California Institute for Regenerative Medicine

Ray Therapeutics is a biotechnology company focused on developing innovative optogenetic gene therapies aimed at treating patients with blinding diseases. The company is dedicated to advancing technology that facilitates the understanding and study of rare retinal diseases. By harnessing optogenetics, Ray Therapeutics aims to empower medical professionals to effectively cure and manage inherited retinal disorders, thereby improving the quality of life for affected individuals.

Tr1x is a biotechnology company focused on developing cellular therapies for the treatment of autoimmune disorders. Utilizing an exclusive platform, Tr1x creates innovative cell therapy products aimed at recalibrating the immune system to restore balance and promote long-term tolerance in patients. The company leverages advancements in genetic engineering and T-cell biology to design scalable and effective curative solutions that target inflammation and autoimmunity. Through its work, Tr1x aims to provide transformative therapies that address the needs of individuals suffering from autoimmune and inflammatory diseases.

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 biotechnology company focused on developing innovative gene therapies for neurological disorders, including chronic pain and ophthalmic diseases. The company utilizes epigenetic regulation tools within a non-immunogenic platform to create its therapies. Navega's lead program addresses chronic pain through the repression of the Nav1.7 gene, a critical pain gatekeeper, achieving pain relief in multiple preclinical models. This novel approach targets the gene's DNA to downregulate the channel without causing permanent mutations. Additionally, Navega's co-lead program offers a mutation-agnostic strategy to combat all forms of retinitis pigmentosa by preventing neuronal degeneration in the eye through in vivo cellular reprogramming. With over $8 million secured in grants from the NIH and CIRM, alongside a robust intellectual property portfolio developed and in-licensed from UCSD, Navega Therapeutics is positioned to advance its pioneering therapies for the treatment of chronic pain and vision loss.

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.

University of California, Irvine is a higher education institution providing diverse academic and cultural events. It also offers a variety of events such as art exhibitions, music performances, healthcare conferences, theatrical productions, and educational seminars to the general public and its student body.

Amplo Biotechnology is a biopharmaceutical company focused on developing innovative Adeno-Associated Viral (AAV) therapies aimed at treating defects related to the neuromuscular junction. These defects can lead to severe health issues, including paralysis and difficulties with breathing and swallowing. The company's lead program, AAV-Dok7, was created by Professor Yuji Yamanashi's research team at the Institute of Medical Science at the University of Tokyo, and it holds promise for addressing various congenital myasthenic syndromes and other rare, severe diseases. Amplo's therapies are administered through intravascular injection, which initiates specific molecular events necessary for effective treatment, thereby enhancing the potential for curative outcomes in affected 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.

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 prominent public research university established in 1920, renowned for its diverse academic programs and distinguished faculty. It attracts students globally, particularly in fields such as medicine, film, and the liberal arts. The UCLA School of Medicine is affiliated with one of the nation's leading hospitals, while the film school is recognized for producing successful filmmakers and actors. The university offers a comprehensive range of disciplines, including education, architecture, engineering, public affairs, and law. UCLA is characterized by a vibrant student life, with numerous organizations and activities available on campus and in the surrounding Westwood area, which features museums, theaters, and dining options. Admissions are competitive, with varying requirements across its programs. UCLA is committed to the creation and dissemination of knowledge, aiming to contribute positively to society.

The University of California (UC) is a renowned public research university system established in 1868, with its flagship campus at UC Berkeley. With over 280,000 students, 227,000 faculty and staff, and more than 2 million alumni worldwide, UC is a global leader in education and research. It offers a wide range of undergraduate, graduate, and professional programs across its 10 campuses, fostering interdisciplinary study and collaboration in advanced learning environments. UC's diverse academic community contributes significantly to the economic and social well-being of California and the nation, driving innovations in fields like biotechnology, computer science, and environmental sustainability. Additionally, UC's athletic teams, known as the Golden Bears, represent the university in various sports at the NCAA level.

