Experimental stem cell gene therapy may give a new lease of life for patient with sickle cell disease – News-Medical.Net

For Evie Junior, living with sickle cell disease has been like running a marathon.

"But it's a marathon whereas you keep going, the trail gets rockier and then you lose your shoes," the 27-year-old said. "It gets harder as you get older. Things start to fail and all you can think about is how much worse it's going to get down the road."

In sickle cell disease, a genetic mutation causes the blood-forming stem cells -- which give rise to all blood and immune cells -- to produce hard, sickle-shaped red blood cells. These misshapen cells die early, leaving an insufficient number of red blood cells to carry oxygen throughout the body.

Because of their sickle shape, these cells also get stuck in blood vessels, blocking blood flow and resulting in excruciating bouts of pain that come on with no warning and can leave patients hospitalized for days.

The disease affects 100,000 people in the United States and millions around the world, the majority of whom are of African or Hispanic descent. It can ultimately lead to strokes, organ damage, and early death.

As a child growing up in the Bronx, New York, Junior had to have his gall bladder and spleen removed due to complications from the disease, but he refused to let his condition limit him. He played football, basketball, and baseball during the day, even though on some nights he experienced pain crises so severe he couldn't walk.

"It was just really routine if I had a sickle cell crisis," he said. "Going to the emergency room, staying in the hospital, coming out in a few days, and then getting back to normal life."

When he was 24 and living in Portland, Oregon, Junior began working as an emergency medical technician. He adopted the same mentality -- trying to treat his pain episodes the best he could, and hoping they would resolve overnight so he could get back to work.

Around that time, though, the crises became harder to manage. He developed pericarditis, an inflammation in the layers of tissue around his heart, and needed six weeks to recover.

"The big worry with sickle cell disease is that you're going to die young from some type of complications or damage to your organs," he said. "In the last couple of years, I've been seeing that slowly happen to me and I can only suspect that it's going to keep getting worse. I want to create a better future for myself."

In July 2019, in pursuit of that future, Junior enrolled in a clinical trial for an experimental stem cell gene therapy for sickle cell disease. The study is led by UCLA Broad Stem Cell Research Center physician-scientists Dr. Donald Kohn and Dr. Gary Schiller and funded by the California Institute for Regenerative Medicine.

The therapy, developed by Kohn over the past 10 years, is intended to correct the mutation in patients' blood-forming stem cells to allow them to produce healthy red blood cells. Kohn has already applied the same concept to successfully treat several immune system deficiencies, including a cure for a form of severe combined immune deficiency, also known as bubble baby disease.

But sickle cell disease has proven more difficult to treat with gene therapy than those other conditions. Junior volunteered for the trial knowing there was a chance the therapy wouldn't cure him.

"Even if it doesn't work for me, I'm hoping that it can be a cure later down the road for millions of people," he said.

In July 2020, Junior received an infusion of his own blood-forming stem cells that had been genetically modified to overcome the mutation that causes his disease.

"The goal of this treatment is to give him a future, let him plan for college, family or whatever he wants without worrying about getting hospitalized because of another pain crisis," said Kohn, a distinguished professor of microbiology, immunology and molecular genetics, pediatrics, and molecular and medical pharmacology at the David Geffen School of Medicine at UCLA.

Three months after his treatment, blood tests indicated that 70% of Junior's blood stem cells had the new corrected gene. Kohn and Schiller estimate that even a 20% correction would be enough to prevent future sickle cell complications.

Junior said he hasn't had a pain crisis since undergoing the treatment and he has more energy and feels breathless often.

"I noticed a big difference in my cardiovascular endurance in general -- even going for a light jog with my dogs, I could feel it," he said.

Junior and his doctors are cautiously optimistic about the results.

It's too early to declare victory, but it's looking quite promising at this point. Once we're at six months to a year, if it looks like it does now, I'll feel very comfortable that he's likely to have a permanent benefit."

Dr. Donald Kohn, Physician-Scientist, University of California - Los Angeles Health Sciences

After a lifetime of dealing with the unwelcome surprises of the disease, Junior is even more cautious than his doctors. But as the weeks pass, he's slowly allowing a glimmer of hope that he could soon be someone who used to have sickle cell disease. For him, that hope feels like "a burst of happiness" that's followed by thoughts of all the things he could do with a healthy future: pursue his dream of becoming a firefighter, get married and start a family.

"I want to be present in my kids' lives, so I've always said I'm not going to have kids unless I can get this cured," he said. "But if this works, it means I could start a family one day."

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Hematologist Discusses the Impact a Myeloma CAR T-Cell Approval Would Have on the Treatment Landscape – DocWire News

Ankit Kansagra, MD, an assistant professor in theDepartment of Internal Medicineat UT Southwestern Medical Center and assistant director of theOutpatient Stem Cell Transplant Program, discusses chimeric antigen receptor T-cell agents in the pipeline for multiple myeloma (MM) and how these therapies may impact the treatment landscape pending future approvals.

In part two of this interview with Dr. Kansagra, available December X, he discusses potential new combination therapy options for MM.

DocWire News: Dr. Kansagra, can you discuss some of the CAR T-cell therapies in development for multiple myeloma, including their targets, clinical trial data that weve seen, and your expectations for any future FDA approvals?

Dr. Kansagra: In multiple myeloma, a few of the CAR T-cell therapy targets, which in the most developments, have been the BCMA-targeted CAR T-cell therapies. Those have been most exciting because they have made it to the phase I to phase II trials, especially the registrational studies from Celgene or Bluebird, BMS, the bb2121 compound or the Janssen compound 4538, being farthest out in the clinical development for CAR T-cell therapy. There are certainly a few other CAR T-cell therapies for multiple myeloma, which have grown, and theyre probably in the earlier development of therapy. An example being the CD38 CAR T-cell therapy, the SLAMF CAR T-cell therapy, and GPR5CD CAR T-cell therapy. Those are the three different targets which are being evaluated as T-cell targets.

