Ray Therapeutics is a company that creates novel optogenetic gene therapies for patients with blinding diseases. The company develops technology to study and learn about rare retinal diseases, allowing doctors to cure and treat inherited retinal diseases using optogenetics.
Tr1x is a biotechnology company focused on developing cellular therapies for the treatment of autoimmune disorders. The company leverages its proprietary platform to create advanced cell therapy products aimed at recalibrating the immune system and restoring homeostasis. By exploring genetic engineering and T-cell biology, Tr1x seeks to develop scalable and effective cellular cures that target inflammation and autoimmunity. Its mission is to provide innovative solutions for autoimmune and inflammatory diseases, ultimately transforming the lives of patients affected by these conditions.
Cure Rare Disease is an independent non-profit foundation located in Woodbridge, Connecticut, established in 2017. The foundation focuses on developing custom therapeutics for individuals with rare genetic disorders, particularly those facing muscular dystrophy. Through collaborations with leading researchers and clinicians, Cure Rare Disease creates effective, life-saving treatments tailored to the specific needs of patients. The organization also serves as a resource hub for families and advocates, providing critical information and support. By leveraging partnerships with charitable donors, the foundation aims to accelerate research and improve the quality of life for those diagnosed with conditions deemed untreatable.
Navega Therapeutics is a preclinical stage company focused on developing gene therapies for neurological disorders, including chronic pain, and ophthalmic diseases. The company utilizes epigenetic regulation tools through a non-immunogenic platform to achieve its therapeutic goals. Its lead program targets chronic pain by repressing the Nav1.7 gene, which acts as a gatekeeper for pain sensation, demonstrating pain relief in multiple preclinical models. Unlike traditional approaches that target proteins or RNA, Navega's method focuses on the DNA of Nav1.7, allowing for downregulation without permanent genomic mutations. Additionally, the co-lead program addresses retinitis pigmentosa through a mutation-agnostic strategy aimed at preventing neuronal degeneration in the eye via in vivo cellular reprogramming. Navega Therapeutics has secured over $8 million in grants from the NIH and CIRM, and with a robust intellectual property portfolio developed in-house and in-licensed from UCSD, the company is positioned to advance innovative treatments in the future.
Tenaya Therapeutics is a developer of novel therapies designed to offer treatment for heart disease. The company's therapies address heart failure through multipronged efforts that target the fundamental cellular pathologies present in diseased cardiac muscle and that leverage cutting-edge research in cardiac development and regeneration, enabling physicians to regenerate heart tissue, and additional programs focused on cardiomyopathies.
NysnoBio is a biopharmaceutical company focused on developing innovative therapies for Parkinson's disease through the exploitation of the Parkin protein and its E3 ubiquitin ligase activity. The company gained recognition for its pioneering research, including the discovery of the Parkin gene and its role in Parkinson's, as well as demonstrating its enzymatic structure and utilizing adeno-associated virus (AAV) vectors in gene therapy. NysnoBio is dedicated to creating small molecule activators of Parkin and employing gene therapy strategies aimed at preserving dopamine neurons in affected patients. Through these efforts, the company seeks to provide effective treatment options for individuals suffering from Parkinson's disease, ultimately improving their quality of life.
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.
Maro Bio is a biotech company.
The University of California, Los Angeles (UCLA), established in 1920, is a prominent public research university located in Los Angeles, California. Known for its distinguished faculty and diverse academic offerings, UCLA attracts students globally, particularly to its renowned School of Medicine and esteemed Film and Theatre department. The university provides a comprehensive range of programs in liberal arts, sciences, humanities, and professional fields such as Education, Architecture, Engineering, Public Affairs, and Law. UCLA emphasizes a vibrant student life with numerous organizations and activities, complemented by the cultural richness of its Westwood surroundings, which include museums, theaters, and dining options. The university also manages an endowment to support its educational mission, ensuring a reliable source of funding for current and future initiatives. Admissions to UCLA can be highly competitive, varying by program, with financial assistance available for qualifying students through loans, grants, and scholarships.
CellFE is a company developing a microfluidics based device to deliver gene-editing molecules. CellFE developed microfluidic device and process that can efficiently deliver gene-editing molecules into the human cells.
