An effective andinnovative way to treat people with sickle cell anemia using gene therapy may soon be available thanks to efforts by several pharmaceutical companies, a Bloomberg report says.

Sickle cell anemia, a genetic defect that causes red blood cells to form in theshape ofa sickle, hinders the bodys ability to adequately distribute oxygen. This is due to atypical hemoglobin molecules, which is the protein in blood that transports oxygen. Sickle cell disease can be extremely painful, causing blood cells to get trapped in blood vessels and lead to heart failure, debilitating fatigue, strokes and blood clots.About 100,000 people suffer from sickle cell anemia in the U.S,with African Americansbeing disproportionately affected by this condition.

New developments with gene therapy, however, could work to have a positive impact on these symptoms. One of the innovative manufacturers, Bluebird Bio, stole the show at the annual conference of the American Society of Hematology in Florida. Its product, LentiGlobin, debuted positive results; in 17 patients treated with LentiGlobin,more than 40 percent of the hemoglobin in patients' red blood cells appearedin a healthier form thanks to gene therapy, per the article.

Bluebird isnt the only biotechnology making strides in gene therapies. Another potential treatment being researched is based on the technology called CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats), a gene-editing tool that is being used for a wide range of biomedical applications.

Documented in an NPR report, sickle cell patient Victoria Gray recently became the first person in the U.S. to have billions of her own cells genetically edited with CRISPR and reintroduced into her body. These cells will hopefully produce fetal hemoglobin to compensate for the faulty hemoglobin in Grays red blood cells. The trial is being expanded to include more patients and is being conducted by Vertex Pharmaceuticals and CRISPR Therapeutics of the Boston area.

Current treatments for sickle cell include blood and bone marrow transfusions and medication. Studies on gene therapy treatments have been encouraging so far, but there is more testing to be done before either CRISPR or LentiGlobin hits the market.

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Gene therapy could be a revolutionary new treatment for sickle cell disease - The Hill

Recommendation and review posted by Bethany Smith

New York City, NY: Dec 12, 2019 Published via (Wired Release) The CRISPR Technology Market Report characterizes and briefs perusers about its items, applications, and particulars. The examination records key organizations working in the market and furthermore features the key changing course received by the organizations to keep up their quality. By utilizing SWOT investigation and Porters five power examination instruments, the qualities, shortcomings, openings, and malediction of key organizations are out and out referenced in the report. Each and every driving player in this worldwide market is profiled with subtleties, for example, item types, business outline, deals, fabricating base, candidate, applications, and particulars.

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Thermo Fisher Scientific, Inc.Merck KGaAGenScript CorporationIntegrated DNA Technologies, Inc.Horizon Discovery GroupAgilent Technologies, Inc.Cellecta, Inc.GeneCopoeia, Inc.New England Biolabs, Inc.Origene Technologies, Inc.

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CRISPR Technology Market: 2020 With Top Competitors Analysis And Insights - Sound On Sound Fest

Recommendation and review posted by Bethany Smith

BERKELEY, Calif.--(BUSINESS WIRE)--GenEdit, Inc., a developer of a novel polymer nanoparticle technology platform for non-viral- and non-lipid-based delivery of gene therapies, today announced that it has entered into a worldwide, exclusive license and collaboration agreement with Editas Medicine, Inc., a leading genome editing company. GenEdit has developed a comprehensive delivery system for CRISPR-based therapeutics, including gene knockout and gene repair therapies, to enable safer delivery options with improved efficiency.

"This license and collaboration agreement further validates the strength of our intellectual property portfolio and the potential of GenEdits technology," said Kunwoo Lee, Ph.D., co-founder and chief executive officer of GenEdit. "We are pleased to establish our relationship with Editas Medicine as they leverage our technology to develop potential genomic medicines."

Under the terms of the agreement, GenEdit has granted Editas Medicine an exclusive worldwide license, with rights to sublicense, to GenEdits Cpf1-based technologies. In return for these rights, GenEdit will receive undisclosed upfront and development milestone payments, including royalties on net sales of products incorporating the licensed intellectual property. In addition, GenEdit and Editas Medicine will collaborate on evaluating delivery of Cpf1-based technologies with GenEdits nanoparticle platform. Editas Medicine will provide research funding and have an option to continue development after the initial collaboration period.

GenEdits nanoparticle platform consists of a proprietary non-viral, non-lipid library of polymers that efficiently encapsulate and deliver cargo [RNA, DNA, protein and/or ribonucleic acid-protein complexes (RNP)] to specific tissues. The company screens the library to identify initial hits and then uses computational analysis and medicinal chemistry for iterative lead optimization. The company has used this platform to identify multiple candidate polymers for efficient and specific delivery of gene editing to a range of tissues.

"Compared to viral vectors and lipid-based nanoparticles, our approach has the potential for better targeting, more cargo, and lower manufacturing cost," said Timothy Fong, Ph.D., chief scientific officer of GenEdit. "In particular, our approach has the potential to enable in vivo gene editing of multiple tissues with CRISPR and expand the potential of gene therapies to treat more diverse sets of diseases."

About GenEdit

GenEdit was founded to transform the delivery of gene and gene editing therapies. We have synthesized the NanoGalaxy library of polymers that can encapsulate RNA, DNA, protein and/or RNP. Through advanced screening methods, computational analysis and iterative medicinal chemistry, we have demonstrated efficient delivery of gene editing cargo to specific tissues. We seek development partnerships for specific tissues and/or gene targets while advancing our internal pipeline of gene editing therapies.

For more information, please visit http://www.genedit.com.

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GenEdit and Editas Medicine Enter into Exclusive License and Collaboration Agreement for Nanoparticle Gene Therapy Delivery - Business Wire

Recommendation and review posted by Bethany Smith

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Global Cryonics Technology Market 2019 by Manufacturers, Countries, Type and Application, Forecast to 2025 - Industry News Releases

Recommendation and review posted by Bethany Smith

Dec. 12, 2019 06:05 UTC

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced new data from the Phase III FeDeriCa study which showed the investigational fixed-dose combination (FDC) of Perjeta (pertuzumab) and Herceptin (trastuzumab) with hyaluronidase, administered by subcutaneous (SC) injection in combination with intravenous (IV) chemotherapy, demonstrated non-inferior levels of Perjeta in the blood (pharmacokinetics) and comparable efficacy and safety to standard IV infusions of Perjeta plus Herceptin and chemotherapy in eligible people with HER2-positive early breast cancer (EBC).

These new data, from a primary analysis of the FeDeriCa study, will be presented in a spotlight session (Abstract #PD4-07) at 7:00 a.m. CST today at the 2019 San Antonio Breast Cancer Symposium (SABCS).

SC administration of the FDC takes approximately eight minutes for the initial loading dose and approximately five minutes for each subsequent maintenance dose. This is compared to approximately 150 minutes for infusion of a loading dose of Perjeta and Herceptin using the standard IV formulations, and between 60-150 minutes for subsequent maintenance infusions of the two medicines.

This fixed-dose subcutaneous combination has the potential to provide a quicker and less invasive method of administration for people with HER2-positive breast cancer being treated with Perjeta and Herceptin, said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. This is the first time that we have brought together two of our targeted antibodies as a single subcutaneous injection that can be administered in just minutes.

The FeDeriCa study met its primary endpoint, with SC administration of the FDC showing non-inferior levels of Perjeta in the blood during a given dosing interval (Ctrough) when compared to IV administration of Perjeta. The geometric mean ratio (GMR, a type of average used when assessing pharmacokinetics) for the primary endpoint was 1.22 (90% CI 1.14-1.31) with the lower limit of the 90% CI of the GMR=1.140.80 (the pre-specified non-inferiority margin). A secondary endpoint of non-inferior Ctrough of Herceptin was also met, with blood concentrations for people receiving the FDC non-inferior to those receiving IV Herceptin (GMR=1.33 [90% CI 1.24-1.43]; lower limit of 90% CI of GMR=1.240.80). A non-inferiority endpoint was chosen for the study to ensure that people were receiving sufficient dosing with Perjeta and Herceptin as compared to the established IV doses at the same treatment intervals. In addition, rates of total pathological complete response (pCR), a secondary endpoint, were comparable between the treatment arms, with 59.7% of patients receiving the FDC and 59.5% of patients treated with IV Perjeta and Herceptin achieving a total pCR a difference of 0.15% (95% CI -8.67-8.97).

The safety profile of the FDC in combination with chemotherapy was comparable to that of IV administration of Perjeta plus Herceptin and chemotherapy, and no new safety signals were identified, including no meaningful difference in cardiac toxicity. The most common adverse events in both arms were alopecia, nausea, diarrhea and anemia.

In previous studies, SC administration has been shown to be strongly preferred by the majority of patients compared to IV administration of the same medicine, with the most common reason being that administration required less time in the clinic. In the PHranceSCa study, Genentech is currently investigating patient preference for SC administration of the FDC compared to standard IV administration of Perjeta and Herceptin in people with HER2-positive EBC. Interim results of this Phase II study will be presented at a future medical meeting.

About the FeDeriCa study

FeDeriCa is an international, multicenter, two-arm, randomized, open-label, Phase III study evaluating the pharmacokinetics, efficacy and safety of SC injection of the FDC of Perjeta and Herceptin in combination with chemotherapy, compared with standard IV infusions of Perjeta and Herceptin in combination with chemotherapy in 500 people with HER2-positive EBC who are being treated in the neoadjuvant (before surgery) and adjuvant (after surgery) settings. The primary endpoint of the study is minimum levels of Perjeta in the blood during a given dosing interval (Ctrough). Secondary endpoints include safety; minimum levels of Herceptin in the blood during a given dosing interval (Ctrough); and total pCR, meaning there is no tumor tissue detectable in the tissue removed at the time of surgery. The safety profile of Perjeta and Herceptin FDC was comparable with that of Perjeta and Herceptin administered intravenously.

About HER2-positive breast cancer

Breast cancer is one of the most common cancers among women worldwide. According to the American Cancer Society, approximately 271,000 people in the United States will be diagnosed with breast cancer, and more than 42,000 will die from the disease in 2019. Breast cancer is not one, but many diseases based on the biology of each tumor. In HER2-positive breast cancer, there is excess HER2 protein on the surface of tumor cells. Approximately 15-20% of breast cancers are HER2-positive based on the result of a diagnostic test.

About the FDC of Perjeta and Herceptin

The FDC of Perjeta and Herceptin is a new SC formulation that combines Perjeta and Herceptin with Halozyme Therapeutics Enhanze drug delivery technology.

Trastuzumab in the FDC is the same monoclonal antibody as in IV Herceptin and pertuzumab in the FDC is the same monoclonal antibody as in IV Perjeta. The mechanisms of action of Perjeta and Herceptin are believed to complement each other as both bind to the HER2 receptor, but in different locations. The combination of Perjeta and Herceptin is thought to provide a more comprehensive, dual blockade of the HER signaling pathways.

Halozymes Enhanze drug delivery technology may enable and optimize SC drug delivery for appropriate co-administered therapeutics. The technology is based on a proprietary recombinant human hyaluronidase PH20 (rHuPH20), an enzyme that temporarily degrades hyaluronan a glycosaminoglycan or chain of natural sugars in the body, to aid in the dispersion and absorption of other injected therapeutic drugs.

Current Perjeta and Herceptin IV Indication Statements and Important Safety Information

Perjeta Indication Statements

Perjeta (pertuzumab) is a prescription medicine approved for use in combination with Herceptin (trastuzumab) and chemotherapy for:

Perjeta (pertuzumab) is a prescription medicine approved for use in combination with Herceptin (trastuzumab) and docetaxel in people who have HER2-positive breast cancer that has spread to different parts of the body (metastatic) and who have not received anti-HER2 therapy or chemotherapy for metastatic breast cancer.

Important Safety Information

Side effects with Perjeta

Most serious side effects of Perjeta

Perjeta may cause heart problems, including those without symptoms (such as reduced heart function) and those with symptoms (such as congestive heart failure).

Receiving Perjeta during pregnancy can result in the death of an unborn baby and birth defects.

Other possible serious side effects

Most common side effects

The most common side effects of Perjeta when given with Herceptin and chemotherapy as part of an early breast cancer regimen before surgery are:

Side effects may vary based on chemotherapy regimen.

The most common side effects of Perjeta when given with Herceptin and chemotherapy as part of an early breast cancer regimen after surgery are:

The most common side effects of Perjeta when given with Herceptin and docetaxel for treatment of breast cancer that has spread to other parts of the body (metastatic) are:

Patients are encouraged to report side effects to Genentech and the FDA. Report side effects to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch. Report side effects to Genentech at (888) 835-2555.

Patients should talk to a healthcare professional for more information about the benefits and risks of Perjeta.

Please see the Perjeta full Prescribing Information for additional Important Safety Information, including most serious side effects, at http://www.perjeta.com.

Herceptin Indication Statements

Adjuvant Breast Cancer

Herceptin is approved for the treatment of early-stage breast cancer that is Human Epidermal growth factor Receptor 2-positive (HER2+) and has spread into the lymph nodes or is HER2-positive and has not spread into the lymph nodes. If it has not spread into the lymph nodes, the cancer needs to be estrogen receptor/progesterone receptor (ER/PR)-negative or have one high-risk feature.* Herceptin can be used in several different ways:

Patients are selected for therapy based on an FDA-approved test for Herceptin.

*High risk is defined as ER/PR-positive with one of the following features: tumor size greater than 2 cm, age less than 35 years, or tumor grade 2 or 3.

Metastatic Breast Cancer

Herceptin has two approved uses in metastatic breast cancer:

Patients are selected for therapy based on an FDA-approved test for Herceptin.

Important Safety Information

Possible serious side effects with Herceptin

Not all people have serious side effects, but side effects with Herceptin therapy are common.

Although some people may have a life-threatening side effect, most do not.

A patients doctor will stop treatment if any serious side effects occur.

Herceptin is not for everyone. A patient should be sure to contact their doctor if they are experiencing any of the following:

HEART PROBLEMS

These include heart problemssuch as congestive heart failure or reduced heart functionwith or without symptoms. The risk for and seriousness of these heart problems were highest in people who received both Herceptin and a certain type of chemotherapy (anthracycline). In a study of adjuvant (early) breast cancer, one patient died of significantly weakened heart muscle. A patients doctor will check for signs of heart problems before, during, and after treatment with Herceptin.

