tumor is cancerous and remove all or as much of the tumor as safely as possible. At UVa Childrens Hospital, the latest technologies are utilized to help perform surgery, including intraoperative MRI, navigation, ultrasound and minimally invasive endoscopic surgery. Based on the types of cells found in the brain tumor, additional treatments may be needed. These therapies may include chemotherapy, radiation therapy, proton therapy, stem cell rescue and bone marrow transplantation and/or supportive care for rehabilitation.

More recent treatment options have focused on precision medicine and targeted drug therapy. Targeted drug treatments can cause brain tumor cells to die by blocking abnormalities present within these cells. These drugs are changing how brain tumors are treated while improving outcomes. Current research is focused on understanding the molecular basis of tumor formation and discovery of new targets for treatment.

At UVa, we are committed to providing the best neurosurgical care for children through our multidisciplinary brain tumor team, consisting of neurosurgery, neurology, pediatric oncology and radiation oncology.

For more information, visit childrens.uvahealth.com/services/pediatric-neurosurgery.

Dr. Hasan R. Syed and Dr. John Jane Jr. are pediatric neurosurgeons at UVa Childrens Hospital.

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"I tried four natural face masks in one week. Here’s exactly what they did for my skin." – Mamamia

By daniellenierenberg

Also, I think we're all trying to be kinder to the environment with our lifestyle choices, and unlike most sheet masks on the market, it's made with100 per cent natural cotton fibres that will biodegrade six months after use. The packaging itself is recyclable too.

If you've ever watched one of those videos of a baby smiling with glee after they try chocolate for the first time, that was me with chemical exfoliation. Unlike scrubs which use psychical exfoliation to buff away at dead skin, chemical exfoliants like AHAs, glycolic acid and lactic acid dissolve dead skin and sebum which can cause breakouts and dull skin.

And when it comes to products which do just that, this cult-favouritemask is one of my personal picks.

Not only does it kind of smell, and look, like theinside of a pumpkin pie, the wonderful combo of glycolic acid, fruit stem cells, Vitamin C, Manuka honey andantioxidant-rich pumpkin puree works to give you the deliciously radiant skin you crave.

In terms of tingliness, there was less sensation than the CannaCell Glow Mask but the results were equally as impressive. My pores appeared tighter, and the texture of my skin felt smoother, brighter and just... better.

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At 16, Shes a Pioneer in the Fight to Cure Sickle Cell Disease – The New York Times

By daniellenierenberg

In the months after the gene therapy infusion at Boston Childrens, her symptoms disappeared. But doctors had given her blood transfusions while she regrew her own red blood cells, so it was not clear if the absence of symptoms was because of the gene therapy or the transfusions.

As she recovered, Helen returned to her passion: dancing. One day, she came back from her school dance group and told her mother, My legs hurt. It feels funny. Ms. Cintron smiled. Thats soreness, she explained. Helen laughed. She had only known pain from sickle cell.

Helen was scheduled for her six-month checkup on Dec. 16. By then, all the transfused cells were gone, leaving only blood made by stem cells in her own marrow. The doctors would finally tell her whether the therapy was working.

The day before, she and her parents visited the New England Aquarium in Boston. She was able to stay outside on a cold, blustery day, watching one seal bully the others, barking and fighting. When Helen mentioned that her hands were cold, Ms. Cintrons stomach clenched in fear. But it was just a normal thing to feel on a winter day.

The next morning, Dr. Esrick delivered the news. Helens total hemoglobin level was so high it was nearly normal a level she had never before achieved even with blood transfusions. She had no signs of sickle cell disease.

Now you are like me, her father told her. I jump in the pool, I run. Now you can do it, too!

Her family, accustomed to constant vigilance, is only now getting used to normal life.

On Dec. 23, Helen and her mother flew to the familys new home in Arizona.

Helen recently described her transformed outlook on Facebook.

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At 16, shes a pioneer in the fight to cure sickle cell disease at Boston Childrens – Boston.com

By daniellenierenberg

BOSTON Helen Obando, a shy slip of a girl, lay curled in a hospital bed in June waiting for a bag of stem cells from her bone marrow, modified by gene therapy, to start dripping into her chest.

The hope was that the treatment would cure her of sickle cell disease, an inherited blood disorder that can cause excruciating pain, organ damage and early death.

Helen, who at 16 was the youngest person ever to undergo the therapy, was sound asleep for the big moment.

It was a critical moment in medical science.

For more than a half-century, scientists have known the cause of sickle cell disease: A single mutation in a gene turns red blood cells into rigid crescent or sickle shapes instead of soft discs. These misshapen cells get stuck in veins and arteries, blocking the flow of blood that carries life-giving oxygen to the body and causing the diseases horrifying hallmark: episodes of agony that begin in babyhood.

Millions of people globally, a vast majority of them Africans, suffer from sickle cell disease. Researchers have worked for decades on improving treatment and finding a cure, but experts said the effort has been hindered by chronic underfunding, in part because most of the estimated 100,000 people in the United States who have the disease are African American, often poor or of modest means.

The disease also affects people with southern European, Middle Eastern or Asian backgrounds, or those who are Hispanic, like Helen.

This is the story of two quests for a sickle cell cure one by the Obando family and one by a determined scientist at Boston Childrens Hospital, Dr. Stuart Orkin, 73, who has labored against the disease since he was a medical resident in the 1970s.

Like many others affected by sickle cell, the Obando family faced a double whammy: not one but two children with the disease, Helen and her older sister, Haylee Obando. They lived with one hope for a cure, a dangerous and sometimes fatal bone marrow transplant usually reserved for those with a healthy sibling as a match. But then they heard about a potential breakthrough: a complex procedure to flip a genetic switch so the body produces healthy blood.

Scientists have been experimenting with gene therapy for two decades, with mixed success. And it will be years before they know if this new procedure is effective in the long term. But if it is, sickle cell disease could be the first common genetic disorder to be cured by manipulating human DNA.

Four weeks after the infusion of stem cells, Helen was strong enough to be discharged. At home, in Lawrence, Massachusetts, on a sofa with her mother by her side, she put a hand over her eyes and started to sob. She and her family wondered: Would it work? Was her suffering really over?

A Familys Nightmare

Sheila Cintron, 35, and Byron Obando, 40, met when she was in the eighth grade and he was a high school senior. They fell in love. Haylee, their first child, was born in 2001, when Cintron was 17.

When a newborn screening test showed that Haylee had the disease, her father asked, Whats sickle cell?

They soon found out.

As the family gathered for her first birthday party, Haylee started screaming inconsolably. They rushed her to the hospital. It was the first of many pain crises.

Doctors warned the parents that if they had another baby, the odds were 1 in 4 that the child would have sickle cell, too. But they decided to take the chance.

Less than two years later, Helen was born. As bad as Haylees disease was, Helens was much worse. When she was 9 months old, a severe blockage of blood flow in her pelvis destroyed bone. At age 2, her spleen, which helps fight bacterial infections, became dangerously enlarged because of blocked blood flow. Doctors surgically removed the organ.

After Helen was born, her parents decided not to have any more children. But four years later, Cintron discovered she was pregnant again.

But they were lucky. Their third child, Ryan Obando, did not inherit the sickle cell mutation.

As Ryan grew up, Helens health worsened. When he was 9, Helens doctors suggested a drastic solution: If Ryan was a match for her, he might be able to cure her by giving her some of his bone marrow, though there would also be major risks for her, including death from severe infections or serious damage to organs if his immune system attacked her body.

As it turned out, Ryan matched not Helen but Haylee.

The transplant succeeded, but her parents asked themselves how they could stand by while one daughter was cured and the sicker one continued to suffer.

There was only one way to get a sibling donor for Helen: have another baby. In 2017, the couple embarked on another grueling medical journey.

Obando had a vasectomy, so doctors had to surgically extract his sperm from his testicles. Cintron had 75 eggs removed from her ovaries and fertilized with her husbands sperm. The result was more than 30 embryos.

Not a single embryo was both free of the sickle cell gene and a match for Helen.

So the family decided to move to Mesa, Arizona, from Lawrence, where the cold, which set off pain crises, kept Helen indoors all winter. The family had already sold their house when they heard that doctors at Boston Childrens were working on sickle cell gene therapy.

Cintron approached Dr. Erica Esrick, a principal investigator for the trial. But the trial wasnt yet open to children.

Figuring Out the Science

Nothing had prepared Orkin for the suffering he witnessed in his 30s as a medical resident in the pediatric hematology ward at Boston Childrens. It was the 1970s, and the beds were filled with children who had sickle cell crying in pain.