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’s pipeline includes various NK- and T-cell immuno-oncology programs, such as FT516 for acute myeloid leukemia and B-cell lymphoma, FT596 for B-cell lymphoma and chronic lymphocytic leukemia, and FT538 for multiple myeloma. Additional programs include FT500, FT576, FT819, and FT-ONO1, targeting solid tumors and hematologic malignancies. Fate Therapeutics is also collaborating with Ono Pharmaceutical Co. Ltd. and Janssen Biotech, Inc. to advance its off-the-shelf iPSC-derived CAR T-cell therapies and other innovative treatments. Founded in 2007, the company leverages insights from leading scientists to create and modulate stem cells for therapeutic applications, aiming to restore health through advanced cellular therapies.
ReviR Therapeutics is a genomics-focused company that develops innovative RNA-based medicines aimed at treating cancer, rare genetic disorders, and infectious diseases. The company leverages an artificial intelligence-driven platform to enhance the discovery of therapeutics that target human RNAs. By combining advanced computational techniques with genomic insights, ReviR Therapeutics strives to create transformative therapies that can significantly impact patient care and improve health outcomes for millions.
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.
Diseases of the neuromuscular junction have profound health effects including paralysis, breathing and swallowing difficulties. Amplo Biotechnology plans to develop multiple, potentially curative, Adeno-Associated Viral (AAV) therapies for the neuromuscular junction with an initial focus on congenital myasthenic syndromes. Amplo’s lead program, AAV-Dok7, was developed by Professor Yuji Yamanashi's group of the Institute of Medical Science at the University of Tokyo, and is applicable to multiple rare, severe diseases.
Neurona Therapeutics Inc. is a biotechnology company based in South San Francisco, California, that specializes in developing cell-based therapies for neurological disorders. Founded by neuroscientists and stem cell pioneers at The University of California, San Francisco, the company focuses on creating therapeutic compositions of specific types of neurons for targeted delivery into the injured nervous system. Drawing on nearly two decades of research, Neurona seeks to harness particular subpopulations of neurons capable of integrating and repairing dysregulated neural circuits. With a dedicated team of scientists and advisors, the company aims to accelerate the development of breakthrough treatments for patients suffering from significant unmet medical needs in the realm of chronic neurological diseases.
AcuraStem Inc is a biotechnology company, founded in 2016 and based in Monrovia, California, that focuses on developing a precision medicine platform aimed at addressing Amyotrophic Lateral Sclerosis (ALS). The company utilizes complex cellular models derived from ALS patients to evaluate disease progression and test existing FDA-approved therapeutics and their combinations. By employing advanced cellular reprogramming and artificial intelligence technologies, AcuraStem seeks to transform the standard of care for neurodegenerative diseases, enabling drug discovery companies to assess their therapeutic candidates more effectively. The team comprises experts, including PhDs, professors, and experienced professionals from the pharmaceutical industry, all dedicated to finding innovative treatments for this challenging health issue.
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.
Aspen Neuroscience, Inc. is a biotechnology company focused on developing induced pluripotent stem cells (iPSCs) to create patient-specific therapies for neurological conditions, particularly Parkinson's disease. Founded in 2018 and based in San Diego, California, the company is advancing its pipeline of products, which includes ANPD001, an autologous neuron replacement therapy for sporadic Parkinson's disease, and ANPD002, a gene-edited autologous neuron replacement therapy targeting familial forms of the disease. Aspen Neuroscience employs innovative genomic approaches in conjunction with stem cell biology to address diseases with significant unmet medical needs, aiming to provide restorative cell therapies that can modify the progression of Parkinson's disease and potentially extend to other affected organs.
Luxa Biotechnology (LuxaBio) is a clinical-stage biotechnology firm developing a unique adult RPE stem cell (RPESC) therapy for dry AMD.
