Category Archives: Stem Cell Clinic

New CEO, check. $172M round, check. Wugen’s off-the-shelf cell therapies are ready for takeoff – FierceBiotech

When Dan Kemp was plottinghis next move after Takeda, he was blown away by data from Wugen, a biotech working on off-the-shelf natural killer (NK) cell therapies. Now, after four months in the CEO seat, hes ready to take those treatments to the next level with a $172 million financing.

The proceeds will bankroll the development of the companys memory NK cell platform and advance its lead program, WU-NK-101, through a phase 1/2 trial in acute myeloid leukemia (AML) and into new studies in solid tumors. The funding will also support Wugens broader pipeline, including an allogeneic CAR-T treatment for T-cell leukemia and lymphoma.

Wugen is one of several biotechs pursuing NK cell therapies to go where CAR-T treatments cannot. Despite its success in blood cancers, CAR-T has faced challenges in solid tumors. And all four of the FDA-approved CAR-T treatments are autologous, meaning theyre made from a patients own cells, which stops them from being widely available.

RELATED: Catamaran Bio sets sail with $42M to create off-the-shelf CAR-NK treatments

The biggest differentiator [of NK cell treatments] from CAR-T is the fact that there is this continual concern around safety. Cytokine release syndrome or neurotoxicity appear to be unavoidable consequences of CAR-T cell therapy, Kemp said, referring to side effects of CAR-T that happen when the treatment activates the immune system too strongly.

T-cell therapy developers have learned to expect these effects and try to manage them rather than avoid them. But NK cell treatments may become a safer alternative.

On the NK side of things, weve seen no toxicity at all; its a pristine safety profile, Kemp said. Thats consistent with other NK cell products that are in the clinic as well.

And thats not allWugen reckons its approach could have an advantage over other NK cell treatments. Its platform generates memory NK cells, which are better at killing cancer cells and last longer in the body than conventional NK cells.

RELATED: Sanofi inks $358M Kiadis takeover to acquire NK-cell platform

Conventional NK cells, like those derived from stem cells, cord blood or peripheral blood, must be engineered to provide sufficient potency to drive any clinical efficacy, Kemp said. Memory NK cells and WU-NK-101 have shown significant efficacy in AML without any engineering at all.

We essentially prime the cells into a superpotent phenotype and expand them so we can actually leverage the innate ability of NK cells themselves to have true clinical potency, he added.

That said, the company plans to combine its NK cell treatments with other cancer-fighting drugs and make engineered NK cell products that could work even better, Kemp said.

Moving forward, Wugen will start a global, multicenter study for WU-NK-101 and file an IND for its CAR-T program in T-cell leukemia and lymphoma later this year, Kemp said. Trials of WU-NK-101 in solid tumors will follow in 2022. As it ramps up its pipeline, the company will aggressively build its team. It currently has 40 staffers across sites in St. Louis and San Diego.

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New CEO, check. $172M round, check. Wugen's off-the-shelf cell therapies are ready for takeoff - FierceBiotech

Arbor Strengthens Focus on Therapeutics with Key Additions to Leadership Team – Yahoo Finance

- Pam Stetkiewicz, Ph.D., Appointed Chief Operating Officer - Kathryn McCabe, Ph.D., Named SVP, Head of Business Development

CAMBRIDGE, Mass., July 20, 2021 (GLOBE NEWSWIRE) -- Arbor Biotechnologies, an early-stage life sciences company discovering and developing the next generation of genetic medicines, announced today the appointments of Pam Stetkiewicz, Ph.D., as Chief Operating Officer, and Kathryn McCabe, Ph.D., as SVP, Head of Business Development. These appointments further expand Arbors leadership team and strengthen its focus on therapeutics.

Bringing Pam and Katy on at this time represents a significant milestone for Arbor as we drive our genetic medicines portfolio to the clinic and partner with leading companies to bring engineered cell therapies to patients, said Devyn Smith, Ph.D., CEO, Arbor Biotechnologies. Their scientific expertise, business acumen, and extensive experience in cell therapy and gene editing will help us execute on this strategy to develop therapeutics with our tailored library of CRISPR-based genetic editors and modifiers.

Pam Stetkiewicz is joining Arbor from Flagship Pioneering, where she was Senior Vice President, Global Program Leader at Flagship Pioneering Medicines. Dr. Stetkiewicz brings more than 20 years of extensive life-sciences pharmaceutical experience with recent experience at Editas Medicine as Vice President, Program and Alliance Management. At Editas, she led the team that filed the first IND for an in vivo CRISPR therapeutic (EDIT-101 for LCA10). Prior to Editas, Dr. Stetkiewicz worked at Novartis Institute of Biomedical Research for 13 years, in a variety of roles across science, alliance, project and portfolio management. Her last role at Novartis was as Executive Director, in Strategic Alliances which involved early business development and collaborations with external companies. She received her Ph.D. from Johns Hopkins University and a B.S. from the University of Rhode Island.