Orkin knew there was a solution to the puzzle of sickle cell, at least in theory: Fetuses make hemoglobin the oxygen-carrying molecules in blood cells with a different gene. Blood cells filled with fetal hemoglobin do not sickle. But the fetal gene is turned off after a baby is born, and an adult hemoglobin gene takes over. If the adult gene is mutated, red cells sickle.

Researchers had to figure out how to switch hemoglobin production to the fetal form. No one knew how to do that.

Orkin needed ideas. Supported by the National Institutes of Health and Howard Hughes Medical Institute, he kept looking.

The breakthrough came in 2008. The cost of gene sequencing was plummeting, and scientists were finding millions of genetic signposts on human DNA, allowing them to home in on small genetic differences among individuals. Researchers started doing large-scale DNA scans of populations, looking for tiny but significant changes in genes. They asked: Was there a molecular switch that flipped cells from making fetal to adult hemoglobin? And if there was, could the switch be flipped back?

They found a promising lead: an unprepossessing gene called BCL11A.

In a lab experiment, researchers blocked this gene and discovered that the blood cells in petri dishes started making fetal instead of adult hemoglobin.

Next they tried blocking the gene in mice genetically engineered to have human hemoglobin and sickle cell disease. Again, it worked.

Patients came next, in the gene therapy trial at Boston Childrens that began in 2018.

The trial run by Dr. David Williams, an expert in the biology of blood-forming stem cells at Boston Childrens, and Esrick has a straightforward goal: Were going to reeducate the blood cells and make them think they are still in the fetus, Williams said.

Doctors gave adult patients a drug that loosened stem cells immature cells that can turn into red blood cells from the bone marrow, their normal home, so they floated free in the bloodstream. Then they extracted those stem cells from whole blood drawn from the patient.

The researchers used a disabled genetically engineered AIDS virus to carry information into the stem cells, flipping on the fetal hemoglobin gene and turning off the adult gene. Then they infused the treated stem cells into patients veins. From there, the treated cells migrated into the patients bone marrow, where they began making healthy blood cells.

With the success in adults, the Food and Drug Administration said Boston Childrens could move on to teenagers.

When her mother told her about the gene therapy trial, Helen was frightened. But the more she thought about it, the more she was ready to take the risk.

In the months after the gene therapy infusion at Boston Childrens, her symptoms disappeared.

Helen was scheduled for her six-month checkup Dec. 16. Helens total hemoglobin level was so high it was nearly normal a level she had never before achieved, even with blood transfusions. She had no signs of sickle cell disease.

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What is Mantle Cell Lymphoma and How Is It Treated? – Dana-Farber Cancer Institute

By daniellenierenberg

Last Updated on January 10, 2020

Medically reviewed by Ann S. LaCasce, MD, MMSc

Mantle cell lymphoma is a rare, often aggressive form of non-Hodgkin lymphoma (NHL), a cancer that involves white blood cells known as lymphocytes, which help protect the body from disease. It is named for its origins in the mantle zone a ring of cells within the lymph nodes where B cells (a type of lymphocyte) grow and take on specialized functions. It comprises about 6% of all cases of NHL, usually arises during an individuals early 60s, and is more common in men than women.

The most common symptoms of mantle cell lymphoma include:

At the time of diagnosis,nearly all patients have disease that has spread beyond its initial site.

For most patients, the cause of the disease is unknown, but rates are higher among farmers and people from rural areas.

Itoccurs when B lymphocytes acquire genetic mutations that alter their functionand growth. One such abnormality, found in 90% of cases, causes B lymphocytesto overproduce cyclin D1, a protein that spurs the cells growth. Othermutations can interfere with B cells ability to produce infection-fightingantibodies, leaving patients vulnerable to certain diseases.

A definitive diagnosis requires a biopsy of an affected lymph node or other involved tissue.

Doctors use a variety of scans to determine the diseases stage, or how far it has advanced. These include:

Treatment for mantle cell lymphoma varies depending on patients age and overall health and the stage of the disease. Patients who have yet to develop symptoms and who have a relatively small amount of slow-growing disease may be recommended for active surveillance close monitoring of their health through regular checkups and lab tests. When lymphoma-related symptoms appear or tests show a worsening of the disease, active treatment may begin.

The initial treatment for aggressive mantle cell lymphoma in younger patients often includes a combination of chemotherapy drugs in conjunction with an antibody-based treatment, often followed by a stem cell transplant using patients own stem cells. Older, less-fit patients may undergo less intensive chemotherapy sometimes followed by a prolonged course of antibody therapy.

Other treatments may include drugs known as BTK inhibitors such as acalbrutinib and ibrutinib, which interfere with lymphoma cells internal growth signals.

In patients who relapse after treatment or dont respond to initial treatment, a variety of options may be available, including:

Clinical trials are currently underway of CAR T-cell therapy for patients with mantle cell lymphoma. The therapy, which uses genetically modified immune system T cells to attack tumor cells, has been shown to be effective in patients with other forms of non-Hodgkin lymphoma. Other trials are testing drugs known as bispecific antibodies, artificial proteins that can bind simultaneously to two surface proteins on cells, and targeted agents directed against specific cancer-related proteins.

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I chased lemurs around Madagascar to help stave off their extinction – Massive Science

By daniellenierenberg

In 2019, Massive Science covered some ground. We wrote about climate change killing off biodiversity, galaxies eating one another, parakeet mate selection, and on and on. We polled the Massive staff for their favorite stories of the year, both in what we worked on and in the outside world. But first, our top five most popular articles of the year:

#5

After another devastating intergovernmental report on wildlife loss, Cassie Freund wrote this urgent call for action. It's the very real end of the world, why isn't anyone acting?

#4

Neuroscience is Massive's bread-and-butter, so Claudia Lopez-Lloreda's story on fish giving up and the brain cells responsible checked a lot of boxes. Next time you quit on something, you'll know who's at fault.

#3

"Unexpected science" is another angle our writers have gotten serious mileage out of, and Darcy Shapiro's article on gorilla teeth, snacks, and how that changes human history is a classic of the genre.

#2

Definitely another "unexpected science" entry, Molly Sargen's story combined math, bridge building, and breakfast food. Now we know our audience likes that and more breakfast food science will be coming in 2020.

#1

It's got it all: space and a vague sex angle. What more could you want? You might say that Mackenzie Thornbury's article went viral. We won't though.

Sometimes though, what we think is cool and what you all think is cool doesn't match up. We're not mad about though, we know disagreement is natural. Not mad at all. Here are our personal faves that we think you should give a second shot. No pressure though!

Or, as it was more affectionately known in the Slack channel: Babies...in...SPAAAAAAACE.

Another Cassie Freund work on the actual human effort to get around conservation efforts that other humans are employing to save the planet.

"Connecting brains" is a sub-genre of our normal neuroscience work and Jordan Harrod wrote one of the best ones we've ever seen.

Yeah, the science is cool, the writing is great, but you know what really spiced up Luyi Cheng's debut article? The gifs.

We love all Our Science Heroes equally, but there's something about du Chtelet. If a man had had her adventurous, influential life that included standing on Newton's shoulders and having Voltaire as a kept man, there'd be movies made about that man's life. This is our pitch for a du Chtelet biopic. Hollywood, please call us.

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From stylish clothes to indoor gardening, 9 tips and trends to try this winter – Commercial Appeal

By daniellenierenberg

Jean Chen Smith, Correspondent for Memphis Commercial Appeal Published 6:00 a.m. CT Jan. 10, 2020

During the first part of winter, the holidays can be all-consuming from shopping to stressing to spending time with friends and family.But there's so much more to the season than that.

Here are nine tips and trends to keep things fresh this winter:

With shorter days and busy schedules, it becomes more difficult to eat delicious and nutritious meals. Heres where Fresh n Lean comes in. This healthy and convenient service was conceptualized by founder Laureen Asseo while preparing meals for friends and family during a time when her father was facing serious health concerns. The meal delivery service provides specialized categories such as Keto, Paleo and Vegan using the highest quality organic ingredients without preservatives or added sugar.The cost is as low as $11 a meal, and menu choices include dishes such as Coconut Chicken Curry with Mixed Vegetables and Chile Lime Salmon with Broccoli.The dishes are never frozen and can be heated up in less than 3 minutes.

Details: freshnlean.com

Bring the outdoors inside with the AeroGarden Harvest Elite Slim.(Photo: AeroGarden)

With the AeroGarden Harvest Elite ($125), you can grow an indoor garden of gourmet herbs, heirloom salad greens, red heirloom cherry tomatoes or cascading petunias. Easy to use and sleek with a premium brushed stainless-steel finish, the AeroGarden requires minimal care and can be set on vacation mode, all the while dressing up the kitchen countertop.

