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Antiviral cellular therapy for enhancing T-cell reconstitution before or after hematopoietic stem cell transplantation … – Nature.com

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Antiviral cellular therapy for enhancing T-cell reconstitution before or after hematopoietic stem cell transplantation ... - Nature.com

Somite Raises $5.3M As Five Leading Scientists Join Forces To Incorporate AI In Stem Cell Therapy – PR Newswire

[[To comply with academic institution guidelines, the founders' academic affiliations and roles are listed only at the end of the statement.]]

BOSTON and AUSTIN, Texas, April 16, 2024 /PRNewswire/ --Somite, a venture-backed company aiming to become the OpenAI of stem cell biology, announces that it has raised $5.3M in pre-seed funding. The round was led by Israel's preeminent Venture fund TechAviv, and joined by renowned Austin-based VCs Next Coast Ventures, Trust Ventures and Texas Venture Partners as well as NY-based Lerer Hippeau and others. The funds will be used to continue development of Somite's proprietary AlphaStem AI platform, building Somite labs and bringing the first therapeutic asset to phase 1 clinical trials.

Founded in October 2023, Somite is building AI foundation models to produce human tissue at scale for cell therapies. These therapies have the potential to cure a wide range of diseases that involve the loss or deficiency of cell populations, such as Diabetes, Obesity, and Muscular Dystrophies.

Somite Raises $5.3M in Pre-seed Round to Transform Cell Therapy with Al

The founding team comprises five distinguished experts in their respective fields: Dr. Micha Breakstone, a seasoned AI entrepreneur who successfully sold Chorus.ai for $575 million, serves as the CEO. Joining him are the CTO,Dr. Jonathan Rosenfeld, who pioneered AI scaling laws at MIT,along with Boston-based scientistsDr. Olivier Pourquie, Dr. Allon Klein, and Dr. Cliff Tabin, who bring expertise in developmental biology, stem cells and computational biology.

Cell therapy, a revolutionary approach to treating medical conditions, involves replacing missing, damaged, or diseased cells. While recent strides in stem cell research have opened new avenues for producing various human cell types, challenges persist in terms of efficiency, scalability, and robustness across existing protocols. Somite.ai stands out as the premier company excelling in the efficient production of cells derived from somites, crucial embryonic structures giving rise to musculoskeletal cells. These include muscle, brown adipose, cartilage, bone, tendon, and dermis. Leveraging its expertise, Somite is pioneering the development of a digital twin of the embryoa computational model mirroring real embryo development and behavior. Drawing from data-rich sources such as scRNA-Seq, scATAC-seq, and gene expression databases, the digital twin empowers Artificial Intelligence to swiftly uncover innovative protocols, identify regulators of cell differentiation, and conduct rapid optimization cycles.

Somite's proprietary digital twin not only surfaces actionable insights but also expedites protocol iterations. Somite also builds on work performed in the Pourquie laboratory where production of somite-derived lineages such as human satellite and brown adipose cellsin vitro was optimized using computational analysis and AI, allowing a critical increase in purity without the need for sorting procedures.

Since its inception, Somite has rapidly achieved noteworthy milestones, including gaining acceptance to the esteemed Blavatnik Harvard Life Labs and securing intellectual property for groundbreaking patents.

"The future of medicine lies at the intersection of AI and biology," says Micha Breakstone, CEO and Co-Founder of Somite. "With Somite's AlphaStem platform we have the unique opportunity to both unlock the governing principles of cell differentiation and introduce therapies to cure tens of millions of people. This funding round is only one of many first steps in our exciting journey."

"We are proud to partner with Somite and have high conviction in the exceptional team and transformative solution for stem cell therapy," says Yaron Samid, Founder and Managing Partner at TechAviv. "I am captivated by Somite's potential to redefine the boundaries of medical innovation. They are committed to advancing their groundbreaking stem cell therapy technology and have the power to transform the lives of millions of people by leveraging AI to produce human tissue for cell therapies."

