California Institute for Regenerative Medicine

Ray Therapeutics develops optogenetic gene therapies to restore vision in patients with blinding retinal diseases. The company designs therapies that use bioengineered light-sensitive proteins delivered to targeted retinal cells via intravitreal injections, with the goal of improving visual function across various forms of retinal blindness and inherited retinal diseases. By combining gene delivery with optogenetic control, Ray Therapeutics aims to provide durable, long-term benefits from a single treatment, addressing diseases that lead to severe retinal degeneration. The company focuses on advancing research and development in this niche field to enable patients with limited or no light perception to regain functional sight, supporting efforts to study rare retinal conditions and translate laboratory insights into potential clinical therapies.

Tr1x specializes in developing cellular therapies aimed at treating autoimmune disorders. The company employs its proprietary platform to create innovative cell therapy products designed to reset the immune system and restore balance, thereby promoting long-term tolerance.

Cure Rare Disease is a non-profit organization based in Woodbridge, Connecticut, founded in 2017 to develop custom therapeutics aimed at treating individuals with rare genetic disorders, particularly muscular dystrophy. The organization focuses on creating effective, life-saving treatments specifically tailored for patients who are often told there are no available cures for their conditions. Through collaborations with renowned researchers and clinicians, as well as support from charitable donors, Cure Rare Disease strives to enhance research and provide crucial resources and information to families and advocates affected by these disorders. Their commitment is to empower patients in their fight for long, fulfilling lives despite their health challenges.

Navega Therapeutics is a preclinical stage company developing innovative gene therapies for neurological disorders, with a focus on chronic pain and ophthalmic diseases. The company's proprietary platform employs epigenetic regulation tools to achieve non-immunogenic, long-lasting effects. Navega's lead program targets Nav1.7, a key pain gatekeeper gene, aiming to relieve chronic pain without introducing permanent mutations in the genome. Additionally, they are developing a mutation-agnostic approach to treat all forms of retinitis pigmentosa through in vivo cellular reprogramming.

Tenaya Therapeutics is a preclinical stage biotechnology company based in South San Francisco, California, specializing in the development of innovative therapies for heart disease, particularly heart failure. Established in 2016, the company employs a multipronged approach to address the fundamental cellular pathologies associated with cardiac muscle disease. Tenaya Therapeutics advances its product candidates through three primary platforms: a cellular regeneration platform that reprograms cardiac fibroblasts into cardiomyocytes using proprietary transcription factors, a gene therapy platform for targeted delivery of therapeutic payloads to cardiac fibroblasts, and a precision medicine platform that offers personalized treatment strategies. Through its research, the company aims to enable physicians to regenerate heart tissue and tackle various forms of cardiomyopathies effectively.

Entos develops next-generation genetic medicines using their proprietary Fusogenix proteolipid vehicle (PLV) drug delivery system. Fusogenix PLVs are formulated with FAST proteins to enable the delivery of nucleic acid into target cells through direct fusion.

The University of California, Irvine Extension is an academic institution offering diverse educational programs and cultural events to both students and the wider community.

Amplo Biotechnology is a biotechnology company developing adeno-associated viral therapies aimed at curing defects in neuromuscular junctions, with an initial focus on congenital myasthenic syndromes. Its lead program, AAV-Dok7, originated with a research team from the Institute of Medical Science at the University of Tokyo and is designed to be delivered via intravascular injection to trigger molecular events that address paralysis, breathing, and swallowing difficulties caused by neuromuscular junction defects.

ReviR Therapeutics is a genomics-focused company that specializes in the development of RNA therapeutics aimed at treating cancer, rare genetic disorders, and infectious diseases. The company utilizes an artificial intelligence-driven platform to enhance the discovery of novel RNA-targeting medicines, thereby facilitating the delivery of innovative therapies to a wide patient population. ReviR Therapeutics combines advanced computational techniques with its expertise in RNA biology, positioning itself as a leader in the evolving field of RNA-based therapeutics.

NysnoBio is a biotechnology company focused on developing innovative therapies for serious medical conditions, with a particular emphasis on Parkinson's disease. The company was the first to identify the Parkin gene's association with Parkinson's disease and to demonstrate that Parkin functions as an E3 ubiquitin ligase. NysnoBio has pioneered several key advancements, including publishing the crystal structure of essential parts of enzymology and utilizing adeno-associated virus (AAV) in gene therapy. Their approach involves developing small molecule activators for the Parkin protein and employing gene therapy to preserve dopamine neurons, aiming to provide effective treatments for Parkinson's disease patients.

