List of Stem Cells Companies with Phase 2 Active Clinical Trial - 53
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7 Hills Pharma Houston, Texas, United States | 7 Hills Pharma is a clinical-stage immunotherapy company developing a platform of novel, oral small molecules for the treatment of cancer and prevention of infectious diseases. Our compounds are first-in-concept allosteric integrin activators that leverage a unique mechanism of action to stabilize the cell-cell interactions required to mount an effective immune response. Our lead clinical candidate, 7HP349, is the only reported systemically safe immune stimulant that can activate both cellular and humoral immunity. |
Aegle Therapeutics Woburn, Massachusetts, United States | Aegle Therapeutics Corp. is a clinical-stage biotechnology company developing novel extracellular vesicle (“EV”) therapies to address rare and serious diseases and disorders with significant unmet medical needs. Aegle’s proprietary platform technology safely isolates native extracellular vesicles (“EVs”) from stem cells. These EVs carry complex assemblies of biologic molecules such as proteins and nucleic acids that can induce a wide variety of effects in recipient cells, including the promotion of regenerative healing, while reducing inflammation and modulating the immune system. Aegle’s EV therapy has the potential to treat a broad range of indications in multiple therapeutic areas, including dermatology, immunology-based diseases, protein deficient disorders and others. |
Affimed N.V. Heidelberg, Germany | At Affimed, we are committed to improving outcomes for patients with cancer. Our scientific team has been devoted to unlocking the power of the innate immune system for nearly two decades to provide a unique approach to fighting cancer. With our unparalleled expertise in innate immunity and innate cell engager-based medicines, we aim to overcome the limitations and challenges faced by current immuno-oncology (I-O) therapies. |
Ageless Biotech Las Vegas | Ageless Biotech (ABT) is in the development of discardable, minimally manipulated umbilical tissue based regenerative medicine biologics to address all forms of Osteoarthritis via intra-articular procedures in cooperation with leading scientists and medical professionals. Our ambition is to become a leading global biopharmaceutical company, focused on the discovery, development and commercialization of innovative medicines that will improve people's lives. We believe that the formation of life itself is contained within after birth tissues. ABT believes that it is morally, religiously and politically acceptable to use afterbirth materials that would be considered discardable in virtually every country. Furthermore, we believe this to be the right course to addressing problems that a vast majority of the population will face at some point during their lifespan. |
AVM Biotechnology Seattle, Washington, United States | AVM is a clinical-stage biotech company located in Seattle WA led by Dr. Theresa Deisher, a biotech veteran with a productive history including 47 patents and 4 discoveries in clinical trials. The company's lead drug AVM0703, an innovative formulation of dexamethasone, when given at suprapharmacologic doses has a novel mechanism of action to mobilize the body's own natural supercharged immune cells. AVM0703 triggers the production and release of endogenous gamma delta+ Natural Killer T cells and could be the first choice for no-option cancer, autoimmunity, and infectious disease. AVM Biotechnology has a Scientific Advisory Board including well-respected leaders in cancer and immunology. The company is committed to developing products that improve outcomes without additional suffering because side effects from treatments should never be worse than the diseases themselves. |
BioGenCell Netanya, Israel | BioGenCell was founded with the vision of developing safe, effective, and long-lasting effective treatment for incurable, blood vessel diseases that will be both accessible and patient friendly. Our disruptive TRACT technology platform provides a comprehensive approach for rapid manufacturing and commercialization of cell-therapies. |
BioRestorative Therapies Melville, New York | BioRestorative Therapies, Inc. (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders: • Disc/Spine Program (brtxDISC™): Our lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a person’s own) cultured mesenchymal stem cells collected from the patient’s bone marrow. We intend that the product will be used for the non-surgical treatment of protruding and bulging lumbar discs in patients suffering from chronic lumbar disc disease. The BRTX-100 production process involves collecting a patient’s bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure, BRTX-100 is to be injected by a physician into the patient’s damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. We have received clearance from the Food and Drug Administration to commence a Phase 2 clinical trial using BRTX-100 to treat chronic lower back pain due to degenerative disc disease related to protruding/bulging discs. • Metabolic Program (ThermoStem®): We are developing a cell-based therapy to target obesity and metabolic disorders using brown adipose (fat) derived stem cells to generate brown adipose tissue (“BAT”). BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in the body may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes. |