DocWire News: How do you see the approval of these CAR T-cell therapy impacting the treatment landscape for multiple myeloma?

Dr. Kansagra: I think its going to be a huge improvement in our momentum of our treatment options. We have already seen cell therapy in myeloma have impressive results in terms of the response rates. I think the first important step is you have these patients who have got six or seven different lines of treatment, and now they are getting a novel product or a novel mechanism of action and also novel target and seeing an impressive response rate. That was amazing. Thats step number one.

Step number two is, as we have got further into the clinical development of CAR T-cell therapy, we have seen the safety of these products because that is extremely important that our products are safer.

Then the third thing which we have seen is that long-term follow-ups are not there, but what we have started seeing is that our responses, which could last up to a year or a year and a half for the population, where we would have usually seen maybe barely a response in a matter of months.

I think those are exciting times for our patients with multiple myeloma, where they have failed a lot of therapies. I think the more exciting times are going to come when we will start seeing these CAR T-cell therapies, potentially even in earlier lines of treatment options, where they could use maybe as a second-line treatment or as a first-line treatment after stem cell transplant or in lieu of stem cell transplant, maybe we can have deeper and longer remission rates.

DocWire News: With some of these agents potentially coming to market, do you foresee any challenges, either associated with adverse events or the ability to make these treatments widely available to patients?

Dr. Kansagra: Access to care is certainly near and dear to me, and thinking about those challenges is extremely, extremely important. I think were going to probably face challenges in a lot of different ways.

The first thing is, obviously, how can we get our patients to the centers who are giving CAR T-cell therapy? How are we going to bring them? We know from our autologous stem cell transplant over the last three to four decades, that still not every eligible transplant patient is referred to a transplant center, for whatever reasons. There are multiple reasons; there are socioeconomic reasons; there are distance reasons. But a lot of them are fixable reasons. There are some which are unfixable, but there are some fixable. I think the first and the foremost important thing is going to be to get our patients to a place who is delivering CAR T-cell therapy. Thats the challenge number one.

Challenge number two is, once they are in there, making sure that they are able to get that thing. So it means theyre not coming too late in their game, so trying to make sure theyre referred in earlier points, so that processes in place, that insurance approval has got started, if we need to work on the sociodemographic issues, how are they going to stay in a particular area? What is the social help, what is the family help theyre going to need? If they had referred earlier on, thats another, I call it, bottleneck that we need to think of that. Thats where we need to act on it.

The hard thing is obviously the cost. We dont know what is going to be the cost of the myeloma CAR T-cell therapy, or what is the price of those things. We can certainly estimate that its not going to be as cheap given the three CAR-Ts, which are not FDA-approved. I think its going to be expensive. You will have to think of the cost of care model of how we are going to work with this.

Last but not least of the challenges are the CAR-T itself. These are in the logistical challenge bucket. Then there are the challenges in the CAR-T landscape or the product itself. We still know that these are second-generation CAR T-cell therapies. They dont work for everybody. They have a high response rates, but they dont last that long. We hope to see longer remissions. An example I give, in comparison to large-cell lymphoma, we had 50% of the people who plateaued out, now coming up to about three years. In myeloma, we havent obviously made it to three years since the CAR T-cell therapy have started, but we do worry that there is a tail end of the curve that people are already relapsing to it. Obviously, that goes to the product itself or the construct itself, which needs to be developed in multiple different ways. I think of them as two major challenges ahead of us.

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Coronavirus Updates: The Latest Treatments and Vaccines – GovTech

(TNS) - Scientists at Bay Area universities, laboratories, biotechnology companies and drug manufacturers are fashioning drug concoctions out of blood plasma, chimpanzee viruses and cells taken from bone marrow in the race to rid the world of COVID-19.

The microbial treasure hunt is not just to find a cure which may not be possible but to control the debilitating health problems caused by the coronavirus.

Major progress has been made this year. The antiviral drug remdesivir, produced in Foster City, has improved recovery times, and the steroid dexamethasone has cut the number of deaths in severely ill patients.

What follows is a list of some of the most promising medications and vaccines with ties to the Bay Area:


and Immunity

Mesenchymal stem cells / UCSF and UC Davis Medical Center:

UCSF Dr. Michael Matthay is leading a study of whether a kind of stem cell found in bone marrow can help critically ill patients with severe respiratory failure, known as ARDS. Matthay hopes the stem cells can help reduce the inflammation associated with some of ARDS' most dire respiratory symptoms, and help patients' lungs recover.

In all, 120 patients are being enrolled at UCSF Medical Center, Zuckerberg San Francisco General Hospital, the UC Davis Medical Center in Sacramento and hospitals in Oregon and Texas. He said the trial, which includes a small number of ARDS patients who don't have COVID-19, should have results by summer or fall 2021. So far, 28 patients are enrolled in San Francisco.

Lambda-interferon / Stanford University:

Lambda-interferon is a manufactured version of a naturally occurring protein that had been used to treat hepatitis, and researchers hoped it would help patients in the early stages of COVID-19.

Stanford researchers completed their trial of lambda-interferon and found that it did not boost the immune system response to coronavirus infections.

"That trial did not find any difference in outcomes between the treatment and placebo," said Yvonne Maldonado, chief of pediatric infectious diseases at Lucile Packard Children's Hospital at Stanford, where 120 patients were enrolled in the trial. "It didn't work."