About SomiteSomite.aiis a venture-backed company aiming to become the OpenAI of stem cell biology, developing AI foundation models to produce human tissue for cell therapies at scale for diseases such as diabetes, obesity, and muscular dystrophies. Somite's AI platform, AlphaStem, fuels a virtuous cycle: It enables new cell therapies, generating massive data that further improve the platform, empowering even faster therapy creation with broader applications.

Incorporated in Oct. 2023, Somite.ai has raised $5.3m to date.

Somite Management Team:

Scientific Co-founders:

Media Contact: [emailprotected]

SOURCE Somite Therapeutics

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Somite Raises $5.3M As Five Leading Scientists Join Forces To Incorporate AI In Stem Cell Therapy - PR Newswire

Cell Therapies Now Beat Back Once Untreatable Blood Cancers. Scientists Are Making Them Even Deadlier. – Singularity Hub

Dubbed living drugs, CAR T cells are bioengineered from a patients own immune cells to make them better able to hunt and destroy cancer.

The treatment is successfully tackling previously untreatable blood cancers. Six therapies are already approved by the FDA. Over a thousand clinical trials are underway. These arent limited to cancerthey cover a range of difficult medical problems such as autoimmune diseases, heart conditions, and viral infections including HIV. They may even slow down the biological processes that contribute to aging.

But CAR T has an Achilles heel.

Once injected into the body, the cells often slowly dwindle. Called exhaustion, this process erodes therapeutic effect over time and has dire medical consequences. According to Dr. Evan Weber at the University of Pennsylvania, more than 50 percent of people who respond to CAR T therapies eventually relapse. This may also be why CAR T cells have struggled to fight off solid tumors in breast, pancreatic, or deadly brain cancers.

This month, two teams found a potential solutionmake CAR T cells more like stem cells. Known for their regenerative abilities, stem cells easily repopulate the body. Both teams identified the same protein master switch to make engineered cells resemble stem cells.

One study, led by Weber, found that adding the protein, called FOXO1, revved up metabolism and health in CAR T cells in mice. Another study from a team at the Peter MacCallum Cancer Center in Australia found FOXO1-boosted cells appeared genetically similar to immune stem cells and were better able to fend off solid tumors.

While still early, these findings may help improve the design of CAR T cell therapies and potentially benefit a wider range of patients, said Weber in a press release.

Heres how CAR T cell therapy usually works.

The approach focuses on T cells, a particular type of immune cell that naturally hunts downs and eliminates infections and cancers inside the body. Enemy cells are dotted with a specific set of proteins, a kind of cellular fingerprint, that T cells recognize and latch onto.

Tumors also have a unique signature. But they can be sneaky, with some eventually developing ways to evade immune surveillance. In solid cancers, for example, they can pump out chemicals that fight off immune cell defenders, allowing the cancer to grow and spread.

CAR T cells are designed to override these barriers.

To make them, medical practitioners remove T cells from the body and genetically engineer them to produce tailormade protein hooks targeting a particular protein on tumor cells. The supercharged T cells are then grown in petri dishes and transfused back into the body.

In the beginning, CAR T was a last-resort blood cancer treatment, but now its a first-line therapy. Keeping the engineered cells around inside the body, however, has been a struggle. With time, the cells stop dividing and become dysfunctional, potentially allowing the cancer to relapse.

To tackle cell exhaustion, Webers team found inspiration in the body itself.

Our immune system has a cellular ledger tracking previous infections. The cells making up this ledger are called memory T cells. Theyre a formidable military reserve, a portion of which resemble stem cells. When the immune system detects an invader its seen beforea virus, bacteria, or cancer cellthese reserve cells rapidly proliferate to fend off the attack.

CAR T cells dont usually have this ability. Inside multiple cancers, they eventually die offallowing cancers to return. Why?

In 2012, Dr. Crystal Mackall at Stanford University found several changes in gene expression that lead to CAR T cell exhaustion. In the new study, together with Weber, the team discovered a protein, FOXO1, that could lengthen CAR Ts effects.

In one test, a drug that inhibited FOXO1 caused CAR T cells to rapidly fail and eventually die in petri dishes. Erasing genes encoding FOXO1 also hindered the cells and increased signs of CAR T exhaustion. When infused into mice with leukemia, CAR T cells without FOXO1 couldnt treat the cancer. By contrast, increasing levels of FOXO1 helped the cells readily fight it off.

