UM, CUHK jointly hold symposium on stem cells and regenerative medicine – gcs.gov.mo

The Faculty of Health Sciences (FHS) of the University of Macau (UM) and the School of Medicine of the Chinese University of Hong Kong (CUHK) jointly held the 10th CUHK International Symposium on Stem Cell Biology and Regenerative Medicine and the 3rd Guangdong-Hong Kong-Macau Greater Bay Area International Conference on Regenerative Medicine. The two-day event attracted more than 100 renowned scholars, clinical medical experts, industry professionals, researchers and students from overseas, mainland China, Hong Kong, and Macao. They engaged in in-depth discussions on the advancements in the field of stem cells and regenerative medicine, as well as their clinical translation.

In his speech, Ge Wei, vice rector of UM, said that the Guangdong-Hong Kong-Macao Greater Bay Area is an important region for the development of medical science and technology in China. To support the countrys implementation of the Greater Bay Area development strategy, UM is committed to biomedical research and the training of outstanding talent in regenerative medicine, providing impetus to the sustainable development of the biomedical industry in the Greater Bay Area. The conference not only deepened the academic exchanges and cooperation between UM and CUHK, but also provided a valuable opportunity for students from both universities to exchange ideas.

Rocky Tuan, vice-chancellor and president of CUHK, said in his speech that regenerative medicine is a major breakthrough in biomedicine in the 21st century. Significant advances in stem cell biology and the development of smart biomaterial engineering have provided a solid foundation for future tissue regeneration to treat injuries and diseases. The conference provided a platform for the exchange of innovative research ideas and an opportunity to inspire students and young scientists to learn and collaborate.

The symposium featured eight keynote lectures and 39 sub-forum presentations, delivered by domestic and foreign experts in stem cell biology, regenerative medicine, bioengineering and other cutting-edge fields, which marked the highest number of presentations in the history of the event. During the keynote lecture session, Tuan presented the latest developments in stem cell technology and social problems that need to be solved; Qin Ling, fellow of the American Institute for Medical and Biological Engineering and renowned professor of orthopaedics and traumatology, shared the research progress of biodegradable metal-derived magnesium in bone regeneration and its clinical translation; Chang Jiang, fellow of the UK Royal Society of Chemistry and professor at the Chinese Academy of Sciences, talked about the prospects for the application of bioactive ceramics and composite materials in different tissue regeneration and disease treatment. In addition, a student forum was held during the symposium for the first time, providing students from both universities with a platform for academic exchanges and presentation of their research results.

Xu Renhe, associate dean of UMs FHS, and Li Gang, professor in CUHKs School of Medicine, served as co-chairs of the organising committee of the symposium. Co-organisers of the symposium included the Prince of Wales Hospital and the Center for Research Excellence of CUHK. Chuxia Deng, dean of UMs FHS also attended the event.

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UM, CUHK jointly hold symposium on stem cells and regenerative medicine - gcs.gov.mo

Human Neuron Model Paves the Way for New Alzheimer’s Therapies – Weill Cornell Medicine Newsroom

Weill Cornell Medicine scientists have developed an innovative human neuron model that robustly simulates the spread of tau protein aggregates in the braina process that drives cognitive decline in Alzheimer's disease and frontotemporal dementia. This new model has led to the identification of novel therapeutic targets that could potentially block tau spread.

The preclinical study, published April 5 in Cell, is a significant advancement in Alzheimer's disease research.

Dr. Li Gan. Credit: John Abbott

Currently no therapies can stop the spread of tau aggregates in the brains of patients with Alzheimers disease," said lead study author, Dr. Li Gan, director of the Helen and Robert Appel Alzheimers Disease Research Institute and the Burton P. and Judith B. Resnick Distinguished Professor in Neurodegenerative Diseases in theFeil Family Brain and Mind Research Instituteat Weill Cornell Medicine. Our human neuron model of tau spread overcomes the limitations of previous models and has unveiled potential targets for drug development that were previously unknown.

Human pluripotent stem cells can develop into any cell of the body and can be coaxed to become neurons to model brain diseases in a lab dish. However, it had been nearly impossible to model tau propagation in these young neurons, as tau propagation requires decades in aging brains.

Celeste Parra Bravo

Dr. Gans team used CRISPR technology to modify the genomes of human stem cells, prompting them to express forms of tau associated with diseased aging brains. "This model has been a game-changer, simulating tau spread in neurons within weeksa process that would typically take decades in the human brain," Dr. Gan said.

In their quest to halt tau propagation, Dr. Gan's team employed CRISPRi screening to disable 1,000 genes to ascertain their roles in tau spread. They discovered 500 genes that have a significant impact on tau abundance.

Dr. Shiaoching Gong

CRISPRi technology allowed us to use unbiased approaches to look for drug targets, not confined to what was previously reported by other scientists, said one of the lead study authors Celeste Parra Bravo, a neuroscience doctoral candidate in the Weill Cornell Graduate School of Medical Sciences working in the Gan lab.