Fate Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing programmed cellular immunotherapies for cancer and immune disorders. The company specializes in NK- and T-cell immuno-oncology programs, with a diverse pipeline that includes candidates such as FT516 and FT596 for treating various hematologic malignancies, FT538 for acute myeloid leukemia and multiple myeloma, and FT500 for advanced solid tumors. Fate Therapeutics employs advanced techniques involving induced pluripotent stem cells (iPSCs) to create engineered product candidates, which are designed to be off-the-shelf solutions. The company has established strategic collaborations with notable partners, including Ono Pharmaceutical Co. Ltd. and Janssen Biotech, to enhance its research and development capabilities. Founded in 2007, Fate Therapeutics aims to leverage cutting-edge science to create innovative therapies that restore health by modulating the properties of stem cells.

CellFE Inc. is a biotechnology company based in San Carlos, California, established in 2017. It specializes in the development of a microfluidics-based device designed to deliver gene-editing molecules into human cells. This innovative platform enhances the manufacturing and development of cell therapies by utilizing rapid cell compression techniques that facilitate the uptake of therapeutic molecules from surrounding fluids. By enabling the transport of hard-to-deliver molecules, CellFE aims to improve the effectiveness of treatments for various disorders, supporting medical researchers in their efforts to advance patient care and outcomes.

The University of California, San Francisco (UCSF) is a prominent educational institution established in 1897, specializing in health sciences. It offers a range of programs across various fields, including biological, biomedical, pharmaceutical, nursing, social and behavioral sciences, dentistry, and medicine. UCSF comprises several schools, including the School of Medicine, School of Dentistry, School of Nursing, School of Pharmacy, and Continuing Medical Education. The university is known for its significant contributions to research and development, particularly in areas such as advanced cytometry, automated digital pathology, and single-cell genomics. With an endowment of $873 million, UCSF supports a robust academic and research environment, fostering innovation and the advancement of medical technologies. The institution has successfully secured over $40 million in extramural funding for its research initiatives, further enhancing its capabilities and impact in the biomedical field.

Stanford University, established in 1855, is a renowned research and teaching institution located in Stanford, California. It comprises seven schools offering a wide range of undergraduate and graduate programs, with a strong emphasis on law, medicine, education, and business. The university is known for its rigorous admissions process, selecting students based on academic excellence, intellectual curiosity, and personal experiences. Stanford provides extensive financial aid to both undergraduate and graduate students. It fosters a vibrant campus life, with over 11,000 students living on campus, and offers a rich array of cultural and athletic programs, including art exhibitions, performing arts, and over 40 varsity and club sports.

Maro Bio is a biotech company.

The University of California, Los Angeles (UCLA) is a renowned public research university offering comprehensive academic programs across various fields. It is known for its prestigious School of Medicine and highly regarded Film School, with notable alumni in both industries. UCLA also excels in liberal arts, sciences, humanities, and professional programs such as Education, Architecture, Engineering, Public Affairs, and Law. The university provides financial aid to qualified students and offers continuing education courses taught by published scholars. Located in Westwood, Los Angeles, UCLA boasts a vibrant student life with numerous on-campus organizations and nearby cultural attractions.

Founded in 1869, the University of California is a public research university system comprising ten campuses. It offers a wide range of academic programs and is renowned for its innovative research, driving advancements in biotechnology, computer science, arts, and architecture. With over 280,000 students and more than 2 million alumni worldwide, UC's impact extends beyond academia, influencing local governments, schools, environmental protection, and space exploration.

Neurona Therapeutics is a biotechnology company developing cell-based therapies for treating neurological disorders. Founded by neuroscientists at UCSF in 2008, the company focuses on generating therapeutic compositions of specific nerve cells for targeted delivery into the injured nervous system.

AcuraStem is a biotechnology company based in Monrovia, California, founded in 2016. The company specializes in developing a precision medicine platform that utilizes complex cellular models derived from Amyotrophic Lateral Sclerosis (ALS) patients' cells. AcuraStem employs advanced cellular reprogramming and artificial intelligence technologies to create patient-specific treatments for neurodegenerative diseases. Their platform is designed to evaluate disease progression and test existing therapeutics, as well as combinations of therapeutic approaches, to identify effective treatments that could slow disease progression. The company's team includes PhDs, professors, tech entrepreneurs, and veterans from the drug industry, all working towards addressing the challenges posed by ALS.

Salk Institute for Biological Studies conducts research on molecular biology and genetics, neurosciences, and plant biology. The research is conducted under the guidance of 59 faculty investigators and more than 850 scientific staff, including visiting scientists, postdoctoral fellows, and graduate students. The institute’s operates to identify therapies and treatments for a range of diseases such as cancer, AIDS, Alzheimer's disease, cardiovascular disorders, anomalies of the brain, and birth defects. Six key areas represent strategic research priorities: chemistry and proteomics, stem cell biology, cell biology, regulatory biology, metabolic research, and computational and theoretical biology. The Institute has trained more than 2,700 scientists, out of which five have won Nobel prizes. Salk is home to nine Howard Hughes Medical Investigators and 14 members of the National Academy of Sciences. NCI Cancer Center, Crick-Jacobs Center, Glenn Center for Aging Research, Helmsley Center for Genomic Medicine, Waitt Advanced Biophotonics Center, and Glenn Center for Aging Research are some of the research centers operated by Salk Institute for Biological Studies. The Salk Institute for Biological Studies was established in the 1960s by Jonas Salk and is based in California, United States.