Analyzing genes related to FOXO1, the team found they were mostly connected to immune cell memory. Its likely that adding the gene encoding FOXO1 to CAR T cells promotes a stable memory for the cells, so they can easily recognize potential harmbe it cancer or pathogenlong after the initial infection.

When treating mice with leukemia, a single dose of the FOXO1-enhanced cells decreased cancer growth and increased survival up to five-fold compared to standard CAR T therapy. The enhanced treatment also tackled a type of bone cancer in mice, which is often hard to treat without surgery and chemotherapy.

Meanwhile, the Australian team also zeroed in on FOXO1. Led by Drs. Junyun Lai, Paul Beavis, and Phillip Darcy, the team was looking for protein candidates to enhance CAR T longevity.

The idea was, like their natural counterparts, engineered CAR T cells also need a healthy metabolism to thrive and divide.

They started by analyzing a protein previously shown to enhance CAR T metabolism, potentially lowering the chances of exhaustion. Mapping the epigenome and transcriptome in CAR T cellsboth of which tell us how genes are expressedthey also discovered FOXO1 regulating CAR T cell longevity.

As a proof of concept, the team induced exhaustion in the engineered cells by increasingly restricting their ability to divide.

In mice with cancer, cells supercharged with FOXO1 lasted longer by months than those that hadnt been boosted. The critters liver and kidney functions remained normal, and they didnt lose weight during the treatment, a marker of overall health. The FOXO1 boost also changed how genes were expressed in the cellsthey looked younger, as if in a stem cell-like state.

The new recipe also worked in T cells donated by six people with cancer who had undergone standard CAR T therapy. Adding a dose of FOXO1 to these cells increased their metabolism.

Multiple CAR T clinical trials are ongoing. But the effects of such cells are transient and do not provide long-term protection against exhaustion, wrote Darcy and team. In other words, durability is key for CAR T cells to live up to their full potential.

A FOXO1 boost offers a wayalthough it may not be the only way.

By studying factors that drive memory in T cells, like FOXO1, we can enhance our understanding of why CAR T cells persist and work more effectively in some patients compared to others, said Weber.

Image Credit: Gerardo Sotillo, Stanford Medicine

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Cell Therapies Now Beat Back Once Untreatable Blood Cancers. Scientists Are Making Them Even Deadlier. - Singularity Hub

Stem Cell Treatment Helped A Man Who Was Paralyzed From The Neck Down To Stand On His Own – Bored Panda

Long gone are the days of people being fearful of innovation and writing off any medical advance as witchcraft. However, it is hard not to believe in magic and science when doctors and scientists are making incredible discoveries that help patients improve their life quality and even make miraculous recoveries. Chris Barr is one of those lucky people who, thanks to science, is able to walk again after a severe spinal cord injury.

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Seven years ago, Chris Barr was having a regular day, just like a hundred other days gone by. The avid surfer was catching waves at a local beach until something went terribly wrong. One wave was particularly dangerous and threw Chris off the board. Soon, he realized that he was injured and the solemn expression on his doctors faces told him the news was going to be hard to swallow.

At the hospital, Chris learned about his life altering diagnosis he was paralyzed from the neck down. But Chris was determined to fight and believed that one day he will be able to regain some control of his body. However, even in his wildest dreams, he never imagined just how advanced his recovery will be and that he will be walking again thanks to an innovative stem cell treatment.

I never dreamed I would have a recovery like this, Chris shared his delight. I can feed myself. I can walk around. I can do day-to-day independent activities.

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Chris was the first patient in the Mayo Clinic study. It consisted of collecting stem cells from his own stomach fat and expanding them in the laboratory to 100 million cells. After that, the cells were injected into Chris lumbar spine. The treatment continued for over five years during which Chris saw a significant improvement in his quality of life, gaining more and more independence with each injection.