One discovery includes the UFMylation cascade, a cellular process involving the attachment of a small protein named UFM1 to other proteins. This process's connection to tau spread was previously unknown. Post-mortem studies of brains from patients with Alzheimers disease found that UFMylation is altered, and the team also found in preclinical models that inhibition of the enzyme required for UFMylation blocks tau propagation in neurons.

We are particularly encouraged by the confirmation that inhibiting UFMylation blocked tau spread in both human neurons and mouse models, said paper co-author Dr. Shiaoching Gong, associate professor of research in neuroscience in the Appel Instituteat Weill Cornell Medicine.

Many Alzheimers disease treatments initially show promise in mouse models but do not succeed in clinical trials, Dr. Gan said. With the new human cell model, she is optimistic about the path ahead. "Our discoveries in human neurons open the door to developing new treatments that could truly make a difference for those suffering from this devastating disease."

Many Weill Cornell Medicine physicians and scientists maintain relationships and collaborate with external organizations to foster scientific innovation and provide expert guidance. The institution makes these disclosurespublic to ensure transparency. For this information, see profile forDr. Li Gan.

This research was supported in part by the National Institute of Neurological Disorders and Stroke, the National Institute on Aging and the National Institute of General Medical Sciences, all part of the National Institutes of Health, through grant numbers U54NS100717, R01AG072758, R01AG054214, R01AG074541, R25GM130494, and R01AG064239. Additional support was provided by the Rainwater Charitable Foundation and the JPB Foundation.

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Newark teenager suffering from a rare form of cancer who is having treatment funded by the NHS matched with stem … – LincsOnline

A teenager given a terminal diagnosis just before Christmas has now been told 95% of the cancer gone after experimental treatment.

Dan Evans, 18, is now looking to Israel for his next stage of treatment.

A major community fundraiser was launched to pay for the treatment after the teenager was told he had exhausted all avenues available on the NHS and to go home and make memories.

But money was not needed when the NHS agreed to fund the combination of drugs which had proved successful in trials for Dan.

And he is showing a positive response to treatment, having been told 95% of his cancer is gone.

Defying doctors and his diagnosis, a small part of the cancer is still visible on Dans neck but it has low activity.

Its still early days, but Dan certainly is on the right path now and its certainly a different path to the one he was on just a few months ago, said his dad, Mark.

As one of the final stages and vital to his recovery and to stay in remission once he gets to that stage, Dan is in need of a stem cell transplant. That process is due to start on May 27.

The NHS went on a world wide search for a matching donor. In such a short time, four stem cell donors have been found in Israel, however, the family needs to await confirmation on availability.

Dan had previously done the Ancestry test to discover more about his family history and roots, which showed him to be 1% connected to Israel.

Mark added: I am still concerned but I just feel that it is going to be OK, he is going to be fine.

After everything he has gone through, it just has to be fine, there is no other option really, it has to be fine,

Because there is still a little bit of cancer there, we are always going to be a little nervous, it is only natural.

The day he is finally in remission will be wonderful, absolutely wonderful and I cant wait.

Dan, a former pupil at Sir William Robertson Academy in Welbourn, was diagnosed with stage four Primary Mediastinal B Cell Lymphoma (PMBCL) in December 2022.

After several failed treatments, the family was told there were no more viable treatments available on the NHS and was told to take him home and make memories.

Instead, through research, Dan, who lives in Newark with his family, found a combination of drugs that had proved successful in trial, but not approved on the NHS.

His family set up a GoFundMe page with a 100,000 target and raised nearly half of their target amount in a short time before Dan was given the treatment for free on the NHS through compassionate use.

Dan had the first dose of treatment on January 26 and ever since, what once was a terminal diagnosis, has been improving and defying the rules of life and medicine.

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Newark teenager suffering from a rare form of cancer who is having treatment funded by the NHS matched with stem ... - LincsOnline

Global Regenerative Medicine Market to Touch Valuation of USD 472.95 Billion by 2032, at 25.86% CAGR: Astute … – GlobeNewswire

New Delhi, April 01, 2024 (GLOBE NEWSWIRE) -- According to the latest research by Astute Analytica, theglobal regenerative medicine market is projected to reach US$ 472.95 billion by 2032, up from US$ 59.68 billion in 2023, at a CAGR of 25.86% during the forecast period 20242032.

Regenerative medicine is rapidly gaining momentum worldwide. Cell therapies lie at the heart of this revolution, capturing a dominant share of the market. This trend is bolstered by numerous clinical trials, advancements in the field, and the potential for highly personalized treatment approaches. Currently, over 1,000 stem cell clinical trials are registered globally, demonstrating a strong commitment to exploring the therapeutic applications of cells. North America, with the U.S. at the helm, holds a leading position in the market. This is due to factors like a high volume of clinical studies, generous government support, and a regulatory system conducive to research and development. Moreover, insurance companies are increasingly considering regenerative therapies for coverage, improving accessibility for patients.

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While cell therapies spearhead the market, other promising areas are gaining traction. Tissue engineering is poised for significant growth, propelled by innovations in biomaterials and 3D bioprinting. Gene therapy is another exciting frontier around 30 gene therapies have been approved worldwide, and countless others remain in the development pipeline, demonstrating the immense potential of this field.