BlueSphere Bio Pittsburgh, Pennsylvania, United States | BlueSphere Bio was founded to unlock the potential of personalized T cell therapy for the treatment of cancer by harnessing the unique specificity of each patient’s own T cells for the particular molecular fingerprint of each patient’s own cancer through our novel TCXpress technology. Our proprietary TCXpress platform is an elegant, rapid approach that exploits the patient’s own immune defenses to attack their cancer specifically, and is designed to leave normal tissues untouched, thereby reducing harmful side effects. The platform and implementation allows us to deliver rapid, personalized T cell treatments for a variety of cancer indications. |
BRIM Biotechnology Taipei City, Taiwan | BRIM is a clinical-stage company developing novel peptide treatments for hard-to-treat diseases. BRIM’s first-in-class assets are based on its innovative stem cell regenerative PDSP technology platform, which has the potential to be effective in multiple therapy areas and indications. |
Carina Biotech Mawson Lakes, South Australia | Carina Biotech An Australian clinical stage immunotherapy company established to research and develop chimeric antigen receptor T cell (CAR-T) therapies to treat solid cancers. Carina are working towards producing broad-spectrum CAR-T therapies that can be used to treat multiple solid cancers yet are patient-specific and result in little, if any, off-cancer damage. Using its proprietary platforms, Carina is also developing technologies to improve access to, and infiltration of, solid cancers, and to enhance CAR-T cell manufacturing. We are working to… 1. Expand the clinical indications for T cell therapies 2. Improve the commercial viability of T cell therapies by developing supporting technologies that make T cell therapies more effective or economically viable Headquartered in Adelaide, South Australia, Carina has strong T cell R&D capability across a network of research providers and collaborators including leading scientists at the UniSA, the Women's & Children's Hospital in Adelaide, the University of Adelaide, the Royal Prince Alfred Hospital in NSW and the Seattle Children's Hospital in the United States. |
CartiHeal Kefar Sava, Israel | CartiHeal, a privately-held medical device company with headquarters in Israel, develops proprietary implants for the treatment of cartilage and osteochondral defects in traumatic and osteoarthritic joints. CartiHeal’s cell-free, off-the-shelf implant is CE marked for use in cartilage and osteochondral defects. Agili-C has been implanted in over 400 patients with cartilage lesions in the knee, ankle and great toe in a series of trials conducted in leading centers in Europe and Israel. In these trials, the implant was used to treat a broad spectrum of cartilage lesions, from single focal lesions to multiple and large defects in patients suffering from osteoarthritis. In the United States the Agili-C implant is not available for sale - it is an investigational device limited for use in the IDE study. |
Cellestia Biotech Basel, Basel-Stadt, Switzerland | Cellestia Biotech is pioneering innovative therapeutics based on novel modes of action to treat autoimmune diseases and multi-drug resistant cancers. Our clinical stage drug candidate, CB-103, has demonstrated both safety and biological activity in humans. Along with ongoing Phase 2 IITs for multi-drug resistant cancers, CB-103 is also poised to enter a Proof-of-Concept clinical study for graft-versus-host disease (GvHD) |
CellOrigin Biotech Hangzhou, Zhejiang | CellOrigin Biotech has a long term focus on iPSC-derived innate immune cells and its applications in new cancer immune cells. Dr. Jin Zhang, the scientific co-founder of CellOrigin used to be trained as a research fellow at the Boston Children’s Hospital and Harvard Medical School. His team worked closely with clinicians at the First Affiliated Hospital of Zhejiang University and for the first time reported the induced pluripotent stem cell or iPSC-derived CAR-macrophages (CAR-iMac), and its applications in cancer immunotherapies. As for now, CellOrigin Biotech holds its proprietary technologies for iPSC-derived CAR-Macrophage. With this platform, they are collaborating with research groups in genome engineering and synthetic biology at Harvard and MIT to fully unleash the potential of iPSC-derived immune cells, which are highly editable, expandable and clonal. Eventually, they would like to achieve a goal of bring more effective, universal and safe immune cell products to cancer patients, especially for those with solid tumors. The investigator initiated trials has been initiated at the First Hospital of Zhejiang University. The core proprietary technology platform and the core patents including the engineered macrophages from pluripotent stem cells has been authorized and is in the process of entering the US, Australia, South Africa, etc. |