Mr. Barrs recovery story was published in the Nature Communications journal, as part of the research on the effects of stem cells in spinal cord injuries. The study claims that out of ten patients participating in the trial, seven saw positive effects in recovery from their injuries. The patients expressed that they have noticed increased strength in muscle motor groups and increased sensation to pinpricks and light touch. Each patient moved at least one level on the American Spinal Injury Association (ASIA) Impairment Scale. The scale has five levels detailing a patients ability to function. The other three patients, sadly, showed no improvement but they did not get worse.

These findings give us hope for the future, Dr. Mohamad Bydon, a neurosurgeon and the lead author of the study shared. The doctor, who is the director of The Christopher Reeve foundation, has dedicated his lifes work to curing spinal cord injuries, and is very hopeful of this trials future.

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The participants ranged from 18 to 65, all spinal injuries were either from the neck or back down. They all had their stem cells harvested from the stomach. Patient 1, Chris, had the most remarkable recovery of them all.

At the beginning of the study, Chris was on a ventilator, completely immobile. As the study progressed, he started using a harness and then even started taking some steps on his own. Besides stem cell treatment, Chris was doing hard work with his physiotherapist.

We waited, we didnt intervene right away, as many studies in this space do, the doctor noted. The earliest we treated anyone was seven months after their injury and the latest was 22 months. The researchers wanted to give the body time to try and recover on its own.

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Despite the incredible findings, the road to this new treatment being approved is still a long way away, as there needs to be more research done so doctors could understand how stem cells operate. To us, it might seem like magic or a miracle but for doctors, it means long years of trial and error, hoping that in their lifetime they will be able to help those in need.

Patients are always looking for a cure. Were not there today, but we have to continue this research in order to get there, Dr. Bydon pondered.

The second part of the study, involving more patients, is now underway, giving hope to those who have heard the horrible words, Youll never walk again. Hopefully, in a few years, those people will take their first steps yet again and say, Take that, bad luck!

As for Chris, he is delighted that he was able to be a part of this groundbreaking study. He might never surf again but at least he is able to take longer and longer walks without any assistance.

Im just thrilled that there are people taking bold steps to try and do research to cure this. Its been a wild ride and its not over yet.

What do you think of this? Do you think stem cell therapy is the future or does this sound like science fiction to you?

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Stem Cell Treatment Helped A Man Who Was Paralyzed From The Neck Down To Stand On His Own - Bored Panda

Anemia: Symptoms, Causes & Treatment – Cleveland Clinic

How do I take care of myself?

While some types of anemia are short-term and mild, others can last a lifetime. Regardless, there are several things you can do to help manage symptoms. Here are some suggestions:

If you have anemia, you should check with your provider if your symptoms get worse despite treatment or if you notice changes in your body that may be new symptoms.

Anemia may increase your risk of a heart attack. Call 911 if you have the following symptoms:

Anemia may affect your body in many ways. It may happen for many different reasons. If you have anemia, here are some questions you may want to ask your healthcare provider:

A note from Cleveland Clinic

Anemia happens when you dont have enough red blood cells or your red blood cells arent working as well as they could. Some people are born with forms of anemia, but most people who have anemia develop the condition over time. Thats why its important to keep track of changes in your body. For example, we all have days when we feel worn out. But if youre feeling very tired for several days despite getting rest, consider talking to your healthcare provider.

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Anemia: Symptoms, Causes & Treatment - Cleveland Clinic

Abu Dhabi Stem Cells Center partners with Red Crescent to enhance bone marrow transplant accessibility for patients – Abu Dhabi Media Office

Abu Dhabi Stem Cells Center (ADSCC) has partnered with Emirates Red Crescent (ERC) to enhance accessibility to bone marrow transplants for patients in need, providing them with treatment and support via the centres dedicated Abu Dhabi Bone Marrow Transplant (BMT) Programme.

Under the partnership, ADSCC will provide medical consultations to patients referred by the medical committee of ERC. In addition, both entities will join efforts in fundraising initiatives aimed at supporting and financing BMT procedures for underprivileged patients.