INFUSION REACTIONS, including:

These signs usually happen within 24 hours after receiving Herceptin.

A patient should be sure to contact their doctor if they:

Are a woman who could become pregnant, or may be pregnant

Herceptin may result in the death of an unborn baby or birth defects. Contraception should be used while receiving Herceptin and for seven months after a patient's last dose of Herceptin. If a patient is or becomes pregnant while receiving Herceptin or within seven months after their last dose of Herceptin, the patient should immediately report Herceptin exposure to Genentech at (888) 835-2555.

Have any signs of SEVERE LUNG PROBLEMS, including:

A patients doctor may check for signs of severe lung problems when he or she examines the patient.

Have LOW WHITE BLOOD CELL COUNTS

Low white blood cell counts can be life threatening. Low white blood cell counts were seen more often in patients receiving Herceptin plus chemotherapy than in patients receiving chemotherapy alone.

A patients doctor may check for signs of low white blood cell counts when he or she examines the patient.

Side effects seen most often with Herceptin

Some patients receiving Herceptin for breast cancer had the following side effects:

A patient should contact their doctor immediately if they have any of the side effects listed above.

Patients are encouraged to report side effects to Genentech and the FDA. Report side effects to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch. Report side effects to Genentech at (888) 835-2555.

Patients should talk to a healthcare professional for more information about the benefits and risks of Herceptin.

Please see the Herceptin full Prescribing Information for additional Important Safety Information, including most serious side effects, at http://www.herceptin.com.

About Genentech in breast cancer

Genentech has been advancing breast cancer research for more than 30 years with the goal of helping as many people with the disease as possible. Our medicines, along with companion diagnostic tests, have substantially improved outcomes for HER2-positive breast cancer. As our understanding of breast cancer biology rapidly improves, we are working to identify new biomarkers and approaches to treatment for other subtypes of the disease, including triple-negative and hormone receptor-positive.

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191211006028/en/

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Genentech's Fixed-dose Subcutaneous Combination of Perjeta and Herceptin Comparable to Intravenous Formulations in People With HER2-positive Breast...

Recommendation and review posted by Bethany Smith

By Michele Munz, St. Louis Post-Dispatch

Amy Chastain didnt think she would be able to be a mom. She didnt think her child would be born with the same debilitating disease as her.

She didnt think she would live long enough to see this day, when a drug could change her life and most importantly, her sons.

At age 40, Chastain is pushing the limits. The average life expectancy for someone with cystic fibrosis is 41. Chastain had a serious scare two years ago, when she spent more than a month in the hospital because she got so ill. She needed a feeding tube and oxygen tank. She faced the need for a lung transplant.

A breakthrough drug was approved Oct. 21 for 90% of teen and adult patients with cystic fibrosis, a genetic life-threatening disease that causes thick mucus to build up in the lungs and other organs. The drug Trikafta is the first therapy to show dramatic improvement in lung function for a majority of those with the disease.

Knowing the drug showed promising results in studies, families had been waiting anxiously for months for approval by the federal government, which came faster than expected.

Today marks a tremendous breakthrough and exciting news for people with cystic fibrosis, said Dr. Preston Campbell, president of the Cystic Fibrosis Foundation. This milestone is the result of an extraordinary community coming together against great odds, and we are overjoyed that this will mean more people will have effective treatments for their disease.

Chastain began taking the pill a few weeks after it was approved, and within three days, she said she already felt better. After her morning treatments to clear the mucus that settles in her lungs overnight, she had little to cough up.

She has more energy. Shes able to make her bed, keep her wood floors clean and walk to her car without getting winded and having to take breaks.

I read that it takes effect that quickly, but I didnt believe it, Chastain said. But it is. Its been amazing.

Her son, Kyler, is now 14. Having religiously spent his life doing the breathing treatments that can take two to three hours every day, his lungs are still strong.

At his next appointment with a specialist at St. Louis Childrens Hospital in January, they will begin the process of getting Trikafta for him, too.

While the long-term effects are unknown, the hope is that the drug will help slow the progressive damage of the disease.

Its very exciting to think that he hopefully wont ever get as sick as I am, Chastain said. As a mother, you just cant put in to words what that means to me, that he wont have to go through everything that I have. Hopefully, hell just be able to live a long, healthy life.

But theres more to her story, she says. She might not have become a mom if not for a mistake in her husbands genetic test. Because of the mistake, they falsely believed their child would not be born with cystic fibrosis.

I dont know what happened or why, but Im thankful because, while it took a while to get over my anger, I cant imagine not having Kyler, Chastain said. For whatever reason, he is meant to be here, he is meant to have CF.

Her anger turned to thankfulness, she said, and now its turned to hope.

A verty different time

More than 30,000 people suffer from CF in the United States, with 70,000 affected worldwide according to the Cystic Fibrosis Foundation. In Missouri and Illinois, newborn screening tests show about one in 3,500 babies are born with CF, said Dr. Thomas Ferkol, pediatrics professor at Washington University School of Medicine.

Its considered to be a rare or orphan disease, but its one of the more common inherited diseases, Ferkol said.

In people with CF, mutations in a regulator gene cause a defect in the cells covering surfaces of the body the skin, airways, blood vessels and organs. The cells cant maintain their balance of salt and water, causing mucus in various organs to become thick and sticky.

It leads to damage and ultimately, the destruction of organs, Ferkol said.

In the lungs, mucus clogs the airways and traps germs, leading to infections and other complications. Other problems include decreased sweating, digestive problems, poor growth, diabetes and infertility.

Doctors have treated the disease by addressing the symptoms taking drugs to loosen the mucus, using airway clearing devices several times a day and taking supplements to replace pancreatic enzymes.

Trikafta directly addresses the salt-water imbalance by improving the function of defective proteins. It is the first drug to do so in CF patients with the most common gene mutation 90% have at least one copy of the mutation.

A similar drug, Kalydeco, was approved eight years ago, but it worked in only 8% of patients; and the improvements are not as dramatic.

A study of 403 patients for six months (some taking the drug and some taking a placebo) showed Trifakta normalized chloride levels in sweat, improved lung function by 14% and increased body mass.

A small number of patients participated in the study through Washington University, and though doctors did not know who was receiving the drug or placebo, it was obvious, said pulmonary disease specialist Dr. Daniel Rosenbluth. Other severely ill patients have been able to get early access to the drug.

They feel like they are totally different people, Rosenbluth said. I had a man whose wife kept waking him up at night because he was sleeping soundly and his wife thought he was dead.

Because results were so swift and dramatic, doctors have for nearly a year been telling their patients that a promising drug was coming.

We would tell families, OK, get ready, because we are entering a very different time, Ferkol said.

The Food and Drug Administration reviewed and approved Trifakta in just three months.

Doctors attribute the success to the Cystic Fibrosis Foundation, which over 20 years ago began working with a network of academic centers and organized patients for research studies. This made it quicker and easier for pharmaceutical companies to test drugs in development.

Other rare diseases have been trying to duplicate this model, Rosenbluth said.

The drug, made by Boston-based Vertex Pharmaceuticals, comes with a hefty price tag $311,000 a year. Patients are now wading through the process of seeking coverage through their public or private health insurance.

Keeping up hope

Patients still must continue their daily treatment regimens while taking the new pill, Ferkol stressed. The drug has been studied for only a short time, but the hope is that the drug greatly slows the progression of the disease over ones lifetime.

When Ferkol was a resident doctor in 1985, the life expectancy for a patient with CF was just 23 years old, he said.

This drug is opening up all kinds of possibilities I never dreamed to imagine when I was much younger, Ferkol said. If you can intervene early, before damage has occurred, can that profoundly change the trajectory of the disease? Its going to be a very exciting time.

While its not a cure, it is a drug families and doctors have long been waiting for. Studies are already underway in children ages 6 to 11.

Many families have hoped for a day that we could have something we could do for their children that is going to have the effect that this drug has, Ferkol said.

Hannah Krumrey, 20, of St. Charles, Missouri, is waiting on her insurance to approve her application for the drug; while her older sister, Kayla Krumrey, 21, was able to get the drug early and has already been able to sleep through the night and have more energy to get through the day.

The sisters recently went to Greece, and they hope to be able to travel more together.

Their father died a year ago. Hannah Krumrey wishes she could celebrate the drugs approval with him.

We know how happy he would be. This was like, his dream. This was all he ever wanted, she said. He will still be happy from up there, but I wish he was here to experience it with us.

Hes a blessing

Because Chastains symptoms were not yet severe when she was in her 20s, doctors thought she could safely have a child. But she feared passing on her disease.

A person with CF inherits a faulty gene from both mom and dad. If a person inherits one faulty gene and one normal gene, the person will not have symptoms but is a carrier.

Chastain and her husband decided he would get tested to see if he was a carrier. They were overjoyed when it came back negative, she said.

After two miscarriages, Kyler was born. His newborn screening, which is not always accurate, did not show he had CF.

His bowels were greasy, however, which is a sign of the disease. Chastains concerns grew, and when Kyler was 2 years old, tests confirmed he had CF. Chastain said she was devastated.

I thought I had done everything I was supposed to do to prevent the possibility, she said. I was really mad.

Chastain had watched others in her family suffer worse than she did from the disease, and it frightened her.

I was scared for him. I didnt want that life for him, she said. I didnt know what his disease would be like.

Chastain tried to figure out why her husbands test was negative. She asked to see the results. The hospital, however, said the system showed the couple was a no show the day of the test.

She did not want to identify the hospital.

God couldve stepped in. I dont know, she said. I just know hes a miracle. Hes a blessing. No one is to blame.

Chastain calls her son an outdoor kid. He loves playing basketball, hunting, fishing and riding four-wheelers. At 5 feet, 9 inches tall and 150 pounds, no one would know hes sick.

Chastain says he doesnt talk about stuff, but she can tell he gets worried when shes sick.

After seeing her do better on the new drug, Chastain said Kyler asked her if he would feel different too when he starts taking it.

Hopefully, she told him, it will just keep you feeling the way you do now.

Kyler then asked if she could go hunting with them when she feels better.

The Cystic Fibrosis Foundation is not resting. Just nine days after the new drug was approved, the foundation unveiled its Path to a Cure plan challenging scientists around the world to submit proposals that would accelerate finding a cure and allocating half a billion dollars to fund the research through 2025.

Distributed by Tribune Content Agency, LLC

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New drug appears to slow effects of cystic fibrosis, offering hope to long-suffering patients - Pocono Record

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Then there are intralipids, an emulsion of soybean oil, egg phospholipids and glycerin administered intravenously and described as a way to decrease natural killer cell activation in the immune system and ostensibly aid in embryo implantation. This emulsion is priced around $400 per infusion; typically several are recommended. Side effects include headache, dizziness, flushing, nausea and the possibility of clotting or infection. A meta-analysis last year found that intralipids and other forms of immunotherapy should not be used in routine clinical practice.

Such procedures are often presented to patients in the form of a stack of papers, written in legalese or medical jargon. Resourceful patients might take to the internet to learn more, where searches might deliver densely written scientific articles, and ads might direct them to companies or clinics eager to promote their own brands of add-ons.

Why is all this happening? Its because IVF remains an under-regulated arena, and entrepreneurial doctors and pharmaceutical and life science companies are eager to find new ways to cash in on a growing global market that is projected to be as large as $40 billion by 2024.

It has been 41 years since the first successful birth through IVF, and for the past three decades or so, the multistage IVF procedure and core drug protocols have remained fundamentally unchanged (though, to be sure, the laboratory equipment involved has become more sophisticated).

But clinics know competition for patients is fierce. Before they found add-ons as a way to differentiate themselves, clinics frequently marketed themselves by emphasizing their superior performance (not always with veracity) and by offering to make peoples dreams come true, as we wrote in our paper. Now they boast expanded menus advanced treatments, cutting-edge labs, custom service packages and special financing options.

Under-regulation of IVF dates to before the procedure first became commercially available. Since the Roe v. Wade decision in 1973, the government has done its best to avoid funding anything associated with embryo research, including IVF. As a result, its a field that since its earliest days was shaped by commercial considerations, with early practitioners lobbying for self-policing. The Food and Drug Administration requires a rigorous assessment of safety and efficacy of procedures that manipulate human cells more than minimally, yet no fertility procedure has been deemed to do so.

We are not alone here in the United States. In 2016, the BBC looked at common add-on procedures sold in Britain and uncovered many unsubstantiated claims. Australian media have done similar reporting. Only one Australian state, Victoria, requires IVF clinics to provide their patients with the evidence, or lack of it, behind add-on treatments.

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The Big IVF Add-On Racket - The New York Times

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Lets face it, my days of trampolining are well and truly over. Elspeth, (my best friend) is on the phone to me, close to tears. It seems that since turning 50, she cannot so much as cough, laugh, or jump on a trampoline, without how can I put this delicately? Peeing her pants or stress incontinence as it's more formally known. I had no idea trampolining was so important to you, I say, in a feeble attempt to console her, while simultaneously going on-line to book a check-up with a specialist pelvic physiotherapist, Kate Walsh. This is not going to happen to me. And who knows, if Walshs advice rings true, perhaps she can prevent it happening to Elspeth. Prevention is better than cure and all that.

A few days later, legs akimbo on the couch in the clinic, Walsh, a friendly Liverpudlian whose work for the NHS and in private practice has led her to treat tens of thousands of women over the years with mild incontinence brought on by the peri-menopause, examines my pelvic wall, with what I quickly come to understand is typical candour. I wish I had a Go-Pro on my fingers sometimes, she says, people would be fascinated by what you can tell from inside the pelvis! Hmm. But would they want to see it? I wonder, squeamishly.

Read more: These Second-Skin Foundations Prove Texture Is Just As Important As The Perfect Shade

Walsh has no such qualms. People stop me in shops in the Wirral where I live and theyll whisper, My mum needs to see you, she has a prolapse! And Ill think, Why are you whispering?! Her concern is that our silence around the subject means that so many women are unaware that you can treat stress incontinence and go for years suffering from shame and embarrassment, when often there are simple solutions to hand. I once had an 80 year old woman tell me she keeps pads under her pillow; waits until her husband falls asleep, and then puts them in her underwear. She doesnt want him to know that this is what she has to do now. I find this story terribly sad, and also slightly scary is this the future? until Walsh continues, But we can do things for women who are 80! We can do things whatever your age - we just need to talk about it more.