Celltex Therapeutics Houston, Texas, United States | Celltex Therapeutics Corporation is a Houston, Texas-based biotechnology company focused on spearheading breakthroughs in regenerative medicine using adult, autologous Mesenchymal Stem Cells (MSCs). |
Creative Medical Technology Holdings Inc. Phoenix, Arizona, United States | Creative Medical Technology Holdings, Inc., formerly Jolley Marketing, Inc., is a clinical-stage company. The Company conducts its business operations primarily through its subsidiary, Creative Medical Technologies, Inc. (CMT). The Company is engaged in stem cell research and applications for use to treat male and female sexual dysfunction, infertility, miscarriages and related issues. CMT focuses on completing the testing of its erectile dysfunction (ED) treatment and, if warranted, marketing treatment kits under the name Caverstem to physicians for use with their patients suffering from ED. CMT also intends to test and, if warranted, market licensed stem cell products under its infertility technology license. Procedures for use of the Company's ED stem cell treatment consist of a one-hour out-patient visit in a physician's office. The physician would harvest a patient's bone marrow from the hip using local anesthetic and separate the stem cells using a cell separator. |
Cynata Therapeutics Carlton, Victoria, Australia | Cynata Therapeutics Limited (ASX: CYP) is an Australian clinical-stage stem cell and regenerative medicine company focused on the development of therapies based on Cymerus™, a proprietary therapeutic stem cell platform technology. Cymerus™ overcomes the challenges of other production methods by using induced pluripotent stem cells (iPSCs) and a precursor cell known as mesenchymoangioblast (MCA) to achieve economic manufacture of cell therapy products, including mesenchymal stem cells (MSCs), at commercial scale without the limitation of multiple donors. Cynata’s lead product candidate CYP-001 met all clinical endpoints and demonstrated positive safety and efficacy data for the treatment of steroid-resistant acute graft-versus-host disease (GvHD) in a Phase 1 trial. Clinical trials of Cymerus MSC products in osteoarthritis (Phase 3) and in severe complications arising from COVID-19 (Phase 2) are currently ongoing. Planning is also underway for further clinical trials of Cymerus MSC products in GvHD (through licensee Fujifilm), critical limb ischemia, idiopathic pulmonary fibrosis, renal transplantation, and diabetic foot ulcers. In addition, Cynata has demonstrated utility of its Cymerus MSC technology in preclinical models of numerous diseases, including the clinical targets mentioned above, as well as asthma, heart attack, sepsis, acute respiratory distress syndrome (ARDS) and cytokine release syndrome. |
Editas Medicine Cambridge, Massachusetts, United States of America | At Editas Medicine, we are pioneering the possible. Our mission and commitment is to harness the power and potential of CRISPR gene editing to develop a robust pipeline of medicines for people living with serious diseases around the world. Our goal is to discover, develop, manufacture, and commercialize transformative, durable genomic medicines for many diseases. |
Ensoma Boston, Massachusetts, US | |
EpiBone 760 Parkside Avenue, Brooklyn, New York 11226, US | EpiBone is a revolutionary bone reconstruction company that allows patients to "grow their own bone". EpiBone's pioneering technology utilizes a scan of the patient's bone defect and the patient's own stem cells to construct and cultivate a defect-specific autologous-like bone graft. EpiBone is strategically positioned to provide a superior bone graft that will provide exact defect repair, a simplified surgical procedure, improved bone formation and regeneration, and shorter recovery times, without the complications of foreign body implantation, to the over 900,000 patients who undergo bone-related surgeries each year. |
Evotec Hamburg, DE | Evotec is a biotechnology company that is committed to advancing drug discovery and development. Through flexible business models, we collaborate with pharmaceutical companies, biotechs, foundations, and government agencies on a substantial scale. We believe that by working together, we can make a difference in the lives of patients. Our Pipeline Co-Creation model represents our adaptable, multi-modality, fully integrated end-to-end approach to drive collaborations and services across all phases of drug discovery and development – from discovery of novel targets to achieving Proof of Concept in the clinic and into commercial manufacturing. We aspire to contribute data-driven disease understanding and early disease relevance in humans to bring the probability of success up. We select the right modality, which is then propelled forward on our platforms by our passionate people striving for the fastest and most effective ways towards patient impact. This is enabled by convergence of human ingenuity with data and AI. We also offer specific solutions, products, and CRO/CDMO-like services, always in support of the Biopharma R&D innovator. Our more than 4,000 scientists work closely with numerous partners concurrently, delivering fully integrated research and development portfolios or individual projects with the highest quality standards and efficiency, coupled with great science, passion, engagement, and communication. |