Nexcella Utilize cell therapies to create safe and effective medicines for oncology and other conditions, improving patient outcomes.

City of Hope is a non-profit clinical research center based in Duarte, California, founded in 1913, focused on cancer treatment and diabetes research. It provides access to expert doctors, coordinates clinical trials and treatment programs, and conducts biomedical research to improve patient outcomes. Designated by the National Cancer Institute as a comprehensive cancer center, City of Hope combines patient care with scientific discovery and education, supported by researchers, clinicians, nurses, and staff.

Aspen Neuroscience develops autologous neuron replacement therapies based on induced pluripotent stem cells to treat neurological conditions, starting with Parkinson's disease. The company uses patient-specific restorative cell therapies by combining pluripotent stem cell biology with genomic approaches and artificial intelligence to address both sporadic and familial forms of Parkinson's disease, focusing on autologous neuron replacement to modify disease progression. Based in San Diego, California, the company is a development-stage biotechnology company pursuing expansion of its iPSC-based platform to other brain disorders.

Luxa Biotechnology is a clinical-stage biotechnology company focused on developing a novel therapy using adult retinal pigment epithelial stem cells (RPESCs) for the treatment of dry age-related macular degeneration (AMD). The company's approach involves transplanting new RPE cells under the retina to prevent or slow down vision loss caused by this degenerative disease. LuxaBio is currently in the clinical trial phase, aiming to advance its innovative therapy towards market approval.

Anova Enterprises is a company focused on accelerating clinical development. Their primary goal is to support and expedite the process of bringing new medical treatments and technologies to market. By leveraging their expertise and resources, Anova Enterprises works to streamline the clinical development process and improve the efficiency of clinical trials.

Denovo Biopharma (Hangzhou) Co. Ltd, based in Hangzhou, China, is a privately held biotechnology company specializing in biopharmaceuticals. The company focuses on personalized drug development through a unique platform that enables de novo genomic biomarker discovery using archived clinical samples. This innovative technology is particularly beneficial for late-stage clinical drugs that have previously demonstrated unsatisfactory efficacy. By identifying biomarkers linked to patient responsiveness, Denovo Biopharma assists biotech and pharmaceutical companies in designing targeted clinical trials, which aim to enhance efficacy and reduce adverse effects. Their biomarker discovery capabilities extend across various therapeutic areas, including oncology, metabolic disorders, cardiology, immunology, and neurology.

Children's Hospital Los Angeles (CHLA) is a nonprofit pediatric care facility established in 1901, recognized for its exceptional clinical services. It has earned the distinction of being the best children's hospital on the West Coast and ranks among the top five in the nation according to U.S. News & World Report. CHLA is dedicated to providing comprehensive healthcare to children and is home to The Saban Research Institute, one of the largest and most productive pediatric research centers in the United States. Through its commitment to clinical excellence and research, CHLA plays a vital role in advancing pediatric medicine and improving health outcomes for children.

ImmPACT Bio USA Inc. is a cell therapy company focused on developing engineered T-cells for the treatment of solid tumors. The company specializes in creating chimeric antigen receptor (CAR) T-cell therapies that target specific gene loss features in tumor cells, allowing for the selective destruction of cancerous tissues while preserving healthy ones. This technology aims to enhance tumor specificity, enabling the CAR-T cells to induce cytotoxicity only when engaging with tumor cells, thereby minimizing the safety risks commonly associated with existing CAR-T therapies. ImmPACT Bio's innovative approach seeks to improve treatment outcomes for cancer patients by offering a more precise and safer therapeutic option.

Ray Therapeutics develops optogenetic gene therapies to restore vision in patients with blinding retinal diseases. The company designs therapies that use bioengineered light-sensitive proteins delivered to targeted retinal cells via intravitreal injections, with the goal of improving visual function across various forms of retinal blindness and inherited retinal diseases. By combining gene delivery with optogenetic control, Ray Therapeutics aims to provide durable, long-term benefits from a single treatment, addressing diseases that lead to severe retinal degeneration. The company focuses on advancing research and development in this niche field to enable patients with limited or no light perception to regain functional sight, supporting efforts to study rare retinal conditions and translate laboratory insights into potential clinical therapies.