Neurona Therapeutics Inc. is a pre-clinical biotechnology company based in South San Francisco, California, focused on developing cell-based therapies for neurological disorders. Founded by a team of neuroscientists and stem cell experts from the University of California, San Francisco, Neurona aims to create therapeutic compositions of specific types of neurons for targeted delivery into the injured nervous system. The company is dedicated to addressing chronic neurological diseases through regenerative neural cell therapies, which hold the potential for single-dose curative treatments. Drawing on nearly two decades of research, Neurona believes that certain subpopulations of neurons can effectively integrate and repair dysfunctional neural circuits, thus offering new hope for patients facing significant unmet medical needs.

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 dedicated to providing comprehensive cancer treatment. Established in 1913, it is recognized as one of only 45 comprehensive cancer centers nationwide by the National Cancer Institute. The organization offers a range of services, including access to expert doctors, streamlined appointment scheduling, and participation in clinical trials aimed at improving patient outcomes. Its diverse community consists of research associates, scientists, medical professionals, and support staff who work collaboratively to enhance the lives of individuals affected by cancer. City of Hope's commitment to patient care and innovative research underscores its mission to advance cancer treatment and support those in need.

Aspen Neuroscience, Inc. is a biotechnology company based in San Diego, California, focused on developing innovative therapies for neurological conditions, particularly Parkinson's disease. Founded in 2018, the company specializes in induced pluripotent stem cells (iPSCs) to create patient-specific cell therapies. Its product pipeline includes ANPD001, an autologous neuron replacement therapy for sporadic Parkinson's disease, and ANPD002, a gene-edited version targeting familial forms of the disease. By combining genomic approaches with stem cell biology, Aspen Neuroscience aims to address significant unmet medical needs and modify the progression of Parkinson's disease through personalized and restorative treatments.

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 is a biotechnology company focused on developing innovative optogenetic gene therapies aimed at treating patients with blinding diseases. The company is dedicated to advancing technology that facilitates the understanding and study of rare retinal diseases. By harnessing optogenetics, Ray Therapeutics aims to empower medical professionals to effectively cure and manage inherited retinal disorders, thereby improving the quality of life for affected individuals.

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 is a biotechnology company focused on research and development aimed at creating innovative therapeutics for cancer treatment. The company is dedicated to developing first-in-class compounds that target new drug mechanisms in cancer biology, employing advanced techniques in molecular cancer biology, epigenetics, and cancer stem cell research. Currently, Tachyon is conducting Phase 1 clinical trials to evaluate the safety, pharmacokinetics, and anti-tumor effectiveness of its lead candidate, TACH101, in patients with advanced cancer. Through a commitment to high-quality and scientifically sound practices, Tachyon aims to provide novel treatment options that unlock new pathways for addressing complex cancer cases.

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 is a biotechnology company dedicated to developing therapies for neurodegenerative movement disorders. The company specializes in providing off-the-shelf neuron replacement medicines that serve as effective disease-modifying treatments for various neurological conditions. By focusing on innovative approaches, Kenai Therapeutics aims to equip physicians with advanced techniques for treating disorders such as Parkinson's disease, ultimately enhancing patient care and outcomes in the field of neurology.

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 Corporation is a biotechnology company focused on developing an autologous cell therapy platform aimed at treating a range of human diseases through its proprietary Immune System Programming (ISP) technology. Founded in 2009 and based in Seattle, Washington, Immusoft specializes in modifying the DNA of a patient's immune cells to create biofactories capable of producing gene-encoded medicines, including essential proteins and enzymes that may be missing or defective. The company's innovative approach involves reprogramming B cells, a type of immune cell, which enables them to manufacture and secrete various antibodies. This process not only provides a potential long-term solution for patients but also positions Immusoft at the forefront of biotechnology in addressing rare and infectious diseases.