ExCellThera Montreal, Quebec, Canada | ExCellThera is an advanced clinical-stage company specializing in cell expansion and engineering, providing molecules and bioengineering solutions to expand stem cells. |
Genenta Science Milan, Italy | Genenta (Nasdaq: GNTA) is a clinical stage immuno-oncology company developing a cell-based platform harnessing the power of hematopoietic stem cells to provide durable and safe treatments for solid tumors. Our platform is not tumor type nor target antigen restricted and provides sustained targeted expression of therapeutic payload(s) inside the tumor micro environment. Genenta’s lead product candidate, Temferon™, precisely targets the delivery of interferon-alpha to the tumor micro-environment, minimizing systemic toxicity while breaking tumor-induced immune tolerance. Our treatments are designed as one-time monotherapies, but with the additional potential, when used in combination, to significantly enhance the efficacy of other approved therapeutics. |
Glycostem Oss, The Netherlands | Glycostem Therapeutics is a Dutch biotech company established in 2007. Glycostem Therapeutics has developed the world's first GMP compliant NK cell platform that is ready for industrial scale-up. |
Greenstone Biosciences Palo Alto, California, United States | Discover the limitless potential of human induced pluripotent stem cells (iPSCs) for groundbreaking research at Greenstone, a leading Bay Area biotech company. We combine cutting-edge technology with AI, computational biology, and pharmacogenomics to accelerate drug discovery and development, transforming the future of therapeutics. Our vision is to transform drug discovery from a slow, sequential, and high-failure process into a rapid, integrated, and novel computational platform. By harnessing the power of iPSCs and our advanced technologies, we revolutionize the traditional drug discovery paradigm, paving the way for a faster, more efficient approach. With our curated and diverse collection of iPSCs, sourced from both healthy individuals and those affected by various diseases, we offer a comprehensive platform for disease modeling, uncovering novel mechanisms, and advancing drug discovery. Our high-quality iPSCs provide researchers with patient-specific and disease-specific cells, enabling the development of personalized treatment strategies. At Greenstone, collaboration and innovation are at the core of our values. We proudly offer our iPSCs to academic investigators free of cost, fostering an environment of shared knowledge and progress. Together, we can unlock new insights, accelerate drug discovery, and shape the future of personalized medicine. Join us on the frontier of biomedical research and tap into the immense potential of iPSCs. Together, let's transform drug discovery into a rapid, integrated, and computational platform that propels us towards groundbreaking therapeutics. #Biotech #iPSCs #DrugDiscovery #BiomedicalResearch #PersonalizedMedicine #Innovation |
Immunis Irvine, California, United States | Immunis Inc. is a private biotechnology company focused on the development of treatments for age-related and disease-related immune deficits. Immunis has developed a novel method to grow human cells for clinical use, and direct them to become highly pure populations of defined cells that secrete factors which benefit immune system development, modulation, and health. With age, every person experiences a reduction of factors which benefit immune system development, modulation, and health; our technology allows replacement of those factors. Our investigational product is all natural, all human, and represents a perfectly balanced set of immune modulators in naturally occurring relevant physiological concentrations. Immunis' first clinical trial is a Phase 1/2a study of the effectiveness of STEM in treating muscle atrophy, which every person experiences with age. |
Immunophotonics, Inc. St. Louis MO | Immunophotonics is a privately owned clinical stage biotech that has discovered a novel and simple approach to achieve patient-specific anticancer immunity. The company is currently developing its first asset, the immune-modulating proprietary carbohydrate polymer IP-001 for the treatment of solid tumor cancers. IP-001 has unique physio-chemical and immunological properties that allows it to ignite a systemically active cancer immunotherapy from a routine tumor ablation; a minimal invasive intervention that destroys tumors and in doing so liberates tumor antigens. IP-001 enhances interactions of tumor antigens with the immune system, improves the motility of tumor infiltrating T cells, promotes their surveillance of tumor tissue and can generate long-term protective immunity. |