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Im thrilled to be joining Arbor at this exciting time, said Dr. Stetkiewicz. The company has made significant progress in the discovery and development of innovative therapies, particularly in the genetic medicines space, and I am looking forward to helping fulfill the therapeutic promise of Arbors already impressive discoveries.

Kathryn (Katy) McCabe is joining Arbor from Roche where she was Senior Director of Business Development based in Cambridge, MA. Over the last 20 years, she has combined her scientific knowledge, entrepreneurial spirit, and business experience to help transform novel modalities into new medicines at Roche, Lilly, Baxalta and GSK. Dr. McCabe has focused much of her attention on cell and gene therapy and has closed deals for CAR-T, diabetes cell therapy, in vivo gene editing, and gene therapy as well as led large strategic initiatives in these areas. In addition, she has had close interactions with a number of venture funds as the scientific lead for Lillys limited partnerships. Early in her career, Dr. McCabe led a team of senior scientists to develop stem cells for retina and corneal transplantation. She was a Postdoctoral Fellow in Dr. Marianne Bronners lab at Caltech, received her Ph.D. in Neuroscience and Behavior from the University of Washington, and her B.A from the University of Pennsylvania.

I look forward to finding partners that share our vision of bringing curative therapies to patients, said Dr. McCabe.

About Arbor Biotechnologies Arbor Biotechnologies is an early-stage life sciences company discovering and developing the next generation of genetic medicines. Co-founded by Feng Zhang and David Walt, Arbor uses its proprietary discovery engine to uncover unique CRISPR-based genetic modifiers with differentiated genetic editing and delivery capabilities. Following its strategic partnership with Vertex Pharmaceuticals to accelerate the path to the clinic for Arbors technologies, Arbor recently announced an agreement with Lonza. These partnerships further validate the breadth of applications of Arbors gene editing platform that can be custom tailored to address the underlying pathology of each genetic disease. Arbors pipeline of genetic medicines is focused on bringing curative therapies to all patients with genetic disease.

Media Contact: Kelly Friendly press@arbor.bio

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Arbor Strengthens Focus on Therapeutics with Key Additions to Leadership Team - Yahoo Finance

For Patients with HIV and Cancer, a Multidisciplinary Treatment Approach is Critical – Targeted Oncology

As a medical oncologist with a particular interest in the intersection of HIV and cancer, I have seen how cultural taboos as well as economic and demographic forces can still affect care. I have treated patients who dont wish to disclose that they have HIV, and who are willing to drive a hundred miles or more to refill medications in a different town or state to ensure their anonymity. Economically disadvantaged patients often dont have a complete care network, instead relying on their primary care physician and/or a cancer specialist for their treatment.

In 2018, the National Comprehensive Cancer Network (NCCN) published new NCCN Clinical Practice Guidelines in Oncology intended to help make sure people living with HIV who also receive a cancer diagnosis receive safe, necessary treatment.1 The guidelines highlight the disparities in cancer care for people living with HIV and point to the special considerations these patients require.

At Miami Cancer Institute, part of Baptist Health South Florida, we have opened a new HIV/Cancer Clinic to provide these particularly complex patients with better access to new clinical trials as well as coordinated, multidisciplinary care in one location. The program has also been accepted to the AIDS Malignancy Consortium (AMC), which is funded by the National Cancer Institute, to support our clinical trials and enhance our information sharing with other AMC members.

At various points during their treatment, patients at the Miami Cancer Institute HIV/Cancer Clinic will encounter a full spectrum of specialists including oncologists, infectious disease specialists, pharmacists specialized in oncology and infectious disease, nurse practitioners, clinical nurses, medical assistants, palliative care specialists, oncology dentists, psychologists, social workers and case managers. Our cancer clinics operate under one roof so that patients can see multiple specialists and caregivers in one visit. The team essentially assumes the role of primary care provider for the patient, coordinating every aspect of care and meeting periodically as a team to review the patients status.

Physicians who wish to refer HIV-positive patients for cancer care should seek out centers that have the expertise and capabilities to deal with complex cases. For example, Miami Cancer Institute has already performed an allogeneic stem cell transplant on a patient who has both HIV and an aggressive form of plasmablastic lymphoma. The procedure involved finding a matching donor who also had a specific mutation in a gene that offers resistance to HIV infection. Typically, it can take up to two years before a patient such as this can be declared cured, however, the patient is currently doing well six months after transplantation.

Lastly, I recommend that referring physicians look for centers that have a robust clinical trials program. At Miami Cancer Institute, we are presently recruiting HIV patients for seven clinical trials soon to increase to ten for various conditions including Kaposis Sarcoma, lymphomas, cervical cancer, and lung cancer. Even patients with very aggressive lymphomas may be good candidates for a clinical trial.

Reference:

Reid E, Suneja G, Ambinder R, et al. Cancer in people living with HIV, Version 1.2018, NCCN Clinical Practice Guidelines in Oncology.J Natl Compr Canc Netw. 2018;16(8):986-1017. doi. 10.6004/jnccn.2018.0066.