Regenerative Medicine Market Booms: Chronic Disease Focus Drives Billions in Investment

Chronic diseases like diabetes and heart conditions present massive global health challenges, and regenerative medicine is offering new hope. Treatments for these diseases account for over 30% of the market. Investments in this area are substantial, with venture capital financing surpassing $10 billion in 2021. CRISPR technology, a cornerstone of gene editing within regenerative medicine, was valued at over $2 billion in 2020. Regenerative treatments are also increasingly utilized for bone and joint disorders, making up about 25% of the market. Further, oncology treatments, particularly CAR-T cell therapies, are gaining ground with a market share of over 20%. The growth of biobanks, which store essential cell and tissue materials, is another positive indicator, with an increase of more than 50% between 2018 and 2022.

The regenerative medicine market landscape is shaped by influential companies such as Novartis, Vericel Corporation, Integra Lifesciences, and MiMedx. The intense level of innovation is evident over 10,000 patents related to the field were filed worldwide in 2021. The Asia-Pacific region, with favorable regulations in countries like Japan and South Korea, is emerging as an important hub for regenerative medicine.

Key Findings in Global Regenerative Medicine Market

Stem Cell Therapy Control More than 34% Share of Global Regenerative Medicine Market

Currently, stem cell therapy is one of the most popular regenerative medicines available in world. Wherein, allogeneic stem cell therapies such as Hematopoietic Stem Cell Transplantation (HSCT) and Mesenchymal Stem Cell (MSC) Therapies are a powerhouse, dominating the global market with a substantial 61.4% share. In line with this, several biotech companies are developing allogeneic cell therapies using gene-editing techniques like CRISPR. For example, Vertex Pharmaceuticals and CRISPR Therapeutics are developing an allogeneic stem cell therapy called VCTX210 for type 1 diabetes, currently in early clinical trials. This reflects the appeal of "off-the-shelf" treatments that don't rely on sourcing cells from the individual patient.

The regenerative medicine markets potential is further highlighted by the remarkable number of clinical trials over 3,000 focused on adult stem cells are registered with the WHO. This demonstrates the scientific community's unwavering dedication to exploring these therapies. The FDA's approval of numerous stem cell-based clinical trials adds to the optimism, suggesting a promising path toward innovative treatments for various conditions.

However, navigating the regulatory landscape is critical. In 2016, a concerning trend emerged with 351 US businesses operating 570 clinics offering stem cell treatments, often with unproven claims. This prompted the FDA to strengthen enforcement measures in 2017, emphasizing the importance of rigorous scientific validation and careful oversight. Ensuring patient safety and protecting the field's integrity are paramount. Both investors and healthcare providers must stay keenly aware of the research advancements and regulatory shifts shaping this dynamic market.

Recent Stem Cell Therapies Approved by FDA

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Oncology Treatment to Contribute Almost 50% Revenue Share of Global Regenerative Medicine Market

The alarming statistics on cancer paint a stark picture: an estimated 18.1 million new cases and 9.9 million deaths occurred globally in 2023. This burden falls disproportionately on lower- and medium-income countries, tragically affected by cancers linked to infections. The global economic impact is staggering, projected to reach $25.2 trillion over the next 30 years. The absence of adequate funding for cancer care services in many nations further underscores the urgency of addressing this widespread crisis. With demographic shifts, a staggering 28.4 million cancer cases are expected by 2040, representing a 47% increase from 2020 and resulting in an estimated 16.3 million deaths worldwide.

Regenerative medicine market offers a beacon of hope within this challenging landscape. Oncology is the most significant application area for these therapies, driven by innovations transforming cancer treatment. CAR T-cell therapies, which genetically reprogram a patient's T cells to fight their specific cancer, are revolutionizing care. Successes like the FDA-approved Abecma, Breyanzi, Kymriah, and Yescarta exemplify the potential of this treatment modality.

Stem cell transplants are another pillar of regenerative oncology. These transplants are critical for blood cancers like leukemia and lymphoma, offering the chance to rebuild a patient's blood and immune system after intensive chemotherapy. Additionally, oncolytic viruses like Imlygic, which target and destroy cancer cells while igniting an anti-tumor immune response, are a further avenue of exploration.

Tissue engineering plays a dual role in regenerative oncology. It provides invaluable models for drug testing and offers the potential to repair tissues damaged by cancer treatments. The development of tissue-engineered oral mucosa for oral cancer patients is a testament to this progress. Lastly, nanoparticle delivery systems hold promise for precisely targeting tumors, minimizing harm to healthy tissues. While still in the research phase, it's an area of intense investigation.

United States Stand Tall in Global Regenerative Medicine Market, Contribute More Than

The United States sits at the forefront of the regenerative medicine revolution, and the trajectory is clear. In 2023, the US regenerative medicine market was valued at $20 billion and is projected to grow at an impressive CAGR of 22.39% through 2032. This dominance is fueled by the escalating prevalence of chronic diseases, a strong demand for artificial organs, and a highly developed healthcare system. The U.S. has built a powerhouse of research and development in this field. It accounts for a staggering 60% of the global development pipeline for regenerative therapies. This commitment is further solidified by the NIH's substantial investments: over $2.5 billion in 2021 alone was directed towards the National Institute of Biomedical Imaging and Bioengineering, a key driver of tissue engineering and regenerative medicine research.