IN8bio New York, United States | IN8bio, Inc. is a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of gamma-delta T cell therapies for the treatment of cancer. Gamma-delta T cells are naturally occurring immune cells that embody properties of both the innate and adaptive immune systems, and can intrinsically differentiate between healthy and diseased tissue. IN8bio develops ex vivo-expanded and activated gamma-delta T cells based upon its deep expertise in gamma-delta T cell biology, proprietary genetic engineering, and cell-type specific manufacturing capabilities, which we refer to collectively as our DeltEx platform. IN8bio’s platform employs allogeneic, autologous, and genetically modified approaches to develop novel cell therapies, which are designed to effectively identify and eradicate tumor cells. IN8bio is currently the most clinically advanced gamma-delta T cell company and the first company to bring genetically modified gamma-delta T cells into the clinic. For more information about the Company and its programs, visit www.IN8bio.com. |
Jasper Therapeutics Jasper Therapeutics, 2600 Bridge Pkwy, Redwood City, CA 94065, United States | Jasper Therapeutics is a biotechnology company focused on targeting mast cells and stem cells to treat chronic diseases. Their mission is to make a significant difference in the lives of patients suffering from these conditions. |
KALA BIO Arlington, Massachusetts, United States | Weaving along Hawaii's Na Pali Coast is the famous, breathtaking Kalalau trail. This trail is not only notable for its strenuous path and terrain that is difficult to traverse, but also known for its unsurpassed beauty. Kalalau was early inspiration for us, today representing our innovative spirit and deep commitment to advancing the treatment of eye diseases. KALA BIO is a clinical-stage biopharmaceutical company dedicated to the research and development of innovative therapies for rare and severe diseases of the eye. KALA BIO’s biologics-based investigational therapies utilize KALA BIO’s proprietary mesenchymal stem cell secretome (MSC-S) platform. KALA BIO’s lead product candidate, KPI-012, is a human MSC-S, which contains numerous human-derived biofactors, such as growth factors, protease inhibitors, matrix proteins and neurotrophic factors that can potentially correct the impaired corneal healing that is an underlying etiology of multiple severe ocular diseases. KPI-012 is currently in clinical development for the treatment of persistent corneal epithelial defect (PCED), a rare disease of impaired corneal healing, for which it has received Orphan Drug and Fast Track designations from the U.S. Food and Drug Administration. KALA BIO is also targeting the potential development of KPI-012 for the treatment of Limbal Stem Cell Deficiency and other rare corneal diseases that threaten vision and has initiated preclinical studies to evaluate the potential utility of its MSC-S platform for retinal degenerative diseases, such as Retinitis Pigmentosa and Stargardt Disease. For more information on KALA BIO, please visit www.kalarx.com. |
Lisata Therapeutics Basking Ridge, New Jersey, United States | (Formally Caladrius Biosciences; NASDAQ: CLBS) Lisata Inc., (LSTA) a clinical-stage biopharmaceutical company, focuses on developing and commercializing cellular therapies to reverse disease and/or promote the regeneration of damaged tissue. Its product candidates include HONEDRA, a recipient of SAKIGAKE designation that is in Phase II clinical trial for the treatment of critical limb ischemia; OLOGO, a regenerative medicine advanced therapy for treating no-option refractory disabling angina; CLBS16 that is in Phase IIb clinical trial for the treatment of coronary microvascular dysfunction; and CLBS201, a CD34+ cell therapy for the treatment of pre-dialysis patients with chronic kidney disease. |
Longeveron Inc Miami, Florida, United States | We are a clinical-stage biotechnology company developing cellular therapies for aging-related and life-threatening conditions. Our lead investigational product is Lomecel-B™, which is derived from culture-expanded medicinal signaling cells (MSCs) that are sourced from bone marrow of young healthy adult donors. We believe that by using the same cells that promote tissue repair, organ maintenance, and immune system function, we can develop safe and effective therapies for some of the most difficult diseases and conditions associated with aging. We are currently conducting Phase 1 and 2 clinical trials in the following indications: Aging Frailty, Alzheimer’s disease, the Metabolic Syndrome, Acute Respiratory Distress Syndrome (ARDS), and hypoplastic left heart syndrome (HLHS), which is the current exception to our focus on aging. Our mission is to advance Lomecel-B™ and other cell-based product candidates into pivotal Phase 3 trials, with the goal of achieving regulatory approvals, subsequent commercialization and broad use by the healthcare community. Our philosophy is that healthy aging can be improved through regenerative medicine approaches. Life expectancy has substantially increased over the past century as a result of medical and public health advancements. However, this increase in longevity has not been paralleled by the number of years a person is expected to live in relatively good health, free of chronic disease and disabilities of aging – a period known as healthspan. As we age, we experience a profound decline, in both number and function, in our own MSCs, a decrease in immune system function, decline in blood vessel functioning, chronic inflammation and other issues. Our clinical data suggest that Lomecel-B™ addresses these conditions through multiple mechanisms of action, or MOAs, that simultaneously target key aging-related processes. |