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For Patients with HIV and Cancer, a Multidisciplinary Treatment Approach is Critical - Targeted Oncology

Keeping the physical appointment was critical, the show of support appreciated by Renville County Commissioner – West Central Tribune

When he called the Olivia Hospital and Clinic to postpone his physical, he was urged to keep it. Physicals are important, he was reminded.

Keeping that date proved to be a lifesaving decision.

The physical went well, and shortly after he told his daughter that he was as fit as a horse.

But Dr. Jon Kemp, his primary physician who had urged him to keep the date for the physical, noticed a slight abnormality in a standard blood test. He recommended further testing.

On Dec. 20 Kramer was diagnosed with multiple myeloma.

Thanks to the early diagnosis, Kramer, age 62, has the means of keeping this disease at bay. Its a cancer of the plasma cells in bone marrow, and is the second most common blood cancer.

He is about to undergo a stem cell transplant this week as part of his treatment.

He learned that hes not alone on the journey ahead.

At Tuesdays meeting of the Renville County Board of Commissioners, fellow board members came wearing T-shirts proclaiming: In this county, nobody fights alone.

Organizers of the surprise sold 76 of the T-shirts to show support for Kramer and raise funds for the Renville County Walk in the Park campaign. More than 40 T-shirt wearing supporters joined the meeting via Zoom. Staff in the health department sang a song to express their support, and staff members told him they would keep him in their thoughts and prayers.

Thank you, said Kramer. He told the West Central Tribune that he was totally surprised by the display of support.

He has lots of support from family and friends, and its all-important. Kramer farms in eastern Renville County. He has lined up plenty of helping hands while he undergoes the stem cell transplant, which will sideline him for at least six weeks.

He said doctors are confident the stem cell transplant can knock the cancer into remission. They will be harvesting bone marrow cells and freezing a portion of them to make it possible to perform at least two more transplants in future years as well.

The decision to keep the date of that routine physical made all the difference. Absolutely, said Kramer.

Health providers told him that in too many cases, multiple myeloma is not diagnosed until a patient comes in with a broken leg or other bone, and wondering why. The cancer carves holes and weakens bones as it progresses unbeknownst to the person.

Thanks to the early diagnosis, Kramer said they found only pinholes in his bones, having caught the disease in the first of its three stages. He began chemotherapy in early January, and it has proven effective, he added.

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Keeping the physical appointment was critical, the show of support appreciated by Renville County Commissioner - West Central Tribune

Appia Bio aims for a new kind of off-the-shelf CAR-T with $52M raise – FierceBiotech

Nobel Prizewinner David Baltimore, Ph.D.has always dreamed of turning Los Angeles into a biotech hub. He tried to help it along as president of the California Institute of Technology some 20 years ago, but the idea didnt seem to stick. Now, hes getting another crack at it with a new cancer cell therapy company co-founded with a group of Caltech and Kite Pharma alums.

Appia Bio launches with $52 million to develop off-the-shelf cell therapies for cancer based on hematopoietic, or blood-forming, stem cells. Its platform, dubbed ACUA, allows the programming of stem cells to become invariant natural killer T (iNKT) cells, a powerful subtype of T cell that some folks think of as a hybrid between a natural killer (NK) cell and a T cell, said JeenJoo JJ Kang, Ph.D., CEO of Appia Bio.

Though everybody has iNKT cells in their body, these cells are exceedingly rare. They make up just a fraction of white blood cells found in the peripheral blood, so its hard to produce them in the traditional way, which is to draw blood and expand those cells, Kang said.

[iNKT] cells have multiple tumor cell-killing mechanisms through the natural killer and T cell pathway, she added. Were going to engineer them with a CAR and we think that is positive because it can address more heterogeneous tumor antigen presentations and get around antigen loss.

RELATED: Harper, Seidenberg's Westlake Village BioPartners reels in $500M across 2 funds

Antigen loss or antigen escape is a process in which cancer cells stop expressing the antigen the CAR-T is designed to hunt down, such as CD19. Targeting more than one antigen could disrupt that escape mechanism and prevent relapse. It could also improve the odds of cell therapy working in solid tumorsa major pain point.

Besides solving these problems, Appia also hopes to address the barrier of access. Autologous cell therapiesthat is, those made from a patients own cellsare complex, expensive and time-consuming to make. Some patients dont have enough T cells or T cells of good enough quality to make those treatments.

Producing large amounts of iNKT cells from stem cells that can be stored until theyre needed could make cell therapy available to more patients and at a lower cost.

Being able to mass-produce a cell therapy product that can be dosed to thousands of patients out of a single manufacturing run can significantly affect the economics of what the viable price point is, Kang said. That is another aspect that is a part of our goals: to bring that price down.

RELATED: Catamaran Bio sets sail with $42M to create off-the-shelf CAR-NK treatments

The ACUA technology is based on research out of the laboratory of co-founder Lili Yang, Ph.D., an associate professor at the University of California, Los Angeles who worked under Baltimore as a student and postdoc at Caltech.

