California Institute for Regenerative Medicine

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.

Maro Bio is a biotech company.

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.

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.

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.

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.

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.

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.

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.

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.

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 is a clinical-stage immuno-oncology company focused on developing allogeneic stem cell-based platforms to deliver oncolytic viruses for cancer therapy. It aims to empower the immune system to fight cancer using patented delivery technology that carries oncolytic viruses for targeted treatment of difficult-to-treat malignancies. The company’s pipeline includes CLD-101 for recurrent high-grade gliomas, CLD-201 for solid tumors, and CLD-400 for certain lung cancers and metastatic solid tumors, with CLD-301 in discovery. Its allogeneic cell-based delivery system is designed to protect and potentiate virus therapies, enabling broader oncology applications. Calidi Biotherapeutics is based in San Diego and was founded in 2014.

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.

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.

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.

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.

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.

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.

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.

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.