Details: aerogarden.com/harvest-elite-slim.html

... Or maybe it never left. When more is required of you than the Casual Friday outfit, men will find the Treffort Contemporary Fit Signature Oxford Shirt ($165) is a wardrobe essential for any office.The mens shirts are made with premium 100% Egyptian and Turkish cotton, using organic colorants that boast unique patterns to create soft comfort with minimal creasing. The company strives for eco-friendly production without sacrificing quality.

Details: treffortshirts.com

The SOUVENIR Toulouse Top is inspired by world travel.(Photo: SOUVENIR)

The silk Moroccan-inspired Toulouse Top ($300) bySOUVENIR is a beautiful novelty printed silk top that is super versatile.It can be worn with a structured woven jacket for a business lunch, free flowing out to dinner or as a pullover on vacation.Based in Los Angeles, the brand is inspired by travel and world culture, with a focus on bright colors and fun prints.

Details: escapetosouvenir.com

Just because its cold outside and you need to bundle up doesnt mean you cant look good.For men, Obermeyers Down Snowshirt ($159) is the perfect remedy with itscasual, lightweight fabric and engineered quilting. Featuring certified 550 Fill Power Duck Down, snap placket, cuffs, chest and hand warmer pockets, it can be worn as a layering piece or as a casual top.

Details: obermeyer.com

Orvis men's flannel provides casual warmth.(Photo: Orvis)

Orvis Mens Flat Creek Tech Flannel ($98) is comfortable and made from recycled oyster shells and PET polyester (think plastic bottles) to create an eco-blend that is then brushed to a soft hand.

Details: http://www.orvis.com

Available for both men and women, Gobi Heats outerwear such as the Victoria Heated Puffer Coat and Sahara Mens Heated Jacket are wind- and water-resistant, keeping you warm and dry throughout the season.Stylish and comfortable, the jackets have an easy one-touch LED controller with three heat settings: low, medium and high.

Details: gobiheat.com

Frances Austen cashmere Is the ultimate staple for your winter wardrobe.(Photo: Frances Austen)

Cashmere is one of the most precious and sought-after fibers because of its soft, silken feel.Frances Austenmakes the same heirloom-quality luxury cashmere that your grandmother owned, but at a more reasonable price point. The company partners with Scotland-basedJohnstons of Elgin, sweater manufacturer to big European fashion houses, and only releases two collections a year.The companys best-seller, Raw Edge Crew Neck(prices vary), is spun in Italy and perfectly matches with a pair of dress pants or jeans. It's also available in a colorblock version.

Details: francesausten.com

Lascanas Quilt Panel Moto Jacket ($89) is not only stylish and reasonably priced, it looks like the real deal while it is faux leather. With chic rose gold zippers and a flattering fit, this is a great item for any size and shape.

Details: http://www.lascana.com

Cat lovers will rejoice at how easy it is to feed fresh, human-grade food to their furry friend with Smalls, which ships directly to your door. The company makes freeze-dried raw kibble and treats that are specifically tailored to fit your cats nutritional needs. You simply add warm water, mix and serve.Smalls believes in giving back and works with shelters and animal nonprofits across the United States to provide quality food to cats in need.With their Shelter Program, shelters can order Smalls in bulk at cost-pricing.

Details: smallsforsmalls.com

The new CannaCell line from Andalou Naturals is super-powered by antioxidants from hemp stem cells, which help to defend against free radicals and counteract premature aging due to oxidative damage, pollutionand UV sun exposure. Hemp stem cells and hemp seed oil provide the skin with nourishing proteins, nutrients and minerals so that the products penetrate the skin for optimal results.

Details: andalou.com

Check out Revos 1985 sunglasses collection, which pays homage to the '80s with sixclassic unisex frame styles that not only look great but are also functional. Heralded as the leader in polarized lens technology, the brand was created using technology developed by NASA as solar protection for satellites.

Details: http://www.revo.com

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What will be the biggest scientific breakthrough of 2020? – Stuff.co.nz

By daniellenierenberg

Cheap, re-purposed cancer drugs, negative carbon-emissions technology, calculating how fast the universe is expandingand huge leaps forward in quantum computing.

Will one of these be the biggest scientific breakthrough of 2020?

We asked a handful of New Zealand's top scientists what "Eureka!" moments might be on the cards next year but even with their formidable combined brain power and expertise, some found it hard to answer and hinted it was difficult to sheet home specific advances to any one year.

And, as one scientist says, 2020's most ground-breaking discovery may come as a total surprise, made accidentally by a student in a lab late one night.

READ MORE:*Scientists are baffled: What's up with the universe?*Medicine already in use may help cancer treatments*Roger Hanson: How you figure out the age of the universe*Doing my part not only to be carbon neutral but carbon negative

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2020's most ground-breaking discovery may come as a total surprise, made accidentally by a student in a lab late one night.

Wellington's Gillies McIndoe Research Institute is carrying out cutting-edge research into ways of treating cancer without radiotherapy, chemotherapy or surgery.

The institute's founder and executive director, Dr Swee Tan, believes significant steps will be made in 2020 towards "re-purposing" existing drugs for cancer treatment.

Tan, a plastic surgeon and medical researcher who has received international recognition for dealing with life-threatening and disfiguring conditions, saiddrugs licensed and marketed for a particular treatment often had other benefits.

"They can be re-purposed for another condition, for the treatment of cancer. This is usually with drugs that have been around for some time they are off-patent, so they become generic, which means they cost next to nothing.

"Another advantage is their safety profile is well understood."

Wellington's Gillies McIndoe Research Institute's founder and executive director, Dr Swee Tan, believes significant steps will be made in 2020 towards "re-purposing" existing drugs for cancer treatment.

Globally, 18 million new cancer cases are diagnosed each year, resulting in nearly 10 million deaths.

"In New Zealand alone, new cancer cases are about 25,000 a year, and on top of that there are 11,000 non-melanoma skin cancers.

"The incidence of cancer is predicted to increase by 50 per cent by 2035, which is just around the corner. We are completely unprepared for that."

The increased incidence of cancer largely bowel, breast, prostate and lung cancers, and melanoma is mostly because people are living longer and due to lifestyle and diet, Tan says.

"Part of the problem now is the expense of treatment. In New Zealand, we spend $1 billion a year to treat cancer, and that is just the fiscal cost, that doesn't count emotional or personal.

"The cost is escalating because of the novel cancer drugsand, at some point, we are not going to be able to afford treatment for cancer.

"This creates disparity in access to treatment because some of these drugs are not funded and, if you have the means, you can buy it, but I don't think that is a good way to run a society. I think a society should allow people to access healthcare, regardless of personal circumstance."

Royal Society Te Aprangi/VIMEO

New Zealand is as vulnerable as all countries to the global growth in antimicrobial resistance that is making some diseases untreatable. Dr Siouxsie Wiles, a microbiologist from the University of Auckland, a Royal Society Te Aprangi Councillor and an expert adviser on the report on antimicrobial resistance produced by Royal Society Te Aprangi explains why it is such a big issue for us.

The institute has been undertaking a clinical trial based on drug re-purposing to control cancer stem cells, the proposed origin of cancer.

"It consists of a combination of low-cost, off-patent, oral medications to control cancer stem cells. We believe this would be more effective than using a single drug.

"For the cost of the drug itself, it costs about $4000 a year a patient, compared with the average cancer treatment, which is about $50,000 per patient.

"You have to prove it is effective first. The big issue with drug re-purposing is 'big pharma' is not interested because there is no money in it. The only way that we can realise the potential is for philanthropy and government to support initiatives like this."

Propranolol, a beta-blocker, had been added to the treatment for melanoma and angiosarcoma.

During 2020, GMRI would also be working on treatments for disfiguring keloid scars.

"They can cause quite significant issues with quality of life. They affect about 2 per cent of the general population, but in dark-skinned races, especially from the African continent, incidence can be up to 16 per cent.

"Treatment is really quite unsatisfactory, hence the reason why we are researching a solution. If you do surgery to remove it, almost every single one returns. Sometimes surgery is followed by radiotherapy to prevent recurrence. Topical chemotherapy is also used.

"What we have found is stem cells as the underlying problem. We are doing further work, which may allow us to develop a simpler, more effective, low-cost treatment taken by mouth, or by applying to the keloid lesion directly."

University of Auckland molecular biologist Dr Hilary Sheppard, a specialist in developmental and stem-cell biology, thinks there will be more emphasis next year on the gene-editing of adult cells.