Antiviral drugs

Remdesivir / Gilead Sciences ( Foster City):

Remdesivir, once conceived as a potential treatment for Ebola, was approved by the Food and Drug Administration in October for use on hospitalized COVID-19 patients.

Trademarked under the name Veklury, the drug interferes with the process through which the virus replicates itself. It was one of the drugs given to President Trump and has been used regularly in hospitals under what is known as an emergency use authorization.

It was approved after three clinical trials showed hospitalized coronavirus patients who received remdesivir recovered five days faster on average than those who received a placebo. Patients who required oxygen recovered seven days faster, according to the studies.

Gilead now plans to conduct clinical trials to see how remdesivir works on pediatric patients, from newborns to teenagers, with moderate to severe COVID-19 symptoms. Remdesivir is also being studied with steroids and other drugs to see if it works better as part of a medicinal cocktail. An inhalable form of the drug is also being developed.

Favipiravir / Fujifilm Toyama Chemical ( Stanford University):

This antiviral drug, developed in 2014 by a subsidiary of the Japanese film company to treat influenza, is undergoing numerous clinical studies worldwide, including a trial involving 180 patients at Stanford University.

Stanford epidemiologists are testing favipiravir to see if it prevents the coronavirus from replicating in human cells, halts the shedding of the virus and reduces the severity of infection. Unlike remdesivir, it can be administered orally, so it can be used to treat patients early in the disease, before hospitalization is necessary.

The Stanford study has so far enrolled about 90 patients, who are given the drug within 72 hours of when they were first diagnosed with COVID-19. Half of them get a placebo. People can enroll by emailing treatcovid@stanford.edu.

Monoclonal antibodies

REGN-COV2 / Regeneron Pharmaceuticals / Stanford School of Medicine:

The REGN-COV2 cocktail is the same one Trump received, and Stanford is one of dozens of locations nationwide where clinical trials are being held. Two separate trials are under way at Stanford one for hospitalized patients, the other for outpatients. A third trial is about to begin for people who aren't sick but are in contact with carriers of the virus.

Regeneron halted testing on severely ill patients requiring high-flow oxygen or mechanical ventilation after the independent Data and Safety Monitoring Board determined that the drug was unlikely to help them.

The drug is a combination of two monoclonal antibodies lab-made clones of the antibodies produced naturally in people who have recovered from COVID-19. The antibodies bind to the virus' spike protein and block the virus' ability to enter cells.

Dr. Aruna Subramanian, professor of infectious diseases at Stanford and lead investigator for the inpatient trial, said the 21 hospitalized patients in the study receive a high dose like Trump, a lower dose or a placebo. Subramanian plans to expand the inpatient trial to 45 patients. The outpatient study has enrolled a little more than 40 of the 60 patients researchers intend to sign up.

"There's enough promising evidence that it helps people early in the infection," Subramanian said. "What we don't know is whether it helps people who are pretty sick but not critically ill."

Bamlanivimab / Eli Lilly / Stanford and UCSF:

Stanford and UCSF are testing the Eli Lilly monoclonal antibodies on outpatients after the pharmaceutical company halted trials on hospitalized COVID-19 patients because of adverse results.

Dr. Andra Blomkalns, chair of emergency medicine at Stanford and the lead in the Eli Lilly outpatient trial, said she is now enrolling older people with comorbidities like heart disease, chronic lung disease, a history of strokes and severe obesity shortly after they test positive.

The hypothesis is that the bamlanivimab monotherapy, which is very similar to the Regeneron monoclonals, might work best early in the infection. Although about 400 patients have been enrolled in the Lilly phase 3 trials nationwide, to date fewer than 10 have been enrolled at Stanford and UCSF.

Matthay, who headed up the Lilly monoclonal study with LY-CoV555 at UCSF, said the cancellation of this inpatient trial was disappointing, but "just because this one did not work, doesn't mean another one won't work for hospitalized patients."

Blomkalns said the testing criteria has been changing. She expects the outpatient trial to open soon to adolescents ages 12 and up to determine whether the drug can be used as a preventive.

Designer monoclonal antibodies / Vir Biotechnology, San Francisco:

Scientists at Vir are studying several types of monoclonal antibodies, including a type engineered to activate T cells, which can search out and destroy cells infected with the coronavirus. A study published in the journal Nature in October found that monoclonals, modified to bind with certain receptors, stimulated T cells and improved the human immune response.

"By observing and learning from our body's powerful natural defenses, we have discovered how to maximize the capacity of antibodies through the amplification of key characteristics that may enable more effective treatments for viral diseases," said Herbert Virgin, the chief scientific officer at Vir and co-author of the study.

A similarly modified monoclonal antibody, leronlimab, is being studied in coronavirus clinical trials by its Washington state drugmaker, CytoDyn, which has developed drugs to treat HIV. The company's chief medical officer is in San Francisco, and the company that does laboratory tests of leronlimab is in San Carlos.

Anti-inflammatory drugs

Colchicine / UCSF ( San Francisco and New York):

The anti-inflammatory drug commonly used to treat gout flare-ups is being studied by scientists at UCSF and New York University. The drug short-circuits inflammation by decreasing the body's production of certain proteins, and researchers hope that it will reduce lung complications and prevent deaths from COVID-19.

Preliminary results from a clinical trial found that "Colchicine can be effective in reducing systemic symptoms of COVID-19 by inhibiting inflammatory biomarkers."

Selinexor / Kaiser Permanente:

Kaiser hospitals in San Francisco, Oakland and Sacramento are studying selinexor, an anticancer drug that blocks a key protein in the cellular machinery for DNA processing. Preliminary findings during the trials indicated that low doses of selinexor helped hospitalized patients with severe COVID-19. The drug has both antiviral and anti-inflammatory properties, and it's administered orally, according to Kaiser's Dr. Jacek Skarbinski.