Denovo Biopharma is a privately held biotechnology company focused on personalizing drug development through innovative biomarker discovery. The company has developed a unique platform and algorithm that enables the retrospective identification of genomic biomarkers using archived clinical samples. This technology is particularly beneficial for late-stage clinical drugs that have previously shown unsatisfactory efficacy, allowing for the identification of biomarkers that correlate with patient responsiveness to specific drug candidates. By leveraging this approach, Denovo Biopharma assists biotech and pharmaceutical companies in designing targeted clinical trials aimed at enhancing drug efficacy and minimizing adverse effects. The platform's applications extend across various therapeutic areas, including oncology, metabolic disorders, cardiology, immunology, and neurology.
Children’s Hospital Los Angeles is a first and largest hospital dedicated specifically to treating children throughout Southern California. CHLA has been named the best children’s hospital on the West Coast and is among the top five in the nation for clinical excellence with its selection to the prestigious U.S. News & World Report Honor Roll. CHLA is home to The Saban Research Institute, one of the largest and most productive pediatric research facilities in the United States.
ImmPACT Bio USA Inc. is a cell therapy company focused on developing engineered T-cells specifically designed to target and treat solid tumors. By utilizing advanced technology, the company aims to create chimeric antigen receptor (CAR) T-cell therapies that exhibit tumor specificity, allowing them to induce cytotoxicity only when interacting with tumor cells while sparing healthy tissues. This selective targeting mitigates the safety risks commonly associated with existing CAR-T therapies, providing a promising approach to cancer treatment that enhances efficacy and reduces potential harm to noncancerous cells.
Ray Therapeutics is a company that creates novel optogenetic gene therapies for patients with blinding diseases. The company develops technology to study and learn about rare retinal diseases, allowing doctors to cure and treat inherited retinal diseases using optogenetics.
Siren Biotechnology is a biotech company focused on innovating cancer treatment through a unique gene therapy platform. By integrating adeno-associated virus (AAV) gene therapy with cytokine immunotherapy, Siren Biotechnology has developed a novel therapeutic approach. This combination aims to address significant challenges in cancer treatment, including delivery methods, efficacy, and toxicity, ultimately enhancing the elimination of tumor cells and stimulating anti-tumor immunity. Through this redesigned modality, the company seeks to transform the landscape of cancer therapies, offering new hope for patients facing this disease.
Eureka Therapeutics, Inc. is a biotechnology company specializing in immunotherapies for cancer treatment. Founded in 2006 and located in Emeryville, California, the company focuses on developing antibody-TCR (AbTCR) and T-cell therapies aimed at solid tumors and hematological malignancies. Eureka has created the ARTEMIS AbTCR T-cell receptor platform and the E-ALPHA antibody discovery platform, the latter being one of the largest human-derived antibody phage libraries, which generates target-specific antibodies, including T cell receptor-mimic antibodies against intracellular targets. The company targets cancer-specific antigens, onco-fetal antigens, mutated oncogenes, and viral oncogenes. To enhance its research capabilities, Eureka Therapeutics has established a strategic collaboration with Lyell Immunopharma, aiming to improve the safety profile of T-cell therapies and develop novel treatments for cancer.
Lineage Cell Therapeutics is a clinical-stage biotechnology company dedicated to developing innovative therapies for degenerative diseases. The company's key product candidates include OpRegen, a therapy aimed at treating dry age-related macular degeneration, currently in Phase I/IIa clinical trials. Another significant candidate is OPC1, an oligodendrocyte progenitor cell therapy being tested for acute spinal cord injuries. Additionally, Lineage is developing VAC2, an allogeneic cancer immunotherapy targeting non-small cell lung cancer, which is also in early clinical trials. The company is working on Renevia, a hyaluronan-based hydrogel for HIV-associated facial lipoatrophy, and has various products in development for orthopedic applications, oncology, and neurological disorders. Founded in 1990 and headquartered in Carlsbad, California, Lineage Cell Therapeutics has also established collaborations to enhance its research and development efforts.