University of Auckland microbiologist,associate professor Siouxsie Wiles, a specialist in infectious diseases and antimicrobial resistance, says there are "desperately" needed breakthroughs in her field next year and beyond, including:

- Rapid "bedside" diagnostic tests that are cheap and can tell the medical practitioner if the patient has a bacterial or viral infection "a bonus if it can tell, if bacterial, what antibiotics would kill the bacterium responsible".

- Effective vaccines for tuberculosis, gonorrhoea, Staphylococcus aureus, Group A and B Streptococci, giardia "I could go on and on".

- Drugs that can kill carbapenemase-producing Enterobacteriaceae "a very scary group of organisms that are becoming untreatable".

"The other breakthroughs we need aren't scientific, they are political," she says.

"[We need] a global agreement on tackling antimicrobial resistance, which would include incentives to bring the pharmaceutical industry back in to antimicrobial development.

"Failing that, nationalisation of pharmaceutical companies so that development of drugs isn't a for-profit initiative."

Phil Doyle/Stuff

University of Auckland microbiologist, associate professor Siouxsie Wiles, says a global agreement on tackling antimicrobial resistance is desperately needed.

University of Auckland molecular biologist Dr Hilary Sheppard, a specialist in developmental and stem-cell biology, thinks there will be more emphasis next year on the gene-editing of adult cells.

"We have seen some major breakthroughs this year, such as the versatile tool which allows for gene-editing with increased precision over existing tools so the technique is becoming more reliable and safer. Hopefully, next year, we will see these newer techniques being tested in clinically relevant cells.

"As part of that, I hope we will see a community-wide discussion about the ethics of gene-editing with a particular focus on adult cells. Personally, I do not think we should be editing germline cells or embryos at least not for the next five years, while the ethical issues are debated.

"Part of gene-editing is knowing what DNA sequence needs to be edited, so I think I hope this could be the year where personalised medicine and individual genotyping takes off.

"Our research focuses on patients with a fragile skin condition called epidermolysis bullosa (EB). We are pushing to get patients with EB genotyped so that we can perform gene-editing on their skin cells we can't do anything useful without this information.

"Currently, we are paying for the genotyping out of our research budgets. Of course, genotyping raises its own ethical and societal issues, so I hope we will see more discussion about this."

In 2020, results should start rolling in from clinical trials using edited T-cells against melanoma and edited bone-marrow cells to treat patients with sickle-cell anaemia, Sheppard says.

"These are very exciting times. I'm sure we'll see more clinical trials targeting previously untargeted disorders soon perhaps for conditions like Duchenne muscular dystrophy and cystic fibrosis."

RNZ

In this podcast, The Detail's Sharon Brettkelly talks to Auckland University physics professor Shaun Hendy about his no-flying mission for the whole of last year.

While Tan, Sheppard and Wiles are looking for discoveries on the tiniest of scales, other scientists are grappling with the biggest question in the universe.

Theoretical cosmologist and University of Auckland professor of physics Richard Easther is among those hoping for a resolution of what has become known as "Hubble tension" a growing disagreement in calculations of how fast the universe is expanding, which has repercussions on its likely age.

The Hubble Constant the number that tells us how fast the universe was expanding has always been hard to measure, he says.

"There was a period of time when a whole different bunch of approaches to measuring it had converged on a single value, which is fascinating. But just over the last couple of years, it seems like there are now two different sets of numbers you get and they've pulled apart a little bit.

"You know, there's this joke that science isn't so much about people saying 'Eureka!' but about someone looking at something and going, 'well, you know, that's funny'.

"This is increasingly resembling one of those moments.

"The numbers are clustering around two values one that would put it in the early to mid-70s [kilometres per second per megaparsec], and one that would put it in the mid- to late 60s, and the uncertainty in the measurements is such that they don't really overlap with each other.

"As the individual measurements get more accurate, the sharpness of the disagreement is growing."

University of Auckland physicist, professor Shaun Hendy, is expecting leaps ahead in clean energy in 2020.

Does it matter? Of course, Easther says.

"There are different physical assumptions that go into the different measurementsandso, if there is a real discrepancy, it would tell us there's something about the expansion of the universe that we don't understand.

"It's hinting at that. The implication seems to be that the story is one step more complicated than current models of the expanding universe might recognise.

"One thought is, that in one set of numbers, there's something that got missed and kind of got away. The other possibility is, there's something kind of physical, that isn't included in our current thinking of the expanding universe.

"The idea that there is something interesting going on is something that cosmologists over the course of the last year have grown substantially more willing to entertain."

Chris Skelton/STUFF

Nicola Gaston is an Associate Professor in the Department of Physics at the University of Auckland and Co-Director of the MacDiarmid Institute for Advanced Materials and Nanotechnology.

At the University of Otago, associate professor Mikkel Andersen, a physicist in the university's Dodd-Walls Centre for Photonic and Quantum Technologies, has been making astounding international discoveries and controlling the movement of individual atoms in a world-first laboratory experiment.

Such control opens up possibilities for a "second quantum technology revolution" and quantum supremacy, something Andersen says will creep closer in 2020.

The first quantum revolution was made possible by the discovery of quantum mechanics in the 1920s, leading to the development of transistors and lasers, the building blocks of all computers.

In the second, he says quantum computers of fewer than 100 atoms will ultimately be able to out-compete "the world's combined conventional computing power".

"Reaching quantum supremacy means that a quantum computer will be able to do calculations that cannot be done on the world's conventional computers. I do not know if it will happen next year, but it will happen eventually.

"In recent years, Google, IBM, Microsoft and a lot of others have invested enormously in development of quantum computers. Quantum supremacy is likely still some years away, but it is one of those things that would clear all the headlines if it happened in 2020."

University of Auckland physicist,professor Shaun Hendy, agrees.

"Google declared quantum supremacy last month they demonstrated that a quantum computer could beat a conventional computer, albeit at a very niche task.

"We'll see more of this next year, as quantum computers start to stretch their legs just don't expect to see one on your phone any time soon."

Ross Giblin

Victoria University of Wellington's professor James Renwick hopes to see breakthroughs in climate change science which reduce greenhouse gas emissions.

Hendy is also expecting leaps ahead in clean energy in 2020.

"We will continue to see the cost of solar and battery technologies fall, to the extent that they will start to disrupt other energy systems. We have seen this already in Australia, where it has become a defining political issue.

"It will play out differently in New Zealand, because our grid is already more than 80 per cent renewable, while many of our industrial energy systems are not. Expect to see some of our big industrial corporates Fonterra, NZ Steel etc moving to greener industrial processes."

Victoria University of Wellington's Professor James Renwick, head of the school of geography, environment and earth sciences, hopes to see breakthroughs in climate change science which reduce greenhouse gas emissions.

He points to work being done at the Cawthron Institute in Nelson into the benefits of using the seaweed Asparagopsis armata as cattle feed. Chemicals in the red seaweed reduce microbes in the stomachs of cattle that make them burp when eating grass.

Renwick is also excited about the use of artificial intelligence (AI) and machine learning to help with severe weather prediction.

"Weather forecasters are totally inundated with information these days, volumes of radar data and satellite data coming through every 10 minutes. So, AI can help in making sense of all that, and what is the most important in determining where, for example, a severe storm will happen."

University of Otago associate professor Mikkel Andersen believes a "second quantum technology revolution" and quantum supremacy will creep closer in 2020.

Auckland University of Technology senior lecturer Dr Mahsa Mohaghegh also foresees huge steps forward in AI and its applications next year and beyond.

"In the medical sector, AI is being used to speed up symptom recognition and diagnosis. Early warning signs can be easily detected, allowing fast reaction.

"Environment and climate monitoring using AI can assist with weather-cycle predictions, frost warnings, and harvest alerts. Automated irrigation is possible using moisture and temperature sensors."

There are also uses in New Zealand's burgeoning space industry, in traffic management and in the "smart home" of the future, she says.

"New Zealand is a leader and frontrunner in the development of AI and related fields. The next 10 years of technology development are set to be exciting."

David White

Auckland University of Technology senior lecturer Dr Mahsa Mohaghegh foresees huge steps forward in AI and its applications next year.

University of Auckland physicist, associate professor Nicola Gaston, co-director of the Victoria University of Wellington-hosted MacDiarmid Institute for Advanced Materials and Nanotechnology, told Stuff scientific discovery did not happen "one year at a time".

"Discoveries that impact on our lives next year will be built on work that has been going on for decades. The biggest discoveries of next year will be the ones that impact on our lives in a decade or two.

"But there is no competition between this fundamental scientific work of discovery and the development of technologies. The two go in tandemand, perhaps in 2020, we can try to appreciate that."

That may be finding a way of moving to negative emissions technologies, or changing the chemistry of materials so they are recyclable and avoid environmental pollution.