VXA-COV2-1 / Vaxart, South San Francisco:

The biotechnology company Vaxart is testing VXA-COV2-1, the only potential vaccine in pill form. It uses the genetic code of the coronavirus to trigger a defensive response in mucous membranes. The hope is that the newly fortified membranes will prevent the virus from entering the body.

"It's the only vaccine (candidate) that activates the first line of defense, which is the mucosa," said Andrei Floroiu, Vaxart's chief executive. He said intravenous vaccines kill the virus after it is inside the body, but this one stops it beforehand.

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Coronavirus Updates: The Latest Treatments and Vaccines - GovTech

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ONLINE: The UW Now – Isthmus

press release: Stream at the WAA YouTube channel.

Dec. 1: As multiple pharmaceutical companies announce initial positive results from vaccine clinical trials, people all over the world are beginning to ask what comes next. Have the vaccines been tested enough? With limited quantities available, who gets first priority? What factors may complicate distribution? Once we have been vaccinated, how long will we be protected?

Join fellow UW-Madison alumni and friends online for a livestream and Q & A with three UW-Madison experts about the development and distribution of COVID-19 vaccines. The talks will be moderated by Mike Knetter, president and CEO of the Wisconsin Foundation and Alumni Association.

R. Alta Charo is the Warren P. Knowles Professor of Law and Bioethics at the University of WisconsinMadison. She is an elected fellow of the National Academy of Medicine, where she serves on its board on health sciences policy and its executive council. Charo served as a member of the Obama-Biden Transition Project, where she was a member of the health and human services review team. She has served as a senior policy adviser in the Office of the Commissioner at the U.S. Food and Drug Administration as well as on several expert advisory boards of organizations with an interest in stem cell research. She is also the cochair of the National Academy of Medicine Forum on Regenerative Medicine and the Committee on Human Gene Editing. She has been a key figure in drafting regulations and guidelines concerning adult, embryonic, and induced pluripotent stem cell research.

James Conway is a pediatric infectious disease specialist and professor of pediatrics. He is the associate director of the UWs Global Health Institute, the medical director of the UW Health Immunization Program, and director of the Office of Global Health at the School of Medicine and Public Health. He is a fellow of the American Academy of Pediatrics (AAP), serving in the sections on infectious diseases and international child health, and received an AAP Special Achievement Award in 2009 for his immunization projects. He is also a member of the board of directors of the Wisconsin AAP chapter (WIAPP), where he serves as chair of the Committee on Immunizations and Infectious Diseases and represents WIAAP on the Wisconsin Council on Immunization Practice.

Jonathan Temte MD87, PhD93 is the associate dean for public health and community egagement at the UW School of Medicine and Public Health. As a family medicine physician and a professor in the Department of Family Medicine and Community Health, Temte has served as a clinician, teacher, and researcher for 25 years. His research includes investigation of the relationships between communities, primary care, and respiratory viruses. An expert in vaccines and immunization policy, Temte has served on the U.S. Advisory Committee on Immunization Practices, also acting as chair of its Evidence-Based Recommendation Work Group. Temte is chair of the Wisconsin Council on Immunization Practices and serves as medical director for Public Health Madison & Dane County. On the national level, Temte is serving an appointment to the Centers for Disease Control and Prevention Board of Scientific Counselors.

More info: https://www.allwaysforward.org/uwnow/. A recording of this livestream will be available on uwalumni.com after the event.

WFAA plans to host The UW Now Livestream weekly, featuring UWMadison faculty and staff with unique expertise.

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Celularity Announces Positive DMC Safety Review and Continuation of its Phase I/II CYNK-001-COVID-19 (CYNKCOVID) Study – PRNewswire

FLORHAM PARK, N.J., Dec. 1, 2020 /PRNewswire/ -- Celularity announced today that the independent Data Monitoring Committee (DMC) completed the first assessment of the ongoing Phase I/II CYNK-001-COVID-19(CYNKCOVID) study (https://clinicaltrials.gov/ct2/show/NCT04365101) with CYNK-001 off-the-shelf, allogeneic, natural killer (NK) cell therapy in adults with COVID-19. The DMC confirmed the absence of dose-limiting toxicities and recommended to move forward with the trial. Additionally, there was no evidence of worsening of inflammatory biomarkers observed. The observed clinical findings justify the continuation of the trial. Enrollment is ongoing in this multi-center clinical study with active sites in Arizona, Arkansas, California, New Jersey, and Washington.

"We are encouraged that an esteemed group of independent experts in COVID-19 and cellular therapy determined that CYNK-001 was safe in the first participants receiving the treatment on the multi-site national study. Our goal now is to rapidly complete enrollment of the study so we can determine the efficacy of this promising treatment for COVID-19 with the epidemic resurging in the United States and few good treatment options for many patients,"said the national PI for the CYNKCOVID clinical trial, Corey Casper, M.D., M.P.H.

"The administration of NK cells may have the potential to both control viral infection while also coordinating a more effective immune response that could lead to strong and lasting protection against viruses. With the increasing incidence of COVID-19 nationwide, Celularity reaffirms our commitment to the development of CYNK-001 as a potential therapeutic treatment for patients with limited treatment options. Through our collaboration with investigators, we anticipate rapid enrollment culminating in the next DMC review of safety and efficacy data," said Robert J. Hariri, M.D., Ph.D., Celularity's Founder, Chairman and Chief Executive Officer.

The Phase I/II CYNK-001-COVID-19 (CYNKCOVID) clinical trial investigating CYNK-001 is continuing to enroll to the next evaluation milestone where the external, independent DMC will review the phase I data for both safety and efficacy. Celularity continues to accumulate safety data on CYNK-001 across a broad platform of programs including COVID-19, as well as hematologic and solid tumor malignancies.