The US FDA has become a gateway for innovation, with notable approvals like Kymriah and Yescarta (CAR T-cell therapies), Luxturna (gene therapy for inherited blindness), and Zolgensma (gene therapy for spinal muscular atrophy) in North America regenerative medicine market. As of January 2023, a total of 27 regenerative medicine products boasts FDA approval, representing major milestones. Clinical studies in the U.S. are abundant, with over 1,200 ongoing trials in 2021, encompassing nearly half of such trials globally. This robust research focuses on stem cell therapies, gene therapies, tissue engineering, and biomaterials.

A flourishing industry, populated with startups and established companies, propels the U.S. market forward. In 2021, U.S.-based regenerative medicine companies garnered an unprecedented $23.1 billion in funding. Significant financing deals for Sana Biotechnology, ElevateBio, and Lyell Immunopharma further underscore the sector's strength.

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Global Regenerative Medicine Market to Touch Valuation of USD 472.95 Billion by 2032, at 25.86% CAGR: Astute ... - GlobeNewswire

Stem Cell Editing Repairs Severe Immunodeficiency – The Scientist

The B and T cells of the adaptive immune system recognize unique features on infectious microbes that enter the body. They accomplish this feat using B-cell and T-cell receptors, which take on various shapes to bind to different antigens on foreign invaders. Recombination activating gene 1 (RAG1) is central to this shapeshifting behavior.1 It shuffles the order of DNA sequences in the genes for these receptors, producing multiple versions of the immune receptors that can bind staggering combinations of antigens. However, some people carry mutations in RAG1 that prevent the enzyme from recombining the DNA sequences that code for these receptors. Without properly functioning receptors, B and T cells fail to develop, leading to severe combined immunodeficiency (SCID), a condition in which even the mildest of infections can prove lethal. In a study published in Science Translational Medicine, researchers developed an efficient method to repair RAG1 genes in immune cell progenitors called hematopoietic stem cells (HSC) taken from SCID patients, and revealed that they could restore immune function in mice.2

When you would like to correct the gene, you have to keep in mind that close to the gene, there are a lot of regulatory elements that are relevant for correct gene expression. -Maria Carmina Castiello, San Raffaele Scientific Institute

Maria Carmina Castiello and Anna Villa, two translational immunologists at the San Raffaele Scientific Institute, set out to overcome some of the challenges with editing the RAG1 gene that researchers previously faced. In the past, scientists have taken healthy, functional HSC and inserted them into SCID-model mice, but they often get destroyed by other types of immune cells that recognize the transplants as foreign.3 Normally, doctors use immunosuppressants like chemotherapy before transplantation to deplete immune cells, but this isnt an option for SCID patients. This disease can be associated with severe organ damage, so the critical conditions of the patients do not allow them to receive chemotherapy, Villa explained.

Castiello and her colleagues took a different approach, modifying a SCID patients own stem cells to express a functional RAG1 gene. While other research groups had successfully added RAG1 to patient HSC, they were unable to properly regulate expression of the gene, and therefore couldnt ensure that the stem cells were safe or would effectively replenish B and T cells.

Introducing the gene into the wrong site in the genome may have partly caused this shortcoming. When you would like to correct the gene, you have to keep in mind that close to the gene, there are a lot of regulatory elements that are relevant for correct gene expression, Castiello said.

Rather than adding a functional copy of RAG1, the researchers decided to modify the existing copy, ensuring that the regulatory networks remained intact. In fact, other researchers succeeded when they took a similar approach to edit RAG2.4

Before Castiello and her team could fix the gene, however, they had to choose their editing strategy. Some researchers use base editing, which modifies single letters in the DNA sequence to correct other genetic disorders of these stem cells, like sickle cell disease and -thalassemia.5

Continue reading below...

However, RAG-1mutations can occur at several different sites within the gene, so base editing wouldnt cover every type of mutation. Instead, the research team used the clustered regularly interspaced short palindromic repeats (CRISPR-Cas9) system to cut out a large section of the mutant gene, and then provided cells with the correct DNA sequence using a lentiviral delivery system. Since the correct sequence was nearly identical to the original gene, the cell could swap the sequences unassisted using homology-directed repair (HDR), a built-in DNA repair pathway that fixes double-strand DNA breaks using complementary DNA as a template.

Once Castiello and her colleagues swapped the HSCs old, mutated coding sequence for a fresh one, they had to test whether the gene produced a functional RAG1 protein. They inserted a backwards green fluorescent protein(gfp) gene flanked by sequences that RAG1 recognizes. Promisingly, they found that the edited RAG1 inverted gfp comparably to RAG1 in HSC from healthy donors, thereby switching it to an on state, resulting in a functional gfp gene.

They next had to check that these edited cells could restore immune function in the body. They transplanted these edited human cells into SCID-model mice and found that B and T cells spiked to levels similar to those seen in mice that received HSC from healthy donors.