"The most important breakthrough of 2020 will be one that none of us sees.

"It'll be a dedicated student or post-doc in a lab somewhere, or up late at night on a computer, who solves the last remaining piece of one of the puzzles that underpin so much of what we hope technology can do for us in the future."

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Kadimastem Finishes Treating Second Group of ALS Patients with AstroRx in Phase 1/2a Trial – ALS News Today

By daniellenierenberg

Kadimastem has finished treating its second group of participants in a Phase 1/2a clinical trial testing the safety and preliminary efficacy of AstroRx, aninvestigational stem cell therapy for amyotrophic lateral sclerosis (ALS).

AstroRx is an off-the-shelf cell therapy consisting of fully mature astrocytes star-shaped cells derived from human embryonic stem cells that are injected into the fluid surrounding the spinal cord to support damaged motor neurons.

Astrocytes usually help maintain a healthy environment in the brain, but often malfunction in ALS, contributing to disease progression. AstroRx was designed to compensate for diseased astrocytes and prevent the loss of motor nerve cells. The therapys goal is to potentially slow disease progression, improve quality of life, and extend life expectancy.

The U.S. Food and Drug Administration grantedAstroRx orphan drug status in November 2018 for the treatment of ALS, a designation meant to accelerate the development of AstroRx for this rare condition.

Earlier preclinical studiesshowed that AstroRx delayed disease onset, maintained muscle function, and prolonged survival in mice and rat models of ALS. The treatment also was found to be safe, with no signs of toxicity.

The ongoing, open-label, Phase 1/2a trial (NCT03482050) is testing the safety and preliminary signs of efficacy of AstroRx in people with ALS. Underway at the Hadassah Ein-Kerem Medical Center in Israel, the trial is expected to enroll 21 patients, ages 18-70, with early stage disease. Recruitment is ongoing.

The trial was originally designed to test three doses of AstroRx delivered into the spinal canal: a low dose of 100 million cells, a medium dose of 250 million cells, and a high dose with two consecutive administrations of 250 million cells.

However, after promising early efficacy results from the low-dose group (cohort A), Kadimastem is seeking to amend the trial protocol. It wants the third group of patients (cohort C) to receive two consecutive injections of the low dose, instead of the originally planned medium dose. Pending positive safety and efficacy results from the first three cohorts, a fourth group (cohort D) will receive two injections of the medium dose.

Safety and tolerability are the studys primary outcome measures. Secondary goals include changes in patients ALS Functional Rating Scale revised (ALSFRS-R) scores, respiratory muscle strength, hand grip strength, limb muscle strength, and quality of life.

In cohort A, the low-dose group, the therapy was found to be safe, with no serious side effects or dose-limiting toxicities. Participant had increased ALSFRS-R scores in the three months after treatment, suggesting a gain in functional abilities.

The trial has now treated all five ALS patients in cohort B, the second group, with the medium dose. Participants will be monitored for six months to evaluate the therapys safety and preliminary efficacy. The company expects to report the results for this group in August 2020.

Completing treatment for the additional 5 ALS patients in Cohort B, for a total of 10 patients treated with our product in our clinical trial, serves as an additional demonstration of our ability to develop and produce high quality clinical grade cells and takes us a significant step forward in our path to bringing innovative cure to ALS, Rami Epstein, CEO of Kadimastem, said in a press release.

The expected completion of cohort B 6-months follow-up period will allow us to assess the safety and preliminary efficacy of [250 million]cells, compared to that of the lower dose administered in cohort A, he added.

Kadimastem has recruited the first patient of cohort C, who all will receive two AstroRx injections of 100 million cells, separated by 2-3 months. Results from this group are expected during the first half of 2021.

The results of the next treatment group, Cohort C, in which each patient will be treated with two consecutive injections separated by an interval of 2-3 months, will allow us to assess the possible prolonged efficacy of the repeated dose, compared to the single dose treatment provided in cohorts A and B, said Michel Revel, founder and chief scientific officer of Kadimastem.

The results that will be obtained from the different cohorts, will support us in the process of defining the dose and treatment regimen that will lead to most favorable results for patients over time, Revel said.

Alejandra has a PhD in Genetics from So Paulo State University (UNESP) and is currently working as a scientific writer, editor, and translator. As a writer for BioNews, she is fulfilling her passion for making scientific data easily available and understandable to the general public. Aside from her work with BioNews, she also works as a language editor for non-English speaking authors and is an author of science books for kids.

Total Posts: 6

Ins holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Cincias e Tecnologias and Instituto Gulbenkian de Cincia. Ins currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.

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Jasper Therapeutics Announces Expansion of Series A Financing, Bringing Total Corporate Fundraising to More than $50 Million – Business Wire

By daniellenierenberg

PALO ALTO, Calif.--(BUSINESS WIRE)--Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, today announced the expansion of its Series A financing with an additional investment of $14.1 million led by Roche Venture Fund and with participation from other investors, bringing the total company financing to more than $50 million to date. The initial Series A round was led by Abingworth LLP and Qiming Venture Partners USA, with further investment from Surveyor Capital (a Citadel company) and participation from Alexandria Venture Investments, LLC.

Jasper plans to use the proceeds to advance and expand the study of its lead clinical asset, JSP191. A humanized antibody targeting CD117 on hematopoietic stem cells, JSP191 is designed to replace toxic chemotherapy and radiation therapy as conditioning regimens to prepare patients for curative stem cell and gene therapy. JSP191 is the only antibody of its kind in clinical development as a single conditioning agent for people undergoing curative hematopoietic cell transplantation.

This investigational agent is currently being evaluated in a Phase 1/2 dose-escalation and expansion study as a conditioning agent to enable stem cell engraftment in patients with severe combined immunodeficiency (SCID) who received a prior stem cell transplant that resulted in poor outcome. Initial positive results from this ongoing clinical trial were presented in an oral session at the American Society of Hematology (ASH) Annual Meeting in December 2019. Jasper plans to expand the Phase 1/2 clinical study to include patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) receiving hematopoietic cell transplant. The development of JSP191 is supported by a collaboration with the California Institute for Regenerative Medicine (CIRM).

About Hematopoietic Cell TransplantationBlood-forming, or hematopoietic, stem cells are rare cells that reside in the bone marrow and are responsible for the generation and maintenance of all blood and immune cells. These stem cells can harbor inherited or acquired abnormalities that lead to a variety of disease states, including immune deficiencies, blood disorders or hematologic cancers. Replacement of the defective or malignant hematopoietic stem cells in the patients bone marrow by transplantation and engraftment of healthy stem cells is the only cure for most of these life-threatening conditions. Successful transplantation is currently achieved by subjecting patients to toxic treatment with radiation and/or chemotherapy followed by transplantation of a donor or gene-corrected hematopoietic cell graft. These toxic regimens cause DNA damage and lead to short- and long-term toxicities, including unwanted damage to organs and prolonged hospitalization. As a result, many patients who could benefit from a hematopoietic cell transplant are not eligible. New approaches that are effective but have minimal to no toxicity are urgently needed so more patients who could benefit from a curative stem cell transplant could receive the procedure.

Safer and more effective hematopoietic cell transplantation regimens could overcome these limitations and enable the broader application of hematopoietic cell transplants in the cure of many disorders. These disorders include hematologic cancers (e.g., myelodysplastic syndrome [MDS] and acute myeloid leukemia [AML]), autoimmune diseases (e.g., lupus, rheumatoid arthritis, multiple sclerosis and Type 1 diabetes), and genetic diseases that could be cured with genetically-corrected autologous stem cells (e.g., severe combined immunodeficiency syndrome [SCID], sickle cell disease, beta thalassemia, Fanconi anemia and other monogenic diseases).

About JSP191JSP191 (formerly AMG 191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from 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, causing the stem cells to undergo cell death and creating an empty space in the bone marrow for donor or gene-corrected transplanted stem cells to engraft.

Preclinical studies have shown that JSP191 as a single agent safely depletes normal and diseased hematopoietic stem cells, including in an animal model of MDS. This 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 80 healthy volunteers and patients. It is currently being evaluated 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, which is curable only by this type of treatment. For more information about the design of the clinical trial, visit http://www.clinicaltrials.gov (NCT02963064). Clinical development of JSP191 will be expanded to also study patients with AML or MDS who are receiving hematopoietic cell transplant. IND-enabling 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 TherapeuticsJasper Therapeutics is a biotechnology company focused on hematopoietic cell transplant therapies. 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 https://jaspertherapeutics.com.