MEDIPOST Seongnam-si, South Korea | MEDIPOST is founded in 2000, and today MEDIPOST is leading the global stem cell therapeutics field with the world’s first regulatory-approved allogeneic human Umbilical Cord Blood-derived Mesenchymal Stem Cell(hUCB-MSC) product named CARTISTEM® for patients with knee Osteoarthritis(OA), launched in Korean market in 2012. MEDIPOST operates the largest private cord blood bank “CELLTREE®” in Korea with over 255,000 units of private cord blood units under storage. Each year, over 20,000 private cord blood units are collected and stored at CELLTREE®. MEDIPOST’s research and development is focused on novel off-the-shelf, allogeneic cell therapeutics using human Umbilical Cord Blood-derived Mesenchymal Stromal Cells (hUCB-MSCs) with clinical-stage assets in the disease areas of osteoarthritis (OA), broncho-pulmonary dysplasia (BPD) and Alzheimer’s disease (AD). MEDIPOST’s flagship product, CARTISTEM® (allogeneic Umbilical Cord Blood-derived MSCs + hyaluronic acid hydrogel composite) for knee osteoarthritis was approved in 2012 by Korea’s regulatory agency Ministry of Food and Drug Safety (MFDS) with Biologics License Application (BLA) label “Treatment of knee articular cartilage defects in patients with osteoarthritis (ICRS grade IV) as a result of degenerative disease or repeated trauma (without age limit)” PNEUMOSTEM® (allogeneic Umbilical Cord Blood-derived MSCs) for the prevention of Bronchopulmonary Dysplasia (BPD) in premature infants, completed the first-in-human Phase I safety trial in Korea and the randomized, placebo-controlled Phase II clinical trial is ongoing in Korea. Phase I/II clinical trial in the U.S. has been completed confirming safety with positive efficacy signals. MEDIPOST’s 2nd generation human Umbilical Cord Blood-derived Mesenchymal Stromal Cell (hUCB-MSC) pipeline code named SMUP-IA-01 is an off-the-shelf intra-articular injectable cell product for the patients with early to mid-stage knee osteoarthritis (OA). |
Metro International Biotech, LLC Worcester, Massachusetts, United States | Metro International Biotech, LLC is a privately-owned clinical-stage pharmaceutical company that has established the most comprehensive portfolio of proprietary drugs for the treatment of various diseases. |
Neurona Therapeutics South San Francisco, California, United States | Neurona is a biotherapeutics company developing neural cell therapies with single-dose curative potential for the treatment of chronic neurological disorders. Based on a novel neural cell lineage pioneered by the company’s scientific founders, Neurona has built a robust regenerative platform and is developing neuronal, glial, and gene-edited cell therapy candidates that provide long-term repair of dysfunctional neural networks for multiple neurological disorders. |
Nido Biosciences Watertown, Massachusetts, United States | Nido Bio is translating today’s neuroscience breakthroughs into tomorrow’s treatments for severe neurological diseases. Leveraging human genetics, we develop precision medicines that address the fundamental biology of disease and restore healthy cell function. NIDO-361, our clinical-stage candidate, is a treatment for Spinal and Bulbar Muscular Atrophy which is a rare and debilitating neuromuscular disease. Additional pipeline programs center around a novel target with the potential to address multiple disease mechanisms and that has broad clinical application across neurodegenerative and peripheral inflammatory diseases. We are creating a sustainable pipeline for the company by utilizing a functional genomics discovery platform based on human cell lines to identify novel targets. Through our approach we seek to transform patient lives in meaningful ways. |
Novadip Biosciences Mont-Saint-Guibert, Belgium | Novadip Biosciences is a clinical-stage biopharmaceutical company founded to design, develop, and bring to the market a new class of regenerative tissue products to accelerate healing of large bone defects and injuries in a single treatment. Novadip’s lead allogeneic product, NVD-X3, employs a matrix containing multiple bioactive factors that induce accelerated tissue healing. Designed as an “off-the-shelf” product, NVD-X3 can be shipped and stored at room temperature and offers superior intraoperative handling characteristics. Novadip is also developing NVD-003, an autologous therapy derived from ASCs as a potential single treatment to save limbs and restore mobility in patients with congenital pseudarthrosis of the tibia (CPT). CPT is a rare pediatric bone condition. |