Whats intriguing from the study is that we dont need to correct all the stem cells. If we manage to correct at least 10 percent of the stem cells, this is going to give us a therapeutic benefit, said Saravanabhavan Thangavel, a geneticist at the Institute of Stem Cell Research and Regenerative Medicine who was not involved with the work. However, he also mentioned, We need to track the HDR-edited cells long term. The researchers need to ensure that the modified cells persist in the bodies of people with SCID so that their newly gained immunity doesnt wane over time. If, by chance, the HDR-edited cells faded away, they may not have a therapeutic benefit, Thangavel added.

Down the line, the team aims to refine their protocol. We are trying to increase the editing efficiency that we achieve, Castiello said. She also wants to optimize delivery of the gene into the cells by comparing different methods. In this study they used lentiviruses to deliver the DNA template to the stem cells, but they plan to test other strategies like using lipid nanoparticle conduits that conceal the DNA template and fuse with the cell membrane to release the DNA into the cell.

The team will also have to test the safety of this gene editing strategy and find a way to scale up production of the edited stem cells, Castiello added. Then they should be able to test their edited cells in people with the hope of eventually treating the variety of conditions caused by RAG1defects. We are really committed to translating our strategy to the clinic, she said.

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Anna Villa and Maria Carmina Castiello are inventors with two patents involved with editing RAG genes.

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Stem Cell Editing Repairs Severe Immunodeficiency - The Scientist

UCF Researchers Develop Novel Therapy for Incurable Brain Cancer – UCF

College of Medicine researchers are developing a more effective way to treat glioblastoma an aggressive, incurable form of brain cancer. Patients currently live just 12 to 15 months after diagnosis despite surgery, radiation and chemotherapy.

New research led by Kiminobu Sugaya, a stem cell researcher and neuroscientist at UCFs Burnett School of Biomedical Sciences, found that targeting a drug resistant mechanism in cancer stem cells significantly enhanced the efficacy of traditional cancer therapies making them four times more effective against glioblastoma. Current FDA-approved drugs kill less than 25% of glioblastoma cancer stem cells (CSCs).

These cells are a subpopulation of cancer cells that are highly resistant to current therapies. Scientists theorize that cancer returns and spreads because CSCs remain in the body. Thats why they are exploring ways to kill them outright.

Cancer stem cells are bad stem cells that are programed to become a cancer, Sugaya says. They withstand cancer therapies, raise their ugly head, regrow and metastasize.

Sugayas team developed a new drug delivery system by creating a technology that destroys the RNA, or ribonucleic acid, that the stem cells use as a blueprint to produce proteins. This unique strategy inhibits the expression of embryonic stem cell genes that are pivotal in CSCs drug resistance. And because embryonic stem cell genes are not expressed in normal adult cells, this breakthrough approach reduces potential for side effects in healthy cells.

Jonhoi Smith is a doctoral student under Sugaya and the first author on their research paper published in the journal Genes. He said the treatment could increase life expectancy for glioblastoma patients.

This treatment could be a precious gift for glioblastoma patients. When I think about the loved ones Ive lost in my life my father, my grandmother I often wish I could have had more time with them, he says. The idea of offering the potential of a whole new life to people who are facing a death sentence in less than a year means a lot to me.

One of the significant challenges in treating glioblastoma is effectively delivering treatments to the brain. Thats because the brain is protected from external germs and substances by the blood-brain barrier, which can also prevent treatments from reaching brain tissues.

To overcome this obstacle, Sugayas therapy is based on exosomes, nano-sized particles with a lipid membrane that are naturally produced by cells. Exosomes function as cellular communicators, transporting proteins, lipids and genetic material between cells, thereby influencing a wide array of biological processes and functions. Their efficiency in carrying molecules across various parts of the body has inspired scientists to investigate exosomes as potential drug delivery vehicles.

Many current drug delivery systems, including viruses, may cause side effects, Sugaya explains. Were using the bodys natural delivery systems and have developed technologies to modify them to carry therapeutic molecules with targeted delivery to specific tissues.

Marvin Hausman is CEO of Exousia AI, the company that is funding the glioblastoma exosome preclinical research. He heard about Sugayas lab and says that when he visited the lab at UCFs Academic Health Sciences Campus in Lake Nona, he was inspired by its capacity for innovative discoveries.

I have thoroughly analyzed this exosome-based targeted drug delivery system many times, and the potential that this unique technology offers. Hausman says. We are embarking on a revolutionary new development in medicine.

Thanks to funding from Exousia AI, the research is advancing to mouse models carrying human glioblastoma, with preliminary results expected as early as this summer.

Sugaya has dedicated more than 40 years to neuroscience research focused on Alzheimers disease, with an emphasis on stem cells for the last 26 years. He moved to the U.S. after receiving his doctoral degree from the Science University of Tokyo in 1988. He joined UCF as a professor in 2004. His cancer research began in 2010 when he discovered stemness gene expressions, the self-renewing and differentiating property that allows stem cells to grow and spread, in CSCs. He is recognized as an expert in the field of exosome research and recently received Florida Innovation Funding from the State Department of Health for his studies.