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Jasper Therapeutics Announces Expansion of Series A Financing, Bringing Total Corporate Fundraising to More than $50 Million - Business Wire

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Q&A: Cancer Death Rates Are Falling Nationally. Here’s What’s Happening at UVA – University of Virginia

By daniellenierenberg

This week, the American Cancer Society released some very welcome news: the cancer death rate in the U.S. dropped by 2.2% from 2016 to 2017, the largest single-year drop ever recorded.

The drop, which the report attributes to plummeting smoking rates as well as new screening and treatment methods, continues a decades-long trend, as cancer death rates have fallen by nearly 30% since 1991 about 2.9 million fewer deaths.

Dr. Thomas Loughran, director of the University of Virginia Cancer Center, said UVA is in step with this national trend.

The UVA Cancer Center is one of 71 National Cancer Institute-designated treatment centers nationwide and ranked among the nations top 50 cancer centers over each of the past four years (No. 26 last year). The center serves approximately 4 million people in Virginia and West Virginia.

We spoke with Loughran about what he is seeing at UVA and beyond, new treatments and research helping to eradicate cancer, and where he sees cancer treatment in five years.

Q. Why have cancer death rates dropped so significantly?

A. As reports of this latest drop have said, a large part of the decline can be attributed to declining rates of lung cancer. The importance of preventing cancer particularly behavioral interventions like stopping smoking has become more prominent, and there have been remarkable declines in smoking across the United States.

This is a very important focus for us at UVA. We serve a large geographical area 90 contiguous counties in Virginia and West Virginia, including rural Appalachia. Southwest Virginia in Appalachia still has high smoking rates, and as a result, high rates of lung cancer. Education, screening and tobacco cessation programs are critically important, especially in those areas.

Q. What advances in treatment have contributed to falling cancer death rates, nationally and at UVA?

A. Screening technology, especially for the more common cancers like lung, colorectal, prostate and breast cancer, has improved. The latest report probably doesnt fully reflect recent implementation of lung cancer screening using a low-dose CT scan, recommended for high risk individuals and especially those with a history of heavy smoking. That has only been around a few years, and its impact will likely show up in future reports.

The second big factor is the development of immunotherapy [cancer treatments that utilize and help the patients immune system]. UVA has invested quite a lot of institutional resources in becoming a state-of-the-art immunotherapy center, and I am proud to say we are a leader in the field.

We have created a Cancer Therapeutics Program to support the development of new therapies. Dr. Craig Slingluff, who leads that program, is a surgical oncologist internationally famous for immunotherapy treatments for melanoma. To strengthen this program, we have recruited a cadre of leading physician scientists from across the country. Dr. Karen Ballen came here to lead our stem cell and bone marrow transplant program. Dr. Lawrence Lum, the scientific director of the transplant program, has developed a novel therapy using antibodies that bind to both T-cells [patient cells that can kill cancer cells] and tumor cells, forming a bridge between the two that helps the T-cells kill the cancer cells. Dr. Trey Lee is a leader in CAR-T cell therapy.

I could keep going; there are so many great people working on this. We also have a new Good Manufacturing Practice lab, supported by a grant from the commonwealth, that will help us grow and modify T-cells as needed and give them to patients under sterile conditions. That just opened and we are very excited about that program.

Q. What other areas of research have shown great promise?

A. Some of our work in nanotechnology is really unique and exciting. [Biomedical engineering professor] Mark Kester directs UVAs nanoSTAR Institute, which is working on delivering cancer therapies by nanotechnology basically, engineering at a very small scale. For example, nanoliposomes a sort of delivery system for cancer therapy are actually smaller than individual cells and can therefore penetrate cancer cells and release treatment from inside those cells.

We are very excited about early phase trials testing this technology on solid tumors, and we also hope to use it to treat patients with acute leukemia over the next few years.

Q. Looking ahead, where do you see the next big gains coming from?

A. Immunotherapy has revolutionized cancer treatment, but why some patients respond well and some dont remains puzzling. I hope that we can begin to discover why some patients are reacting to these newer treatments differently than others. Once we figure out why some patients respond to immunotherapy, we can begin to make improvements that could benefit a larger percentage of patients with these deadly cancers.

CAR T Cell therapy one method of immunotherapy is very effective against leukemia, lymphoma and cancers of the blood, but not yet against solid tumors. Over the next five years, I hope we can determine how to deliver these T-cells to solid tumors such as those found in lung, colorectal and other common cancers again to make this advance more widely applicable to a larger number of patients.

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Stem cell therapy on skin: Mechanisms, recent advances and …

By daniellenierenberg

Highlights

Skin stem cells and the mechanism of skin repair: illustrating how skin stem cells are involved in the skin repair process.

Cell products for skin repair: the history of skin repair products and newly emerging medications applying stem cell biology.

Drug reviewing issue of skin cell products: the disputations of dermatological drug development and critical recommendations.

Stem cell products and its clinical applications have been widely discussed in recent years, particularly when the Japanese induced pluripotent stem cells founder Dr. Yamanaka was awarded as Nobel Prize laureate in 2013. For decades, major progresses have been achieved in the stem cell biology field, and more and more evidence showed that skin stem cells are involved in the process of skin repair. Stem/progenitor cells of the epidermis are recognized to play the most essential role in the tissue regeneration of skin. In this review, we first illustrated basic stem cell characteristics and various stem cell subtypes resided in the skin. Second, we provided several literatures to elucidate how stem/progenitor cells collaborate in the process of skin repair with the evidence from animal model studies and invitro experiments. Third, we also introduced several examples of skin cell products on the pharmaceutic market and the ongoing clinical trials aiming for unmet medical difficulties of skin. Last but not least, we summarized general reviewing concerns and some disputatious issues on dermatological cell products. With this concise review, we hope to provide further beneficial suggestions for the development of more effective and safer dermatological stem/progenitor cell products in the future.

Cell therapy

Dermatology

Drug reviewing

Recommended articlesCiting articles (0)

2017 Food and Drug Administration, Taiwan. Published by Elsevier Taiwan LLC.

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White blood cells: Function, ranges, types, and more – Medical News Today

By daniellenierenberg

White blood cells circulate around the blood and help the immune system fight off infections.

Stem cells in the bone marrow are responsible for producing white blood cells. The bone marrow then stores an estimated 8090% of white blood cells.

When an infection or inflammatory condition occurs, the body releases white blood cells to help fight the infection.

In this article, learn more about white blood cells, including the types and their functions.

Health professionals have identified three main categories of white blood cell: granulocytes, lymphocytes, and monocytes. The sections below discuss these in more detail.

Granulocytes are white blood cells that have small granules containing proteins. There are three types of granulocyte cells:

These white blood cells include the following:

Monocytes are white blood cells that make up around 28% of the total white blood cell count in the body. These are present when the body fights off chronic infections.

They target and destroy cells that cause infections.

According to an article in American Family Physician, the normal range (per cubic millimeter) of white blood cells based on age are:

The normal range for a pregnant women in the 3rd trimester is 5,80013,200 per cubic millimeter.

If a person's body is producing more white blood cells than it should be, doctors call this leukocytosis.

A high white blood cell count may indicate the following medical conditions:

Surgical procedures that cause cells to die can also cause a high white blood cell count.

If a person's body is producing fewer white blood cells than it should be, doctors call this leukopenia.

Conditions that can cause leukopenia include:

Doctors may continually monitor white blood cells to determine if the body is mounting an immune response to an infection.

During a physical examination, a doctor may perform a white blood cell count (WBC) using a blood test. They may order a WBC to test for, or rule out, other conditions that may affect white blood cells.

Although a blood sample is the most common approach to testing for white blood cells, a doctor can also test other body fluids, such as cerebrospinal fluid, for the presence of white blood cells.

A doctor may order a WBC to:

The following are conditions that may impact how many white blood cells a person has in their body.

This is a condition wherein a person's body destroys stem cells in the bone marrow.

Stem cells are responsible for creating new white blood cells, red blood cells, and platelets.

This is an autoimmune condition wherein the body's immune system destroys healthy cells, including red and white blood cells.

HIV can decrease the amount of white blood cells called CD4 T cells. When a person's T cell count drops below 200, a doctor might diagnose AIDS.

Leukemia is a type of cancer that affects the blood and bone marrow. Leukemia occurs when white blood cells rapidly produce and are not able to fight infections.

This condition causes a person's body to overproduce some types of blood cells. It causes scarring in a person's bone marrow.

Whether or not a person needs to alter their white blood cell count will depend on the diagnosis.

If they have a medical condition that affects the number of white blood cells in their body, they should talk to a doctor about the goals for their white blood cell count, depending on their current treatment plan.