Ossium Health, Inc. is engaged in the development and manufacturing of cell therapy products aimed at treating leukemia, other blood cancers, and tissue damage, as well as addressing radiation poisoning. The company offers a range of innovative therapies, including HPC, Marrow, which features HLA-matched bone marrow stem cells for hematopoietic reconstitution in blood cancer patients; Chimera, which enhances organ transplantation; and Chymalis, composed of mesenchymal stem cells that promote the healing of damaged bone and muscle tissue. Additionally, Ossium provides bone marrow cells for research purposes in oncology and immunology. Founded in 2016 and headquartered in San Francisco, California, Ossium Health focuses on improving health outcomes and longevity through advanced 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 developing innovative therapeutics for cancer treatment. The firm specializes in creating selective compounds that target significant drug targets within cancer biology, employing advanced methodologies in molecular cancer biology, epigenetics, and cancer stem cell biology. Currently, Tachyon is conducting Phase 1 clinical trials to evaluate the safety, pharmacokinetics, and anti-tumor effectiveness of its lead compound, TACH101, in patients with advanced cancer. The company's research and development efforts aim to unlock new pathways for treating challenging cancer types, ensuring that its programs are advanced in a timely and scientifically rigorous manner.
Genascence is a clinical-stage biotechnology company focused on gene therapy to address prevalent musculoskeletal diseases, particularly osteoarthritis. This condition is the leading cause of disability and chronic pain, posing significant health risks, including opioid addiction. Genascence's innovative approach aims to improve the treatment of osteoarthritis by alleviating symptoms and slowing disease progression, ultimately enhancing patient care and recovery outcomes. The company's research and development efforts are directed toward providing effective solutions for both common and rare disorders associated with musculoskeletal health.
Kenai Therapeutics is a biotechnology company dedicated to developing therapies for neurodegenerative movement disorders. The company specializes in off-the-shelf neuron replacement therapies, which serve as effective disease-modifying treatments for various neurological conditions. By providing innovative therapeutic options, Kenai Therapeutics aims to equip healthcare professionals with advanced methods for treating disorders such as Parkinson's disease.
Rubedo Life Sciences, Inc. is a biopharmaceutical company based in Sunnyvale, California, focused on developing therapies that target senescent cells responsible for various age-related diseases. Established in 2018, the company utilizes its proprietary ALEMBIC drug discovery platform to create a pipeline of innovative therapeutic candidates. These candidates include novel small molecules designed to selectively address senescent cells, which contribute to the progression of several chronic conditions, including pulmonary, dermatological, oncological, neurodegenerative, and fibrotic diseases. Rubedo's leadership team comprises experienced professionals from the fields of chemistry, technology, and life sciences, bringing a wealth of knowledge in drug development and commercialization.
Immusoft Corporation is a biotechnology company based in Seattle, Washington, focused on developing autologous cell therapies for various human diseases through its proprietary Immune System Programming (ISP) technology. Founded in 2009, the company modifies a patient's immune cells, specifically B cells, to create miniature drug factories that produce gene-encoded medicines, or biologics. By reprogramming these cells, Immusoft's technology enables the in vivo delivery of therapeutics that can replace missing or defective enzymes and proteins. The core ISP technology was initially developed at the California Institute of Technology and is exclusively licensed to Immusoft. This innovative approach aims to improve treatments for rare and infectious diseases by harnessing the body's own immune system.
Angiocrine Bioscience has developed a cellular platform for amplifying stem and progenitor cells, in both human and mouse models, by recapitulating the proliferative stimulation of the in vivo vascular niche in vitro. The expansion capacity is on an unprecedented scale when compared to any other competing technology, and it is achieved without the requirement for costly media additives. This process can meet the needs of a broad range of therapeutic, diagnostic, and research applications. We look forward to the expansive application of this technology to help fulfill the promise of tissue-specific engineering and organ-regeneration.
Calidi Biotherapeutics, Inc. is a clinical-stage company based in San Diego, California, that specializes in immuno-oncology services. Since its incorporation in 2014, the company has focused on developing innovative oncolytic virus-based immunotherapies aimed at treating challenging malignancies. Calidi utilizes a patented allogeneic cell-based delivery system designed to enhance the effectiveness of oncolytic viruses, enabling targeted therapies that provide new treatment options for patients with cancer. Through its advanced stem cell-based platforms, Calidi aims to pioneer the commercialization of these novel therapies, contributing to the evolution of cancer treatment.
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, founded in 2015. The company focuses on developing therapeutic drugs aimed at enhancing the effectiveness of anti-cancer therapies. Its lead product, LUT014, is a proprietary small molecule B-Raf inhibitor designed to facilitate the administration of epidermal growth factor receptor (EGFR) inhibitors. This innovative approach helps mitigate the common cutaneous side effects associated with EGFR treatments, thereby improving the overall quality of life for patients undergoing cancer therapy.