About NK CellsNK cells are innate immune cells with an important role in early host response against various pathogens. Multiple NK cell receptors are involved in the recognition of infected cells. Studies in humans and mice have established that there is robust activation of NK cells during viral infection, regardless of the virus class, and that the depletion of NK cells aggravates viral pathogenesis.

About CYNK-001CYNK-001 is an investigational cryopreserved allogeneic, off-the-shelf NK cell therapy developed from placental hematopoietic stem cells. CYNK-001 is being investigated as a potential treatment option in adults with COVID-19, as well as for various hematologic cancers and solid tumors. NK cells are a unique class of immune cells, innately capable of targeting cancer cells and interacting with adaptive immunity. CYNK-001 cells derived from the placenta are currently being investigated as a treatment for acute myeloid leukemia (AML), multiple myeloma (MM), and glioblastoma multiforme (GBM).

About Celularity Celularity, headquartered in Florham Park, N.J., is a next-generation Biotechnology company leading the next evolution in cellular medicine by developing off-the-shelf allogeneic cellular therapies. Celularity's innovative approach to cell therapy harnesses the unique therapeutic potential locked within the cells of the post-partum placenta. Through nature's immunotherapy engine the placenta Celularity is leading the next evolution of cellular medicine with placental-derived T cells, NK cells, and pluripotent stem cells to target unmet and underserved clinical needs in cancer, infectious and degenerative diseases. To learn more visit celularity.com

Media ContactFactory PR Email: [emailprotected]

SOURCE Celularity, Inc.

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Celularity Announces Positive DMC Safety Review and Continuation of its Phase I/II CYNK-001-COVID-19 (CYNKCOVID) Study - PRNewswire

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Mustang Bio to Host Key Opinion Leader Call on MB-106 for the Treatment of Relapsed or Refractory B-Cell Non-Hodgkin Lymphoma – GlobeNewswire

WORCESTER, Mass., Dec. 01, 2020 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that it will host a key opinion leader (KOL) call on MB-106 for the treatment of relapsed or refractory B-cell non-Hodgkin lymphoma on Wednesday, December 9, 2020, at 1:00 p.m. EST.

The call will feature presentations by KOLs Mazyar Shadman, M.D., M.P.H., Fred Hutchinson Cancer Research Center (Fred Hutch), and Brian Till, M.D., Fred Hutch, who will discuss the interim Phase 1/2 data on MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell non-Hodgkin lymphoma that the company is developing in collaboration with Fred Hutch. Data from this study have been selected for a poster presentation at the 62nd American Society of Hematology Annual Meeting.

During the call, Drs. Shadman and Till will also discuss the modified cell manufacturing process that was co-developed by Fred Hutch and Mustang Bio, as well as the correlative science observed in the study to date. The Mustang team will then give a corporate update on its pipeline and future plans. Following the formal presentations, the Mustang team, along with Drs. Till and Shadman, will be available for questions.

To register for the call, please click here.

About Dr. ShadmanMazyar Shadman, M.D., M.P.H., is an associate professor at the University of Washington (UW) and Fred Hutch. He is a hematologic malignancies expert who specializes in treating patients with lymphoma / chronic lymphocytic leukemia (CLL). He is involved in clinical trials using novel therapeutic agents, immunotherapy (CAR T cell), and stem cell transplant for treatment of lymphoid malignancies with a focus on CLL. He also studies the clinical outcomes of patients using institutional and collaborative retrospective cohort studies. Dr. Shadman received his M.D. from Tehran University in Iran. He finished internal medicine internship and residency training at the Cleveland Clinic in Cleveland, Ohio. He completed his training in hematology and medical oncology fellowships at UW and Fred Hutch. Dr. Shadman also earned an M.P.H. degree from UW and was a fellow for National Cancer Institutes cancer research training program at Fred Hutch, where he studies cancer epidemiology.

About Dr. TillBrian Till, M.D., is an Associate Professor in the Clinical Research Division of Fred Hutch and Department of Medicine at UW. His laboratory focuses on developing chimeric antigen receptor (CAR)-based immunotherapies for non-Hodgkin lymphoma and understanding why CAR T cell therapies work for some patients but not for others. He led the first published clinical trial testing CAR T cells as a treatment for lymphoma patients. Dr. Till also has a clinical practice treating patients with lymphoma and attends on the stem cell transplantation and immunotherapy services at the Seattle Cancer Care Alliance.

About Mustang BioMustang Bio, Inc. is a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for X-linked severe combined immunodeficiency (XSCID), also known as bubble boy disease. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission (SEC). Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.mustangbio.com.

ForwardLooking StatementsThis press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.

Company Contacts:Jaclyn Jaffe and William BegienMustang Bio, Inc.(781) 652-4500ir@mustangbio.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com

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Exploring the Challenges and Opportunities of Harvesting CTCs – Technology Networks

This cluster of circulating tumor cells (CTCs, shown in red) originated from the blood of a breast cancer patient. Credit: Min Yu (Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at USC), National Cancer Institute/USC Norris Comprehensive Cancer Center

Liquid biopsies involve sampling and analyzing bodily fluids, such as blood, urine or saliva, to look for signs of cancer or other diseases. A range of disease biomarkers can be detected, including circulating tumor cells (CTCs), circulating tumor DNA (ctDNA), exosomes and proteins. The information gleaned from liquid biopsies could help clinicians diagnose, monitor and treatcancer more efficiently, providing insights not possible with traditional surgical biopsies.