Appias founding team also includes Pin Wang, Ph.D., of the University of Southern California and Kite alums Edmund Kim, Ph.D., and Jeff Wiezorek, M.D. Kim, who led corporate development at Kite, is Appias chief operating officer. Wiezorek,who led cell therapy development at Kite, is Appias chief medical officer.

Gilead acquired Kite in 2017, the same year its first CAR-T therapy, Yescarta, scored FDA approval. Later on, Wiezorek and Kims search for their next project coincided with the maturation of Yangs technology at UCLA.

Bringing the two together made a lot of sense, so we did that, Baltimore said. Kang came on board from The Column Group, the VC shop she joined in 2015 after completing her Ph.D. atyou guessed itCaltech.

RELATED: AACR: A look at next-gen CAR-T therapies for blood cancers

With the series A round in the bank, the team will turn to company building and advancing its lead programs in blood cancers and solid tumors, with plans to be in the clinic by the end of 2023, Kang said. 8VC led the financing, while Two Sigma Ventures, Sherpa Healthcare Partners and Freeflow Ventures also chipped in.

The company is setting up shop in Los Angeles County.

Im overjoyed that were getting space in Culver City and the people that we found really appreciate Los Angeles, Baltimore said. Maybe we can make it a biotech hubwe'll seebut at least we can get this company going."

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Appia Bio aims for a new kind of off-the-shelf CAR-T with $52M raise - FierceBiotech

Experimental treatments risk further medicalisation of menopause – The BMJ

Many women experience debilitating menopause symptoms. Fledgling interventions aimed at delaying menopause or treating symptoms are attracting commercial interest, but robust evidence of potential benefit and harms for patients is lacking. Sally Howard reports

Ovarian tissue cryopreservation and transplantation (OTCT)surgical procedures successfully used to restart ovarian function in young women who have undergone chemotherapy1are being offered to healthy women in the United Kingdom with a view to reinstate their pre-menopausal endocrine function. But robust evidence of effectiveness and safety in this indication is lacking, and some experts see this as overmedicalisation of menopause.

The private Birmingham based clinic Profam charges healthy women between 7000 and 11000 (8000-12500; $9500-$15000) for OTCT. Before menopause, a slice of oocyte rich ovary is laparoscopically resected, cryopreserved, and then regrafted into the pelvis or subcutaneously in the forearm after menopause.

Meanwhile, patients in Canada are being advertised a proposed surgical treatment for early menopause that would involve transferring newly recognised germline stem cells from the ovarian lining to the ovary despite limited knowledge of their development into new oocytes.2 Fertility CARE: The IVF Center, a clinic based in Florida, US, markets the treatment OvaPrime as a means of stopping your biological clock, which is on the cusp of being approved for mainstream use just over the northern border.3

Were seeing something like the commercial big bang that happened with IVF in the 1990s, says Evelyn Telfer, a biologist at the University of Edinburgh who researches the clinical potential of germline oocyte precursor cells. Private IVF clinics have proliferated since the 1990s, and some now sell a plethora of non-evidence based add-on interventions. This risks what we saw with IVFpatients paying a lot of money for treatments not ready to be rolled out, she says.

ProFams cofounder Simon Fishel, an IVF scientist,

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Experimental treatments risk further medicalisation of menopause - The BMJ

Biopreservation Market Know the diverse technological advancements in the biopreservation – BioSpace

Biopreservation is a process for conserving the tissues, organs and cells along with maintaining their integrity and functionality at different temperatures for a prolonged period of time. The necessary products for biopreservation are cryo bags, tubes, refrigerators, liquid nitrogen tanks, and freezers. These biopreservation equipment have applications in stem cell, DNA, and plasma and tissue research which helps the market to grow steadily. Various researches being carried out in cell therapy and the increasing number of bio banks also encourage market expansion. Currently, the geriatric population is affected with many disorders related to their lifestyle, namely cardiovascular disease, chronic illness, hypertension, and cancer. Biopreservation applications such as drug discovery, regenerative medicines, and bio banking help the consumers or patients during their life span and even at the time of death.

The enormous growth in the global biopreservation market is accelerated by the rising healthcare expenditure, increasing trend of conserving cord blood stem cells of newborns, and the growing investments in research and development pertaining to this field. A considerable healthcare spending is expected to drive the gene banks, bio banks and hospitals to focus on biopreservation. High costs of advanced techniques and stability issues such as tissue injury during thawing and freezing have been considered as some of the primary factors restraining the biopreservation market growth. One of the prominent names in the industry, BioLife Solutions has signed a ten years business supply agreement with Bellicum Pharmaceuticals for manufacturing, marketing of proprietary tissue and cell, and various cellular immunotherapies which target solid tumors and blood cancers. The influential regions for the biopreservation market are North America, Europe and Asia Pacific. Asia Pacific is expected to offer significant growth opportunities to market players, mostly driven by the demand arising from India and China.

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Global Biopreservation Market: Overview

Biopreservation involves maintaining the functionality and integrity of cells, tissues, and organs outside their natural environment for an extended period of storage at different temperatures. For instance, vaccines save 3 million lives every year in the U.S, however, vaccines worth US$ 20 million are wasted each year due to inadequate storage and improper refrigeration.