Angiocrine Bioscience is a biotechnology company focused on developing innovative cellular therapies for regenerative medicine and hematopoietic stem cell gene therapy. The company has created a unique cellular platform that enhances the amplification of stem and progenitor cells in both human and mouse models by mimicking the in vivo vascular niche in vitro. This technology allows for an unprecedented scale of cell expansion without the need for expensive media additives, making it suitable for various therapeutic, diagnostic, and research applications. Angiocrine Bioscience aims to leverage its E-CEL technology, which involves genetically modified cultured endothelial cells, to advance treatment options in regenerative medicine and improve therapeutic procedures for physicians.

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.

University of California, Irvine is a higher education institution providing diverse academic and cultural events. It also offers a variety of events such as art exhibitions, music performances, healthcare conferences, theatrical productions, and educational seminars to the general public and its student body.

Lutris Pharma is a biopharmaceutical company based in Tel Aviv-Yafo, Israel, focused on developing innovative therapies to enhance the effectiveness of anti-cancer treatments. Established in 2015, the company has created a proprietary small molecule drug known as LUT014, which acts as a topical B-Raf inhibitor. This drug facilitates the use of epidermal growth factor receptor (EGFR) inhibitors while minimizing the common cutaneous side effects typically associated with such treatments. By addressing these side effects, Lutris Pharma aims to improve both the efficacy of anti-cancer therapies and the overall quality of life for patients undergoing treatment 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 Inc. is a pre-clinical biotechnology company based in South San Francisco, California, focused on developing cell-based therapies for neurological disorders. Founded by a team of neuroscientists and stem cell experts from the University of California, San Francisco, Neurona aims to create therapeutic compositions of specific types of neurons for targeted delivery into the injured nervous system. The company is dedicated to addressing chronic neurological diseases through regenerative neural cell therapies, which hold the potential for single-dose curative treatments. Drawing on nearly two decades of research, Neurona believes that certain subpopulations of neurons can effectively integrate and repair dysfunctional neural circuits, thus offering new hope for patients facing significant unmet medical needs.

Ray Therapeutics is a biotechnology company focused on developing innovative optogenetic gene therapies aimed at treating patients with blinding diseases. The company is dedicated to advancing technology that facilitates the understanding and study of rare retinal diseases. By harnessing optogenetics, Ray Therapeutics aims to empower medical professionals to effectively cure and manage inherited retinal disorders, thereby improving the quality of life for affected individuals.

BioEclipse Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in San Francisco, California, focused on developing innovative immune-oncology therapeutics for cancer treatment. The company specializes in a proprietary approach that combines activated immune cells with an adapted oncolytic virus, which selectively targets and destroys malignant cells. This method enhances the immune system's ability to recognize and attack tumors, leading to curative treatments across a wide range of tumor types. BioEclipse aims to improve clinical outcomes and the quality of life for cancer patients, striving to advance the field of oncology toward making cancer a curable disease. Established in 2006 and previously known as ConcentRx Inc., the company rebranded in 2017 to reflect its commitment to pioneering immune therapies without requiring the reengineering of patients' own cells.

Navega Therapeutics is a preclinical stage biotechnology company focused on developing innovative gene therapies for neurological disorders, including chronic pain and ophthalmic diseases. The company utilizes epigenetic regulation tools within a non-immunogenic platform to create its therapies. Navega's lead program addresses chronic pain through the repression of the Nav1.7 gene, a critical pain gatekeeper, achieving pain relief in multiple preclinical models. This novel approach targets the gene's DNA to downregulate the channel without causing permanent mutations. Additionally, Navega's co-lead program offers a mutation-agnostic strategy to combat all forms of retinitis pigmentosa by preventing neuronal degeneration in the eye through in vivo cellular reprogramming. With over $8 million secured in grants from the NIH and CIRM, alongside a robust intellectual property portfolio developed and in-licensed from UCSD, Navega Therapeutics is positioned to advance its pioneering therapies for the treatment of chronic pain and vision loss.