Siren Biotechnology is a biotech company focused on developing innovative cancer treatments through a unique platform that integrates AAV gene therapy with cytokine immunotherapy. By combining these two transformative therapeutic approaches, Siren aims to address significant challenges in cancer treatment, such as delivery, efficacy, and toxicity. This novel modality seeks to enhance the elimination of tumor cells while simultaneously stimulating anti-tumor immunity, thereby offering a promising new strategy for cancer therapy. Through this integrated approach, Siren Biotechnology aspires to advance the effectiveness of cancer treatments and improve patient outcomes.

Eureka Therapeutics, Inc. is a biotechnology company based in Emeryville, California, specializing in the development of immunotherapies for cancer treatment. The company focuses on creating innovative T-cell therapies using its proprietary ARTEMIS AbTCR T-cell receptor platform and the E-ALPHA antibody discovery platform, which has become one of the largest human-derived antibody phage libraries. Eureka Therapeutics aims to target cancer-specific antigens, onco-fetal antigens, mutated oncogenes, and viral oncogenes, improving the safety and efficacy of T-cell therapies. The company has established a strategic collaboration with Lyell Immunopharma to enhance its research and development efforts in this field. Founded in 2006, Eureka Therapeutics is dedicated to advancing the discovery and development of novel cancer therapeutics.

Lineage Cell Therapeutics is a clinical-stage biotechnology company headquartered in Carlsbad, California, that specializes in the development and commercialization of innovative cellular therapies aimed at addressing unmet medical needs associated with degenerative diseases. The company’s leading product candidates include OpRegen, designed for the treatment of dry age-related macular degeneration, and OPC1, which targets acute spinal cord injuries. Additionally, Lineage is advancing VAC2, an allogeneic cancer immunotherapy aimed at non-small cell lung cancer. Other notable developments include Renevia, a hyaluronan-based hydrogel for treatment of HIV-associated facial lipoatrophy, and HyStem, a biomaterial for cell retention and delivery. Lineage also explores various therapeutic products for oncology and neurological disorders, alongside research programs focused on vision restoration and demyelination. Established in 1990, the company was formerly known as BioTime, Inc., and underwent a rebranding in August 2019.

Ossium Health, Inc. is a biotechnology company that specializes in developing and manufacturing advanced cell therapy products aimed at treating blood cancers, improving organ transplantation, and repairing tissue damage caused by radiation exposure. Established in 2016 and based in San Francisco, the company has created a range of therapies, including HPC, Marrow, which consists of human leukocyte antigen (HLA)-matched bone marrow stem cells for hematopoietic reconstitution in blood cancer patients. Additionally, Ossium offers Chimera, designed to enhance organ transplantation, and Chymalis, which utilizes mesenchymal stem cells from bone marrow to facilitate the healing of damaged bone and muscle tissue. The company also supplies bone marrow cells for research in oncology, immunology, and related fields, focusing on improving health and longevity through innovative bioengineering techniques.

Combangio is a clinical-stage biotechnology company focused on developing regenerative biotherapeutics based on mesenchymal stem cell secretomes.

Tachyon Therapeutics develops innovative compounds to enhance cancer treatments. It is currently conducting Phase 1 clinical trials to evaluate the safety and efficacy of its lead compound, TACH101.

Genascence is a clinical-stage biotechnology company developing gene therapies for musculoskeletal diseases. Its work targets osteoarthritis, aiming to treat the condition, alleviate symptoms, and slow structural disease progression to improve patient outcomes. The company also pursues genetic therapies for rare disorders and other prevalent diseases, with the goal of delivering safer and more effective treatment options for affected individuals.

Kenai Therapeutics specializes in developing innovative therapies for neurodegenerative movement disorders. The company focuses on providing off-the-shelf neuron replacement medicines as effective disease-modifying treatments, enabling doctors to employ new techniques for treating neurological conditions such as Parkinson's.

Rubedo Life Sciences, Inc. is a biopharmaceutical company based in Sunnyvale, California, focused on developing innovative therapies that target senescent cells implicated in age-related diseases. Established in 2018, the company utilizes its proprietary ALEMBIC drug discovery platform to create a pipeline of therapeutic candidates. These candidates are designed to selectively address senescent cells, as well as pro-inflammatory and pro-fibrotic cell populations that contribute to various chronic conditions, including pulmonary, dermatological, oncological, neurodegenerative, and fibrotic diseases. Rubedo's leadership team comprises industry experts with significant experience in drug development and commercialization across both large pharmaceutical companies and biotech firms.

Immusoft is a biotechnology company that develops autologous cell therapies using its Immune System Programming (ISP) technology. Founded in 2009 and headquartered in Seattle, Washington, the company reprograms patients' cells to produce gene-encoded medicines for treating various diseases.

Angiocrine Bioscience has developed a cellular platform that expands stem and progenitor cells by recapitulating the proliferative signals of the in vivo vascular niche in vitro. The expansion capacity is notable relative to other approaches and is achieved without costly media additives, enabling a broad range of therapeutic, diagnostic, and research applications centered on tissue-specific engineering and organ regeneration. The platform uses cultured endothelial cells that are genetically modified via insertion of the adenovirus gene, enabling physicians to tailor treatments and procedures. The technology supports applications in regenerative medicine and hematopoietic stem cell gene therapy.