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UCF Researchers Develop Novel Therapy for Incurable Brain Cancer - UCF

New Positive Data Presented on Briquilimab Conditioning in Patients with Fanconi Anemia – GlobeNewswire

REDWOOD CITY, Calif., March 15, 2024 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (Nasdaq: JSPR) (Jasper), a biotechnology company focused on development of briquilimab, a novel antibody therapy targeting c-Kit (CD117) to address mast cell driven diseases such as chronic spontaneous urticaria (CSU) and chronic inducible urticaria (CIndU), announced additional positive Phase 1b/2a data on briquilimab as a conditioning agent in the treatment of Fanconi Anemia (FA).

The data was presented at the 2024 Stanford Medicine Center for Definitive and Curative Medicine Symposium on March 13, 2024, in Palo Alto, California.

The ongoing investigator initiated Phase 1b/2a clinical trial is evaluating a conditioning regimen that includes intravenous briquilimab as a potential treatment for FA patients in bone marrow failure. Data from the study show that briquilimab infusion has a promising safety profile and appears to be well tolerated in patients with FA, with all six patients treated achieving full donor engraftment and full blood count recovery.

We continue to be encouraged by the results from Stanford Medicine's Phase 1b/2a study in Fanconi Anemia, which demonstrates the potential of briquilimab to serve as a key component of non-toxic conditioning regimens for stem cell transplant, said Edwin Tucker, Chief Medical Officer of Jasper. Wed like thank our collaborators at Stanford Medicine for their work evaluating briquilimab in this vulnerable patient population.

About Briquilimab

Briquilimab (formerly JSP191) is a targeted aglycosylated monoclonal antibody that blocks stem cell factor from binding to the cell-surface receptor c-Kit, also known as CD117, thereby inhibiting signaling through the receptor. This inhibition disrupts the critical survival signal, leading to the depletion of the mast cells via apoptosis which removes the underlying source of the inflammatory response in mast cell driven diseases such as chronic urticaria. Jasper is currently conducting clinical studies of briquilimab as a treatment in patients with CSU or with CIndU. Briquilimab is also currently in clinical studies as a treatment for patients with LR-MDS and as a conditioning agent for cell and gene therapies for rare diseases. To date, briquilimab has a demonstrated efficacy and safety profile in more than 145 dosed participants and healthy volunteers, with clinical outcomes as a conditioning agent in severe combined immunodeficiency (SCID), acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), FA, and sickle cell disease (SCD).

About Fanconi Anemia

Fanconi Anemia (FA) is a rare but serious blood disorder that prevents the bone marrow from making sufficient new red blood cells. The disorder can also cause the bone marrow to make abnormal blood cells. FA typically presents at birth or early in childhood between five and ten years of age. Ultimately, it can lead to serious complications, including bone marrow failure and severe aplastic anemia. Cancers such as acute AML and MDS are other possible complications. Treatment may include blood transfusions or medicine to create more red blood cells, but a hematopoietic stem cell transplant (HSCT) is currently the only cure.

About Phase 1/2 clinical trial (NCT04784052)

The Stanford sponsored, investigator initiated Phase 1/2 study is an open-label clinical trial evaluating briquilimab as a potential treatment for FA patients in bone marrow failure (BMF) requiring allogeneic transplant. Utilizing briquilimab, the regimen eliminates the need for busulfan chemotherapy or total body irradiation. Participants with FA with BMF receive allo-HCT with TCR+ T-cell/CD19+ B-cell depleted hematopoietic grafts from 10/10 unrelated, 9/10 unrelated or haploidentical family donors. A 0.6 mg/kg dose of briquilimab is administered in combination with standard FA dosing of anti-thymocyte globulin (ATG), cyclophosphamide, fludarabine, and rituximab as lymphodepletion. The primary outcomes include safety, efficacy, and engraftment success.

About Jasper

Jasper is a clinical-stage biotechnology company developing briquilimab, a monoclonal antibody targeting c-Kit (CD117) as a therapeutic for chronic mast and stem cell diseases such as chronic urticaria and lower to intermediate risk MDS and as a conditioning agent for stem cell transplants for rare diseases such as SCD, FA and SCID. To date, briquilimab has a demonstrated efficacy and safety profile in more than 145 dosed participants and healthy volunteers, with clinical outcomes as a conditioning agent in SCID, acute myeloid leukemia, MDS, FA, and SCD. For more information, please visit us atwww.jaspertherapeutics.com.

