List of Stem Cells Companies with Phase 3 Active Clinical Trial - 22

AIVITA Biomedical is an Irvine, CA based company focused on the advancement of commercial and clinical-stage programs utilizing curative and regenerative medicines. We leverage our unique expertise in stem cell growth and directed, high-purity differentiation to develop safe, efficient and economical manufacturing processes for therapeutic development. These proprietary capabilities enable us to turn therapeutic discovery into reality, developing revolutionary cell-based treatments and healthcare products for the patients who need them. AIVITA is developing treatments for cancer, vision loss, and is bringing next-generation skincare formulations to consumers through its commercial product offerings.

BioLineRx Ltd., a clinical-stage biopharmaceutical development company, focuses on oncology. Its development pipeline consists of clinical-stage therapeutic candidates, which include BL-8040, a peptide for the treatment of solid tumors, hematological malignancies, and stem cell mobilization; AGI-134, an immuno-oncology agent in the development for solid tumors; and BL-5010, a customized, proprietary, pen-like applicator for the non-surgical removal of skin lesions. The company has collaboration agreement with MSD for the cancer immunotherapy field; MD Anderson Cancer Center to investigate the combination of BL-8040 with KEYTRUDA (pembrolizumab) in pancreatic cancer; and Genentech Inc. to investigate the combination of BL-8040 for solid tumors. BioLineRx Ltd. was founded in 2003 and is headquartered in Hevel Modi'in, Israel.

bluebird bio, Inc., a biotechnology company, engages in researching, developing, and commercializing of transformative gene therapies for severe genetic diseases and cancer. Its gene therapy programs include LentiGlobin for the treatment of ß-thalassemia and sickle cell disease; and Lenti-D for the treatment of cerebral adrenoleukodystrophy. The company’s product candidates in oncology include bb2121 and bb21217, which are chimeric antigen receptor T (CAR T) cell product candidates for the treatment of multiple myeloma. It has a strategic collaboration with Bristol-Myers Squibb to discover, develop, and commercialize disease-altering gene therapies in oncology; and Regeneron Pharmaceuticals, Inc. to discover, develop, and commercialize various immune cell therapies for cancer. The company also has collaborations with Medigene AG to discover T cell receptor (TCR) product candidates in the field of cancer; Gritstone Oncology, Inc. to discover TCR product candidates in the field of cancer; TC BioPharm Limited to research and develop gamma delta CAR T cells directed at hematologic and solid tumor targets; Forty Seven, Inc. to pursue clinical proof-of-concept for an antibody-based conditioning regimen; and Novo Nordisk A/S to develop in vivo genome editing treatments for genetic diseases, as well as clinical trial collaboration with Magenta Therapeutics. The company was formerly known as Genetix Pharmaceuticals, Inc., and changed its name to bluebird bio, Inc. in September 2010. bluebird bio, Inc. was founded in 1992 and is headquartered in Cambridge, Massachusetts.

BrainStorm Cell Therapeutics Inc. (NASDAQ) develops autologous stem cell therapies for neurodegenerative disorders such as ALS, MS, and Parkinson’s disease. These diseases have limited treatment options and represent unmet medical needs. NurOwn® is BrainStorm’s proprietary process for differentiating autologous mesenchymal stem cells (MSC) into neurotrophic factor (NTF)-secreting cells, transplanted at or near the affected tissue site. Our technology converts MSCs into a living drug delivery system for NTFs. We have proof-of-concept in various animal models of neurodegenerative diseases, including Parkinson's, Huntington's, ALS, MS, and peripheral nerve injury. We have completed two single-arm clinical trials in ALS patients in Israel, demonstrating good safety and tolerability profiles and strong efficacy signals. We also finalized a Phase 3 randomized, double-blind, placebo-controlled clinical trial at multiple US sites, supported by a $16 million USD grant from the California Institute for Regenerative Medicine (CIRM) and another grant from the ALS Association. Currently, we are planning a Phase 3b clinical trial of NurOwn® in ALS under a Special Protocol Assessment (SPA) with the U.S. Food and Drug Administration (FDA). For more information, visit www.brainstorm-cell.com

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.

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.

CorestemChemon is a global biotechnology corporation centered on stem cell-based new drug development and non-clinical research divisions. Motivated by the urgent need for medication for patients, the bio-pharmaceuticals division remains dedicated to research and development. In 2014, the unit successfully commercialized Neuronata-R inj, a stem cell therapy for the rare disease Amyotrophic Lateral Sclerosis (ALS), making it the fifth stem cell therapy approved for use globally. Alongside ALS treatment, the division has pipelines for various diseases such as lupus, Neuromyelitis Optica (NMO), and multiple system atrophy (MSA). Leveraging the success of the ALS stem cell therapy, the unit plans to concentrate on developing treatments for rare and incurable neurological and autoimmune diseases to dominate new market.

CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom.

Direct Biologics is a market-leading innovator in regenerative medicine.

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.

Gameto is a biotechnology company whose mission is to redefine female reproductive health by developing therapies that improve lives. Women’s health is a vastly underserved therapeutic area. Most conditions have few treatment options, and those available often cause frequent complications and offer limited clinical benefit. We aim to change that. Gameto is committed to using cutting-edge scientific advances to deliver better solutions to patients. Using cellular engineering, we have developed a platform to produce female reproductive cell lines. We use proprietary combinations of transcription factors to reprogram hiPSCs into engineered ovarian and endometrium cell lines that mimic the functions of endogenous cells, producing and responding to all hormones. Our team of cellular engineers is using this platform to advance a portfolio of cellular therapeutics for female reproductive diseases. Our first program, Fertilo, is a therapeutic for IVF and egg freezing, aiming to make the process shorter, safer and more effective. Additional applications of our platform include, an organoid of the female reproductive system, Deovo, and a cell-based therapeutic to ameliorate the health consequences of primary ovarian insufficiency and menopause, Ameno.