Calidi Biotherapeutics, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on developing innovative immunotherapies for challenging malignancies. Established in 2014, the company specializes in creating oncolytic virus-based treatments that utilize a proprietary allogeneic cell-based delivery system. This technology aims to enhance the efficacy of oncolytic viruses, allowing for targeted cancer therapies that improve patient outcomes. By leveraging its patented delivery platforms, Calidi seeks to advance the treatment of cancer through effective and novel approaches in immuno-oncology.

The University of California, Irvine Extension is an academic institution offering diverse educational programs and cultural events to both students and the wider community.

Lutris Pharma is an Israel-based biopharmaceutical company focused on improving anti-cancer therapy. It develops drugs to enhance treatment effectiveness, including a proprietary topical B-Raf inhibitor LUT014 that enables administration of epidermal growth factor receptor inhibitors without the cutaneous side effects that typically interrupt therapy. Founded in 2015 and based in Tel Aviv-Yafo, Lutris Pharma aims to improve treatment continuity and patient quality of life for those treated with EGFR inhibitors.

Greenstone Biosciences is a drug discovery platform focused on accelerating the development of effective therapies for patients. By leveraging advanced technologies, including artificial intelligence and human-induced pluripotent stem cells, the company aims to transform the traditional drug discovery process, which is often slow and prone to high failure rates. Greenstone Biosciences seeks to create a rapid and integrated computational platform that can efficiently deliver safe and effective therapeutics, addressing diseases with significant unmet medical needs. Through its innovative approach, the company is dedicated to improving patient outcomes and advancing therapeutic options.

Excision BioTherapeutics Inc. is a life science company based in Philadelphia, Pennsylvania, established in 2015. The company is dedicated to the development and commercialization of gene editing therapeutics aimed at treating life-threatening diseases caused by neurotropic viruses. Utilizing CRISPR-based technology, Excision BioTherapeutics focuses on creating advanced gene-editing medicines that can eradicate or disrupt viral genes in human patients. The company's mission is to advance these therapeutics into safe and effective treatments, significantly improving the lives of individuals affected by viral infections globally.

Neurona Therapeutics is a biotechnology company developing cell-based therapies for treating neurological disorders. Founded by neuroscientists at UCSF in 2008, the company focuses on generating therapeutic compositions of specific nerve cells for targeted delivery into the injured nervous system.

Ray Therapeutics develops optogenetic gene therapies to restore vision in patients with blinding retinal diseases. The company designs therapies that use bioengineered light-sensitive proteins delivered to targeted retinal cells via intravitreal injections, with the goal of improving visual function across various forms of retinal blindness and inherited retinal diseases. By combining gene delivery with optogenetic control, Ray Therapeutics aims to provide durable, long-term benefits from a single treatment, addressing diseases that lead to severe retinal degeneration. The company focuses on advancing research and development in this niche field to enable patients with limited or no light perception to regain functional sight, supporting efforts to study rare retinal conditions and translate laboratory insights into potential clinical therapies.

BioEclipse Therapeutics is a clinical-stage oncology company developing immuno-oncology therapeutics using its proprietary technology. It aims to improve cancer patient outcomes by harnessing insights into the human immune system, pairing activated immune cells with adapted oncolytic viruses that selectively target and kill malignant cells.

Navega Therapeutics is a preclinical stage company developing innovative gene therapies for neurological disorders, with a focus on chronic pain and ophthalmic diseases. The company's proprietary platform employs epigenetic regulation tools to achieve non-immunogenic, long-lasting effects. Navega's lead program targets Nav1.7, a key pain gatekeeper gene, aiming to relieve chronic pain without introducing permanent mutations in the genome. Additionally, they are developing a mutation-agnostic approach to treat all forms of retinitis pigmentosa through in vivo cellular reprogramming.

City of Hope is a non-profit clinical research center based in Duarte, California, founded in 1913, focused on cancer treatment and diabetes research. It provides access to expert doctors, coordinates clinical trials and treatment programs, and conducts biomedical research to improve patient outcomes. Designated by the National Cancer Institute as a comprehensive cancer center, City of Hope combines patient care with scientific discovery and education, supported by researchers, clinicians, nurses, and staff.

Cytonus Therapeutics, Inc. is a biotechnology company founded in 2018 and located in Columbus, Ohio. The company specializes in developing immune-oncology and gene therapies aimed at addressing devastating and rare diseases. Cytonus employs a synthetic biology approach through its proprietary Cargocyte technology, which enables precision delivery of therapeutics and in vivo production of innovative treatments. By integrating AI-driven drug discovery methods, Cytonus is focused on creating first-in-class therapeutics to treat immune-related disorders effectively.