Forward-Looking Statements

Certain statements included in this press release that are not historical facts are forward-looking statements for purposes of the safe harbor provisions under the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements are sometimes accompanied by words such as believe, may, will, estimate, continue, anticipate, intend, expect, should, would, plan, predict, potential, seem, seek, future, outlook and similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These forward-looking statements include, but are not limited to, statements regarding briquilimabs potential, including its potential as a conditioning agent in the treatment of FA and FA patients in bone marrow failure and its safety profile, its potential to serve as a key component of non-toxic conditioning regimens for stem cell transplant and its potential to address mast cell driven diseases such as CSU and CIndU. These statements are based on various assumptions, whether or not identified in this press release, and on the current expectations of Jasper and are not predictions of actual performance. These forward-looking statements are provided for illustrative purposes only and are not intended to serve as, and must not be relied on by an investor as, a guarantee, an assurance, a prediction or a definitive statement of fact or probability. Many actual events and circumstances are beyond the control of Jasper. These forward-looking statements are subject to a number of risks and uncertainties, including general economic, political and business conditions; the risk that the potential product candidates that Jasper develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all; the risk that clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release; the risk that Jasper will be unable to successfully market or gain market acceptance of its product candidates; the risk that prior study results may not be replicated; the risk that Jaspers product candidates may not be beneficial to patients or successfully commercialized; patients willingness to try new therapies and the willingness of physicians to prescribe these therapies; the effects of competition on Jaspers business; the risk that third parties on which Jasper depends for laboratory, clinical development, manufacturing and other critical services will fail to perform satisfactorily; the risk that Jaspers business, operations, clinical development plans and timelines, and supply chain could be adversely affected by the effects of health epidemics; the risk that Jasper will be unable to obtain and maintain sufficient intellectual property protection for its investigational products or will infringe the intellectual property protection of others; and other risks and uncertainties indicated from time to time in Jaspers filings with the SEC, including its Annual Report on Form 10-K for the year ended December 31, 2023 and subsequent Quarterly Reports on Form 10-Q. If any of these risks materialize or Jaspers assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements. While Jasper may elect to update these forward-looking statements at some point in the future, Jasper specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Jaspers assessments of any date subsequent to the date of this press release. Accordingly, undue reliance should not be placed upon the forward-looking statements.

Contacts:

Joyce Allaire (investors) LifeSci Advisors 617-435-6602 jallaire@lifesciadvisors.com

Alex Gray (investors) Jasper Therapeutics 650-549-1454 agray@jaspertherapeutics.com

Lauren Walker (media) Real Chemistry 646-564-2156 lbarbiero@realchemistry.com

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New Positive Data Presented on Briquilimab Conditioning in Patients with Fanconi Anemia - GlobeNewswire

BioCardia and StemCardia Announce Biotherapeutic Delivery Partnership – Diagnostic and Interventional Cardiology

March 15, 2024 BioCardia, Inc., a biotechnology company focused on advancing late-stage cell therapy interventions for cardiovascular disorders, andStemCardia, Inc., a biotechnology company focused on cell and gene therapy to re-muscularize the failing heart, today announced a long-term partnership to advance StemCardias investigational pluripotent stem cell product candidate for the treatment of heart failure.

Under the partnership, BioCardia is the exclusive biotherapeutic delivery partner for StemCardias cell therapy candidate through studies expected to result in FDA approval of an investigational new drug application (IND) and the anticipated Phase I/II clinical development to follow.

BioCardia has established safe and minimally invasive delivery of cellular medicines directly into the heart, said Chuck Murry, MD, PhD, StemCardias Founder and CEO. Having worked with BioCardia to successfully deliver our bona fide cardiac muscle cells in large animal models of heart failure, we are excited for this partnership to accelerate clinical development and broaden future commercial access to an off-the-shelf heart regeneration treatment.

StemCardias team encompasses recognized leaders in the field of cardiac regenerative medicine who are pursuing an elegant strategy to repair the failing heart. We look forward to supporting their efforts with our experienced team and proven, proprietary Helix biotherapeutic delivery system, said BioCardia CEO Peter Altman, PhD. This partnership is expected to enhance future treatment options for millions of people suffering from heart failure, offset the costs of biotherapeutic delivery development for our own programs, and provide our investors with meaningful revenue sharing should our efforts together contribute to StemCardias successful therapeutic development.

For more information:www.biocardia.com

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BioCardia and StemCardia Announce Biotherapeutic Delivery Partnership - Diagnostic and Interventional Cardiology

Striving to Support Gender Equality in Health Care – City of Hope

Nadia Carlesso, M.D., Ph.D.

As a member of the newest class of 24 women selected nationwide to participate in the Carol Emmott Fellowship, Nadia Carlesso, M.D., Ph.D., wants to take this unique and competitive opportunity to move the needle in advancing diversity, equity and inclusion (DEI) on both sides of the research bench.

The Carol Emmott Foundation is a national nonprofit organization that is dedicated to achieving fully inclusive gender equality in health care leadership and governance. The fellowship includes a 14-month experience in which fellows can receive mentorship for their personal projects that support gender equity in health care.

Based on the Duarte, California, campus, Carlesso joined City of Hope in 2016. She is currently professor and chair of the Department of Stem Cell Biology and Regenerative Medicine.

As a scientist in the stem cell field and as chair of the Department of Stem Cell Biology and Regenerative Medicine, I feel the responsibility and commitment to contribute, in the measure I can, to decreasing health disparities in the areas of innovative cell and gene therapies, Carlesso said.

Carlesso said health disparities exist in some part due to the prohibitive costs associated with getting treatment and participating in clinical trials.

