California Institute for Regenerative Medicine

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.

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.

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.

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.

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.

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.

Siren Biotechnology is a biotechnology company focused on cancer treatment through an integrated therapeutic modality that combines adeno-associated virus–based gene therapy with cytokine immunotherapy. The approach delivers targeted cytokines to tumors, aiming to activate both innate and adaptive immune responses while addressing toxicity and off-target effects. By uniting gene delivery with immunotherapy in a single modality, the company seeks to enhance tumor cell elimination and improve long-term anti-tumor immunity, potentially reducing recurrence in cancer patients.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.