A person can lower their white blood cell count by taking medications such as hydroxyurea or undergoing leukapheresis, which is a procedure that uses a machine to filter the blood.

If a person's white blood cell count is low due to cancer treatments such as chemotherapy, a doctor may recommend avoiding foods that contain bacteria. This may help prevent infections.

A person can also take colony-stimulating factors. These may help prevent infection and increase the number of white blood cells in the body.

White blood cells are an important part of the body's immune system response. There are different types of white blood cell, and each has a specific function in the body.

Certain conditions can affect the number of white blood cells in the body, causing them to be too high or too low.

If necessary, a person can take medication to alter their white blood cell count.

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Tacitus Therapeutics Launches in Collaboration with Mount Sinai to Develop Stem Cell Therapies for Life-Threatening Diseases – PRNewswire

By daniellenierenberg

NEW YORK, Jan. 9, 2020 /PRNewswire/ -- Tacitus Therapeutics, a clinical-stage company, has launched in collaboration with the Mount Sinai Health System to develop stem cell therapies initially targeting blood cancers and related clotting disorders. Their first therapy, HSC100, currently is being investigated in a Phase I clinical trial1.

Tacitus is building upon technology developed by and exclusively licensed from Mount Sinai. Based on research by scientific co-founders Ronald Hoffman, M.D., and Camelia Iancu-Rubin, Ph.D., the technology includes proprietary cell expansion, differentiation and engineering methods. Together, these methods manufacture healthy cells that overcome the limitations of traditional allogeneic, or donor, cell transplantations.

Blood cancers comprise about 10% of new cancer cases in the U.S. each year, and almost 60,000 people die from blood cancer complications annually. Most blood cancers start in the bone marrow, where blood is produced. A common therapy for such blood cancers is a hematopoietic stem cell (HSC) treatment or, as more commonly referred to, bone marrow transplantation. In this process, doctors infuse healthy HSCs into the patient's bloodstream, where they migrate to the bone marrow to grow or engraft.

HSCs for this process can be collected from bone marrow, circulating blood, or umbilical cord blood (CB) of healthy donors. While HSC transplants are common, significant barriers to success exist, including high levels of graft-versus-host disease, low numbers of healthy cells obtained from CB, and increased risk of bleeding due to delayed megakaryocyte, or platelet, engraftment.

Hoffman and Iancu-Rubin are pioneers of bone marrow cell therapy treatments, and development of this technology was enabled by the New York State Stem Cell Science program, NYSTEM. As a New York State Department of Health initiative, NYSTEM awarded a $1 million grant to Hoffman in 2010 that supported the original research underpinning this platform technology. In 2015, NYSTEM awarded Hoffman and Iancu-Rubin an $8 million grant to translate the technology from the laboratory into the clinic, where it is currently in clinical trial1.

Hoffman also serves as Director of the Myeloproliferative Disorders Research Program and Professor of Medicine (Hematology and Medical Oncology) and Iancu-Rubin is Associate Professor of Pathology at the Icahn School of Medicine and Director of the Cellular Therapy Laboratory at Mount Sinai Hospital.

"Promising discoveries by Mount Sinai scientific thought leaders may lead to new, essential cell-based therapies that will broadly benefit patients," said Erik Lium, Executive Vice President and Chief Commercial Innovation Officer, Mount Sinai Innovation Partners. "We're pleased to be collaborating with Tacitus to launch the next stage of development for these technologies."

"Tacitus is committed in its mission to advance next-generation cell therapies with curative potential," said Carter Cliff, CEO of Tacitus. "Based on our founders' solid foundation of research, we are translating these discoveries into broad clinical practice as we look to dramatically improve the standard of care for patients with life-threatening conditions."

About HSC100

HSC100 is an investigational therapy based on allogeneic hematopoietic stem cells (HSC) expanded from umbilical cord blood. HSC100 is being investigated currently in an open-label Phase I clinical trial1 in the United States for treatment of hematological malignancies. The success of unmanipulated cord blood as a source of stem cells has been hampered by the small number of stem cells present in a single cord, leading to delayed engraftment and frequent graft failure. Our proprietary technology includes the use of an epigenetic modifier, valproic acid, to expand the number and the quality of HSCs found in cord blood collections. For more information on HSC100 clinical trials, please visit http://www.clinicaltrials.gov.

1ClinicalTrials.gov identifier NCT03885947.

About Tacitus Therapeutics

Tacitus Therapeutics is a clinical-stage biotechnology company developing advanced medicines for treatment of blood cancers, immune disorders and other intractable disease conditions. Our mission is to pioneer best-in-class therapies using proprietary cell expansion, differentiation and engineering platform technologies that overcome the limitations of traditional cell transplantation. Initial targets include a lead clinical program (HSC100) investigating the treatment of blood cancers, followed by preclinical programs to address clotting disorders and other serious unmet medical needs. For additional information, please visit http://www.tacitustherapeutics.com.

About Mount Sinai Health System

The Mount Sinai Health System is New York City's largest integrated delivery system, encompassing eight hospitals, a leading medical school, and a vast network of ambulatory practices throughout the greater New York region. Mount Sinai's vision is to produce the safest care, the highest quality, the highest satisfaction, the best access and the best value of any health system in the nation. The Health System includes approximately 7,480 primary and specialty care physicians; 11 joint-venture ambulatory surgery centers; more than 410 ambulatory practices throughout the five boroughs of New York City, Westchester, Long Island, and Florida; and 31 affiliated community health centers. The Icahn School of Medicine is one of three medical schools that have earned distinction by multiple indicators: ranked in the top 20 by U.S. News & World Report's "Best Medical Schools", aligned with a U.S. News & World Report's "Honor Roll" Hospital, No. 12 in the nation for National Institutes of Health funding, and among the top 10 most innovative research institutions as ranked by the journal Nature in its Nature Innovation Index. This reflects a special level of excellence in education, clinical practice, and research. The Mount Sinai Hospital is ranked No. 14 on U.S. News & World Report's "Honor Roll" of top U.S. hospitals; it is one of the nation's top 20 hospitals in Cardiology/Heart Surgery, Diabetes/Endocrinology, Gastroenterology/GI Surgery, Geriatrics, Gynecology, Nephrology, Neurology/Neurosurgery, and Orthopedics in the 2019-2020 "Best Hospitals" issue. Mount Sinai's Kravis Children's Hospital also is ranked nationally in five out of ten pediatric specialties by U.S. News & World Report. The New York Eye and Ear Infirmary of Mount Sinai is ranked 12th nationally for Ophthalmology, Mount Sinai St. Luke's and Mount Sinai West are ranked 23rd nationally for Nephrology and 25th for Diabetes/Endocrinology, and Mount Sinai South Nassau is ranked 35th nationally for Urology. Mount Sinai Beth Israel, Mount Sinai St. Luke's, Mount Sinai West, and Mount Sinai South Nassau are ranked regionally. For more information, visit http://www.mountsinai.org or find Mount Sinai on Facebook, Twitter and YouTube.

About Mount Sinai Innovation Partners (MSIP)

MSIP is responsible for driving the real-world application and commercialization of Mount Sinai discoveries and inventions and the development of research partnerships with industry. Our aim is to translate discoveries and inventions into health care products and services that benefit patients and society. MSIP is accountable for the full spectrum of commercialization activities required to bring Mount Sinai inventions to life. These activities include evaluating, patenting, marketing and licensing new technologies building research, collaborations and partnerships with commercial and nonprofit entities, material transfer and confidentiality, coaching innovators to advance commercially relevant translational discoveries, and actively fostering an ecosystem of entrepreneurship within the Mount Sinai research and health system communities. For more information, please visit http://www.ip.mountsinai.orgor find MSIP onLinkedIn, Twitter, Facebook,Medium, and YouTube.

Media Contacts:

Mount Sinai Cynthia Cleto Mount Sinai Innovation Partners (646) 605-7359 cynthia.cleto@mmsm.edu

Tacitus TherapeuticsJoleen RauRau Communications(608) 209-0792232130@email4pr.com

SOURCE Tacitus Therapeutics

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Jasper Therapeutics Raises Additional $14.1M in Series A Financing – FinSMEs

By daniellenierenberg

Jasper Therapeutics, Inc., a Palo Alto, Calif.-based biotechnology company focused on hematopoietic cell transplant therapies, expanded its Series A financing with an additional investment of $14.1m.

The round was led by Roche Venture Fund with participation from other investors. This brought the total company financing to more than $50m to date.

The initial Series A round was led by Abingworth LLP and Qiming Venture Partners USA, with further investment from Surveyor Capital (a Citadel company) and participation from Alexandria Venture Investments, LLC.