Ossium Health San Francisco, California, United States | Building a Healthier Future Our mission is to improve the health, vitality, and longevity of human beings through bioengineering. Who We Are At Ossium, we believe that science is humanity’s best weapon in the fight against disease, and we embrace the challenge of mobilizing the world’s healthcare ecosystem to win that fight. In pursuit of this goal, we empower our employees, maintain the highest standards of excellence, and are a force for good. PRINCIPLES The values we live by Impact We are mission-driven and results-oriented. We believe that a healthier world is a better one and that our work should drive positive change. Truth Embracing the truth empowers us to learn from both our successes and our mistakes. We follow the data wherever it leads. Ownership We hold ourselves and each other to the highest bar. We care deeply about the work we do and know that even the smallest details can make a difference. |
Pelage Pharma 907 Westwood Boulevard, Suite 384, Los Angeles, CA 90024 | Pelage is a clinical-stage regenerative medicine company developing novel treatments for hair loss, including androgenetic alopecia. Based on discoveries in stem cell biology made by Pelage's scientific co-founders, the company's novel approach is designed to reactivate dormant hair follicle stem cells and restore the body's ability to grow hair naturally. |
Q Therapeutics Salt Lake City, Utah | Q Therapeutics is a biotechnology company based out of 417 S Wakara Way, Salt Lake City, UT, United States. |
Rege Nephro Co., Ltd. 46-29 Yoshidashimoadachi-cho, Sakyo-ku, Med-Pharm Collaboration bldg. Kyoto University, Kyoto, Kyoto 606-8501, JP | Rege Nephro is a clinical-stage biotech company developing cell therapy for chronic kidney disease (CKD) and small molecule for autosomal dominant polycystic kidney disease (ADPKD), and founded in 2019. There are 13 million patients with CKD in Japan, of which 340,000 are severe CKD patients forced to undergo artificial dialysis. The healthcare cost of dialysis is $12 billion, 4% of the total healthcare cost of Japan. However, there is still no curative treatment for CKD. Rege Nephro tries to treat patients suffering from CKD with cell therapy using nephron progenitor cells differentiated from iPS cells. |
Regenerative Patch Technologies Portola Valley, California, United States | Regenerative Patch Technologies LLC (RPT) is a CA-based biotechnology company focused on the development of stem cell-based therapies designed to reverse blindness caused by advanced dry age related macular degeneration (AMD). |
ReNeuron Bridgend, Wales, United Kingdom | Our focus at ReNeuron is on our CustomEx exosome technology platform, producing exosomes with unique tissue targeting tropisms to deliver a payload of choice to a preferred cell type. Our mission is to partner and develop novel exosome therapeutics for diseases with significant unmet needs. |
Rocket Pharmaceuticals New York, New York, United States of America | Rocket Pharmaceuticals (NASDAQ: RCKT), Ltd is a biotechnology company focused on seeking cures through molecular and gene therapy. We are developing first-in-class treatments for patients with rare and often devastating orphan diseases using a lentiviral-based and adeno-associated virus (AAV) therapy approach. We have developed rewarding partnerships with a number of leading international research institutions as well as with centers of manufacturing and cell processing expertise, while building an internal team to drive the research, development, clinical and regulatory programs. Ultimately we aim to launch and globally commercialize innovative gene therapies in disease areas with high unmet need. |
Seraxis Germantown, Maryland, United States | Seraxis is a privately owned biotechnology company. Our GMP lab is located in Germantown, Maryland, in the heart of the vibrant BioHealth Capital Region. Launched in early 2013, Seraxis used proprietary technologies to develop a cell replacement therapy, SR-01, that is more effective and safer than embryonic stem cell-derived therapies. Our highly pure insulin-producing cells are micro-encapsulated through a proprietary technology that enables the function of human pancreas cells in type-1 diabetes models without the need for immuno-suppression. Seraxis intends to bring SR-01 to the clinic in order to provide a cure for the devastating disease of diabetes. |
Smart Immune Paris, Île-de-France, France | Smart Immune is a clinical-stage biotechnology company developing ProTcell, a thymus-empowered T-cell therapy platform to fully and rapidly re-arm the immune system, enabling next-generation allogeneic T-cell therapies for all. The company is a spin-off from the Imagine Institute of Genetic Diseases, and was founded in 2017 by Karine Rossignol, PharmD-HEC, Marina Cavazzana, MD-PhD and Isabelle André, PhD to transform outcomes for patients with life-threatening diseases such as high-risk blood cancers and primary immunodeficiencies. Smart Immune has clinical partnerships with leading institutions in the US and Europe. The ProTcell platform, already in Phase I/II clinical trials, enables the accelerated recovery of a complete immune repertoire in patients fighting cancer and infection. The ProTcell platform introduces potent, allogeneic T-cell progenitors which are then differentiated by the thymus into fully functional T-cells, to ultimately develop an ‘off the shelf’ T-cell medicine. The company is headquartered in Paris, France, at Paris Biotech Santé. |