Biopreservation safeguards the stability, purity, and quality of biospecimens saved in hospitals, biobanks, and gene banks. For instance, preservation of red blood cells (RBCs) is required for the ready availability of safe blood for blood transfusion needs. The biopreservation of RBCs for clinical purposes can be divided on the basis of techniques used to attain biologic stability and safeguard a viable state after extended storage times.

Global Biopreservation Market: Key Trends

The major factors driving the global biopreservation market include increasing R&D expenditure, increasing number of sperm and egg banks, increasing demand for preserving the stem cells of newborns, and rising adoption of regenerative medicine.

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Across the world, the increasing healthcare expenditure for health and well-being will stimulate the growth of the biopreservation market. This is because healthcare expenditure accounts for a significant part of the developmental budget of most countries. According to data from the World Bank, public healthcare spending is expected to rise at a substantially high rate, which along with a substantial healthcare spending will be an important driver for gene banks, hospitals, and biobanks, which are the key end-users of biopreservation market.

A large population afflicted with chronic disorders such as cardiovascular diseases, diabetes, cancer, and hypertension as well as lifestyle diseases will bolster the markets growth. The increasing public and private spending on, medical goods and services, rising disposable income, increasing demand for biobanking services for the preservation of cells, tissues, and organs, and rising disposable income are also expected to further enhance the growth of the global biopreservation market.

Global Biopreservation Market: Market Potential

In a recent development in the biopreservation industry, BioLife Solutions, a leading name in developing, manufacturing, and marketing of proprietary cell and tissue has entered into a ten year business supply agreement with Bellicum Pharmaceuticals. The latter is a leading name in the development of cellular immunotherapies for cancers and inherited blood disorders. On account of this supply agreement, BioLifes CryoStor cell freeze media is incorporated into Bellicums production process for various cellular immunotherapies that targets blood cancers and solid tumors.

In another industry development, BioLife Solutions has entered into a partnership with transportation firm MNX and expects heightened demand for its biologistics services with the entry of more cell therapies into the clinic.

Global Biopreservation Market: Regional Outlook

The global biopreservation market can be analyzed with respect to the regional segments of North America, Europe, Asia Pacific, and Rest of the World. In North America, the U.S. accounts for almost half the revenue of the region. This is due to the increasing demand for detection of chronic diseases, government stipulations for the ethical usage of biological samples, and introduction of newer of biopreservation methodologies.

Asia Pacific biopreservation market, driven by India and China will display a sustainable growth over the next couple of years

Global Biopreservation Market: Competitive Analysis

Some of the key companies operating in the global Biopreservation market include Thermo Fischer Scientific Inc., VWR Corporation, Lifeline Scientific Inc., BioCision LLC, Custom Biogenic Systems Inc., Princeton Cryotech Inc., Sigma-Aldrich Corporation, Biolife Solutions Inc., Cesca Therapeutics Inc., Core Dynamics Ltd., and So-Low Environmental Equipment Co. Inc.

Top companies in the market are focused on mergers and acquisitions, practicing effective services, and develop new products to stay competitive in the biopreservation market. Expanding geographical reach and developing a broad product portfolio with respect to refrigerators, freezers, and consumables is also leading to the increased market share of some of the top players.

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Biopreservation Market Know the diverse technological advancements in the biopreservation - BioSpace

Reawakened immune cells attack cells tied to diseases of aging – STAT – STAT

Scientists have started to test whether natural killer, or NK, cells can be trained to go after hard-to-cure blood cancers in human patients. But making these sentinels of the innate immune system a potential boon to human health spans might be simpler: Rather than needing to be genetically engineered or primed with synthetic antibodies, they just need to be turned on.

In mice, researchers reported on Monday in the journal Med, activating NKT cells can eliminate the senescent cells partly responsible for many diseases of aging. If the results hold up, they could offer a promising alternative to senolytics experimental drugs that destroy these zombified cells that pile up and pollute your tissues as you get older. Although dozens of such drugs have postponed or even reversed diseases of aging in mouse experiments, clinical trials have thus far underwhelmed.

Its an interesting approach that works in experimental animals with two different conditions, said geriatrician James Kirkland of the Mayo Clinic, whose discovery that giving old mice senescent cell-crushing compounds makes the animals live longer, healthier lives, helped take senolytics from backwater to boomtown. Were going to need multiple ways of getting at senescent cells, he said. Any step forward is important, and this is quite a nice step forward. But he cautioned that a single senolytic strategy is unlikely to work for all age-related conditions.

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For decades, scientists had largely ignored senescent cells old and arrested in a permanent state of suspended animation dismissing them as a quirk of evolution, a clever way for the body to keep damaged cells from proliferating into cancer. But more recently, Kirkland and other researchers established that senescence is actually a driver of the decrepitude that comes with old age. As cells stop dividing, they dont exactly go dormant. In their zombie-like state, they start spewing a cocktail of toxic molecules that cause inflammation, damage surrounding tissues, and contribute to diseases like osteoarthritis, atherosclerosis, diabetes, and Alzheimers.