The reason its more common around our mid forties onwards is because our hormone levels are changing. Oestrogen in particular is very important for the integrity of the soft tissues, and the support it gives, so once your oestrogen levels drop, the tissue of the vagina becomes more papery, she explains. Thats when vaginal atrophy sets in, the thinness sets in, and problems take hold.

Sounds like hell, I say. What can we do to make sure This Does Not Happen? Firstly the front wall of the vagina, which doesnt support the bladder as well as it should because of the change of hormones, needs to be strengthened. We should all be doing pelvic floor exercises, either with the help of a device like Elvie or without - theyre easily mastered but require regular practice if youre going to see improvements. When we laugh, cough or sneeze, we generate pressure in the abdomen, says Walsh, And this squeezes the bladder. Normally the vaginal wall supports the fascia and the muscles come up, suppress the urethra and keep you continent, without you even knowing it. But with stress incontinence, in the case of your friend, you lack the ability to tighten up.

Read more: Feeling Low? Here Are 7 Ways To Ease Seasonal Affective Disorder

To strengthen the fascia, a treatment like ThermiVas, which Walsh performs regularly, via the insertion of a warm wand which emits radio frequency technology to tighten the internal walls of the vagina, is great, as theres no down time and most see an instant improvement.

There are now so many options, says Walsh, from injecting a filler into the urethral sphincter, to meche, where a tape is added to the neck of the bladder so it has additional support. At the first hint of a problem, you should see a doctor. And be prepared to be told to put the work in yourself. Recently I attended a conference in Scotland where they debated the complications surrounding meche, says Walsh. And what it boils down to is, we all need to try the more sustainable solutions first treatments like radiofrequency, and exercising at home.

Get ready to start squeezing.

Try: If youre worried about losing control of your bladder and find yourself going more often to the loo as a precautionary measure, try not to. Walsh says youre just creating a sense of panic, and making the situation worse by creating an overactive bladder. Never go just in case and never leave it until youre bursting - aim for something in between. You need to show your bladder whos boss and for most of us that means six to eight times within 24 hours.

Buy: My Pelviva is a new pelvic floor muscle trainer that you insert like a tampon into your vagina. It then sends electric pulses to stimulate and strengthen the muscles. 84% of women reported improved bladder control after 12 months of use.

Do: If you get the chance to book in with Kate Walsh, do! You can find her in clinics across Merseyside and in London at Mallucci London.

More from British Vogue:

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How To Deal With Stress Incontinence Because It's More Common Than You Might Think - British Vogue

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Have you been dreaming of getting pregnant, without success? Have you gone through several hormone injections or in vitro fertilization cycles? Or do you have any period problems? Do you complain of night sweats, hot flushes, mood swings, pain during intercourse, increased anxiety or irritability, or other menopause-related symptoms? Are you feeling stressed or anxious during the onset of menopause?

If your answer is yes to any of the above questions, you may consider fertility enhancement or relief of menopause-related symptoms with traditional Chinese medicine treatments, including acupuncture and herbs, which can improve your potential for pregnancy or alleviate your distress when menopausal symptoms occur.

Based on the Western and traditional Chinese medical principles of female health and the family medicinal traditions of Dr Xian-Yang Lai, the Chinese Medical Clinic can precisely tailor personal Chinese medication that is specific to your individual circumstances. The treatment will optimise your fertility potential or rebalance your body, mind, and energy from your imbalanced conditions caused by menopause and/or other external factors.

The Chinese Medical Clinic has successfully treated fertility problems by enhancing the growth and development of ovarian follicles, and improving ovulation or menopausal conditions by rebalancing hormone levels. For more information contact the Chinese Medical Clinic, 1 Nile Lodge, Lower Salthill, Galway, phone 087 799 7866 or visit http://www.CMCgalway.com

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Fertility enhancement and menopausal relief with acupuncture and herbs - Galway Advertiser

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Led by researchers at Baylor College of Medicine, a study published in the journal Cell Stem Cell reveals a new mechanism that contributes to adult bone maintenance and repair and opens the possibility of developing therapeutic strategies for improving bone healing.

Adult bone repair relies on the activation of bone stem cells, which still remain poorly characterized, said corresponding author Dr. Dongsu Park, assistant professor of molecular and human genetics and of pathology and immunology at Baylor. Bone stem cells have been found both in the bone marrow inside the bone and also in the periosteum the outer layer of tissue that envelopes the bone. Previous studies have shown that these two populations of stem cells, although they share many characteristics, also have unique functions and specific regulatory mechanisms.

Of the two, periosteum stem cells are the least understood. It is known that they comprise a heterogeneous population of cells that can contribute to bone thickness, shaping and fracture repair, but scientists had not been able to distinguish between different subtypes of bone stem cells to study how their different functions are regulated.

In the current study, Park and his colleagues developed a method to identify different subpopulations of periosteum stem cells, define their contribution to bone fracture repair in live mouse models and identify specific factors that regulate their migration and proliferation under physiological conditions.

Periosteal stem cells are major contributors to bone healing

The researchers discovered specific markers for periosteum stem cells in mouse models. The markers identified a distinct subset of stem cells that contributes to life-long adult bone regeneration.

We also found that periosteum stem cells respond to mechanical injury by engaging in bone healing, Park said. They are important for healing bone fractures in the adult mice and, interestingly, their contribution to bone regeneration is higher than that of bone marrow stem cells.

In addition, the researchers found that periosteal stem cells also respond to inflammatory molecules called chemokines, which are usually produced during bone injury. In particular, they responded to chemokine CCL5.

Periosteal stem cells have receptors molecules on their cell surface that bind to CCL5, which sends a signal to the cells to migrate toward the injured bone and repair it. Deleting the CCL5 gene in mouse models resulted in marked defects in bone repair or delayed healing. When the researchers supplied CCL5 to CCL5-deficient mice, bone healing was accelerated.

The findings suggested potential therapeutic applications. For instance, in individuals with diabetes or osteoporosis in which bone healing is slow and may lead to other complications resulting from limited mobility, accelerating bone healing may reduce hospital stay and improve prognosis.

Our findings contribute to a better understanding of how adult bones heal. We think this is one of the first studies to show that bone stem cells are heterogeneous and that different subtypes have unique properties regulated by specific mechanisms, Park said. We have identified markers that enable us to tell bone stem cell subtypes apart and studied what each subtype contributes to bone health. Understanding how bone stem cell functions are regulated offers the possibility to develop novel therapeutic strategies to treat adult bone injuries.

Reference

Ortinau et al. (2019) Identification of Functionally Distinct Mx1+SMA+ Periosteal Skeletal Stem Cells. Cell Stem Cell. DOI: https://doi.org/10.1016/j.stem.2019.11.003

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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New Mechanism of Bone Maintenance and Repair Discovered - Technology Networks

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DUBLIN--(BUSINESS WIRE)--The "Global Blood and Bone Marrow Cancer Treatment Market Size, Market Share, Application Analysis, Regional Outlook, Growth Trends, Key Players, Competitive Strategies and Forecasts, 2019 to 2027" report has been added to ResearchAndMarkets.com's offering.

The global blood and bone marrow cancer treatment market was valued at US$ 38.8 Bn in 2018 and is expected to reach US$ 74.9 Bn by 2027, expanding at a CAGR of 7.7% from 2019 to 2027.

Market Insights

Blood cancer begins in the bone marrow which is the integral source of stem cells which later are differentiated in different types of blood cells in the human body. Researchers at Bristol Myers Squibb Company have stated that approximately 1.85 million new cases of blood cancer will be diagnosed by 2040 throughout the globe.

Lymphoma is the largest indication segment for blood and bone marrow cancer treatment market. It is prevalent in 2 types Hodgkin lymphoma and Non-Hodgkin lymphoma throughout the globe. The chief variables responsible for its rising prevalence worldwide are increasing prescription of immunosuppressant drugs for treating chronic infections and genetic mutations. Leukemia occurs when the DNA of immature white blood cells gets damaged due to exposure to ionizing radiation, hazardous chemicals, smoking, etc. The prevalence rate of leukemia is highly variable across different ethnic groups with men to women ratio of 1.4.

Chemotherapy is reigning the therapy segment for blood and bone marrow cancer treatment market. The key parameter hold responsible for its increasing demand is the availability of its generic version at affordable cost, drastically reducing the healthcare burden on ailing patients. Oncologists prefer to use them in combination therapy either with radiotherapy or immunotherapy to treat patients showing resistance to first-line drug therapy. Immunotherapy will be the fastest-growing segment during the forecast period owing to its promising drug pipeline for the treatment of blood cancer.

North America representing a market share of 34.6% is dominating the regional segment for blood and bone marrow cancer treatment market. The chief contributing factor for its market supremacy is a growing incidence of blood cancer. As per the research citings of the Leukemia and Lymphoma Society (CDC) figures after every 3 minutes, 1 person in the U.S. is diagnosed with blood cancer. In 2019, approximately 176,200 people in the U.S. are diagnosed with blood cancer in the United States. Europe holds a market share of 30.8% owing to the supportive regulatory framework provided by the European Medical Agency for the development and sale of medication for the treatment of blood cancer. The Asia Pacific accounts for 18.4% market share on account of rising public health awareness related to blood cancer & its treatment and developing healthcare infrastructure.

Key Market Movements:

Key Topics Covered:

Chapter 1. Preface

1.1. Report Scope and Description

1.1.1. Purpose of the Report

1.1.2. Target Audience

1.1.3. USP and Key Offerings

1.2. Research Scope

1.3. Research Methodology

1.3.1. Phase I-Secondary Research

1.3.2. Phase II-Primary Research

1.3.3. Approach Adopted

1.3.4. Top-down Approach

1.3.5. Bottom-up Approach

1.3.6. Phase III-Expert Panel Review

1.3.7. Assumptions

1.4. Market Segmentation

Chapter 2. Executive Summary

2.1. Global Blood and Bone Marrow Cancer Treatment Market Portraiture

2.2. Global Blood and Bone Marrow Cancer Treatment Market, by Indication, 2018 (US$ Bn)

2.3. Global Blood and Bone Marrow Cancer Treatment Market, by Therapy, 2018 (US$ Bn)

2.4. Global Blood and Bone Marrow Cancer Treatment Market, by Geography, 2018 (US$ Bn)

Chapter 3. Blood and Bone Marrow Cancer Treatment Market: Dynamics and Future Outlook

3.1. Market Overview

3.2. Drivers

3.3. Challenges

3.4. Opportunities

3.5. Attractive Investment Proposition, by Geography, 2018

3.6. Competitive Analysis: Global Blood and Bone Marrow Cancer Treatment Market, by Key Players, 2018

Chapter 4. Global Blood and Bone Marrow Cancer Treatment Market, by Indication

4.1. Overview

4.2. Multiple Myeloma

4.3. Leukemia

4.4. Lymphoma

4.5. Others

Chapter 5. Global Blood and Bone Marrow Cancer Treatment Market, by Therapy

5.1. Chemotherapy

5.2. Immunotherapy

5.3. Stem Cell Transplant

5.4. Radiotherapy

5.5. Pipeline Analysis

5.5.1. Phase III Drug

5.5.1.1. Eltrombopag

5.5.1.2. Avatrombopag

5.5.1.3. Hetrombopag

5.5.1.4. Omidubicel

5.5.1.5. Fedratinib

5.5.1.6. ATIR101

5.5.1.7. Pegylated Proline Interferon Alpha-2b

5.5.2. Tabular Representation of Phase II and I Pipeline Drugs

Chapter 6. Global Blood and Bone Marrow Cancer Treatment Market, by Geography

6.1. Overview

6.2. North America Blood and Bone Marrow Cancer Treatment Market Analysis, 2017- 2027

6.3. Europe Blood and Bone Marrow Cancer Treatment Market Analysis, 2017 - 2027

6.4. Asia Pacific Blood and Bone Marrow Cancer Treatment Market Analysis, 2017 - 2027

6.5. Latin America Blood and Bone Marrow Cancer Treatment Market Analysis, 2017 - 2027

6.6. Middle East and Africa Blood and Bone Marrow Cancer Treatment Market Analysis, 2017 - 2027

Chapter 7. Company Profiles

7.1. AstraZeneca, Plc.

7.1.1. Business Description

7.1.2. Financial Information (Subject to data availability)

7.1.3. Product Portfolio

7.1.4. News Coverage

7.2. Celgene, Inc.

7.3. Bristol Myers Squibb & Company

7.4. Eli Lilly & Company

7.5. Johnson & Johnson Company

7.6. F.Hoffman La-Roche Ltd.

7.7. Merck & Co., Inc.

7.8. Novartis AG

7.9. Pfizer, Inc.

7.10. Varian Medical Systems, Inc.

For more information about this report visit https://www.researchandmarkets.com/r/pi0qoz

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Global Blood and Bone Marrow Cancer Treatment Market Trends & Analysis During the Forecast Period, 2019-2027 - ResearchAndMarkets.com - Business...

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JSP191, a humanized antibody targeting CD117, is designed to replace or reduce the toxicity of chemotherapy and radiation therapy as a conditioning regimen to prepare patients for hematopoietic cell transplantation. The Phase 1 clinical trial is evaluating JSP191 as a conditioning agent to enable stem cell transplantation in patients with severe combined immunodeficiency (SCID) who received a prior stem cell transplant that had poor outcomes.

Life-threatening disorders such as SCID, and other conditions including autoimmune diseases and hematologic cancers, can be cured by hematopoietic cell transplantation, and those with certain genetic diseases can be cured with stem cell-directed gene therapies. However, the number of patients who can benefit from these approaches is limited because of the severe toxicity of the chemotherapy used for pre-transplant conditioning that is needed to allow room in the bone marrow for the stem cells to engraft, said Judith Shizuru, M.D., Ph.D., co-founder and member of the Board of Directors of Jasper Therapeutics. We are encouraged by the initial Phase 1 study results of JSP191 in these fragile patients with SCID and plan to expand clinical development of this antibody beyond patients with SCID. We expect to initiate clinical trials of JSP191 in 2020 to evaluate it as a conditioning agent in patients undergoing hematopoietic cell therapy for acute myeloid leukemia, myelodysplastic syndrome and Fanconi anemia, and IND-enabling studies for sickle cell disease and autoimmune indications.