Greenstone Biosciences is a drug discovery platform focused on accelerating the development of effective therapies for patients with urgent unmet medical needs. The company leverages advanced technologies, including artificial intelligence and human-induced pluripotent stem cells, to transform the traditional drug discovery process. By integrating these innovative approaches, Greenstone aims to streamline what is typically a slow and high-failure endeavor into a more rapid and efficient system, ultimately delivering safe and effective therapeutics to patients.
Excision BioTherapeutics Inc. is a life science company based in Philadelphia, Pennsylvania, established in 2015. The company specializes in developing 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 is committed to advancing these therapeutics to ensure they are safe and effective, with the goal of significantly improving the lives of individuals affected by viral infections worldwide.
Neurona Therapeutics Inc. is a biotechnology company based in South San Francisco, California, that specializes in developing cell-based therapies for neurological disorders. Founded by neuroscientists and stem cell pioneers at The University of California, San Francisco, the company focuses on creating therapeutic compositions of specific types of neurons for targeted delivery into the injured nervous system. Drawing on nearly two decades of research, Neurona seeks to harness particular subpopulations of neurons capable of integrating and repairing dysregulated neural circuits. With a dedicated team of scientists and advisors, the company aims to accelerate the development of breakthrough treatments for patients suffering from significant unmet medical needs in the realm of chronic neurological diseases.
Ray Therapeutics is a company that creates novel optogenetic gene therapies for patients with blinding diseases. The company develops technology to study and learn about rare retinal diseases, allowing doctors to cure and treat inherited retinal diseases using optogenetics.
BioEclipse Therapeutics is a clinical-stage biopharmaceutical company based in San Francisco, California, focused on developing curative immuno-oncology therapeutics for cancer patients. The company’s flagship product, CRX-100, represents a novel combination therapy that enhances the immune system's ability to combat cancer without the need to reengineer patients' cells. By utilizing a proprietary approach that combines activated immune cells with an adapted oncolytic virus, BioEclipse Therapeutics aims to selectively target and destroy malignant cells while improving tumor visibility to the immune system. The company's mission is to advance clinical outcomes and enhance the quality of life for cancer patients, with the ultimate goal of transforming cancer into a curable disease. BioEclipse Therapeutics was established in 2006 and was previously known as ConcentRx Inc before rebranding in 2017.
Navega Therapeutics is a preclinical stage company focused on developing gene therapies for neurological disorders, including chronic pain, and ophthalmic diseases. The company utilizes epigenetic regulation tools through a non-immunogenic platform to achieve its therapeutic goals. Its lead program targets chronic pain by repressing the Nav1.7 gene, which acts as a gatekeeper for pain sensation, demonstrating pain relief in multiple preclinical models. Unlike traditional approaches that target proteins or RNA, Navega's method focuses on the DNA of Nav1.7, allowing for downregulation without permanent genomic mutations. Additionally, the co-lead program addresses retinitis pigmentosa through a mutation-agnostic strategy aimed at preventing neuronal degeneration in the eye via in vivo cellular reprogramming. Navega Therapeutics has secured over $8 million in grants from the NIH and CIRM, and with a robust intellectual property portfolio developed in-house and in-licensed from UCSD, the company is positioned to advance innovative treatments in the future.
Cytonus Therapeutics, Inc. is a biotechnology company founded in 2018 and based in Columbus, Ohio. The company focuses on developing immune-oncology and gene therapies aimed at treating devastating and rare diseases. Cytonus combines artificial intelligence-driven drug discovery with a unique delivery system known as Cargocyte, which utilizes synthetic biology for precision treatment. The company's mission centers on addressing immune-related disorders through innovative approaches that enable targeted delivery and in vivo production of first-in-class therapeutics.