That realization spurred the creation of at least two dozen companies developing ways to systematically purge the body of senescent cells. Senolytics attracted this wave of investment because it promises a scintillating and fundamental shift in medicine away from the one-drug-one-target-one-disease paradigm of the last century, toward correcting a root cause behind many of them with a single treatment.

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One of those researchers is the new studys senior author, Anil Bhushan of the University of California, San Francisco. In 2019, his lab traced the progression of type 1 diabetes in mice and human pancreatic cells. They discovered that signatures of senescence preceded the onset of disease. When his team removed the senescent pancreatic cells in mice, their metabolism stabilized and their diabetes symptoms went away.

What Bhushan took away from that study was that senescence didnt just happen when the bodys biological clocks wound down too far. It occurred in acute diseases too. Thered been talk in the field that an immune surveillance system maintained tissue homeostasis, said Bhushan. We postulated that that system was failing in the disease state and gradually failing in aging, so the only time wed see senescent cells is when this system fails.

To test that hypothesis, his team first went looking for clues as to the identity of their indolent immune cells. By comparing the transcriptional profile which genes were turned on and off in senescent pancreatic cells to healthy ones, they uncovered that the senescent ones boosted production of their antigen-presenting machinery. These are the proteins that, if a cell were infected with a pathogen, would shuttle little bits of the bacteria or virus to its surface, displaying them for immune cells to find. Then they cross-referenced those results with an analysis of senescent stem cells that accumulate in the fat tissues of obese mice who are fed a chronic, high-fat diet. Those cells also upregulated antigen-presenting molecules, and one in particular: CD1d. That was the lock, said Bhushan. And once wed found it, the key was then obvious.

Only one kind of immune cell binds to CD1d invariant natural killer T cells, or iNKTs. Comprising less than 1% of all peripheral blood immune cells, iNKTs are rare but critical components of the bodys surveillance system, scanning for infected and defective cells in need of removal. When they find them, iNKTs expel torrents of cytokines, which signal to other immune cells to do the dirty work. Bhushan figured that something was interfering with that process. And though he wasnt sure what it was, he knew there was a way to fix it.

In the early 1990s, Japanese scientists from Kirin Brewerys pharmaceutical research lab, looking for anticancer treatments in the porous bodies of marine sponges collected in the Okinawan sea, purified a lipid compound called -galactosylceramide. And they discovered that when CD1d grabs onto this -GalCer lipid, it turns on iNKTs like crazy in mice.

So Bhushans team shot up some of their diet-induced obese mice with -GalCer. Within days, the levels of senescent cells in the mouse fat tissues had dropped. So did their fasting glucose. Their insulin sensitivity also improved. Their metabolism started to look normal.

To see how generalizable the effect was, they repeated the experiment with mice whose lung tissues had been damaged by a chemotherapy drug a common model for idiopathic pulmonary fibrosis, a serious and incurable human lung disease, and one of the nastier complications of Covid-19. In those mice, -GalCer successfully activated iNKTs, again resulting in the removal of senescent cells. The treated mice had fewer damaged cells, and they also lived longer than the control group.

Finally, Bhushan and his colleagues looked at how well activated iNKT cells could tell senescent human cells from healthy ones when cultured together. After 18 hours, 100% of the senescent cells had been destroyed; the vast majority of healthy cells went unscathed. That could give the iNKT approach a potential advantage over the senolytics drugs already in development.

Most of them are repackaged cancer drugs that work by flipping on senescent cells self-destruct buttons. But because zombie cells share a lot of molecular features with their fully animated counterparts, those drugs run the risk of creating lots of collateral damage. Clinical trials of one such drug, 17-DMAG, were abandoned due to toxic side effects in the kidney and brain. Other groups are trying to solve this by engineering a different kind of immune cell, the CAR-T cell, to become a better anti-aging treatment. But CAR-Ts come with their own dangerous side effects and are expensive to make.

Bhushan is optimistic that by returning the cells best trained to suss out senescent cells to the ranks of active immune duty, both these safety and cost concerns can be ameliorated. We have this built in specificity of the immune cells part of their job is telling senescent cells apart from healthy ones were just helping them do their job, he said.

Its still an open question. But clinical trials to answer it could be underway by the end of next year. Bhushans initial discoveries are now being developed by a biotech startup called Deciduous Therapeutics, which he co-founded in 2018. Deciduous is backed by 8VC, CRV, and Laura Demings Longevity Fund, and has until now, been operating in stealth. CEO and co-founder Robin Mansukhani told STAT that the company has been focusing on developing compounds that can best stimulate human iNKTs, whose receptors are structurally a bit different from those of a mouse. He expects Deciduous to file its first investigational new drug application to start human testing within the next 18 months, likely for a metabolic disease or fibrotic lung disorder.