Details of the oral presentation follow:

Abstract Title: Non-Genotoxic Anti-CD117 Antibody Conditioning Results in Successful Hematopoietic Stem Cell Engraftment in Patients with Severe Combined Immunodeficiency (abstract #800) Session Name: 721. Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities: Innovative Approaches in Allogeneic Transplantation for Pediatric or Nonmalignant DisordersPresenter: Rajni Agarwal, M.D., Associate Professor of Pediatrics and Stem Cell Transplantation, the Stanford University School of MedicineTime: 3:00 p.m. ETLocation: W311EFGH, Level 3, Orange County Convention Center

About Stem Cell Transplantation

Blood-forming, or hematopoietic, stem cells are 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. Successful transplantation of hematopoietic stem cells is the only cure for most of these life-threatening conditions. Replacement of the defective or malignant hematopoietic stem cells in the patients bone marrow is currently achieved by subjecting patients to toxic treatment with radiation and/or chemotherapy that cause DNA damage and lead to short- and long-term toxicities, including immune suppression and prolonged hospitalization. As a result, many patients who could benefit from a stem 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 JSP191

JSP191 (formerly AMG191) 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 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 dose-escalation 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.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on enabling safer conditioning and therapeutic agents that expand the application of curative hematopoietic stem cell transplants and gene therapies. Jasper Therapeutics lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a stem cell transplant. For more information, please visit us at http://www.jaspertherapeutics.com.

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Jasper Therapeutics Announces Upcoming Data Presentation on Lead Program, JSP191, at 61st American Society of Hematology (ASH) Annual Meeting &...

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DetailsCategory: DNA RNA and CellsPublished on Tuesday, 10 December 2019 10:25Hits: 254

GDA-201 demonstrated early evidence of clinical activity in patients with non-Hodgkin lymphoma, with multiple complete responses observed

Research on mechanism of action for the NAM technology platform provides further scientific rationale for stem cell engraftment and patient outcomes reported in previous Phase 1/2 clinical study of omidubicel

BOSTON, MA, USA I December 9, 2019 I Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to finding cures for blood cancers and serious blood diseases, today announced updated results from a Phase 1 clinical study of GDA-201, an investigational, natural killer (NK) cell-based cancer immunotherapy for the treatment of patients with non-Hodgkin lymphoma (NHL) and multiple myeloma (MM), at the 61st Annual Meeting of the American Society of Hematology (ASH), which is being held December 710 in Orlando, FL. Data from 22 patients in the ongoing study showed GDA-201 in combination with monoclonal antibodies was generally well tolerated and demonstrated early evidence of clinical activity in heavily pre-treated patients, including five complete responses observed among nine patients with NHL. Gamida Cell plans to initiate a Phase 1/2 multi-dose, multi-center study of GDA-201 in patients with NHL in 2020.

NK cells are increasingly recognized as a potential breakthrough approach in immunotherapy, and the data reported today provide early evidence that GDA-201 has the potential to be an important new treatment option, said Veronica Bachanova, M.D., Ph.D., Associate Professor of Medicine in the Division of Hematology, Oncology and Transplantation at the University of Minnesota and principal investigator of the study through the Masonic Cancer Center. Given the population of heavily pre-treated patients with advanced disease, its particularly encouraging to witness multiple complete responses. I look forward to the continued development of this investigational therapy.

New research was also presented today on the mechanism of action of Gamida Cells NAM-based cell expansion platform, which is designed to enhance the number and functionality of allogeneic donor cells. These data provide further scientific rationale for the favorable stem cell engraftment and patient outcomes observed in the Phase 1/2 clinical study of omidubicel, the companys advanced cell therapy currently in Phase 3 clinical development as a potential life-saving treatment option for patients in need of an allogeneic bone marrow transplant.

These mechanism of action data reinforce the transformative potential of our NAM therapeutic platform, which can be used to expand multiple cell types. Specifically for omidubicel, this research suggests that NAM modulates certain gene expression pathways that, collectively, mimic the hypoxic environment of the bone marrow to help preserve stem cell function and long-term engraftment ability, said Tracey Lodie, Ph.D., chief scientific officer of Gamida Cell. We expect to build on our findings by characterizing the metabolites produced when we expand stem cells to make omidubicel, and we are also beginning to conduct similar mechanism of action studies with GDA-201.

GDA-201 Phase 1 Clinical Data Presented at ASH

The oral presentation, Results of a Phase 1 Trial of GDA-201, Nicotinamide-Expanded Allogeneic Natural Killer Cells (NAM-NK) in Patients with Refractory Non-Hodgkin Lymphoma (NHL) and Multiple Myeloma (MM) (Abstract #777), described data from the Phase 1 clinical study of GDA-201 in heavily pre-treated patients with advanced NHL and MM. Twenty-two patients were enrolled in the study, including nine patients with NHL and 13 patients with MM. Of these 22 patients, all were evaluable for safety and 21 were evaluable for response (NHL = 9; MM = 12).

In the study, cell therapy using GDA-201 with monoclonal antibodies was generally well tolerated and demonstrated early evidence of clinical activity. Of the nine patients with NHL, five achieved a complete response and one achieved a partial response. Among the patients with MM, one patient achieved a complete response, and five patients achieved stable disease.

GDA-201 was generally well tolerated, with no graft vs. host disease (GvHD), no tumor lysis syndrome, no neurotoxicity and no marrow aplasia observed. No dose limiting toxicities were observed. Hypertension and hematologic events were the most common Grade 3/4 adverse events observed. Most non-hematologic toxicities were attributed to cyclophosphamide/fludarabine, which was used as a pre-conditioning treatment.

NAM Therapeutic Platform Mechanism of Action Data Presented at ASH

The poster presentation, Nicotinamide (NAM) Modulates Transcriptional Signature of Ex Vivo Cultured UCB CD34+ Cells (Omidubicel) and Preserves Their Stemness and Engraftment Potential (Abstract #3718), included transcriptome, transcription factor, and pathway analysis to elucidate the pathways leading to the preservation of engraftment after ex vivo expansion of CD34+ hematopoietic stem cells derived from umbilical cord blood (the starting point for omidubicel) compared to CD34+ cells grown in the absence of NAM.

Analyses showed that the presence of NAM reduced the expression of genes involved in the production of reactive oxygen and nitrogen species, suggesting that cell stress was minimized during expansion. In addition, NAM also decreased growth factor pathways responsible for activation and differentiation of hematopoietic stem cells, suggesting NAM expanded cells while keeping them in an undifferentiated state. The presence of NAM also led to a decrease in the expression of genes responsible for matrix-metallo proteinase secretion, simulating the microenvironment of the bone marrow. Additionally, NAM led to an increased expression of telomerase genes, which is believed to enable cells to remain in a more quiescent, stem-like state. These data provide further scientific rationale for the favorable stem cell engraftment and patient outcomes that were observed in the Phase 1/2 clinical study of omidubicel.

About GDA-201 GDA-201 (formerly known as NAM-NK) is being developed as an innate natural killer (NK) cell immunotherapy for the treatment of hematologic and solid tumors in combination with standard-of-care antibody therapies. NK cells have the ability to kill tumor cells, representing a novel immunotherapeutic approach to cancer treatment. GDA-201 is designed to address key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs of NK cells expanded in culture. GDA-201 is in Phase 1 development in patients with refractory non-Hodgkin lymphoma and multiple myeloma.1 For more information on the clinical study of GDA-201, please visit http://www.clinicaltrials.gov.

About Omidubicel Omidubicel (formerly known as NiCord), the companys lead clinical program, is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has also received Orphan Drug Designation in the U.S. and EU. In a Phase 1/2 clinical study, omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated.2 A Phase 3 study evaluating omidubicel in patients with leukemia and lymphoma is ongoing in the U.S., Latin America, Europe and Asia.3 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia.4 The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit http://www.clinicaltrials.gov.

GDA-201 and omidubicel are investigational therapies, and their safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.

About the NAM Therapeutic Platform Gamida Cells proprietary NAM-based cell expansion platform is designed to enhance the number and functionality of donor cells in culture, enabling the creation of potentially transformative therapies that move beyond what is possible with existing approaches. The NAM therapeutic platform leverages the unique properties of nicotinamide to enable the expansion of multiple cell types including stem cells and natural killer (NK) cells with appropriate growth factors to maintain the cells original phenotype and potency. This can enable the administration of a therapeutic dose of cells with the potential to improve patient outcomes.

About Gamida Cell Gamida Cell is an advanced cell therapy company committed to finding cures for patients with blood cancers and serious blood diseases. We harness our cell expansion platform to create therapies with the potential to redefine standards of care in areas of serious medical need. For additional information, please visit http://www.gamida-cell.com.

1ClinicalTrials.gov identifier NCT03019666. 2 Horwitz M.E., Wease S., Blackwell B., Valcarcel D. et al. Phase I/II study of stem-cell transplantation using a single cord blood unit expanded ex vivo with nicotinamide. J Clin Oncol. 2019 Feb 10;37(5):367-374. 3 ClinicalTrials.gov identifier NCT02730299 4 ClinicalTrials.gov identifier NCT03173937

SOURCE: Gamida Cell

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Gamida Cell Announces Results from Phase 1 Study of GDA-201 and New Mechanism of Action Data at ASH 2019 Annual Meeting | DNA RNA and Cells | News...

Recommendation and review posted by Bethany Smith

PALO ALTO, Calif.--(BUSINESS WIRE)--Jasper Therapeutics, Inc., a new biotechnology company focused on enabling safer conditioning and therapeutic agents that expand the application of curative hematopoietic stem cell transplants and gene therapies, today announced the launch of the company with a $35 million total Series A financing. Abingworth LLP and Qiming Venture Partners USA served as lead investors, with further investment from Surveyor Capital (a Citadel company) and participation from Alexandria Venture Investments, LLC. The proceeds will be used to advance the clinical development of the companys lead product candidate, JSP191, which is designed to replace or reduce the toxicity of chemotherapy and radiation therapy as a conditioning regimen to prepare patients for hematopoietic cell transplant.

Jaspers development of JSP191 is also supported by a collaboration with the California Institute for Regenerative Medicine (CIRM), which has been funding the program and is committed to providing a total of $23 million in grant support. As part of the Series A financing, Amgen, which discovered JSP191 (formerly AMG191), has licensed worldwide rights to Jasper that also include translational science and materials from Stanford University.

Jasper was co-founded by Judith Shizuru, M.D., Ph.D., a hematopoietic stem cell transplant expert at Stanford University, and Susan Prohaska, Ph.D., a Stanford University-trained immunologist, stem cell biologist and early-stage drug development professional. Dr Shizurus CIRM-funded lab advanced the understanding of the ability of anti-CD117 to impact hematopoietic stem cells and, together with the Lucile Packard Childrens Hospital Stanford and University of California, San Francisco (UCSF) pediatric transplant teams, was the first to study an anti-CD117 antibody in the clinic as a conditioning agent. That humanized antibody, now called JSP191, was first studied for conditioning for transplant in immune-deficient patients in collaboration with Amgen, UCSF and CIRM.

Stem cell transplantation is a potential curative therapy for people with hematologic cancers, autoimmune diseases, and debilitating genetic diseases. However, the pre-transplant conditioning required to prepare patients for transplant involves highly toxic chemotherapy, which can be life-threatening and limits the number of people who are able to benefit, said Dr. Shizuru, co-founder and member of the Board of Directors of Jasper Therapeutics. JSP191 is the only anti-CD117 antibody to demonstrate safety and efficacy in severely ill patients receiving stem cell transplant in the clinic. We plan to expand clinical development to patients receiving transplants for acute myeloid leukemia/ myelodysplastic syndrome or autoimmune diseases and to patients receiving stem cell-directed gene therapies.

Dr. Shizuru added, With an experienced executive team of biotech veterans and a strong syndicate of healthcare-focused investors, Jasper Therapeutics is well positioned to achieve our vision of building a leading biotech company starting with JSP191 and expanding to other novel therapies for immune modulation, graft engineering and cell and gene therapies.

JSP191 is currently being evaluated in an ongoing Phase 1 clinical trial as a conditioning agent to enable stem cell transplantation in patients with severe combined immunodeficiency (SCID) who received a prior stem cell transplant that failed. This severe genetic immune disorder leaves patients without a functioning immune system. Interim results of the study will be presented in an oral presentation (abstract #800) on Monday, December 9, at the 61st American Society of Hematology (ASH) Annual Meeting & Exposition in Orlando, Fla. Clinical studies to evaluate the safety and efficacy of JSP191 as a conditioning agent in patients undergoing hematopoietic cell therapy for hematologic cancers are planned for 2020.

Founding Management Team

Dr. Shizuru and Mr. Lis are joined on the Jasper Therapeutics Board of Directors by Kurt von Emster, Managing Partner of Abingworth LLP, and Anna French, Ph.D., Principal at Qiming Venture Partners USA. Dr. Prohaska is a Board observer.

With our investment in this program, were able to realize our mission of fast-tracking stem cell treatments by helping academic researchers rapidly advance the most promising discoveries in the lab into the clinics and to drug development with commercialization partners, said Maria T. Millan, M.D., President and CEO of CIRM. Jaspers two co-founders took a novel antibody with unique properties and moved it from the bench to the bedside relatively quickly, and were thrilled to partner with this talented team to potentially impact a broad group of people who could benefit from stem cell therapy.

About Stem Cell Transplantation

Blood-forming, or hematopoietic, stem cells are 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. Successful transplantation of hematopoietic stem cells is the only cure for most of these life-threatening conditions. Replacement of the defective or malignant hematopoietic stem cells in the patients bone marrow is currently achieved by subjecting patients to toxic doses of radiation and/or chemotherapy that cause DNA damage and lead to short- and long-term toxicities, including immune suppression and prolonged hospitalization. As a result, many patients who could benefit from a stem 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 JSP191

JSP191 (formerly AMG191) 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 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 dose-escalation 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.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on enabling safer conditioning and therapeutic agents that expand the application of curative hematopoietic stem cell transplants and gene therapies. Jasper Therapeutics lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a stem cell transplant. For more information, please visit us at http://www.jaspertherapeutics.com.