StemCyte Inc 13800 live oak ave, baldwin park, california, united states | |
Steminent Biotherapeutics Taipei, Taiwan | Steminent Biotherapeutics Inc. is the leading stem cell clinical development company in Taipei, with subsidiaries in San Diego and Shanghai, dedicated to the development of novel cellular therapeutics for the treatment of diseases with unmet or under-served medical needs. Steminent utilizes advanced, proprietary processes and know how to isolate, purify, amplify and manufacture standardized stem cell products of the highest quality for research and clinical development in multiple indications. Steminent’s Stemchymal® allogeneic cell therapy R&D program has generated a portfolio of clinical stage therapeutic candidates for multiple diseases including: Phase II; Spinocerebellar Ataxia (PolyQ SCA), Phase I; Osteoarthritis of the Knee, and Phase I: Acute Liver Failure. 幹細胞醫藥產業先驅的仲恩生醫,以推動異體間葉幹細胞治療為核心,擁有世界一流的幹細胞研發團隊與技術。獨家核心技術平台Stemchymal®,以領先全球的幹細胞分離、純化、增生技術,應用於自體及異體的脂肪間葉幹細胞治療。 仲恩生醫以最嚴謹的製藥規範與品質管理,不斷累積科學實證,並透過國內外頂尖醫學中心臨床試驗證實仲恩生醫幹細胞產品的安全與療效。除此,仲恩生醫透過與臨床醫師及國內外合作夥伴緊密的合作,持續地針對臨床未被滿足的需求,持續開發Stemchymal®優質細胞產品於急性肝衰竭及其他適應症之應用,期許未來多國的二期臨床試驗的結果,以及在其他不治和難治病症上的努力和不斷的進步,將可盡早實現幹細胞藥物在臨床應用的價值,嘉惠無藥可醫之病患。 2016年藉由台日再生醫療的策略結盟,深化台日雙方的合作關係,更促進再生醫學產業化和實用化的發展。2018年向美日兩國申請的臨床試驗二期申請案,相繼於7月14日和7月29日正式通過。一旦於日本完成異體間葉幹細胞新藥的二期臨床試驗,將依日本再生醫藥專法申請孤兒藥資格及最高7至10年暫時性藥證許可。 |
TC BioPharm Holytown, North Lanarkshire, Scotland, United Kingdom | TC BioPharm is a publicly traded, clinical-stage cell therapy company developing advanced allogeneic CAR-T cell therapy products for the treatment of cancer, as well as developing gamma delta T cell therapies for the treatment of infectious disease. We have a growing team based in our Glasgow office, alongside a wide range of scientific advisors, and partner organisations. |
Telios Pharma, Inc. 275 Shoreline Drive, Suite 325, Redwood City, California 94065, US | Telios Pharma, Inc. is a clinical-stage biopharmaceutical company based in Redwood City, California, specializing in developing therapies for mast cell-mediated inflammatory diseases. Founded in 1993, the company focuses on small-molecule drugs and has a team of 51 to 100 employees. Its annual revenue is estimated between $10 million and $25 million. The company's pipeline includes innovative Bruton’s tyrosine kinase (BTK) inhibitors. Notable candidates are TL-925, a topical eye drop for dry eye disease currently in Phase 2 trials, and M-7583, an oral BTK inhibitor for primary myelofibrosis. TL-925 has shown promising results, providing rapid relief from symptoms and significant improvements in clinical measures. Telios Pharma aims to address significant unmet needs in both dry eye disease and myelofibrosis, targeting markets with limited existing therapies. |
Tempo Therapeutics 3030 Bunker Hill Street, Suite 104, San Diego, CA 92109 | Tempo Therapeutics is a biotechnology company that specializes in tissue engineering using proprietary MAP material science technology to build functional tissue and organs within patients in real time for disease treatment. |
Tenaya Therapeutics South San Francisco, California, United States | Tenaya Therapeutics, Inc. develops therapeutics for heart failure. It offers cellular regeneration platform, that enables in vivo reprogramming of cardiac fibroblasts into cardio myocytes by delivering proprietary transcription factors; gene therapy platform, that enables the targeted delivery of a payload to cardiac fibroblasts; and precision medicine platform, which provides personalized medicine approach to treat heart disease. The company was incorporated in 2016 and is based in South San Francisco, California. |
Vor Biopharma Cambridge, Massachusetts, United States | We are a cell therapy company that combines a novel patient engineering approach with targeted therapies to provide a solution for patients suffering from hematological malignancies. |
Xintela AB Lund, Skåne, Sweden | Xintela develops medical products in stem cell therapy and targeted cancer therapy based on the Company's cell surface marker integrin α10β1 which is found on mesenchymal stem cells and on certain aggressive cancer cells. The stem cell marker is used to select and quality-assure the patent-protected stem cell product XSTEM®, which is in clinical development for treatment of knee osteoarthritis and difficult-to-heal leg ulcers. The company produces XSTEM for the clinical studies in its GMP-approved manufacturing facility. In cancer therapy, which is run by the wholly owned subsidiary Targinta AB, therapeutic antibodies, targeting integrin α10β1 (First-in-Class) are being developed for the treatment of triple-negative breast cancer and the brain tumor glioblastoma. |