Immusoft is a biotechnology company that develops autologous cell therapies using its Immune System Programming (ISP) technology. Founded in 2009 and headquartered in Seattle, Washington, the company reprograms patients' cells to produce gene-encoded medicines for treating various diseases.

Juvena Therapeutics is a biopharmaceutical company developing protein-based therapies to promote tissue rejuvenation and repair in elderly individuals. Founded in 2017, the company focuses on treating age-related conditions such as sarcopenia, wound healing, and neural regeneration.

Rocket Pharmaceuticals is a biotechnology company focused on developing gene therapies for rare pediatric diseases. Its clinical-stage programs include treatments for Fanconi Anemia, Leukocyte Adhesion Deficiency-I, Pyruvate Kinase Deficiency, and Infantile Malignant Osteopetrosis.

Rocket Pharmaceuticals is a biotechnology company focused on developing gene therapies for rare pediatric diseases. Its clinical-stage programs include treatments for Fanconi Anemia, Leukocyte Adhesion Deficiency-I, Pyruvate Kinase Deficiency, and Infantile Malignant Osteopetrosis.

Poseida Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, dedicated to developing innovative therapeutics for patients with significant unmet medical needs. The company specializes in genome engineering technologies and is focused on creating targeted treatments, including a pipeline of autologous and allogeneic chimeric antigen receptor T cell (CAR-T) therapies aimed at addressing hematological malignancies and solid tumors. Additionally, Poseida is advancing gene therapies for rare and life-threatening diseases, such as Ornithine transcarbamylase deficiency and methylmalonic acidemia, along with therapies for genetic liver diseases. Founded in 2014, Poseida Therapeutics is committed to improving patient outcomes through its cutting-edge research and development efforts.

AiVita Biomedical, Inc. is a biotechnology company focused on advancing commercial and clinical-stage programs that utilize curative and regenerative medicines. Founded in 2016 and based in Irvine, California, the company develops innovative treatments for cancer and vision loss, along with a line of rejuvenating skincare products. Its primary therapeutic candidate is a patient-specific immunotherapy aimed at treating advanced cancers, particularly glioblastoma and ovarian cancer. Additionally, AiVita Biomedical is exploring a preclinical program involving human stem cells to create sheets of retinal tissue for treating degenerative retinal diseases. The company’s portfolio also includes personalized vaccines for infectious diseases, enhancing the medical system's ability to combat emerging health threats. Through its expertise in stem cell growth and differentiation, AiVita Biomedical aims to deliver safe and effective therapies to patients in need.

Sangamo Therapeutics is a clinical-stage biotechnology company focused on developing genomic therapies aimed at transforming the lives of patients with serious health conditions. The company employs advanced technologies in genome editing, gene therapy, gene regulation, and cell therapy to create innovative treatment options. Sangamo's diverse product pipeline addresses a range of diseases, including hemophilia, various central nervous system disorders, HIV, lysosomal storage disorders, and hemoglobinopathies. Additionally, the company has prioritized neurology-related programs targeting chronic neuropathic pain, prion disease, and other neurological conditions. Other ongoing initiatives involve treatments for Fabry disease, renal transplant, inflammatory bowel disease, and multiple sclerosis, reflecting Sangamo's commitment to addressing critical unmet medical needs.

Fate Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing programmed cellular immunotherapies for cancer and immune disorders. The company specializes in NK- and T-cell immuno-oncology programs, with a diverse pipeline that includes candidates such as FT516 and FT596 for treating various hematologic malignancies, FT538 for acute myeloid leukemia and multiple myeloma, and FT500 for advanced solid tumors. Fate Therapeutics employs advanced techniques involving induced pluripotent stem cells (iPSCs) to create engineered product candidates, which are designed to be off-the-shelf solutions. The company has established strategic collaborations with notable partners, including Ono Pharmaceutical Co. Ltd. and Janssen Biotech, to enhance its research and development capabilities. Founded in 2007, Fate Therapeutics aims to leverage cutting-edge science to create innovative therapies that restore health by modulating the properties of stem cells.

Medeor Therapeutics, Inc. is a biotechnology company focused on researching, developing, and commercializing innovative cellular immunotherapies aimed at enhancing outcomes for organ transplant recipients. Based in San Mateo, California, Medeor Therapeutics has developed a specialized immunotherapy product for kidney transplant recipients known as MDR-10X. The company's cellular immunotherapies target organ transplant immune tolerance and immuno-oncology, providing healthcare providers with advanced techniques to preserve or improve kidney function and mitigate the risk of graft rejection more effectively than traditional immunosuppression therapies. Incorporated in 2012, Medeor Therapeutics is dedicated to transforming transplant outcomes through personalized treatment strategies.