City of Hope is a non-profit clinical research center dedicated to providing comprehensive cancer treatment. Established in 1913, it is recognized as one of only 45 comprehensive cancer centers nationwide by the National Cancer Institute. The organization offers a range of services, including access to expert doctors, streamlined appointment scheduling, and participation in clinical trials aimed at improving patient outcomes. Its diverse community consists of research associates, scientists, medical professionals, and support staff who work collaboratively to enhance the lives of individuals affected by cancer. City of Hope's commitment to patient care and innovative research underscores its mission to advance cancer treatment and support those in need.

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 Corporation is a biotechnology company focused on developing an autologous cell therapy platform aimed at treating a range of human diseases through its proprietary Immune System Programming (ISP) technology. Founded in 2009 and based in Seattle, Washington, Immusoft specializes in modifying the DNA of a patient's immune cells to create biofactories capable of producing gene-encoded medicines, including essential proteins and enzymes that may be missing or defective. The company's innovative approach involves reprogramming B cells, a type of immune cell, which enables them to manufacture and secrete various antibodies. This process not only provides a potential long-term solution for patients but also positions Immusoft at the forefront of biotechnology in addressing rare and infectious diseases.

Juvena Therapeutics, Inc. is a biopharmaceutical company based in San Mateo, California, founded in 2017. The company focuses on developing protein-based therapeutics aimed at promoting tissue rejuvenation, primarily targeting age-related conditions such as sarcopenia, wound healing, and neural regeneration. Juvena Therapeutics utilizes a drug discovery platform that screens for restored cellular functions to create innovative therapeutics that facilitate tissue regeneration and repair. By decoding secreted proteins, the company aims to address various chronic and rare diseases, ultimately enhancing the quality of life for elderly patients.

Rocket Pharmaceuticals, Inc. is a biotechnology company dedicated to developing innovative gene therapies for rare and severe pediatric diseases. The company specializes in lentiviral vector and adeno-associated virus platforms to target genetic disorders. Its lead program focuses on treating Fanconi Anemia, a serious condition that leads to bone marrow failure. In addition to this, Rocket is actively researching therapies for several other bone marrow-derived disorders, including Pyruvate Kinase Deficiency, Leukocyte Adhesion Deficiency-I, and Infantile Malignant Osteopetrosis. Furthermore, the company is developing an AAV-based gene therapy for Danon Disease, a multi-organ disorder associated with increased risk of cardiovascular complications. To advance its research, Rocket Pharmaceuticals maintains numerous collaborations and license agreements with prestigious institutions, enhancing its capabilities in gene therapy development. The company is headquartered in New York, New York.

Rocket Pharmaceuticals, Inc. is a biotechnology company dedicated to developing innovative gene therapies for rare and severe pediatric diseases. The company specializes in lentiviral vector and adeno-associated virus platforms to target genetic disorders. Its lead program focuses on treating Fanconi Anemia, a serious condition that leads to bone marrow failure. In addition to this, Rocket is actively researching therapies for several other bone marrow-derived disorders, including Pyruvate Kinase Deficiency, Leukocyte Adhesion Deficiency-I, and Infantile Malignant Osteopetrosis. Furthermore, the company is developing an AAV-based gene therapy for Danon Disease, a multi-organ disorder associated with increased risk of cardiovascular complications. To advance its research, Rocket Pharmaceuticals maintains numerous collaborations and license agreements with prestigious institutions, enhancing its capabilities in gene therapy development. The company is headquartered in New York, New York.

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, Inc. is a medical research and development company based in Morrisville, North Carolina, focusing on the creation and commercialization of human tissue-based products for regenerative medicine and vascular surgery. Established in 2004, the company specializes in developing acellular extracellular matrices derived from banked vascular smooth muscle cells, which are decellularized to prevent rejection. These engineered tissues are designed for use as vascular grafts, providing solutions for patients requiring vascular repair or replacement. Humacyte aims to deliver universally implantable bioengineered human tissues and organs, addressing various diseases, injuries, and chronic conditions while advancing the field of medicine.