Immusoft Corporation is a biotechnology company based in Seattle, Washington, focused on developing autologous cell therapies for various human diseases through its proprietary Immune System Programming (ISP) technology. Founded in 2009, the company modifies a patient's immune cells, specifically B cells, to create miniature drug factories that produce gene-encoded medicines, or biologics. By reprogramming these cells, Immusoft's technology enables the in vivo delivery of therapeutics that can replace missing or defective enzymes and proteins. The core ISP technology was initially developed at the California Institute of Technology and is exclusively licensed to Immusoft. This innovative approach aims to improve treatments for rare and infectious diseases by harnessing the body's own immune system.
Juvena Therapeutics, Inc. is a biopharmaceutical company based in San Mateo, California, founded in 2017. The company specializes in developing protein-based therapeutics aimed at promoting tissue rejuvenation and addressing age-related diseases in the elderly. Its innovative platform focuses on discovering novel therapeutics that restore cellular functions, targeting conditions such as sarcopenia, wound healing, and neural regeneration. By decoding secreted proteins, Juvena Therapeutics aims to provide solutions for chronic and rare diseases, enhancing the quality of life for aging populations.
Rocket Pharmaceuticals is a biotechnology company dedicated to developing gene therapies aimed at treating rare pediatric diseases. The company's primary focus is on its lead program, a lentiviral-based gene therapy for Fanconi Anemia, a genetic disorder that leads to bone marrow failure. In addition to this, Rocket is advancing therapies for several other conditions, including Pyruvate Kinase Deficiency, Leukocyte Adhesion Deficiency-I, and Infantile Malignant Osteopetrosis, all of which are currently in various stages of clinical and preclinical trials. The company is also engaged in an adeno-associated virus program targeting Danon disease, a serious multi-organ disorder. Rocket Pharmaceuticals collaborates with various academic and research institutions, enhancing its research capabilities and expanding its therapeutic portfolio. The company is based in New York, New York.
Rocket Pharmaceuticals is a biotechnology company dedicated to developing gene therapies aimed at treating rare pediatric diseases. The company's primary focus is on its lead program, a lentiviral-based gene therapy for Fanconi Anemia, a genetic disorder that leads to bone marrow failure. In addition to this, Rocket is advancing therapies for several other conditions, including Pyruvate Kinase Deficiency, Leukocyte Adhesion Deficiency-I, and Infantile Malignant Osteopetrosis, all of which are currently in various stages of clinical and preclinical trials. The company is also engaged in an adeno-associated virus program targeting Danon disease, a serious multi-organ disorder. Rocket Pharmaceuticals collaborates with various academic and research institutions, enhancing its research capabilities and expanding its therapeutic portfolio. The company is based in New York, New York.
Poseida Therapeutics 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 utilizes non-viral gene engineering technologies to create a pipeline of targeted therapies, including both autologous and allogeneic chimeric antigen receptor T cell (CAR-T) product candidates aimed at treating hematological malignancies and solid tumors. Additionally, Poseida is advancing gene therapies for rare and life-threatening conditions, such as Ornithine transcarbamylase deficiency, methylmalonic acidemia, and various genetic liver diseases. Founded in 2014, Poseida is committed to addressing critical health challenges through its focused research and development efforts.
AiVita Biomedical 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 treatments for cancer, including a patient-specific immunotherapy targeting advanced cancers such as glioblastoma and ovarian cancer. Additionally, AiVita Biomedical is engaged in a preclinical program that involves human stem cells differentiated into retinal tissue sheets aimed at treating degenerative retinal diseases. The company's offerings also include a line of rejuvenating skincare products under the ROOT OF SKIN brand, which focuses on anti-aging solutions. Through its expertise in stem cell growth and differentiation, AiVita Biomedical aims to create safe and effective manufacturing systems that support its diverse therapeutic pipeline.
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’s pipeline includes various NK- and T-cell immuno-oncology programs, such as FT516 for acute myeloid leukemia and B-cell lymphoma, FT596 for B-cell lymphoma and chronic lymphocytic leukemia, and FT538 for multiple myeloma. Additional programs include FT500, FT576, FT819, and FT-ONO1, targeting solid tumors and hematologic malignancies. Fate Therapeutics is also collaborating with Ono Pharmaceutical Co. Ltd. and Janssen Biotech, Inc. to advance its off-the-shelf iPSC-derived CAR T-cell therapies and other innovative treatments. Founded in 2007, the company leverages insights from leading scientists to create and modulate stem cells for therapeutic applications, aiming to restore health through advanced cellular therapies.