ViaCyte, Inc. is a regenerative medicine company based in San Diego, California, that specializes in developing cell replacement therapies for diabetes. The company’s primary product candidates, VC-01 and VC-02, aim to treat type 1 diabetes by implanting pancreatic beta-cell precursors derived from stem cells within an encapsulation device. This innovative approach allows the implanted cells to produce insulin in response to blood glucose levels while minimizing the risk of immune rejection, thereby eliminating the need for immunosuppressants. ViaCyte's goal is to provide a long-term solution that can free both type 1 and type 2 diabetes patients from dependence on insulin and reduce associated complications such as hypoglycemia and cardiovascular issues. Founded in 1999, ViaCyte was previously known as Novocell, Inc., and changed its name in 2010. The company also operates an additional facility in Athens, Georgia.

Poseida Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, dedicated to developing innovative therapeutics for patients with significant unmet medical needs. The company specializes in genome engineering technologies and is focused on creating targeted treatments, including a pipeline of autologous and allogeneic chimeric antigen receptor T cell (CAR-T) therapies aimed at addressing hematological malignancies and solid tumors. Additionally, Poseida is advancing gene therapies for rare and life-threatening diseases, such as Ornithine transcarbamylase deficiency and methylmalonic acidemia, along with therapies for genetic liver diseases. Founded in 2014, Poseida Therapeutics is committed to improving patient outcomes through its cutting-edge research and development efforts.

Mustang Bio, Inc. is a clinical-stage biopharmaceutical company dedicated to developing innovative cell and gene therapies aimed at curing hematologic cancers, solid tumors, and rare genetic diseases. The company’s pipeline includes gene therapy programs for rare genetic disorders and chimeric antigen receptor (CAR) T cell therapies targeting various malignancies. Notable developments include MB-107, a potential curative gene therapy for X-linked severe combined immunodeficiency, and several CAR T therapies such as MB-102 for acute myeloid leukemia, MB-106 for B-cell lymphomas, and programs targeting glioblastoma and solid tumors like breast and pancreatic cancers. Mustang Bio has established collaborations with institutions such as Nationwide Children’s Hospital and Beth Israel Deaconess Medical Center to advance its research initiatives. Founded in 2015 and headquartered in New York, the company operates as a subsidiary of Fortress Biotech, Inc.

Humacyte specializes in developing human tissue-based products for regenerative medicine and vascular surgery. It creates acellular extracellular matrices using banked vascular smooth muscle cells, which are then decellularized to eliminate rejection risks. These matrices serve as off-the-shelf tissue-engineered grafts for patients requiring vascular repair or replacement.

BrainStorm Cell Therapeutics Inc. is a biotechnology company focused on developing and commercializing adult stem cell therapies for neurodegenerative diseases. Their proprietary technology, NurOwn, utilizes a patient's own mesenchymal stem cells, which are engineered to produce neurotrophic factors that promote neuronal survival. The company is actively advancing NurOwn through clinical trials for conditions such as amyotrophic lateral sclerosis (ALS), multiple sclerosis (MS), and Parkinson's disease. Currently, NurOwn is in a Phase III clinical trial for ALS and a Phase II trial for MS, with plans for preclinical studies targeting Parkinson’s disease, Huntington’s disease, and autism spectrum disorder. BrainStorm has partnered with Catalent for the manufacturing of NurOwn, ensuring the production of this autologous cellular therapy. Founded in 2000 and headquartered in New York, BrainStorm Cell Therapeutics was previously known as Golden Hand Resources Inc. before rebranding in 2004.

SanBio is a regenerative medicine company focused on developing cell-based therapies for various medical conditions. The company is advancing several innovative products, notably SB623, which is in a Phase 2b clinical trial aimed at treating chronic motor impairments due to stroke. This product is being developed in collaboration with Sumitomo Dainippon Pharma Co., Ltd. and is also undergoing a global Phase 2 trial for motor impairment resulting from traumatic brain injury in the United States and Japan. Additionally, SanBio is exploring other therapeutic candidates, including SB618, which targets peripheral neuropathy, and SB308, designed for muscular dystrophy. The company's research encompasses a range of neurological and degenerative conditions, emphasizing its commitment to pioneering regenerative therapies.

Scaled Biolabs Inc. is a biotechnology company based in San Francisco, California, specializing in research and development solutions for cell therapies. Established in 2016, the company has developed a Cell Therapy Discovery Engine that leverages microfluidics, automation, and machine learning to facilitate the discovery of advanced cell-based medicines. This innovative platform allows leading biotech and pharmaceutical firms to explore a wide range of experimental parameters simultaneously, yielding valuable insights into cellular processes. By miniaturizing laboratory environments, Scaled Biolabs accelerates the development and manufacturing of novel cell therapies, which enhances clinical outcomes and reduces time to market. Additionally, the company is engaged in creating its own cell therapy assets, including a program focused on iPSC-derived dopaminergic neurons aimed at treating Parkinson’s disease.