Investors pumped the brakes on senolytics after one of the biggest and brightest stars of the nascent sector and another Longevity Fund portfolio company, Unity Biotechnology, announced last August that its lead drug candidate had failed to reduce knee pain in patients with osteoarthritis. The experimental drug was immediately and unceremoniously dumped, along with nearly one-third of Unitys staff.

But despite the recent slowdown, Mansukhani remains optimistic. The issue in the field has always been what is the actual immune system process behind senescence clearing?he said. And I feel like weve uncovered that.

Mayos Kirkland cautions that deciphering one chapter of the immune system users manual isnt likely to be the whole story. Senescence can be caused by lots of things aging, yes, but also obesity, chemotherapy drugs, and radiation. There are about 40 to 50 different things that can push a cell into a death spiral, said Kirkland. That makes it really hard to define what a senescent cell is, because its molecular makeup depends on how its senescence was induced.

But the good news, according to Kirkland, is that all the fundamental aging processes mitochondria powering down, oxygen radicals disfiguring DNA, rampant inflammation, the spiral toward senescence appear to be tightly interlinked. Its looking increasingly like if you hit one part of this network of things going on, you affect all the rest, and usually in a positive way, he said.

Kirkland, together with his team at Mayo, have had some success with a cocktail of dasatinib and quercetin. In 2019, they reported positive results from a Phase 1 pilot study of nine diabetic kidney disease patients senescent cells were reduced. A Phase 2 study is now underway. And his team has several more trials for serious conditions, including osteoporosis and Alzheimers disease, in the pipeline. Kirkland serves as a scientific adviser to a new senolytics company called NRTK Biosciences that has yet to receive funding and anti-aging supplement company Elysium Health.

But the real question for the future, said Kirkland, is which interventions can you combine to get an additive, synergistic effect? Something that actually alleviates not just one disease of aging, but many, or even all of them? Figuring out how immune cells interplay with senescence is going to be the start of looking for those combinations.

Thats whats next for Bhushans lab a painstaking process of deleting different antigen-displaying peptides across dozens of types of immune cells, and mapping out the almost infinitely complex network of interactions brewing in the toxic stew surrounding senescent cells.

We know we only have part of the story, Bhushan said. We know we can fix whatever is going wrong with iNKTs, but we still dont know exactly what that is.

Science Writer

Megan Molteni is a science writer for STAT, covering genomic medicine, neuroscience, and reproductive tech.

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Global Stem Cell Therapy Market Analysis and Forecast (2019-2024) The Courier – The Courier

During the projected period, the world stem cell therapy market is projected to rise to a CAGR of 10.6 percent and by 2024, its market size will reach 214.5 million USD. The global demand for stem cell treatment is driven mainly by increasing awareness of the therapeutic power of stem cells and the growth of stem cell banking and processing infrastructure. Due to the easy production scale process and the growing commercialization of allogeneic treatments, the industry has experienced strong demand for allogeneic therapies in the past couple of years. Two kinds of stem cell therapy are available, allogeneic and autologous. Both are larger in the allogeneic segment, and in the coming years, are expected to expand rapidly on the market due to their comprehensive therapies, increased marketing of allogeneic products, easy processing, and the increasing number of clinical trials relating to allogeneic therapies.

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The market was segmented based on therapeutic application into gastrointestinal disease, musculoskeletal disorders, cardiovascular disease, and injury. Musculoskeletal problems have led to the highest revenues in the market because musculoskeletal disorders and bone and joint diseases are on the increase and because stem cell products are available for treating musculoskeletal disease and because patients are increasingly favored for effective and early-treatment strategies.

Adipose originating from tissue-derived mesenchymal stem cells, cord blood cells, and bone marrow-derived mesenchymal stem cells were also categorized into a global stem cell therapy market by cell source. Of all categories, mesenchymal stem cells originating from bone marrow are used more and more for therapeutic purposes. The primary drivers of growth in stem cell therapy are increased awareness about the therapeutic potential of stem cell systems, development of stem cell banking and processing facilities, development of advanced cell analysis techniques based on genome, and the increase of private-government investment in stem cell therapy development. Globally, more than 50,000 transplants are carried out annually and they expand each year, according to the World Health Organization (WHO). The growth of the market also fosters a growing prevalence of chronic diseases, regulatory support across developing countries, and technical innovation in health care, cell therapy, and the discovery of new stem cell lines. Any of the diseases that can be treated by stem cell treatment may include osteoarthritis, multiple sclerosis, heart failure, hearing loss, and cerebral palsy. For example, the WHO predicts that by 2050 there will be a disabling hearing loss for an estimated 900 million people. In addition, the preventable cause of 60 percent childhood hearing loss.

Furthermore, the growth of the stem cell treatment market is stimulated by restrictions to traditional organ transplants such as organ donor dependence, risk of infection, immunosuppression rejection and threats and increasing pipelines for new applications and the development of medicinal products. North America is the the biggest market in stem cell therapy and the fastest growth in the market is seen. Factors that contribute to growth in the US stem cell therapeutics market include the an increasing number of trials to determine products therapeutic potential, increased chronic disease prevalence, a growing patient base for target diseases, increased public awareness of the therapeutics potential of therapy, and increased public-private grants for research and funding. Europe is expected to see substantial growth in stem cell therapy in the coming years.