Medeor Therapeutics, Inc. is a biotechnology company based in San Mateo, California, focused on researching, developing, and commercializing personalized cellular immunotherapies. Founded in 2012, the company specializes in creating innovative treatments aimed at improving outcomes for kidney transplant recipients. Under the MDR-10X brand, Medeor Therapeutics develops cellular immunotherapy products that enhance organ transplant immune tolerance and advance immuno-oncology. These therapies are designed to better preserve or improve kidney function and reduce the risk of graft rejection compared to traditional immunosuppressive drugs.
Poseida Therapeutics 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 utilizes non-viral gene engineering technologies to create a pipeline of targeted therapies, including both autologous and allogeneic chimeric antigen receptor T cell (CAR-T) product candidates aimed at treating hematological malignancies and solid tumors. Additionally, Poseida is advancing gene therapies for rare and life-threatening conditions, such as Ornithine transcarbamylase deficiency, methylmalonic acidemia, and various genetic liver diseases. Founded in 2014, Poseida is committed to addressing critical health challenges through its focused research and development efforts.
Mustang Bio, Inc. is a clinical-stage biopharmaceutical company dedicated to developing innovative cell and gene therapies aimed at treating hematologic cancers, solid tumors, and rare genetic diseases. The company has a diverse pipeline that includes gene therapy programs for rare genetic disorders and various chimeric antigen receptor (CAR) T cell therapies targeting both hematologic malignancies and solid tumors. Notable projects include MB-107, a gene therapy aimed at treating X-linked severe combined immunodeficiency, which is currently undergoing Phase 1/2 clinical trials. Other therapies under development include MB-102 for acute myeloid leukemia, MB-106 for B-cell lymphomas, and multiple CAR T programs targeting glioblastoma, prostate cancer, and pancreatic cancer. Mustang Bio also collaborates with institutions such as Nationwide Children’s Hospital and Beth Israel Deaconess Medical Center for research and development in gene editing and oncolytic viruses. Established in 2015 and headquartered in New York, Mustang Bio operates as a subsidiary of Fortress Biotech, Inc.
Humacyte, Inc. is a medical research and development company based in Morrisville, North Carolina, focused on creating and commercializing human tissue-based products for regenerative medicine and vascular surgery. The company specializes in designing acellular extracellular matrices, which are crafted in vitro from banked vascular smooth muscle cells and decellularized to prevent rejection. These products serve as tissue-engineered grafts for patients requiring vascular repair or replacement. Humacyte's innovative approach aims to provide universally implantable bioengineered human tissues and organs, addressing various diseases, injuries, and chronic conditions while enhancing patient care and transforming medical practices. Founded in 2004, the company is committed to advancing solutions for vascular diseases and the replacement of anatomical conduits.
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.
Scaled Biolabs Inc. is a biotechnology company founded in 2016 and based in San Francisco, California, specializing in research and development solutions for cell therapies. The company has developed a Cell Therapy Discovery Engine that leverages microfluidics, automation, and machine learning to enable biotech and pharmaceutical firms to explore numerous parameters across extensive experimental processes. This innovative platform allows for the simultaneous execution of multiple experiments in a miniaturized lab setting, leading to deeper biological insights and the identification of optimal conditions for cellular processes. Scaled Biolabs also engages in the internal development of cell therapy assets, including a program focused on induced pluripotent stem cell-derived dopaminergic neurons for treating Parkinson's disease. Through its technologies, Scaled Biolabs aims to streamline the manufacturing process of complex medicines, reduce time to market, and enhance clinical outcomes for patients.
Orchard Therapeutics is a biopharmaceutical company based in the United Kingdom that specializes in developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which modifies a patient's own hematopoietic stem cells to create a targeted treatment administered in a single dose. Orchard's portfolio includes Strimvelis, the first autologous gene therapy approved by the European Medicines Agency for the treatment of adenosine deaminase-severe combined immunodeficiency (ADA-SCID). Additionally, it is advancing clinical programs for various conditions, including metachromatic leukodystrophy, Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, and transfusion-dependent beta-thalassemia. The company also has an extensive preclinical pipeline targeting mucopolysaccharidosis types I, IIIA, and IIIB. Orchard Therapeutics collaborates with leading institutions in gene therapy research, enhancing its efforts to transform the lives of patients with rare diseases in the UK, European Union, and the United States. Founded in 2015 and headquartered in London, it aims to provide sustainable clinical benefits through its innovative therapies.