As part of its efforts in the liquid biopsy space, ANGLE has developed the Parsortix system. The technology captures CTCs from a blood sample and enables a variety of downstream analyses to be completed. In a recent study from the University Medical Centre Hamburg-Eppendorf, Parsortix was used to successfully harvest CTCs to investigate the causes of brain metastasis in non-small cell lung cancer (NSCLC) patients.We spoke to Andrew Newland, CEO, ANGLE, to learn more about the study, the challenges of harvesting CTCs and how the Parsortix system overcomes some of these challenges.Andrew also discussed the value of characterizing CTCs in clinical trials for new drugs.Anna MacDonald (AM): What advantages do liquid biopsies offer over traditional surgical biopsies?Andrew Newland (AN): Liquid biopsies are based on a blood test. This has multiple advantages but in particular: firstly they are non-invasive requiring only a simple blood draw and do not require an invasive surgical procedure, with the associated costs and potential complications. Secondly they can be repeated often to check on current status of the disease, and response to treatment, whereas the tissue biopsy can only be done once for a given cancer site. This enables personalized medicine with treatment tailored to the patients current condition.AM: Can you explain what circulating tumor cells are, how they can be used to detect and monitor cancers, and why they are so challenging to isolate?AN: CTCs are cancer cells that have left the primary site (or the secondary sites) and are circulating in the blood stream as seeds to spread the cancer in the metastasis process. If they can be captured and harvested for analysis, they can be investigated to determine the current status of the cancer. This provides the information needed for personalized treatment tailored to the patients current condition the right drug at the right time. The CTCs are challenging to isolate because they are very few in number, generally around one CTC for one billion blood cells.AM: Can you give an overview of how the Parsortix system works to capture and harvest CTCs? How does this approach compare to other methods?AN: The Parsortix system captures CTCs using a patented microfluidic structure which separates the CTCs based on their larger size and lack of deformability. Key advantages of the Parsortix system compared to alternative CTC approaches are that it is epitope-independent (does not rely on antibodies to capture CTCs) and captures all types of CTCs including those that are mesenchymal. It also allows easy harvest of the CTCs (recovery from the system) for analysis unlike filtration systems, which have the added problems of clogging up and lack of purity in addition to problems of harvesting the cells for analysis.AM: What benefits does it offer over biological affinity-based methods of CTC isolation?AN: The affinity-based systems are based on binding antibody-coated magnetic beads to the CTCs. These systems fail to capture the clinically significant EMTing and mesenchymal CTCs as they do not express the cell surface markers targeted by the antibodies. In addition, the antibody capture process kills the cells negating the ability to culture the CTCs and potentially changing the RNA and protein expression of the cells.AM: Parsortix was used in a recent study that investigated the causes of brain metastasis in NSCLC patients. Can you tell us more about the study and the significance of the findings?AN: Brain metastasis is where the primary cancer has spread to the brain. Treatment is difficult because it is not known how the cancer is developing in the brain and a surgical tissue biopsy to investigate this is too dangerous. It had been thought not to be possible to harvest CTCs from brain metastasis due to the blood-brain barrier. However University of Hamburg-Eppendorf demonstrated that this was possible using the Parsortix system.AM: ANGLE has recently applied for FDA clearance for Parsortix, for use in metastatic breast cancer. If approved, what difference could this make to patients and clinicians?AN: This would be the first ever FDA cleared platform for harvesting CTCs from patient blood for subsequent analysis. It would open up a whole range of possible diagnostic uses to tailor treatment for metastatic breast cancer patients, that can be validated with additional clinical studies. Despite being recommended in the US National Cancer Guidelines, 50% of MBC patients are too sick, the tumor too inaccessible or insufficient tissue is available for a successful tissue biopsy of the metastatic site. For these patients there is no current information on the cancer to guide treatment. A liquid biopsy would open up alternatives for these patients based on a simple blood test.All MBC patients would benefit from the potential to have repeat biopsies using the Parsortix system to tailor their treatment more effectively. The liquid biopsies can be used to determine whether a drug is being effective, to select which drugs would be effective and to monitor patients in remission to determine, in advance, whether there are signs indicating a risk of relapse. Such early detection may allow treatment to reduce relapse.AM: What value can the characterization of CTCs bring to clinical trials?AN: Analysis of CTCs in clinical trials for new drugs, may allow the identification of likely patient responders so that drugs can be targeted. CTCs enable DNA, RNA and protein analysis providing a more complete picture of the cancer than DNA analysis alone. CTCs may also reduce the time and costs of drug trials by providing early information on drug effectiveness as well as enabling faster enrolment. Liquid biopsies allow serial monitoring of patients over different time periods, which is not possible with tissue biopsies as these cannot be repeated. This longitudinal monitoring may provide more accurate and more timely information of the performance of the drug facilitating clinical trials.AM: In what areas do you envisage liquid biopsies are likely to make the most impact? Will there still be a place for surgical biopsies?AN: The Parsortix liquid biopsy is intended to be additive to surgical tissue biopsies. Tissue biopsies are the current gold standard and will likely continue where the tissue is accessible and the procedure not overly invasive. There is no expectation that liquid biopsy will replace tissue biopsy. Liquid biopsies will be used for repeat biopsies, which is not possible with a tissue biopsy (you cannot cut out the same tissue twice), to provide up-to-date information and where the tissue is inaccessible and/or the biopsy is overly invasive (such as brain metastasis).Andrew Newland was speaking to Anna MacDonald, Science Writer, Technology Networks.

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Exploring the Challenges and Opportunities of Harvesting CTCs - Technology Networks

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COVID-19 patients on some cancer therapies may be contagious for months: study – Reuters

A 3D-printed coronavirus model is seen in front of the words coronavirus disease (Covid-19) on display in this illustration taken March 25, 2020. REUTERS/Dado Ruvic/Illustration

(Reuters) - COVID-19 patients who received cancer treatments that suppress their immune system may remain contagious and able to spread the coronavirus for two months or more, according to a study published on Tuesday.