Orchard Therapeutics is a UK-based biopharmaceutical company dedicated to developing and commercializing innovative gene therapies for rare diseases. Its core focus is autologous ex vivo gene therapy, transforming hematopoietic stem cells into a gene-modified drug product for single-administration treatments. The company's portfolio includes Strimvelis, approved by the EMA in 2016, and several programs in advanced registrational studies across various disease areas.

Semma Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing innovative cell therapies for patients with Type 1 diabetes. Founded in 2014, the company is dedicated to creating a transformative treatment that addresses the need for insulin injections by generating functional, insulin-producing beta cells through a proprietary method licensed from the laboratory of Professor Douglas Melton. Semma Therapeutics aims to combine these engineered cells with advanced devices to offer a potential replacement for the missing beta cells in diabetic patients, eliminating the need for immunosuppression. The company's ongoing research seeks to bring this novel therapeutic option to clinical settings, ultimately improving the lives of individuals living with diabetes.

Humacyte specializes in developing human tissue-based products for regenerative medicine and vascular surgery. It creates acellular extracellular matrices using banked vascular smooth muscle cells, which are then decellularized to eliminate rejection risks. These matrices serve as off-the-shelf tissue-engineered grafts for patients requiring vascular repair or replacement.

Capricor Therapeutics is a clinical-stage biotechnology company focused on developing cell and exosome-based therapies for Duchenne muscular dystrophy and other high-need diseases. Its lead program CAP-1002 is an allogeneic cardiac-derived cell therapy evaluated for Duchenne muscular dystrophy and related cardiac conditions. The company also pursues CAP-2003 in preclinical development for inflammatory diseases and explores exosome-based approaches through its StealthX platform to advance next-generation therapeutics. Capricor aims to address unmet medical needs by translating cellular and exosome science into transformative treatments. The company was founded in 2005 and is headquartered in Los Angeles, California.

ImmunoCellular Therapeutics, Ltd. is a clinical-stage biotechnology company specializing in the development of immune-based therapies for various cancers. The company is known for its innovative products, including ICT-107, a dendritic cell immunotherapy designed for newly diagnosed glioblastoma multiforme, a highly aggressive brain cancer. Additionally, ImmunoCellular is advancing ICT-140, which targets tumor-associated antigens in ovarian cancer, and ICT-121, aimed at treating recurrent glioblastoma and other solid tumors by targeting CD133 markers on cancer stem cells. The company also explores Steam-to-T-Cell immunotherapies to enhance cancer treatment. ImmunoCellular Therapeutics has established licensing agreements with prestigious institutions such as the California Institute of Technology and The Johns Hopkins University. Originally named Optical Molecular Imaging, Inc., the company rebranded in November 2006 and is headquartered in Westlake Village, California.

ViaCyte, Inc. is a regenerative medicine company based in San Diego, California, that specializes in developing cell replacement therapies for diabetes. The company’s primary product candidates, VC-01 and VC-02, aim to treat type 1 diabetes by implanting pancreatic beta-cell precursors derived from stem cells within an encapsulation device. This innovative approach allows the implanted cells to produce insulin in response to blood glucose levels while minimizing the risk of immune rejection, thereby eliminating the need for immunosuppressants. ViaCyte's goal is to provide a long-term solution that can free both type 1 and type 2 diabetes patients from dependence on insulin and reduce associated complications such as hypoglycemia and cardiovascular issues. Founded in 1999, ViaCyte was previously known as Novocell, Inc., and changed its name in 2010. The company also operates an additional facility in Athens, Georgia.

Bluebird Bio is a clinical-stage biotechnology company focused on developing gene therapies to treat severe genetic and rare diseases. Its proprietary lentiviral vector platform enables the creation of potentially curative treatments by genetically modifying patients' cells to address the root cause of these conditions.

VistaGen Therapeutics is a clinical-stage biopharmaceutical company focused on developing medicines for central nervous system disorders. Its product candidates include AV-101, an orally available prodrug in Phase 2 for major depressive disorder and related symptoms; PH94B, a neuroactive nasal spray in preparation for Phase III clinical development for acute treatment of anxiety in adults with social anxiety disorder; and PH10, a neuroactive nasal spray planned for Phase 2b development for major depressive disorder. The company pursues licensing and collaboration arrangements to support development and commercialization with partners such as Pherin Pharmaceuticals, BlueRock Therapeutics, Cato Research, and EverInsight Therapeutics for PH94B in Asia. VistaGen was founded in 1998 and is headquartered in South San Francisco, California.

Senti Biosciences is a biotechnology company focused on synthetic biology. It develops gene circuit platforms to engineer therapeutics, with a focus on improving precision and control in cancer treatments.