Semma Therapeutics, Inc. is focused on developing innovative cell therapies for patients with Type 1 diabetes, who typically rely on insulin injections. The company was founded based on groundbreaking research by Professor Douglas Melton, which enabled the generation of functional, insulin-producing beta cells in the laboratory. This technology has been exclusively licensed to Semma Therapeutics, and the company aims to combine these proprietary cells with advanced devices to create a functional replacement for the missing beta cells without the need for immunosuppression. Headquartered in Cambridge, Massachusetts, with an additional office in Boston, Semma Therapeutics operates as a subsidiary of Vertex Pharmaceuticals and is dedicated to advancing this new therapeutic option to improve the lives of diabetes patients.
Humacyte, Inc. is a medical research and development company based in Morrisville, North Carolina, focused on creating and commercializing human tissue-based products for regenerative medicine and vascular surgery. The company specializes in designing acellular extracellular matrices, which are crafted in vitro from banked vascular smooth muscle cells and decellularized to prevent rejection. These products serve as tissue-engineered grafts for patients requiring vascular repair or replacement. Humacyte's innovative approach aims to provide universally implantable bioengineered human tissues and organs, addressing various diseases, injuries, and chronic conditions while enhancing patient care and transforming medical practices. Founded in 2004, the company is committed to advancing solutions for vascular diseases and the replacement of anatomical conduits.
Capricor Therapeutics is a clinical-stage biotechnology company that focuses on the discovery, development, and commercialization of cell and exosome-based therapeutics for the treatment and prevention of diseases. Its CAP-1002 is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy and COVID-19. The company was founded in 2005 and is headquartered in Los Angeles, California.
ImmunoCellular Therapeutics, Ltd. is a clinical-stage biotechnology company based in Westlake Village, California, focused on developing immune-based therapies for the treatment of various cancers. The company specializes in dendritic cell (DC) immunotherapies, with notable products including ICT-107, which targets glioblastoma multiforme, a severe brain cancer; ICT-140, aimed at ovarian cancer; and ICT-121, designed to address recurrent glioblastoma and other solid tumors by targeting CD133 markers. Additionally, ImmunoCellular is advancing Steam-to-T-Cell immunotherapies to enhance cancer treatment outcomes. The company has established license agreements with prominent institutions, including the California Institute of Technology and The Johns Hopkins University, to support its research and development efforts.
Bluebird Bio is a biotechnology company focused on the research, development, and commercialization of innovative gene therapies for severe genetic diseases and cancer. Its 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 collaborates with several partners, including Bristol-Myers Squibb and Regeneron Pharmaceuticals, to discover and commercialize disease-altering therapies. Additional collaborations with organizations such as Medigene AG and Gritstone Oncology focus on developing T cell receptor product candidates for cancer treatment. Founded in 1992 and headquartered in Cambridge, Massachusetts, Bluebird Bio aims to fundamentally address the genetic basis of diseases through its proprietary lentiviral vector gene addition platform. Revenue is generated from collaboration agreements, research fees, license fees, and grants.
VistaGen Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for diseases and disorders of the central nervous system, particularly those related to psychiatric conditions such as depression and anxiety. The company's main product candidates include PH94B, a neuroactive nasal spray poised for Phase III trials aimed at treating acute anxiety in adults with social anxiety disorder, and PH10, another nasal spray in preparation for Phase 2b development for major depressive disorder. Additionally, AV-101, an oral prodrug candidate, is being developed for multiple indications, including major depressive disorder, suicidal ideation, and neuropathic pain. VistaGen has established various licensing and collaboration agreements with other firms to enhance its research and development efforts, including a strategic partnership for the clinical development of PH94B in select Asian markets. Founded in 1998, VistaGen is headquartered in South San Francisco, California.