The company plans to use the proceeds to advance and expand the study of its lead clinical asset, JSP191.

Jasper Therapeutics is a biotechnology company focused on hematopoietic cell transplant therapies. 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 conditioning antibody is designed to enable safer and more effective curative hematopoietic cell transplants and gene therapies.

FinSMEs

09/01/2020

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Stem Cell and Regenerative Medicine Action Awards to be Presented at World Stem Cell Summit on January 23 at the Hyatt Regency Miami – GlobeNewswire

By daniellenierenberg

Miami, FL, Jan. 09, 2020 (GLOBE NEWSWIRE) -- The formal ceremony of the 2020 Stem Cell and Regenerative Medicine Action Awards will take place at a gala reception and dinner on January 23, during the 15th annual World Stem Cell Summit (WSCS) at the Hyatt Regency in Miami. Since 2005, the nonprofit Regenerative Medicine Foundation (RMF) (formerly Genetics Policy Institute) has recognized the stem cell and regenerative medicine community's leading innovators, leaders, and champions through its annual awards reception.

Bernard Siegel, Executive Director of Regenerative Medicine Foundation and founder of the World Stem Cell Summit, said, The 2020 Action Awards will recognize three important organizations that are positively impacting the emerging field of regenerative medicine. We will also honor a retired Major General, who has capped off his military and diplomatic career by promoting the cause of world peace through medicine. All of these distinguished honorees will be recognized for their devotion to improving health and developing cures through advocacy, innovation, leadership and inspiration. In addition, the wounded warrior veterans community of South Florida will also receive special recognition at the event.

Meet the 2020 Stem Cell & Regenerative Medicine Action Award Honorees:

Innovation Award: With the motto, We will not rest until we find a cure, the Cystic Fibrosis Foundation is geared towards the successful development and delivery of treatments, therapies and a cure for every person with cystic fibrosis. CF Foundation has added decades to the lives of people with the disease as a direct result of advances in treatment and care made possible through its innovative business model- venture philanthropy. The Foundation recently unveiled its Path to a Cure research agenda aimed at addressing the root genetic cause of the disease and is currently funding industry programs aimed at gene delivery with the goal of progressing into clinical studies in 2021.

Inspiration Award: Emily Whitehead Foundation is a nonprofit organization committed to raising funds to invest in the most promising pediatric cancer research. Tom and Kari Whitehead founded EWF in honor of their daughter Emily, the first child in the world to receive CAR T-cell therapy, training her own cells to fight cancer. Her inspiring story focused public attention on thepotential for cancer immunotherapy to transform cancer treatment,as well as the need to support lifesaving cancer immunotherapy research. The foundation provides support to pediatric cancer patients and promotes awareness of the disease through education and sharing other inspiring stories.

Advocacy Award: Gift of Life Marrow Registry was established in 1991 by Jay Feinberg and his family after Jay received a life-saving bone marrow transplant. Gift of Life is dedicated to saving lives and facilitating bone marrow and blood stem cell transplants for patients with leukemia, lymphoma, sickle cell and other diseases. In 2019, Gift of Life opened the worlds first apheresis center fully integrated within a registry, the Dr. Miriam and Sheldon G. Adelson Gift of Life-Be The Match Stem Cell Collection Center. With the collection center and rapidly expanding donor database, Gift of Life will launch a biobank to advance cellular therapies using allogeneically sourced cells in 2020.

Leadership Award: Ret. Major General Bernard Burn Loeffke, PhD (US Military) is a highly decorated Special Forces officer, diplomat and medical officer.He survived two helicopter crashes and was wounded in combat. After the Vietnam War, he served as the Army Attach at theU.S. Embassy in Moscow, first Defense Attach at the U.S Embassy in Beijing, a staff officer in theWhite House, and Director of the Commission onWhite House Fellows. His last command was Commanding General of Army South. After 35 years in the military, he became a medical officer traveling the world on relief missions to third and fourth world countries. Presently, at age 85, he champions the hydrocephalus and wounded warrior communities. He continues to serve as an inspiration and supporter of building peaceful international relations through medical partnerships and played a pivotal role as a keynote speaker at the inaugural 2019 World Stem Cell Summit CHINA.He is called the Peace General in Latin America. In China, he is simply known as The General, our Friend.

To learn more about past honorees and details for sponsoring or attending the upcoming 2020 Stem Cell and Regenerative Medicine Action Awards dinner, please visit, https://www.worldstemcellsummit.com/stem-cell-action-awards/

About the World Stem Cell Summit (WSCS)

Produced by the non-profit Regenerative Medicine Foundation (RMF), and in its 15th year, the World Stem Cell Summit will take place January 21-24, 2020, in Miami, Florida in partnership with Phacilitate Leaders World, as part of Advanced Therapies Week. The Summit is the most inclusive and expansive interdisciplinary, networking, and partnering meeting in the stem cell science and regenerative medicine field. With the overarching purpose of fostering translation of biomedical research, funding, and investments targeting cures, the Summit and co-located conferences serve a diverse ecosystem of stakeholders. For more information about the upcoming World Stem Cell Summit in Miami, please visit: http://www.worldstemcellsummit.com.

About the Regenerative Medicine Foundation (RMF)

The nonprofit Regenerative Medicine Foundation fosters strategic collaborations to accelerate the development of regenerative medicine to improve health and deliver cures. RMF unites the worlds leading researchers, medical centers, universities, labs, businesses, funders, policymakers, experts in law, regulation and ethics, medical philanthropies, and patient organizations. We maintain a trusted network of leaders and pursue our mission by producing our flagship World Stem Cell Summit series of conferences and public days, honoring leaders through the Stem Cell and Regenerative Medicine Action Awards, supporting our official journal partner STEM CELLS Translational Medicine (SCTM), promoting solution-focused policy initiatives both nationally and internationally and creating STEM/STEAM educational projects. For more information about RMF, please visit: http://www.regmedfoundation.org.

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Stem Cell and Regenerative Medicine Action Awards to be Presented at World Stem Cell Summit on January 23 at the Hyatt Regency Miami - GlobeNewswire

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Here’s Why CRISPR Therapeutics Stock Jumped 113.2% in 2019 – Motley Fool

By daniellenierenberg

What happened

Shares of CRISPR Therapeutics (NASDAQ:CRSP) rose over 113% last year, according to data provided by S&P Global Market Intelligence. The pharma stock built momentum throughout much of the year, but surged in October ahead of an important data presentation that ultimately lived up to the hype. That allowed the gene-editing stock to easily outperform the 28.8% gain of the S&P 500 in 2019.

The end-of-year rally was driven by promising clinical results for its lead drug candidate. The first two individuals, one with sickle cell disease (SCD) and one with transfusion-dependent beta thalassemia (TDT), dosed with CTX001 achieved functional cures after receiving an initial dose of the gene-editing product. The results need to be proven durable and replicated in a larger number of patients, but the update was about as good as investors could have hoped for at the current stage of development.

Image source: Getty Images.

Both SCD and TDT are caused by structural abnormalities in red blood cells. But these are one of the few cells in the human body that don't contain DNA. That means CRISPR Therapeutics has to harvest stem cells from the bone marrow of patients, apply gene editing to those extracted cells, and then inject the engineered stem cells back into patients (the ex vivo method). If the therapy works, then the engineered stem cells should produce functional red blood cells and potentially result in a cure.

In the early study, the ex vivo approach of CTX001 appeared to do just that. The TDT patient required an average of 16.5 blood transfusions per year in the two years before the clinical trial. Nine months after receiving the gene-editing treatment, the individual was transfusion independent (compared with an expected 12 transfusions) and expressed working copies of hemoglobin on 99.8% of red blood cells.

The SCD patient experienced an average of seven vaso-occlusive crises (painful blockages of blood vessels caused by abnormally shaped red blood cells) per year in the two years before the clinical trial. Four months after receiving the gene-editing treatment, the individual reported no vaso-occlusive crises (compared with an expectation for two such episodes) and expressed working copies of hemoglobin on 94.7% of red blood cells.

The early success of CTX001 bodes well for the ex vivo approach of CRISPR Therapeutics and its partner Vertex Pharmaceuticals(NASDAQ:VRTX), but investors should be careful not to extrapolate the results too broadly. Gene-editing tools that are applied inside the body (in vivo) face significantly steeper obstacles, such as the difficulty of delivering gene-editing payloads to specific tissue types inside the body. There's also the elephant in the room: Scientists are beginning to realize that current-generation CRISPR gene-editing tools don't work all that well.

Nonetheless, CRISPR Therapeutics is the top CRISPR-based gene-editing stock on the market. It has the cash, the partnerships, and the early results to back up its claim to that label.

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