The production of technologically innovative and advanced products is capitalized by major players in the industry, which is strengthened in the stem cell therapy market. Prestige Lyotechnology, a storage system for living cells and tissues, was introduced by Osiris Therapeutics in March 2017. In addition, MEDIPOST announced that in February 2018, the FDA has approved its drug NEUROSTEM for clinical trials for its stem cell-based Alzheimers disease drug. Some of the major players providing services are Osiris Therapeutics, Ing, RTI Surgical, Inc., MEDIPOST Co., Ltd, Nuvasive, Inc., Pharmicell, Ltd, Holostem Terapie Avanzate Srl, JCR Pharmaceuticals Co., Ltd., Anterogen Co., Ltd., and Allosaurus.

Update :

20 years old, a life-saving stem cell transplant was carried out in an anonymous donor in Germany to fight rare blood cancer. Swindons Luke Hope lost his sight in the right eye and got a 30% chance of acute lymphoblastic leukemia survival (ALL). In October, after a match was made with a man from Germany, he received a transplant at Churchill Hospital, Oxford. The UK stem cell transplantation waiting list currently has 2,000 participants.

In some private clinics patients with the healing power of stem cells charge thousands of pounds for unproven and non-regulated treatments. And some of these therapies can cause serious damage to experts. Stem cells can be used to heal damaged tissue and can transform several forms of body cells, from muscle to brain. However, only certain blood disorders, skin grafts, and repair of damaged corneas have been authorized.

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Contact Person- Kundan Kumar Email ID kundan@vynzresearch.com Source: VynZ Research https://www.vynzresearch.com/

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Global Stem Cell Therapy Market Analysis and Forecast (2019-2024) The Courier - The Courier

Cell Therapy Company Raises $160 Million to Advance iPSC-Derived Therapies to Clinic – BioSpace

Century Therapeuticsreceived a $160 million infusion of cash to advance the companys pipeline of induced pluripotent stem cell (iPSC)-derived cell therapies for cancer.

This morning, the Philadelphia-based company announced a Series C financing round that will drive its preclinical pipeline, which includes multiple iPSC-derived CAR-iT and CAR-iNK cell products, into the clinic over the next 12 months. The companys assets are designed to resist host rejection, enhance cell persistence, and allow repeat dosing to provide durable responses in all patients. Century Therapeutics, which launched in 2019, anticipates clinical testing to begin in 2022 and also predicts it will generate multiple Investigational New Drug applications over the next several years.

The companysgenetically engineered, iPSC-derived iNK and iT cell products are designed to specifically target hematologic and solid tumor cancers. Centurys iPSCs, which are stem cells that can be generated from adult stem cells, have unlimited self-renewing capacity, which enables multiple rounds of cellular engineering. According to the company, these engineering rounds will produce master cell banks of modified cells that can be expanded and differentiated into immune effector cells to supply vast amounts of allogeneic and homogeneous therapeutic products. This platform differentiates Century from competitors that are developing cell therapies made from non-renewable donor-derived cells.

The Series C financing round was led by Casdin Capital and include a number of new investors, including Fidelity Management & Research LLC, the Federated HermesKauffmannFunds, RA Capital, Logos Capital, OrbiMed,Marshall Wace, Qatar Investment Authority, Avidity Partners, and Octagon Capital.Founding investorsVersant Ventures and Leaps by Bayer also participated in the latest fundraising.

We are fortunate to be surrounded by such a top-tier group of investors, whose support will enable the acceleration of Century's technology platform into the clinic, Lalo Flores, chief executive officer of Century Therapeutics said in a statement. With this new investor partnership, we are well-positioned to capitalize on the tremendous potential of our integrated iPSC, cell engineering and manufacturing capabilities to develop safer, more effective and more affordable next generation allogeneic cancer therapies.

Eli Casdin, chief investment of Casdin Capital, who joined the Century Therapeutics Board of Directors following this Series C, said he was excited to partner with the cell therapy company.

It's a remarkable and transformative time in the field, with the ability to engineer cells for therapeutic impact now a commercial reality. At the same time, iPSC technology has matured and is now leading the transition from bespoke autologous products to off-the-shelf allogeneic ones, Casdin said in a statement.

For Century Therapeutics, the financing round was announced about one month after the company expanded its capabilities with new laboratory manufacturing facilities in Pennsylvania and New Jersey. In addition to thePennsylvaniaandNew Jerseylocations, Century has a laboratory inHamilton, Ontariospecifically focused on targeting glioblastoma, and recently opened aSeattle-based innovation hub to help advance the company's novel iPSC platform and support the continued pipeline growth and development.

Last year, Century Therapeutics acquired Empirica Therapeutics to leverage its iPSC-derived allogeneic cell therapies against glioblastoma, one of the most common types of primary brain tumor in adults.

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