The U.S. Centers for Disease Control and Prevention (CDC) currently recommends that when patients have compromised immune systems, healthcare workers follow extra precautions such as wearing respirators instead of face masks and isolate patients for up to 20 days after symptoms appear.

In the new study, researchers analyzed sputum and swab samples from 20 immunosuppressed cancer patients infected with the new coronavirus. They found that three were contagious for more than three weeks after their symptoms began, including one who remained contagious for 61 days.

The three patients had received either a stem-cell transplant or therapy with genetically engineered immune cells called CAR T-cells within the previous six months. Two of the three had developed severe COVID-19. None of them had antibodies to the virus.

Current public health recommendations for COVID-19 patients with weak immune systems are based on limited data and may need to be revised, the researchers said in a letter published in the New England Journal of Medicine.

We know from several studies that if youre ... healthy, you are no longer infectious after the first week of illness. But there is very little we know about immunocompromised patients, said Mini Kamboj, one of the studys authors from the Memorial Sloan Kettering Cancer Center. Is that 20 days enough or do we need to exercise precautions for longer than that?

While only a small proportion of cancer patients with COVID-19 are likely to remain contagious for prolonged periods, its a residual risk that we need to address, Kamboj said. We need to keep an open mind about how (much) longer immunocompromised patients could pose an infection risk to others.

Reporting by Manas Mishra in Bengaluru; Editing by Nancy Lapid and Bill Berkrot

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Gain Therapeutics and University of Maryland School of Medicine Announce Research Collaboration – GlobeNewswire

BETHESDA, Md. and BALTIMORE, Nov. 30, 2020 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Gain), today announced a research collaboration with the University of Maryland School of Medicine (UMSOM), to investigate Gains structurally targeted allosteric regulators (STARs) in cellular models of neuronopathic Gaucher disease (nGD) and Parkinsons disease (PD). STARs are proprietary small molecules targeting novel allosteric binding sites on enzymes. These small molecule drug candidates are designed to cross the blood brain barrier and penetrate other hard to treat organs such as bone and cartilage, stabilize the effective enzyme to restore function and reduce toxic substrate. Research will be led by Ricardo A. Feldman, Ph.D., Associate Professor, of Microbiology and Immunology in UMSOM.

Under the terms of the collaboration, UMSOM will investigate Gains STAR candidates in macrophage and neuronal models of nGD and GBA-associated PD. These diseases are characterized by mutations in the GBA gene, where misfolding of the enzyme encoded by GBA (beta-glucocerebrosidase (GCase)) interferes with its normal transport to the lysosome. The research program will aim to further elucidate the mechanism of action of Gains STAR candidates by studying their effect on GCase, including GCases enzyme activity and transport to the lysosome. Additionally, other effects such as prevention of alpha-synuclein aggregation in PD dopaminergic neurons will be evaluated.

We are exceedingly proud to be advancing our work in nGD and Parkinsons in close collaboration with the University of Maryland School of Medicine, said Eric Richman, Chief Executive Officer at Gain. The expertise and experience of UMSOM and Dr. Feldman will be instrumental as we work to further validate the exciting potential of Gains STAR candidate for these devastating diseases. I am confident these foundational studies will bring us closer to a potential new treatment option for those with these disorders.

Dr. Feldman added, Our laboratory has used human induced pluripotent stem cell (iPSC) models of GD and GBA-associated PD to uncover the molecular mechanisms leading to these diseases. We have also developed very sensitive assays to evaluate the therapeutic efficacy of small molecules in reversing the phenotypic abnormalities caused by mutant GBA in the cell types affected by these diseases, including macrophages and neuronal cells. I have been impressed by Gains initial results evaluating the potential of STARs in correcting enzyme misfolding and restoring function, and look forward to working with Gains team to further advance its program to treat these diseases.

Gain and UMSOM intend to report initial data from the collaboration in the first half of 2021.

About Gain Therapeutics, Inc.Gain Therapeutics is redefining drug discovery with its SEE-Tx target identification platform. By identifying and optimizing allosteric binding sites that have never before been targeted, Gain is unlocking new treatment options for difficult-to-treat disorders characterized by protein misfolding. Gain was originally established in 2017 with the support of its founders and institutional investors such as TiVenture, 3B Future Health Fund (previously known as Helsinn Investment Fund) and VitaTech. It has been awarded funding support from The Michael J. Fox Foundation for Parkinsons Research (MJFF) and The Silverstein Foundation for Parkinsons with GBA, as well as from the Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research and Innosuisse. In July 2020, Gain Therapeutics, Inc. completed a share exchange with Gain Therapeutics, SA., a Swiss corporation, whereby GT Gain Therapeutics SA became a wholly owned subsidiary of Gain Therapeutics, Inc. For more information, visit https://www.gaintherapeutics.com/.

Forward-Looking StatementsAny statements in this release that are not historical facts may be considered to be forward-looking statements. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties which may cause results to differ materially and adversely from the statements contained herein. Such statements include, but are not limited to, statements regarding Gain Therapeutics, Inc. (Gain) expected use of the proceeds from the Series B financing round; the market opportunity for Gains product candidates; and the business strategies and development plans of Gain. Some of the potential risks and uncertainties that could cause actual results to differ from those predicted include Gains ability to: make commercially available its products and technologies in a timely manner or at all; enter into other strategic alliances, including arrangements for the development and distribution of its products; obtain intellectual property protection for its assets; accurately estimate its expenses and cash burn and raise additional funds when necessary. Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made. Except as required by law, Gain does not undertake any obligation to update any forward-looking statements to reflect new information, events or circumstances after the date they are made, or to reflect the occurrence of unanticipated events.