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Jasper Therapeutics Launches with $35 Million Series A Financing to Develop and Commercialize Innovative Conditioning Agents and Therapies to...

Recommendation and review posted by Bethany Smith

--(BUSINESS WIRE)--Jasper Therapeutics is a biotechnology company focused on enabling safer conditioning agents and therapeutics to allow for expanded use of curative hematopoietic stem cell transplants and gene therapies. Jasper Therapeutics lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a stem cell transplant. For more information, please visit us at http://www.jaspertherapeutics.com.

Company:

Jasper Therapeutics, Inc

Headquarters Address:

3000 Sand Hill Road B1-145

Menlo Park, CA

Main Telephone:

1-650-254-6687

Website:

https://jaspertherapeutics.com/

Type of Organization:

Private

Industry:

Biotechnology

Key Executives:

Executive Chairman and Interim Chief Executive Officer: William Lis

Chief Business Officer, Chief Financial Officer: Jeet Mahal

Company Contact

Contact:

Jeet Mahal

Phone:

1-650-254-6687

Email:

jmahal@jaspertherapeutics.com

Public Relations

Contact:

Julie Normart

Phone:

1-415-946-1087

Email:

jnormart@w2ogroup.com

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Company Profile for Jasper Therapeutics, Inc - Business Wire

Recommendation and review posted by Bethany Smith

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering -- by editing her genome.

Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy.

"I have hoped for a cure since I was about 11," the 34-year-old told AFP in an email. "Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency."

Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness -- stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 -- pronounced "Crisper" -- a new tool informally known as molecular "scissors."

The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

"This is one patient. This is early results. We need to see how it works out in other patients," said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville. "But these results are really exciting."

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

"It's all developing very quickly," said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practising the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not -- such as making normal red blood cells, in Victoria's case, or making tumor-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the U.S. and a blood disease in the European Union.

They join several other gene therapies -- bringing the total to eight -- approved in recent years to treat certain cancers and an inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

"Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris. "It took a generation for gene therapy to become a reality. Now, it's only going to go faster."

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a "breakthrough period."

"We have hit an inflection point," said Carrie Wolinetz, NIH's associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million -- meaning patients face grueling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion -- and fighting a general infection. "You cannot do this in a community hospital close to home," said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers. They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal -- and his excommunication from the scientific community -- when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

"That technology is not safe," said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr "scissors" often cut next to the targeted gene, causing unexpected mutations.

"It's very easy to do if you don't care about the consequences," Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species -- malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the U.S.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American "biohackers" injecting themselves with Crispr technology bought online.

"Not everyone is a biologist or scientist," she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies' crops?

Charpentier thinks that technology generally tends to be used for the better.

"I'm a bacteriologist -- we've been talking about bioterrorism for years," she said. "Nothing has ever happened."

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2019: the year gene therapy came of age - Japan Today

Recommendation and review posted by Bethany Smith

DURHAM, N.C., Dec. 10, 2019 (GLOBE NEWSWIRE) -- Chimerix, Inc. (Nasdaq: CMRX), a biopharmaceutical company focused on accelerating the development of medicines to treat cancer and other serious diseases, today announced that data relating to its dociparstat sodium (DSTAT) program, formerly known as CX-01, were presented at the 61st American Society of Hematology Annual Meeting, in Orlando, FL.

The poster, titledUpdated Study Results for CX-01, an Inhibitor of CXCL12/CXCR4, With Azacitidine for the Treatment of Hypomethylating Agent Refractory AML and MDS, was presented byEric Huselton, M.D., Assistant Professor of Medicine at the University of Rochester on December 9, 2019.

As reported in the published study abstract, 20 patients with refractory myelodysplastic syndrome (MDS) (n = 9) or refractory acute myeloid leukemia (AML) (n = 11) were enrolled of which 15 were considered evaluable for response with a bone marrow biopsy after cycle 2. Patients received a 7-day continuous infusion of DSTAT (CX-01) at a dose of 0.25 mg/kg/hour, and azacitidine 75 mg/m2 daily days 1-7, in 28-day cycles. The primary objective of this trial was to assess the overall response rate. Half of the patients had high risk cytogenetic abnormalities and 3 had TP53 mutations. Patients had a median of 2 prior lines of therapy (range 1-3) with median of 6 prior cycles of hypomethylating agent (HMA) therapy (range 4-20). Only 4 patients had a confirmed response to prior HMA therapy.

The 15 evaluable patients received a median of 3 cycles of CX-01 and azacitidine (range 2-9). Of 15 evaluable patients, there was 1 CR (complete remission) and 3 bone marrow CRs (mCR, with incomplete peripheral blood count recovery), 9 stable disease, and 2 progressive disease for an overall response rate of 27%. Of the 3 patients with a mCR after cycle 2, two had hematologic improvement of their neutrophil and platelet counts, respectively, by the end of cycle 4. A patient with stable disease also had hematologic improvement in platelets.

The median overall survival of evaluable patients was 221 days. The median overall survival was not significantly different between AML patients at 221 days and MDS patients at 248 days.

Following a minimum of 4 cycles of prior HMA therapy, one would not expect to observe response to subsequent HMA therapy, said Dr. Huselton. These results demonstrate DSTATs potential to improve HMA therapy outcomes in terms of both response and overall survival.

"DSTATs mechanism of action is intended to enhance patient benefit when combined with an active agent, so to observe these results in HMA-refractory patients is promising. In addition to our planned Phase 3 pivotal trial in newly diagnosed AML, this study highlights the potential to develop DSTAT to enhance the benefit of multiple therapies such as azacitidine, in AML and MDS in both front-line and recurrent settings," said Mike Sherman, Chief Executive Officer of Chimerix.

AboutChimerix

Chimerixis a development-stage biopharmaceutical company dedicated to accelerating the advancement of innovative medicines that make a meaningful impact in the lives of patients living with cancer and other serious diseases. The two clinical-stage development programs are dociparstat sodium (DSTAT) and brincidofovir (BCV).

Dociparstat sodium is a potential first-in-class glycosaminoglycan biologic derived from porcine heparin that has low anticoagulant activity but retains the ability to inhibit activities of several key proteins implicated in the retention and viability of AML blasts and leukemic stem cells in the bone marrow during chemotherapy (e.g., CXCL12, selectins, HMGB1). Mobilization of AML blasts and leukemic stem cells from the bone marrow has been associated with enhanced chemosensitivity and may be a primary mechanism accounting for the observed increases in EFS and OS in Phase 2 with DSTAT versus placebo. Randomized Phase 2 data suggest that DSTAT may also accelerate platelet recovery post-chemotherapy via inhibition of platelet factor 4, a negative regulator of platelet production that impairs platelet recovery following chemotherapy. BCV is a lipid conjugate DNA polymerase inhibitor in development as a medical countermeasure for smallpox.For further information, please visit the Chimerix website,www.chimerix.com

CONTACT:

Investor Relations:Michelle LaSpaluto919-972-7115ir@chimerix.com

Will OConnorStern Investor Relations212-362-1200will@sternir.com

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Chimerix Presents Updated Results from Phase 2 Clinical Trial of DSTAT in Refractory Myelodysplastic Syndrome and Acute Myeloid Leukemia at American...

Recommendation and review posted by Bethany Smith

Using immunotherapy with genetically modified T cells that express chimeric antigen receptors or CARs designed to target tumor-associated molecules, have impressive efficacy in the treatment hematological malignancies.

A CAR is a synthetic construct that, when expressed in T cells, mimics T cell receptor activation and redirects specificity and effector function toward a specified antigen.[1]

In the treatment of cancer, this process is accomplished by linking an extracellular ligand-binding domain specific for a tumor cell surface antigen to an intracellular signaling module that activates T cells upon antigen binding.[1]

The presented studies include results from emerging second-generation cellular immunotherapy products that strive to overcome the limitations of existing products such as resistance and reduce toxicity and simplify treatment.

Cellular immunotherapy uses genetic engineering to enhance the ability of the immune system the bodys defense system against infection and disease to kill malignant cells in the blood, the bone marrow, and other sites, in order to keep cancer from coming back.

CAR T-cell TherapyChimeric antigen receptor T-cell therapies, better known as CAR T-cell therapies, are developed by harvesting a patients own T-cells, the immune systems primary cancer-killing cells, engineering them to target proteins specific to the surface of cancer cells, and reintroducing these modified T-cells back into the patients immune system to kill the cancer cells.

First generationFirst-generation CAR T-cell therapies primarily target CD-19, a protein found on the surface of most normal and malignant B cells in B cell cancers such as lymphoma. These therapies have been shown to produce long-term remissions in about one-third of patients with B-cell lymphomas that have not responded to prior therapies.

We are now seeing efforts to enhance the effectiveness of CAR T-cell therapy by designing products capable of attacking multiple targets, expand the availability of cellular immunotherapy to other blood cancers such as multiple myeloma and replace the complex manufacturing process required for CAR T-cell therapy with a uniform off-the-shelf product, noted Gary Schiller, MD, UCLA Health, an academic medical center which includes a number of hospitals and an extensive primary care network in the Los Angeles, California, region.

One of the phase I studies evaluates an off-the-shelf cellular immunotherapy product that targets two proteins found on the surface of lymphoma cells, including its potential to revive previously administered CAR T-cells that have stopped working.

Another study presents preclinical results for one of the first cellular immunotherapies to be based on off-the-shelf natural killer (NK) cells and the first, according to its manufacturer, to be genetically engineered to contain three active anti-tumor components.

The other two studies, also phase I studies, assess novel CAR T-cell therapies for multiple myeloma that test different dual-target strategies.

One investigational agent is genetically engineered to contain two proteins that attach to BCMA, a protein found almost exclusively on the surface of plasma cells, the immune-system cells that become cancerous in multiple myeloma.

The other is designed to target both BCMA and CD-38, another protein found on the surface of plasma cells. In both studies, many patients achieved minimal residual disease (MRD) negativity, which means that using highly sensitive testing fewer than one myeloma cell per 100,000 cells was identified in the bone marrow. Previous studies have shown that patients who achieve this milestone have a lower risk of relapse after more than three years of follow-up.

Dual-targeted CAR T-cell therapiesThe three phase I studies also hint at the possibility that dual-targeted CAR T-cell therapies might result in fewer patients experiencing moderate to severe cytokine release syndrome (CRS), a known adverse effect caused by an immune response in the body to the activated T cells that are attacking the cancer. CRS causes flu-like symptoms such as fever, body aches, and fatigue, and in severe cases can be life-threatening. Treatment with the drug tocilizumab can reduce CRS symptoms.

Dual-Targeted Antibody Elicits Durable ResponsesPatients with B-cell Non-Hodgkin Lymphoma (NHL) that had returned after or failed to respond to a median of three prior therapies showed complete responses (CR) and durable remissions after being treated with an investigational drug called mosunetuzumab (RG7828; Genentech/Roche). [2]

This investigational agent is a humanized, T-cell bispecific antibody designed to engage T cells and redirect their cytotoxic activity against malignant B cells. The drug works by activating the patients own T-cells, stimulating them to attack and kill cancerous B cells to which they have been introduced by the novel antibody.

Mosunetuzumab simultaneously binds to CD3 epsilon (CD3), a component of the T-cell receptor (TCR) complex, and to CD20, a B-cell surface protein expressed in a majority of B-cell malignancies. This results in crosslinking of the TCR, inducing downstream signaling events that leads to B-cell killing.

Among patients whose lymphoma progressed after treatment with CAR T-cell therapy, 22% had complete remissions when treated with mosunetuzumab. This new drug targets two proteins, one on the surface of tumor cells and the other on the surface of the recipients Tcells.

Unlike CAR T-cell therapy, mosunetuzumab is an off-the-shelf immunotherapy product that can be given to patients without having to genetically modify their T cells, noted lead author Stephen J. Schuster, MD, of Abramson Cancer Center at the University of Pennsylvania in Philadelphia.

Mosunetuzumab generates long-lasting responses with a very tolerable safety profile in patients with B-cell non-Hodgkin lymphomas for whom multiple prior treatments have failed and whose prognosis is poor. Of particular interest, we are seeing durable complete remissions in patients whose lymphomas progressed after CAR T-cell therapy, he added.

The researchers observed many remissions continue after patients stop receiving the drug.

I have stopped therapy in some patients after six months and they have remained in remission. Some patients have remained in remission without additional therapy for more than a year, Schuster said.

New treatment options are needed not only for patients in whom CAR T-cell therapy has failed, but also for those patients whose lymphomas are getting worse so quickly that they cannot wait for CAR T-cell manufacturing, which takes several weeks, Schuster explained.

The data presented during the annual meeting of the American Society of Hematology included 270 patients (median age 62, 172 men) enrolled in the phase I trial in seven countries (the United States, Australia, Canada, Germany, South Korea, Spain, and the United Kingdom). All participating patients had B-cell lymphomas that had come back or not responded to a median of three prior therapies. Two-thirds of patients (67%) had fast-growing lymphomas; 85 (31%) patients had more slow-growing forms of the disease. In 30 patients (11%), the cancer was resistant to or returned after an initial response to CAR T-cell therapy; in 77 patients (29%), the disease had progressed after a stem cell transplant.

All patients were treated with mosunetuzumab by intravenous infusion. They had an imaging test at either six weeks or three months after starting therapy to assess the initial response to treatment, and responses continued to be followed every three months thereafter.

Forty-six of 124 patients with fast-growing lymphomas (37%) had measurable decreases in the extent of their cancer (objective response); 24 of 124 patients (19%) saw all detectable tumors disappear (complete response). A higher response rate was observed in patients with higher exposure to mosunetuzumab. Among patients with slow-growing lymphomas, 42 of 67 (63%) had objective responses and 29 of 67 (43%) had complete responses. Both objective response rate and complete response rate were maintained in subgroups of patients at high risk for relapse.

Complete remissions appear to be long lasting, Schuster said.

With a median follow-up of six months since first complete remission, 24 of 29 patients (83%) who achieved complete remissions of their slow-growing lymphomas and 17 of 24 patients (71%) who achieved complete remissions of their fast-growing lymphomas remain free of disease. In some patients whose cancers progressed after receiving CAR T-cell therapy, highly sensitive molecular testing showed that the previously administered CAR T cells increased in number.