We are living in exciting times in terms of the progress and availability of cell and gene therapies potentially curative therapies, she said. But these products have a high cost that can unfortunately widen the care gap. Access to innovative therapies is expensive for many complex reasons. As a community, scientists, technologists and health care professionals must come together to be part of the solution.

There's no easy answer, and not a problem that will be resolved tomorrow, but the conversation has started within scientific circles, and Carlesso wants to further that dialogue and parlay it into actions that can truly make progress.

One step in the right direction is to increase diversity within the scientific community in all aspects, in the name of advancing clinical knowledge.

City of Hope is a diverse and inclusive organization, and I would like to leverage that commitment, Carlesso said. The Emmott Fellowship has given me the opportunity to focus more intently on growing the diversity within our scientific environment.

Innovative biomedical research flourishes when people with different perspectives, experience and skills are empowered to explore new ideas and to work collaboratively and inclusively, she continued. Recruiting, training, retaining and nurturing a workforce representing all dimensions of diversity is critical for the development and implementation of leading-edge therapies that can reach underserved populations. These communities have not had their voices heard: Targeting these communities to be active participants of this specialized workforce and future leaders in this sector is key to decrease barriers to health equity.

Another key step toward this goal is to increase awareness among these communities that their voices are important.

There is not sufficient knowledge out there that they are needed, Carlesso explained. There are cultural biases that have to be overcome. If patients can see themselves in our medical and scientific staff, they will feel more represented and more comfortable that their best interests are at heart.

As an Emmott Fellow, Carlesso said she has identified three areas that she wishes to address in her research project.

The first part is for me to get the numbers, to look at the current diversity within our clinical trials, within our GMP teams, clinical trial coordinators, research nurses, clinicians and basic scientists, she said. At any given time, we have more than 30 investigational new drug applications and 300 clinical trials going on. I want to take advantage of this ideal setting and generate a complete database of the progress weve made, the challenges we still face, and where the bottlenecks are that are compromising diversity and inclusion at all levels.

Establishing a discourse, conversations among peers, is essential to stimulate a reaction toward diversity in research that will benefit all of us, Carlesso said.

A cohesive stem cell research community is critical not only to generate discoveries but also to bring awareness in health disparities and generate synergistic efforts to tackle this problem, she said.

Carlesso maintained that we can empower patients by empowering a diverse workforce, and one way we can do that is to recruit individuals from diverse backgrounds into careers in science, technology, engineering and math (STEM).

A powerful way to give a voice to underserved communities and people of underrepresented backgrounds, and to mitigate health disparities, is to train a workforce that represents them, she said. If underserved families start to have kids going to high school and college doing internships in science and contemplating a carrier in nursing, biomanufacturing, research, then they will have more information, more access and will be less afraid to ask and to pursue more advanced therapeutic solutions or participate to clinical trials.

City of Hope is committed to training the next generation of leaders in stem cells, gene therapy and regenerative medicine, and in fostering an inclusive and safe environment that embraces diversity. Our laboratories host interns every year from the California Institute for Regenerative Medicine bridge program, for example, and we recruit from all communities for our Eugene and Ruth Roberts Summer Student Academy. We also partner with California State Universities in our area and local high schools near the Duarte campus to grow the interest in STEM careers.

As an organization, we need to know that our research has great potential and is helping to close the care gap between socially economically disadvantaged groups and others, Carlesso said. Knowing where we are now is important, so we can define where we are going and how we are going to get there.

I am very excited and thankful to have been chosen for the Emmott Fellowship and the opportunities it represents, she said. I am thrilled and look forward to what I can learn, accomplish and change for the better in my work.

Learn more about the Carol Emmott Fellowship, Carlesso and other members of the Class of 2024, here.

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Striving to Support Gender Equality in Health Care - City of Hope

Transitioning from traditional surgical methods to the innovative use of stem cells – pharmaphorum

Patient lives are being transformed by a transitioning from traditional surgical methods to the innovative use of stem cells.

In todays podcast, host Nicole Raleigh welcomes Dr Jeffrey Gross, founder of ReCELLebrate, neurological surgeon, and expert in the field of regenerative medicine, to discuss this development. Their conversation delves into the recent appetite for the use of stem cells for longevity and explores the health span notion that is being ever more frequently spoken of.

But, essentially, regenerative medicine deals with the functional restoration of specific tissue and/or organ of patients, patients who might be suffering from severe injuries or chronic disease conditions, and who are in a state where the bodys own regenerative responses do not suffice and that is where stem cells, endorsed with indefinite cell division potential and the ability to transdifferentiate into other types of cells, are emerging as a frontline regenerative medicine source.

Yet, when it come to health and vitality, and to epigenetics and prolonged health, scepticism remains and Dr Gross attempts to demystify the landscape for listeners on that, too, including especially the most regenerative source of stem cells: the placenta.

You can listen to episode 121a of thepharmaphorum podcastin the player below, download the episode to your computer, or find it - and subscribe to the rest of the series - iniTunes,Spotify,acast,Stitcher,andPodbean.

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Transitioning from traditional surgical methods to the innovative use of stem cells - pharmaphorum