Gain Therapeutics Investor Contact:Daniel FerryLifeSci Advisors+1 617-430-7576daniel@lifesciadvisors.com

Gain Therapeutics Media Contact:Cait Williamson, Ph.D.LifeSci Communications+1 646-751-4366cait@lifescicomms.com

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Gain Therapeutics and University of Maryland School of Medicine Announce Research Collaboration - GlobeNewswire

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Human Embryonic Stem Cells (HESC) Market 2019 | Analyzing The Impact Followed By Restraints, Opportunities And Projected Developments | DataIntelo -…

DataIntelo, one of the worlds prominent market research firms has released a new report on Global Human Embryonic Stem Cells (HESC) Market. The report contains crucial insights on the market which will support the clients to make the right business decisions. This research will help both existing and new aspirants for Human Embryonic Stem Cells (HESC) market to figure out and study market needs, market size, and competition. The report talks about the supply and demand situation, the competitive scenario, and the challenges for market growth, market opportunities, and the threats faced by key players.

The report also includes the impact of ongoing global crisis i.e. COVID-19 on the Human Embryonic Stem Cells (HESC) market and what the future holds for it. The published report is designed using a vigorous and thorough research methodology and DataIntelo is also known for its data accuracy and granular market reports.

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A complete analysis of the competitive scenario of the Human Embryonic Stem Cells (HESC) market is depicted by the report. The report has a vast amount of data about the recent product and technological developments in the markets. It has a wide spectrum of analysis regarding the impact of these advancements on the markets future growth, wide-range of analysis of these extensions on the markets future growth.

Human Embryonic Stem Cells (HESC) market report tracks the data since 2015 and is one of the most detailed reports. It also contains data varying according to region and country. The insights in the report are easy to understand and include pictorial representations. These insights are also applicable in real-time scenarios.

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Components such as market drivers, restraints, challenges, and opportunities for Human Embryonic Stem Cells (HESC) are explained in detail. Since the research team is tracking the data for the market from 2015, therefore any additional data requirement can be easily fulfilled.

Some of the prominent companies that are covered in this report:

ESI BIOThermo FisherBioTimeMilliporeSigmaBD BiosciencesAstellas Institute of Regenerative MedicineAsterias BiotherapeuticsCell Cure NeurosciencesPerkinElmerTakara BioCellular Dynamics InternationalReliance Life SciencesResearch & Diagnostics SystemsSABiosciencesSTEMCELL TechnologiesStemina Biomarker DiscoveryTakara BioTATAA BiocenterUK Stem Cell BankViaCyteVitrolife

*Note: Additional companies can be included on request

The industry looks to be fairly competitive. To analyze any market with simplicity the market is fragmented into segments, such as its product type, application, technology, end-use industry, etc. Segmenting the market into smaller components helps in understanding the dynamics of the market with more clarity. Data is represented with the help of tables and figures that consist of a graphical representation of the numbers in the form of histograms, bar graphs, pie charts, etc. Another key component that is included in the report is the regional analysis to assess the global presence of the Human Embryonic Stem Cells (HESC) market.

Following is the gist of segmentation:

By Application:

ResearchClinical TrialsOthers

By Type:

Totipotent Stem CellsPluripotent Stem CellsUnipotent Stem Cells

By Geographical Regions

Asia Pacific: China, Japan, India, and Rest of Asia PacificEurope: Germany, the UK, France, and Rest of EuropeNorth America: The US, Mexico, and CanadaLatin America: Brazil and Rest of Latin AmericaMiddle East & Africa: GCC Countries and Rest of Middle East & Africa

You can also go for a yearly subscription of all the updates on the Human Embryonic Stem Cells (HESC) market.

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Below is the TOC of the report:

Executive Summary

Assumptions and Acronyms Used

Research Methodology

Human Embryonic Stem Cells (HESC) Market Overview

Human Embryonic Stem Cells (HESC) Supply Chain Analysis

Human Embryonic Stem Cells (HESC) Pricing Analysis

Global Human Embryonic Stem Cells (HESC) Market Analysis and Forecast by Type

Global Human Embryonic Stem Cells (HESC) Market Analysis and Forecast by Application

Global Human Embryonic Stem Cells (HESC) Market Analysis and Forecast by Sales Channel

Global Human Embryonic Stem Cells (HESC) Market Analysis and Forecast by Region

North America Human Embryonic Stem Cells (HESC) Market Analysis and Forecast

Latin America Human Embryonic Stem Cells (HESC) Market Analysis and Forecast

Europe Human Embryonic Stem Cells (HESC) Market Analysis and Forecast

Asia Pacific Human Embryonic Stem Cells (HESC) Market Analysis and Forecast

Middle East & Africa Human Embryonic Stem Cells (HESC) Market Analysis and Forecast

Competition Landscape

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DataIntelo has a vast experience in designing tailored market research reports in various industry verticals. We also have an urge to provide complete client satisfaction. We cover in-depth market analysis, which consists of producing lucrative business strategies for the new entrants and the emerging players of the market. We make sure that each report goes through intensive primary, secondary research, interviews, and consumer surveys before final dispatch. Our company provides market threat analysis, market opportunity analysis, and deep insights into the current market scenario.

We invest in our analysts to ensure that we have a full roster of experience and expertise in any field we cover. Our team members are selected for stellar academic records, specializations in technical fields, and exceptional analytical and communication skills. We also offer ongoing training and knowledge sharing to keep our analysts tapped into industry best practices and loaded with information.

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Human Embryonic Stem Cells (HESC) Market 2019 | Analyzing The Impact Followed By Restraints, Opportunities And Projected Developments | DataIntelo -...

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