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 biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which aims to modify a patient's hematopoietic stem cells to create a personalized treatment through a single administration. Orchard's product portfolio includes Strimvelis, the first gene therapy approved by the European Medicines Agency for adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company is advancing several clinical programs, including OTL-101 for ADA-SCID, OTL-200 for metachromatic leukodystrophy, OTL-103 for Wiskott-Aldrich syndrome, OTL-102 for X-linked chronic granulomatous disease, and OTL-300 for transfusion-dependent beta-thalassemia. Additionally, Orchard has a robust preclinical pipeline targeting various mucopolysaccharidosis types. Founded in 2015, Orchard Therapeutics collaborates with leading institutions in the field to enhance its research and development efforts.

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, Inc. is a medical research and development company based in Morrisville, North Carolina, focusing on the creation and commercialization of human tissue-based products for regenerative medicine and vascular surgery. Established in 2004, the company specializes in developing acellular extracellular matrices derived from banked vascular smooth muscle cells, which are decellularized to prevent rejection. These engineered tissues are designed for use as vascular grafts, providing solutions for patients requiring vascular repair or replacement. Humacyte aims to deliver universally implantable bioengineered human tissues and organs, addressing various diseases, injuries, and chronic conditions while advancing the field of medicine.

Capricor Therapeutics is a clinical-stage biotechnology company dedicated to the discovery, development, and commercialization of biological therapies for various diseases, particularly focusing on Duchenne muscular dystrophy (DMD) and other rare disorders. The company is advancing its lead product candidate, CAP-1002, an allogeneic cell therapy currently undergoing phase II clinical trials for late-stage DMD. Additionally, CAP-1002 has been explored in trials for other cardiac conditions, including heart failure and post-myocardial infarction complications. Capricor is also developing CAP-2003, which is in pre-clinical stages for treating inflammatory conditions. Founded in 2005, the company is headquartered in Beverly Hills, 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, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on researching, developing, and commercializing gene therapies for severe genetic diseases and cancer. The company's gene therapy programs include LentiGlobin, aimed at treating β-thalassemia and sickle cell disease, and Lenti-D for cerebral adrenoleukodystrophy. In oncology, bluebird bio is developing chimeric antigen receptor T (CAR T) cell therapies, specifically bb2121 and bb21217, for multiple myeloma. The company has formed strategic collaborations with several organizations, including Bristol-Myers Squibb and Regeneron Pharmaceuticals, to advance gene therapies in oncology and immune cell therapies. Additionally, bluebird bio collaborates with various partners to explore T cell receptor product candidates and in vivo genome editing treatments for genetic disorders. Established in 1992 and originally known as Genetix Pharmaceuticals, the company transitioned to its current name in 2010.

VistaGen Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for central nervous system disorders, particularly depressive and social anxiety disorders. The company's lead product candidate, AV-101, is an orally available treatment currently in Phase 2 development for major depressive disorder, targeting patients who have not adequately responded to standard antidepressants. Additionally, VistaGen is advancing PH94B, a neuroactive nasal spray, which is preparing for Phase III clinical trials for the acute treatment of anxiety in adults with social anxiety disorder. Another product in its pipeline, PH10, is being planned for Phase 2b development as a treatment for major depressive disorder. VistaGen has established licensing and collaboration agreements to facilitate the clinical development and commercialization of its products, including a strategic partnership for PH94B in Greater China, South Korea, and Southeast Asia. Founded in 1998, VistaGen is headquartered in South San Francisco, California.

Senti Bio, established in 2016 and headquartered in South San Francisco, specializes in synthetic biotechnology for therapeutic development. The company's core focus is engineering gene circuits, a platform technology that enables precise control over cellular behavior. Senti Bio's innovative approach allows for the creation of therapies with enhanced specificity and control, particularly in oncology. Their product pipeline includes SENTI-202, a targeted cancer therapy using gene-engineered allogeneic CAR-NK cells designed to eliminate cancer cells while sparing healthy bone marrow, and SENTI-301A for treating hepatocellular carcinoma.