This suggests that, in addition to its ability to kill cancerous B cells, mosunetuzumab may also help augment the effect of the prior CAR-T treatment, Schuster noted.

Cytokine-release syndromeIn this study, 29% of patients treated with mosunetuzumab experienced cytokine-release syndrome that was mostly mild.

Cytokine release syndrome or CRS is caused by a large, rapid release of cytokines into the blood from immune cells affected by the immunotherapy. While most patients have a mild reaction, sometimes patients may have a severe, life threatening, reaction.

In 3% of patients, CRS was treated with tocilizumab (Actemra; Genentech/Roche). Four percent of patients experienced moderately severe neurologic side effects. Patients who received higher doses of mosunetuzumab were no more likely to have CRS or neurologic side effects than patients treated at lower doses.

A study of a higher dose of mosunetuzumab is now enrolling patients and long-term follow-up of these patients will ultimately help to better evaluate the durability of response data.

Larger, randomized trials are needed to further confirm these promising data and determine whether the treatment benefit of mosunetuzumab is enhanced when it is used earlier in the course of lymphoma therapy or in combination with other agents, Schuster concluded.

Novel Off-the-Shelf CARPreclinical studies provide the first evidence that cellular immunotherapy for B cell cancers could ultimately become an off-the-shelf product, capable of being uniformly manufactured in large quantities as prescription drugs are.

We have taken the concept of traditional pharmaceutical drug development and applied it to cellular therapy, explained senior author Bob Valamehr, Ph.D, of Fate Therapeutics, a San Diego biopharmaceutical company.

The product called FT596, is among the first cellular immunotherapies to be based on off-the-shelf NK cells the first line of defense of the immune system and is the first cellular immunotherapy to be genetically engineered to contain three active anti-tumor components, Valamehr explained.

Comparable with standard CAR T-cell therapyFT596 demonstrated comparable ability to kill cancerous white blood cells as standard CAR T-cells and, when combined with the drug rituximab (Rituxan; Genentech/Roche), killed cancerous white blood cells that were no longer responding to standard CAR T-cell therapy due to loss of the CD19 antigen target.

The U.S. Food and Drug Administration (FDA) approved Fate Therapeutics Investigational New Drug Application for FT596 in September 2019 and the company hopes to begin a first-in-human phase I clinical trial for the treatment of B-cell lymphoma and chronic lymphocytic leukemia in the first quarter of 2020.

The primary purpose of this trial will be to assess the safety and activity of FT596 in patients.

ManufacturingThe development and manufacturing of FT596 begins with human induced pluripotent stem cells (iPSCs) that are uniquely capable of unlimited self-renewal and can differentiate into more than 200 types of human cells. These iPSCs are genetically engineered, after which a single genetically engineered cell or clone is selected and multiplied in the laboratory to create a master engineered cell line that can be repeatedly used to generate cancer-fighting immune-system cells such as NK and T cells.

Natural Kiler Cells or NK cells are a type of lymphocyte and a component of innate immune system, the bodys first line of defense against infection and disease. Unlike T-cells, which have to be trained to recognize their target and can kill only cells that display that target on their surface, NK cells do not need special preparation before going on the attack and can kill many different types of transformed or infected cells.

NK cells are multifaceted and can be viewed as a jack-of-all-trades when it comes to protecting the host, whereas T cells can act in only one way, Valamehr explained.

But NK cells are also different in other ways. They are inherently limited in their capacity to multiply and expand when infused into patients, and they have a shorter lifespan.

Valamehr and his colleagues used genetic engineering to address these shortcomings. In addition to engineering FT596 to carry a CAR targeting the CD19 protein, which is produced by nearly all B-cell lymphomas and leukemias, they inserted two other novel proteins: CD16, which boosts and broadens the NK cells ability to kill cancer cells, and IL15, which stimulates FT596 to proliferate and persist.

Valamehr explained that FT596 has been designed to address two more limitations of CAR T-cell therapy .

The investigational agent is an off-the-shelf product. As a result, it significantly improves the current patient-by-patient CAR T-cell treatment paradigm by eliminating the time-consuming and costly process that is currently required to treat a patient with CAR T-cells.

The addition of the CD16 protein gives FT596 broader therapeutic activity and versatility. In combination with rituximab, FT596 has the potential to lead to deeper and more durable responses and overcome resistance that hampers the long-term efficacy of CAR T-cell therapy.

Eliminating the high production cost, weeks of manufacturing time, and complex manufacturing process required for CAR T-cell therapy and replacing it with a mass-produced, off-the-shelf product, promises to expand access to effective cell-based cancer immunotherapy to many more patients who may benefit from it, Valamehr concluded.

Results from CARTITUDE-1 in R/R Multiple MyelomaPatients with multiple myeloma who had received a median of five prior therapies, and for whom standard-of-care treatments were no longer working, had a high response rate when treated with the investigational CAR T-cell therapy JNJ-68284528 (JNJ-4528), which targets BCMA, a protein commonly found on the surface of multiple myeloma cancer cells.

These patients participated in a clinical trials (NCT03548207), supported by Janssen Research & Development, designed to characterize safety of and establish the recommended Phase II dose (RP2D) (Phase Ib) and to evaluate the efficacy of JNJ-68284528 (Phase II).

We are seeing a high response rate, with most patients achieving MRD negativity, noted lead study author Deepu Madduri, MD, of The Tisch Cancer Institute at Mount Sinai in New York.

Considering these patients have all received multiple prior therapies, these results are extremely encouraging, Madduri added.

All evaluable patients receiving this CAR T-cell therapy have achieved MRD-negative disease state and 27 of 29 patients are progression free at a median follow-up of six months, Madduri said.

Multiple myeloma is a cancer of plasma cells, which are found in the bone marrow and are part of the immune system, the bodys defense system against infection. Typical signs and symptoms of multiple myeloma may be bone pain or fractures, high levels of calcium in the blood, kidney damage, and anemia. Multiple myeloma affects an estimated 160,000 people each year, occurs most often in people over 60. The disease is slightly more common in men than in women.

Although new therapies for multiple myeloma have recently become available that can extend patients life expectancy, a cure for the disease remains elusive.

We can get the disease into remission, but most patients unfortunately relapse, and outcomes are very poor for patients who have relapsed multiple times, she said.

Researchers explained that JNJ-4528 is a novel CAR T-cell therapy featuring two molecules that bind to BCMA, a protein found on the surface of multiple myeloma cells.

We are learning that every CAR T-cell therapy is different, Madduri said.

JNJ-4528 has a unique CAR T-cell composition in patients, preferentially enriched in CD8 T cells, which are believed to be one of the most important T cells in killing cancer cells, she noted.

This phase Ib/II trial is continuing to enroll patients.

During the 2019 annual meeting of the American Society of Hematology, Madduri reported results for the first 29 patients enrolled.

Patients T-cells were collected and sent to a laboratory where they were genetically engineered to express JNJ-4528. Prior to re-infusing these CAR T-cells, the patients received three days of chemotherapy to make room in their immune systems for the engineered T-cells.

Following chemotherapy, each patient received a single infusion of the JNJ-4528 CAR T-cells.

After a minimum of 28 days, these patients had blood and bone marrow exams, which was followed by exams at six months, and one year after treatment to assess their response. The primary aims of the trial are to assess the therapys safety and to confirm the dose to be tested in a larger, phase II trial.

The median follow-up time in the current analysis is six months. Overall, 100% of patients had a clinical response to JNJ-4528. Moreover, 66% had a stringent complete response, meaning that sensitive laboratory and microscopic tests found no evidence for myeloma proteins or cells in blood, urine, or bone marrow.

Most patients (93%) experienced some form of CRS. One patient had severe (grade 3) CRS, and one patient died from its complications 99 days after the CAR T-cell infusion. In 76% of patients, CRS was treated with tocilizumab.

To see some patients in this heavily pretreated population surviving for a year or more with a one-time treatment and a manageable safety profile is remarkable, Madduri explained.

These patients feel that they have their quality of life back. They no longer have to come into the clinic for weekly treatments and some are well enough to travel, Madduri concluded.

The phase II portion of this study is ongoing to evaluate the overall response rate of patients treated with JNJ-68284528 (JNJ-4528). Additional clinical studies are evaluating the safety and efficacy of JNJ-4528 in different multiple myeloma treatment settings.

BreakthroughEarlier this week the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for JNJ-68284528 (JNJ-4528).

The granting of Breakthrough Therapy Designation for JNJ-68284528 (JNJ-4528) is a significant milestone as we continue to accelerate the global development of this innovative CAR-T therapy in collaboration with Legend Biotech, noted Sen Zhuang, MD, Ph.D., Vice President, Oncology Clinical Development, Janssen Research & Development.

We look forward to continuing to work closely with the U.S. Food and Drug Administration to advance the clinical development program for JNJ-68284528 (JNJ-4528) and ultimately bring this BCMA-targeted immunotherapy to patients living with multiple myeloma who are in need of a new therapeutic option, Zhuang concluded.

Encouraging Results for Dual-Targeted CAR T-Cell TherapyMore than three out of four patients with multiple myeloma that returned or did not respond to at least two therapies remained in remission seven months after treatment with a novel CAR T-cell therapy targeting two proteins that are frequently found on myeloma cells.

Nine patients experiencing sustained remissions in this study, which ws supported by the National Natural Science Foundation of China, the Major Technological Innovation Special Project fund of Hubei Province of China, and Cellyan Therapeutics, were diagnosed with a difficult-to-treat form of multiple myeloma in which the disease has spread beyond the bone marrow.

Roughly one in 10 patients with multiple myeloma develop tumors in the organs or soft tissues such as the blood vessels, muscles, and nerves. These so-called extramedullary tumors respond poorly to treatment, and patients who develop them have a poor outlook and poor health related quality of life (hrQoL)

Our results show that this CAR T-cell product can effectively achieve elimination of extramedullary tumors, said study author Yu Hu, MD, Ph.D, of Union Hospital, Huazhong University of Science and Technology in Wuhan, China.

Although these are preliminary data, they are encouraging for patients with multiple myeloma who have not responded to other therapies, Hu added.

Hu and his colleagues are developing the first CAR T-cell therapy to be genetically engineered to target BCMA and CD38, two proteins found on the surface of plasma cells. Multiple myeloma is a cancer of plasma cells, which are found in the bone marrow and are part of the immune system, the bodys defense system against infection and disease.

Our thinking was that targeting both of these proteins would improve treatment efficacy without increasing toxicity, and induce deeper, more durable remissions, Hu noted.

The first-in-humans phase I trial enrolled 22 patients whose average age was 59, of whom 11 were men. All had multiple myeloma that had returned or not responded to at least three therapies. Nine of the 22 patients had extramedullary tumors. The study aims were to determine the safest and most effective dose of the CAR T-cell therapy as well as to initially evaluate its effectiveness.

Just like in other trials with CAR T-cell therapies, the participating patients received three days of chemotherapy to make room in their immune systems for the engineered T-cells. Then each patient was infused with the dual-targeted CAR T cells. Patients were divided into five groups, with each group receiving a higher dose than the previous one. Depending on the cell dose, patients received either one or two infusions.

At a median of 36 weeks of follow-up, 18 patients (90.9%) had MRD-negative disease. Twelve patients (54.5%) had a stringent complete response, meaning that no plasma cells were detected in the bone marrow. Seven patients (31.8%) had a good or very good partial response, meaning that the level of M-protein (an abnormal protein produced by cancerous plasma cells) in the blood or urine was reduced but still detectable. In eight of the nine patients with extramedullary lesions, these tumors were undetectable on their computed tomography scans. For the 17 patients who remained in remission at seven months after treatment, the median duration of response was 28.8 weeks.

The adverse events observed included 20 patients who experienced CRS, of whom six needed treatment. No serious adverse neurologic effects such as seizures, movement impairment, difficulty speaking or understanding speech, or fatal swelling in the brain were reported.

With this dual-targeted CAR T-cell therapy, we have demonstrated a high response rate, especially a higher rate and longer duration of stringent complete response, compared with other therapies, as well as effective elimination of extramedullary lesions, with no serious neurologic adverse effects and manageable levels of other adverse effects, Hu concluded.

The investigators continue to follow the patients for the next two years. They are also planning to conduct a phase II trial in both China and the United States to test the treatments effectiveness in a larger number of patients.

Clinical trialsA Study of JNJ-68284528, a Chimeric Antigen Receptor T Cell (CAR-T) Therapy Directed Against B-Cell Maturation Antigen (BCMA) in Participants With Relapsed or Refractory Multiple Myeloma (CARTITUDE-1) NCT03548207

References[1] Srivastava S, Riddell SR. Chimeric Antigen Receptor T Cell Therapy: Challenges to Bench-to-Bedside Efficacy. J Immunol. 2018;200(2):459468. doi:10.4049/jimmunol.1701155 [Abstract][2] Schuster SJ, Bartlett NL, Assouline S, Yoon SS, Bosch F, Sehn LH, Cheah CY, Shadman M, et al. Mosunetuzumab Induces Complete Remissions in Poor Prognosis Non-Hodgkin Lymphoma Patients, Including Those Who Are Resistant to or Relapsing After Chimeric Antigen Receptor T-Cell (CAR-T) Therapies, and Is Active in Treatment through Multiple Lines. 61st annual meeting of the American Society of Hematology. Program: General Sessions. Session: Plenary Scientific Session. Hematology Disease Topics & Pathways: antibodies, Follicular Lymphoma, CRS, Diseases, Biological, Therapies, neurotoxicity, Adverse Events, CAR-Ts, Non-Hodgkin Lymphoma, DLBCL, immunotherapy, Lymphoid Malignancies. [Abstract][3] Goodridge JP, Mahmood S, Zhu H, Gaidarova S, Blum R, Bjordahl R, Cichocki F, et al. FT596: Translation of First-of-Kind Multi-Antigen Targeted Off-the-Shelf CAR-NK Cell with Engineered Persistence for the Treatment of B Cell Malignancies. 61st annual meeting of the American Society of Hematology. Program: Oral and Poster Abstracts. Type: Oral. Session: 625. Lymphoma: Pre-ClinicalChemotherapy and Biologic Agents: Targeting Apoptosis Pathways in Lymphoma.[Abstract][4] Madduri D, Usmani SZ, Jagannath S, Singh I, Zudaire E, Yeh TM, Allred AJ, Banerjee A, et al. Results from CARTITUDE-1: A Phase 1b/2 Study of JNJ-4528, a CAR-T Cell Therapy Directed Against B-Cell Maturation Antigen (BCMA), in Patients with Relapsed and/or Refractory Multiple Myeloma (R/R MM). 61st annual meeting of the American Society of Hematology. Program: Oral and Poster Abstracts. Type: Oral Session: 653. Myeloma: Therapy, excluding Transplantation: Novelty in CAR T in Relapsed/Refractory Multiple Myeloma. [Abstract][5] Li C, Mei H, Hu Y, Guo T, Liu L, Jiang H, Tang L, Wu Y, et al. A Bispecific CAR-T Cell Therapy Targeting Bcma and CD38 for Relapsed/Refractory Multiple Myeloma: Updated Results from a Phase 1 Dose-Climbing Trial61st annual meeting of the American Society of Hematology. Program: Oral and Poster Abstracts. Type: Oral. Session: 653. Myeloma: Therapy, excluding Transplantation: Novel Therapy for Relapsed Myeloma. Hematology Disease Topics & Pathways: Biological, Diseases, Adult, Therapies, Lymphoma (any), Adverse Events, CAR-Ts, Elderly, Biological Processes, Technology and Procedures, Cell Lineage, Study Population, Clinically relevant, Lymphoid Malignancies.