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.

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

Innovacell Biotechnologie AG is an innovative biotechnology company that develops personalized cell therapies for treatment of incontinence.

MaaT Pharma has established the most complete approach to restoring patient-microbiome symbiosis to improve survival outcomes in life-threatening diseases. Committed to treating blood cancers and graft-versus-host disease, a serious complication of allogeneic stem cell transplantation, MaaT Pharma has already achieved proof of concept in acute myeloid leukemia patients. Supporting the further expansion of our pipeline into larger indications, we have built a powerful discovery and analysis platform to evaluate drug candidates, determine novel disease targets and identify biomarkers for microbiome-related conditions. Our therapeutics are produced through a standardized cGMP manufacturing and quality control process to safely deliver the full diversity of the microbiome. MaaT Pharma benefits from the commitment of world-leading scientists and established relationships with regulators to spear-head microbiome treatment integration into clinical practice.

Mesoblast (ASX:MSB; Nasdaq:MESO) is developing and commercializing allogeneic cellular medicines to treat serious and life-threatening inflammatory diseases with significant, unmet medical needs. The Company's Phase 3 off-the-shelf mesenchymal lineage cell product candidates are: • RYONCIL™ (remestemcel-L) for steroid-refractory acute graft versus host disease (acute GVHD) • Remestemcel-L for moderate to severe acute respiratory distress syndrome (ARDS) due to COVID-19 infection • REVASCOR® for advanced chronic heart failure, and • MPC-06-ID for chronic low back pain due to degenerative disc disease. The Company’s proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide. Mesoblast’s approach to product development is to ensure rigorous scientific investigations are performed with well-characterized cell populations in order to understand mechanisms of action for each potential indication. Extensive preclinical translational studies guide clinical trials that are structured to meet stringent safety and efficacy criteria set by international regulatory agencies. All trials are conducted under the continuing review of independent Data Safety Monitoring Boards comprised of independent medical experts and statisticians. Mesoblast has an extensive patent portfolio comprising approximately 1,000 patents and patent applications with protection extending through 2040 in all major markets. This intellectual property portfolio covers composition of matter, manufacturing, and therapeutic applications of mesenchymal lineage cells. The Company believes this patent estate provides strong global protection.

Orca Bio is a clinical-stage biotechnology company developing a pipeline of high precision allogeneic cell therapy products that are designed to safely replace patients’ diseased blood and immune system with a healthy one. Our manufacturing platform sorts donor blood with single-cell precision and a high level of purity and speed, enabling us to create proprietary, optimal therapeutic mixtures of immune and stem cells that have the potential to transform allogeneic cell therapy. Our talented team is driven by a passion for science and compassion for patients.

Orchard Therapeutics, a Kyowa Kirin company, is a global gene therapy leader focused on ending the devastation caused by genetic and other severe diseases by discovering, developing, and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patient’s own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease with a single treatment. Founded in 2015, Orchard’s roots go back to some of the first research and clinical developments involving HSC gene therapy. Our team has played a central role in the evolution of this technology from a promising scientific idea to a potentially life-transforming reality. Today, Orchard is advancing a pipeline of HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist. For more information, please visit www.orchard-tx.com.

Priothera - We are dedicated to improving the lives of patients suffering from hematological malignancies by delivering an innovative immune modulator to enhance the curative potential of allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

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.

Thermo Fisher Scientific Inc. is the world leader in serving science, with annual revenue of approximately $40 billion. Our Mission is to enable our customers to make the world healthier, cleaner and safer. Whether our customers are accelerating life sciences research, solving complex analytical challenges, increasing productivity in their laboratories, improving patient health through diagnostics or the development and manufacture of life-changing therapies, we are here to support them. Our global team delivers an unrivaled combination of innovative technologies, purchasing convenience and pharmaceutical services through our industry-leading brands, including Thermo Scientific, Applied Biosystems, Invitrogen, Fisher Scientific, Unity Lab Services, Patheon and PPD.

VistaGen Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in developing and commercializing various medicines with the potential to care for anxiety, depression, and other disorders of the central nervous system (CNS). The company’s CNS pipeline includes PH94B, a rapid-onset neuroactive nasal spray, which is in preparation for Phase III development for the acute treatment of anxiety in adults with social anxiety disorder. Its PH94B product also has potential to treat a range of anxiety disorders, including adjustment disorder with anxiety, postpartum anxiety, post-traumatic stress disorder, preprocedural anxiety, panic, and others. The company’s CNS pipeline also comprises PH10, a rapid-onset neuroactive nasal spray, which is in preparation for Phase 2B clinical development as a stand-alone treatment for major depressive disorder (MDD); and AV-101, an oral N-methyl-D-aspartate receptor antagonist, which is in development in combination with probenecid as a potential treatment of levodopa-induced dyskinesia, MDD, neuropathic pain, and suicidal ideation. It has contract research and development agreement with Cato Research Ltd.; license and option agreements with Pherin Pharmaceuticals, Inc.; license and collaboration agreement with EverInsight Therapeutics Inc.; and license and sublicense agreement with BlueRock Therapeutics, LP. The company was founded in 1998 and is headquartered in South San Francisco, California.