The partnership aims to provide these critical treatments to individuals affected by various blood cancers, blood disorders, and autoimmune diseases like multiple sclerosis, ensuring they receive the highest standard of care and support throughout their treatment process by Abu Dhabi Bone Marrow Transplant program (ADBMT) at ADSCC, which is accredited as a Centre of Excellence in Hematopoietic Stem Cell Transplantation by the Department of Health Abu Dhabi.

The agreement was signed by His Excellency Mohammed Al Fahim, Deputy Secretary General of Support Services Sector at ERC and Dr Maysoon Al Karam, Chief Medical Officer of ADSCC.

His Excellency Mohammed Al Fahim said: The agreement with Abu Dhabi Stem Cells Center embodies the vision of the UAE Red Crescent of enhancing humanitarian partnerships with various local sectors, particularly the healthcare sector, which is currently a priority in the authority's local and international efforts. It reflects the shared goal between both parties of alleviating the suffering of patients, supporting their health needs, and strengthening cooperation to achieve better health outcomes for the Red Crescent's beneficiaries. I would like to express our appreciation for such initiatives that address the health needs of vulnerable groups and commend Abu Dhabi Stem Cell Center's (ADSCC) efforts, and their contributions to advancing treatment and healing. The alignment of visions between the UAE Red Crescent and ADSCC, highlights the importance and potential for strategic partnerships in healthcare.

Dr Maysoon Al Karam said: "The UAEs leadership has always placed the health and wellbeing of its people at the forefront of its priorities, ensuring that high-quality healthcare services are accessible to all. This commitment underscores the essence of our collaboration with the Emirates Red Crescent, reflecting our joint vision to expand the accessibility of advanced medical treatments. Through such collaborations, we aim to bridge the gap in healthcare access, ensuring that state-of-the-art treatments are accessible to those in need. As a Centre of Excellence in Hematopoietic Stem Cell Transplantation, our Abu Dhabi Bone Marrow Transplant Programme has been offering holistic care for autologous and allogenic bone marrow transplant for adults and children since 2020. By providing advanced treatments locally, we eliminate the need for patients to seek medical care abroad. This not only enhances convenience for patients and their families but also plays a vital role in reducing the nation's healthcare expenditures.

To ensure healthcare access in the UAE, ADSCC is dedicated to collaborating with key stakeholders across the nation. This commitment is geared towards achieving our vision of pioneering innovative solutions to discover cures for diseases.

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Abu Dhabi Stem Cells Center partners with Red Crescent to enhance bone marrow transplant accessibility for patients - Abu Dhabi Media Office

ProQR Achieves Successful Defense of New Challenge to its Axiomer™ IP Portfolio

LEIDEN, Netherlands & CAMBRIDGE, Mass., April 19, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced it has again successfully defended against opposition filed against a key patent for its ADAR-mediated RNA editing platform Axiomer™.

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ProQR Achieves Successful Defense of New Challenge to its Axiomer™ IP Portfolio

Tiziana Life Sciences Announces Study Results from Intranasal Anti-CD3 Foralumab in Multiple Sclerosis Patients with PIRA Highlighted in Neurology…

- Intranasal foralumab attenuated microglial activation in patients with non-active secondary progressive multiple sclerosis and progression independent of relapse (PIRA) -- Data presented in a platform session at the Annual Meeting of the American Academy of Neurology in Denver, Colorado -

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Tiziana Life Sciences Announces Study Results from Intranasal Anti-CD3 Foralumab in Multiple Sclerosis Patients with PIRA Highlighted in Neurology...

Ocugen Announces Dosing Completion of Subjects with Geographic Atrophy in Cohort 2 of Phase 1/2 ArMaDa Clinical Trial of OCU410—A Modifier Gene…

MALVERN, Pa., April 19, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that dosing is complete in the second cohort of its Phase 1/2 ArMaDa clinical trial for OCU410 (AAV-hRORA)—a modifier gene therapy candidate being developed for geographic atrophy (GA), an advanced stage of dry age-related macular degeneration (dAMD). GA affects approximately 1 million people in the United States alone.

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Ocugen Announces Dosing Completion of Subjects with Geographic Atrophy in Cohort 2 of Phase 1/2 ArMaDa Clinical Trial of OCU410—A Modifier Gene...