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ASH 2019: Second-gen CAR T-Cell Therapy Overcome Resistance, Reduce Toxicity and Simplify Treatment - OncoZine

Recommendation and review posted by Bethany Smith

The issuance of this patent highlights Lineages dominant position in the field of cell therapy, stated Brian M. Culley, CEO of Lineage. Our efforts to develop new treatments rely on well-characterized and NIH-approved human cell lines. These lines are not genetically manipulated, which avoids the safety concerns associated with genetic aberrations arising from the creation of iPS cells. We believe the Lineage cell lines provide the safest option for our current clinical-stage programs, particularly in immune-privileged anatomical sites such as the eye (OpRegen for the treatment of dry AMD) and spinal cord (OPC1, for the treatment of spinal cord injury). However, the vast intellectual property estate which underlies our cell therapy platform has never been limited to these particular cell lines. As one example, this newly-issued patent provides us with proprietary methods for producing induced pluripotent stem cells, or, as it was practiced by us prior to Yamanaka, Analytical Reprogramming Technology (ART). In certain settings, an ART/iPS approach might offer important advantages, such as for an autologous treatment or when the selection of preferential attributes from a series of iPS lines is desirable. Questions as to which stem cell technology is preferred ultimately will be answered by clinical safety and efficacy and likely will be indication-specific, so we believe it is in the best interest of our shareholders to generate patented technology which enables us to pursue programs in either or both formats which we believe will ensure the highest probability of success.

This patent broadly describes multiple techniques for reprogramming cells of the body back to the all-powerful stem cell state, said Dr. Michael D. West, CEO of AgeX and first inventor on the patent. Perhaps more significantly, it includes certain factors that address some of the difficulties currently encountered with iPS cells. It also reflects the foundational work our scientists have undertaken to apply reprogramming technology to age-reversal, specifically, induced Tissue Regeneration (iTR) which is currently a focus of AgeX product development.

Induced Pluripotent Stem Cells (iPS) are typically derived from adult skin or blood cells which have been reprogrammed or induced to retrace their developmental age and regain the potential to form all of the young cell and tissue types of the body. In 2010 inventors of the -723 patent issued today demonstrated that this reversal of developmental aging even extended to the telomere clock of cell aging. This reprogramming technology provides an alternate source of starting material for the manufacture of potentially any type of human cell needed for therapeutic purposes. Because iPSCs can be derived directly from adult tissues, they can be used to generate pluripotent cells from patients with known genetic abnormalities for drug discovery or as an alternative source of cell types for regenerative therapies.

U.S. Patent No. 10,501,723, entitled Methods of Reprogramming Animal Somatic Cells was assigned to Advanced Cell Technology of Marlborough, Massachusetts (now Astellas Institute for Regenerative Medicine) and licensed to Lineage and sublicensed to AgeX Therapeutics for defined fields of use. Inventors of the patent include Michael D. West, CEO of AgeX and previous CEO of Advanced Cell Technology, Karen B. Chapman, Ph.D., and Roy Geoffrey Sargent, Ph.D.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its proprietary cell-based therapy platform and associated development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally-differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed either to replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical assets include (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase I/IIa development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase I/IIa development for the treatment of acute spinal cord injuries; and (iii) VAC2, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells currently in Phase I development for the treatment of non-small cell lung cancer. Lineage is also evaluating potential partnership opportunities for Renevia, a facial aesthetics product that was recently granted a Conformit Europenne (CE) Mark. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

About AgeX Therapeutics

AgeX Therapeutics, Inc. (NYSE American: AGE) is focused on developing and commercializing innovative therapeutics for human aging. Its PureStem and UniverCyte manufacturing and immunotolerance technologies are designed to work together to generate highly-defined, universal, allogeneic, off-the-shelf pluripotent stem cell-derived young cells of any type for application in a variety of diseases with a high unmet medical need. AgeX has two preclinical cell therapy programs: AGEX-VASC1 (vascular progenitor cells) for tissue ischemia and AGEX-BAT1 (brown fat cells) for Type II diabetes. AgeXs revolutionary longevity platform induced Tissue Regeneration (iTR) aims to unlock cellular immortality and regenerative capacity to reverse age-related changes within tissues. AGEX-iTR1547 is an iTR-based formulation in preclinical development. HyStem is AgeXs delivery technology to stably engraft PureStem cell therapies in the body. AgeX is developing its core product pipeline for use in the clinic to extend human healthspan and is seeking opportunities to establish licensing and collaboration agreements around its broad IP estate and proprietary technology platforms. For more information, please visit http://www.agexinc.com or connect with the company on Twitter, LinkedIn, Facebook, and YouTube.

Forward-Looking Statements

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as believe, may, will, estimate, continue, anticipate, design, intend, expect, could, plan, potential, predict, seek, should, would, contemplate, project, target, tend to, or the negative version of these words and similar expressions. Such statements include, but are not limited to, Lineages exploration of alternative cell therapy platforms. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineages actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including risks and uncertainties inherent in Lineages business and other risks in Lineages filings with the Securities and Exchange Commission (the SEC). Lineages forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading Risk Factors in Lineages periodic reports with the SEC, including Lineages Annual Report on Form 10-K filed with the SEC on March 14, 2019 and its other reports, which are available from the SECs website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191210005404/en/

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Lineage Cell Therapeutics and AgeX Therapeutics Announce Issuance of US Patent for Method of Generating Induced Pluripotent Stem Cells - BioSpace

Recommendation and review posted by Bethany Smith

AgeX Therapeutics, Inc. (NYSE American: AGE) and Lineage Cell Therapeutics, Inc. (NYSE American and TASE LCTX), announced today that the United States Patent and Trademark Office (USPTO) has issued U.S. Patent No. 10,501,723, entitled "Methods of Reprogramming Animal Somatic Cells" covering what is commonly designated "induced Pluripotent Stem (iPS) cells. The issued claims include methods to manufacture pluripotent cells capable of becoming any cell in the body. The patent has an early priority date, having been filed before the first scientific publication of Shinya Yamanaka, for which he won the Nobel Prize for Physiology or Medicine in 2012.

"This patent broadly describes multiple techniques for reprogramming cells of the body back to the all-powerful stem cell state," said Dr. Michael D. West, CEO of AgeX and first inventor on the patent. "Perhaps more significantly, it includes certain factors that address some of the difficulties currently encountered with iPS cells. It also reflects the foundational work our scientists have undertaken to apply reprogramming technology to age-reversal, specifically, induced Tissue Regeneration (iTR) which is currently a focus of AgeX product development." A video describing the significance of the patent in AgeXs product development is available on the AgeX website.

"The issuance of this patent highlights Lineages dominant position in the field of cell therapy," stated Brian M. Culley, CEO of Lineage. "Our efforts to develop new treatments rely on well-characterized and NIH-approved human cell lines. These lines are not genetically manipulated, which avoids the safety concerns associated with genetic aberrations arising from the creation of iPS cells. We believe the Lineage cell lines provide the safest option for our current clinical-stage programs, particularly in immune-privileged anatomical sites such as the eye (OpRegen for the treatment of dry AMD) and spinal cord (OPC1, for the treatment of spinal cord injury). However, the vast intellectual property estate which underlies our cell therapy platform has never been limited to these particular cell lines. As one example, this newly-issued patent provides us with proprietary methods for producing induced pluripotent stem cells, or, as it was practiced by us prior to Yamanaka, Analytical Reprogramming Technology (ART). In certain settings, an ART/iPS approach might offer important advantages, such as for an autologous treatment or when the selection of preferential attributes from a series of iPS lines is desirable. Questions as to which stem cell technology is preferred ultimately will be answered by clinical safety and efficacy and likely will be indication-specific, so we believe it is in the best interest of our shareholders to generate patented technology which enables us to pursue programs in either or both formats which we believe will ensure the highest probability of success."

Induced Pluripotent Stem Cells (iPS) are typically derived from adult skin or blood cells which have been "reprogrammed" or "induced" to retrace their developmental age and regain the potential to form all of the young cell and tissue types of the body. In 2010 inventors of the -723 patent issued today demonstrated that this reversal of developmental aging even extended to the telomere clock of cell aging. This reprogramming technology provides an alternate source of starting material for the manufacture of potentially any type of human cell needed for therapeutic purposes. Because iPSCs can be derived directly from adult tissues, they can be used to generate pluripotent cells from patients with known genetic abnormalities for drug discovery or as an alternative source of cell types for regenerative therapies.

U.S. Patent No. 10,501,723, entitled "Methods of Reprogramming Animal Somatic Cells" was assigned to Advanced Cell Technology of Marlborough, Massachusetts (now Astellas Institute for Regenerative Medicine) and licensed to Lineage and sublicensed to AgeX Therapeutics for defined fields of use. Inventors of the patent include Michael D. West, CEO of AgeX and previous CEO of Advanced Cell Technology, Karen B. Chapman, Ph.D., and Roy Geoffrey Sargent, Ph.D.

About AgeX Therapeutics

AgeX Therapeutics, Inc. (NYSE American: AGE) is focused on developing and commercializing innovative therapeutics for human aging. Its PureStem and UniverCyte manufacturing and immunotolerance technologies are designed to work together to generate highly-defined, universal, allogeneic, off-the-shelf pluripotent stem cell-derived young cells of any type for application in a variety of diseases with a high unmet medical need. AgeX has two preclinical cell therapy programs: AGEX-VASC1 (vascular progenitor cells) for tissue ischemia and AGEX-BAT1 (brown fat cells) for Type II diabetes. AgeXs revolutionary longevity platform induced Tissue Regeneration (iTR) aims to unlock cellular immortality and regenerative capacity to reverse age-related changes within tissues. AGEX-iTR1547 is an iTR-based formulation in preclinical development. HyStem is AgeXs delivery technology to stably engraft PureStem cell therapies in the body. AgeX is developing its core product pipeline for use in the clinic to extend human healthspan and is seeking opportunities to establish licensing and collaboration agreements around its broad IP estate and proprietary technology platforms.

Story continues

For more information, please visit http://www.agexinc.com or connect with the company on Twitter, LinkedIn, Facebook, and YouTube.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its proprietary cell-based therapy platform and associated development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally-differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed either to replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical assets include (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase I/IIa development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase I/IIa development for the treatment of acute spinal cord injuries; and (iii) VAC2, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells currently in Phase I development for the treatment of non-small cell lung cancer. Lineage is also evaluating potential partnership opportunities for Renevia, a facial aesthetics product that was recently granted a Conformit Europenne (CE) Mark. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

Forward-Looking Statements

Certain statements contained in this release are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not historical fact including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates" should also be considered forward-looking statements. Forward-looking statements involve risks and uncertainties. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the business of AgeX Therapeutics, Inc. and its subsidiaries, particularly those mentioned in the cautionary statements found in more detail in the "Risk Factors" section of AgeXs Annual Report on Form 10-K and Quarterly Reports on Form 10-Q filed with the Securities and Exchange Commissions (copies of which may be obtained at http://www.sec.gov). Subsequent events and developments may cause these forward-looking statements to change. AgeX specifically disclaims any obligation or intention to update or revise these forward-looking statements as a result of changed events or circumstances that occur after the date of this release, except as required by applicable law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191210005435/en/

Contacts

Media Contact for AgeX:Bill Douglass Gotham Communications, LLCbill@gothamcomm.com (646) 504-0890

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AgeX Therapeutics and Lineage Cell Therapeutics Announce Issuance of U.S. Patent for Method of Generating Induced Pluripotent Stem Cells - Yahoo...

Recommendation and review posted by Bethany Smith

Lineage Cell Therapeutics and AgeX Therapeutics have been awarded a United States Patent and Trademark Office patent for Methods Of Reprogramming Animal Somatic Cells.

The issuance of this patent highlights Lineages dominant position in the field of cell therapy, stated Brian M. Culley, CEO of Lineage. Our efforts to develop new treatments rely on well-characterized and NIH-approved human cell lines. These lines are not genetically manipulated, which avoids the safety concerns associated with genetic aberrations arising from the creation of iPS cells. We believe the Lineage cell lines provide the safest option for our current clinical-stage programs, particularly in immune-privileged anatomical sites such as the eye (OpRegen for the treatment of dry AMD) and spinal cord (OPC1, for the treatment of spinal cord injury). However, the vast intellectual property estate which underlies our cell therapy platform has never been limited to these particular cell lines. As one example, this newly-issued patent provides us with proprietary methods for producing induced pluripotent stem cells, or, as it was practiced by us prior to Yamanaka, Analytical Reprogramming Technology (ART). In certain settings, an ART/iPS approach might offer important advantages, such as for an autologous treatment or when the selection of preferential attributes from a series of iPS lines is desirable. Questions as to which stem cell technology is preferred ultimately will be answered by clinical safety and efficacy and likely will be indication-specific, so we believe it is in the best interest of our shareholders to generate patented technology which enables us to pursue programs in either or both formats which we believe will ensure the highest probability of success.

This patent broadly describes multiple techniques for reprogramming cells of the body back to the all-powerful stem cell state, said Dr Michael D West, CEO of AgeX and first inventor on the patent. Perhaps more significantly, it includes certain factors that address some of the difficulties currently encountered with iPS cells. It also reflects the foundational work our scientists have undertaken to apply reprogramming technology to age-reversal, specifically, induced Tissue Regeneration (iTR) which is currently a focus of AgeX product development.

Patent 10,501,723 covers induced pluripotent stem cells which includes methods to manufacture iPSs cells that are capable of becoming any cell within the body. This patent has an early priority date having been filed before the first scientific publication, and was assigned to Advanced Cell Technology of Marlborough, Massachusetts and licenced to Lineage as well as being sublicensed to Age X for defined fields of use.

See more here:
Patent Granted To Lineage & AgeX - Anti Aging News

Recommendation and review posted by Bethany Smith

Men are nearly two times more likely to develop blood clots while undergoing testosterone therapy, according to University of Minnesota research.

The study, which was published last month, found that men on testosterone therapy were at a heightened risk of developing a condition commonly known as VTE. With this condition, blood clots form in the veins and travel to the lungs, potentially blocking the blood supply. Men seek testosterone therapy for a variety of reasons, including low testosterone levels and sexual dysfunction.

These findings were shown in both men with and without hypogonadism, a condition where the body does not produce enough testosterone naturally.

There hasnt really been [this] kind of care given to a hypogonadism diagnosis. A lot of other studies either control for it or restrict it to one specific group of men, said Robert Walker, the study's lead and a research assistant at the Universitys Minnesota Population Center.

In the study, researchers separated men with the condition and men without it. By doing so, they were able to see a near doubling of risk in both groups, he said. They were also able to control for factors such as smoking status, obesity and race.

Overall for Americans, there [are] about 1 million cases of VTE annually, Walker said, which includes a lifetime risk of one in 12 people. Symptoms include leg pain, swelling and rashes.

While men typically receive testosterone therapy for hypogonadism, they may also seek it for unwanted weight gain or emotional regulation.

"These findings are important, particularly for the men with no hypogonadism, as they suggest that off-label testosterone use for symptoms of 'male menopause' may be causing harm, said Pamela Lutsey, a co-author of the study and University associate professor in the Division of Epidemiology and Community Health, in an email to the Minnesota Daily.

When men seek testosterone therapy for reasons other than hypogonadism, there is not necessarily an official diagnosis. However, treatment could fix those symptoms, Walker said.

Testosterone therapy is more than just a kind of stroke and heart failure risk factor, we have to take into account other cardiovascular symptoms, he said.

In the future, the researchers can use the same study design to test the effects of testosterone therapy on women, Walker said.

Women who need to take testosterone for one reason or another may also be put at a risk, he said.

Women are often at risk of developing VTE while pregnant because of the pressure on their organs or afterward in the postpartum period, Walker said.

An important next step is for other researchers to reproduce these results, said Richard MacLehose, another co-author of the study and University associate professor in the Division of Epidemiology and Community Health.

Walker said he began this study because of the lack of research papers on the effects of testosterone therapy.

The story of the paper is really just to think before you prescribe, he said.

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Men who receive testosterone therapy nearly twice as likely to develop blood clots, researchers find - Minnesota Daily

Recommendation and review posted by Bethany Smith

NORTHBROOK, Ill., Dec. 11, 2019 (GLOBE NEWSWIRE) -- Clarus Therapeutics, Inc. today announced the appointment of five senior leaders, each of whom bring valuable industry and category experience to the team. Together they strengthen the companys commercialization capability for JATENZO (testosterone undecanoate) capsules, the first-in-class oral testosterone replacement therapy approved by the U.S. Food and Drug Administration (FDA) for the treatment of hypogonadism. See indication and important risk information, including boxed warning below.

Dr. Newmark, Mr. Jaeger, Mr. Rodriguez, Ms. Wright and Mr. Holloway join a management team led by Robert Dudley, Ph.D., Clarus Therapeutics Chief Executive Officer and co-inventor of JATENZO. Dr. Dudleys legacy in mens health includes CEO leadership of the company that developed and launched AndroGel, a product he co-invented and the last major advancement in testosterone replacement therapy delivery.

We are proud to bring Jay, Frank, Jose, LaTonya, and James into an organization that shares their focus on innovation and excellence. They have each demonstrated an ability to connect with medical professionals who are invested in mens health, said Dudley. Their expertise will help us make the long sought-after oral testosterone option widely available to appropriate hypogonadal men.

INDICATION

JATENZO (testosterone undecanoate) capsules, CIII, is an androgen indicated for testosterone replacement therapy in adult males for conditions associated with a deficiency or absence of endogenous testosterone:

Limitation of use

Safety and efficacy of JATENZO in males less than 18 years old have not been established.

IMPORTANT SAFETY INFORMATION

WARNING: INCREASES IN BLOOD PRESSURE

CONTRAINDICATIONS

JATENZO is contraindicated in men with carcinoma of the breast or known or suspected carcinoma of the prostate, in women who are pregnant, in men with a known hypersensitivity to JATENZO or its ingredients, or in men with hypogonadal conditions that are not associated with structural or genetic etiologies as JATENZO has not been established for these conditions and there is a risk of increased blood pressure with JATENZO that can increase the risk of MACE.

WARNINGS AND PRECAUTIONS

ADVERSE EVENTS

The most common adverse events of JATENZO (incidence 2%) are headache (5%), increased hematocrit (5%), hypertension (4%), decreased HDL (3%), and nausea (2%).

DRUG INTERACTIONS

USE IN SPECIFIC POPULATIONS

The safety and efficacy of JATENZO in pediatric patients less than 18 years old have not been established. Improper use may result in acceleration of bone age and premature closure of epiphyses.

There have not been sufficient numbers of geriatric patients involved in controlled clinical studies utilizing JATENZO to determine whether efficacy or safety in those over 65 years of age differs from younger subjects. There is insufficient long-term safety data in geriatric patients utilizing JATENZO to assess the potentially increased risk of cardiovascular disease and prostate cancer.

Please see accompanying full Prescribing Information, including BOXED WARNING on increases in blood pressure.About JATENZO JATENZO is a first-in-class proprietary oral soft gel formulation of testosterone undecanoate for testosterone replacement therapy in adult males for conditions associated with a deficiency or absence of endogenous testosterone.

JATENZOs proprietary formulation is built around testosterone undecanoate a testosterone prodrug that the body converts to testosterone. In the JATENZO pivotal inTUne (investigation of its oralTestosteroneUndecanoate) clinical trial, 87% (n=222) of men treated with JATENZO achieved average circulating levels of testosterone in the normal range for men.

About Clarus Therapeutics, Inc.Clarus is a men's specialty pharmaceutical company developing and preparing for the commercial launch of JATENZO, a product protected by patents issued in the United States and in other major pharmaceutical markets around the world. Clarus owns the worldwide, royalty-free commercialization rights for JATENZO.For more information, please visit:www.clarustherapeutics.com.

Media ContactAmir KhanPhone: (212) 462-8767Email: Amir.Khan@Syneoshealth.com

2019 Clarus Therapeutics, Inc. All rights reserved.

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Clarus Therapeutics Expands Executive Team in Preparation for Landmark Launch of JATENZO (testosterone undecanoate) Capsules CIII - GlobeNewswire

Recommendation and review posted by Bethany Smith

She only appeared in the pilot!

Who is Hosanna Plath? That's been the question on everybody's mind since Welcome to Plathvilledebuted on TLC, the network that once focused on things like childbirth and dinosaur bones, but now focuses on messy relationships, hypogonadism, multiple birth families, and metabolic issues with their accompanying bariatric procedures. So what makes Hosanna Plath stand out from the rest of the Plath clan?

RELATED:Meet The 9-Kid, No-Technology Plath Family Featured In New TLC Show 'Welcome To Plathville'

Welcome to Plathvilleis the latest TLC reality show that focuses on a family that most would consider "strange and unusual." The Plath family led by matriarch Kim and patriarch Barry say on the show they are "devout Christians," and though they don't get into detail about which denomination of Christianity they follow, there are some photos of the family posing in front of a Baptist church. What makes them remarkable, however, is not their adherence to Christianity, but their practice of isolating their children from pop culture. In many of the previews for the show, for example, viewers are left to marvel as the Plath children Hosanna included are left to wonder who people like Justin Bieber and Tom Brady really are.

That said, though the family has no exposure to pop culture, they do have a YouTube channel that promotes their gospel music. Each family member plays an instrument, so let's look at the violinist, Hosanna Plath.

Though Hosanna Plath has an older brother, Ethan who is the officialoldest of the Plath children she's the oldest Plath daughter. She is one of 9 children and would have been one of 10 had her younger brother, Joshua, not been killed in a tragic accident.

Hosanna Plath is known, in her family band, for playing the violin. She's an accomplished violinist and actually turned down going to college to continue to play the violin. (Since she married her husband, Timothy Noble, she's also played as a duet with him.)

In theWelcome to Plathvillepilot, it was revealed that Hosanna Plath had a musical scholarship to college, but turned it down to continue to play with the family band. This decision drew some fire from the fandom, especially since it was later revealed that Kim Plath the matriarch of the family graduated from FSU.

RELATED:Meet Nail Artist Lexi Martone Star Of The New TLC Reality Series 'Unpolished'

In 2016, Hosanna Plath went to the National Quartet Convention. And it was there that she met Timothy Noble, the man who would become her husband. Noble plays piano, and he often plays together with his wife as a duet. They dated for two years before they ultimately got married in June 2019.

According to reports, there'stension within the family because Hosanna Plath was considered the "musical prodigy" of the family. Because she's so talented and accomplished, Hosanna Plath reportedly taught her siblings how to play their respective instruments. So when Hosanna married Timothy and the duo moved to Ohio thus, away from the family the Plath family band seemed to languish. As such, rumors began circulating that there was a fracture in the Plath family.

Sometimes, it really is the devil you know. While therecertainl, is no shortage of people who criticize the Plath family for raising their children including Hosanna Plath so strictly, there are some good reasons for their method. As revealed on the show, Kim Plath drew a hard line against having alcohol served at her son, Ethan's, wedding, because she admitted she was a recovering alcoholic. Her biggest fear, she said, was that her children would walk down the same path that she walked down.

While, no doubt, that is a bit extreme, we can certainly understand the desire to protect your children. We look forward to hearing more from Hosanna Plath in the future.

RELATED:Meet Kim Plath Controversial Matriarch Of Family Raising 9 Kids Technology-Free On TLC's 'Welcome To Plathville'

Bernadette Giacomazzo is an editor, writer, and photographer whose work has appeared in Teen Vogue, People, Us Weekly, The Source, XXL, HipHopDX, The Los Angeles Times, The New York Post, BET.com, and more. She is also the author of The Uprising series and is the CEO of the acclaimed G-Force Marketing & Publicity firm, which has been featured in The Hollywood Reporter and has scored film, television, radio, and print placements for celebrity clientele worldwide.

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Get To Know Hosanna Plath, The 'Welcome To Plathville' Star And Accomplished Violinist - YourTango

Recommendation and review posted by Bethany Smith

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Press Release updated: Dec 11, 2019 15:00 EST

LOS ANGELES, December 11, 2019 (Newswire.com) QY Research has lately come up with a new report titled, Global Hormone Replacement Therapy Market Report, History and Forecast 2014-2025, for the projected period of six years, i.e. between 2019 and 2025. The reports states that the global Hormone Replacement Therapy market was valued at US $15.7 billion in 2018 and is expected to reach US $17.4 billion by the end of 2025. The global market is anticipated to register a sluggish CAGR of 1.5% from 2019 to 2025.

Request a Sample Copy of this Report athttps://www.qyresearch.com/sample-form/form/1123650/global-hormone-replacement-therapy-market

Factors Hampering the Growth of the Global Hormone Replacement Therapy Market

Medical Conditions and Healthcare:Lack of awareness amongst patients regarding hormone deficiencies and imbalances, resulting in serious conditions such as menopause and hypothyroidism, is expected to hamper the growth of the market.

Drugs and Pharmaceuticals:Lack of drug delivery systems, particularly in developing economies and the high cost of therapies and potential side effects are anticipated to restrain the growth of the market.

Risk of Cancer and Heart Disease:Uncertainty among doctors for prescribing Hormone Replacement Therapy due to the risk of heart disease and breast cancers is likely to obstruct the growth of the market.

Availability of Alternate Options A Significant Factor Obstructing Growth

The type segment is further sub-segmented into growth hormone, Estrogen Hormone, Testosterone Hormone, and thyroid hormone. Based on application, the segment is bifurcated into

Due to premature birth resulting in underdevelopment in infants, treatments for growth hormone deficiency has witnessed considerable demand over the years. Increasing awareness among women regarding menopause is likely to boost the growth of the application segment. However, the availability of alternative options in the market, along with the presence of various brands and generic drugs, are likely to impede the growth of the market.

Prevalence of Hormone Related Diseases Likely to foster Regional Market

The global market is bifurcated into Asia-Pacific, Europe, North America, Central America, Middle-east, and Africa. Asia-Pacific is likely to hold a considerable share of the market due to the presence of a huge population and an increase in the prevalence of conditions such as hypothyroidism, menopause, hypogonadism, and hormone deficiency.

Innovation and Product Development A Major Emphasis of Key Players

Some of the top players operating in the market are Pfizer, Eli Lilly, Novo Nordisk, AbbVie, Merck, Bayer, KGaA, Mylan, Novartis, Teva, Abbott, Roche, Ipsen, Endo International, TherapeuticsMD, and ANI Pharmaceuticals. Top players of the market are inclined to incorporate advanced technology and to collaborate for offering innovative products.

Get a Complete Report in your Inbox within 24 hours (USD 2,900): https://www.qyresearch.com/settlement/pre/a2424e7155d3f73613318581f3c173c7,0,1,Global-Hormone-Replacement-Therapy-Market-Report-History-and-Forecast-Breakdown-Data-by-Companies-Key-Regions-Types-and-Applicatio

Source: QY Research, Inc.

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Global Hormone Replacement Therapy Market is Anticipated to Reach US $17.4 Billion by the End of 2025 - The Chestnut Post

Recommendation and review posted by Bethany Smith