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We are a biotechnology company creating a new class of targeted gene therapies for debilitating and life-threatening genetic disorders in both central nervous system (CNS) and non-CNS indications. Current gene therapy approaches have shown dramatic efficacy in several rare diseases but are hindered by imprecise targeting. The inability to selectively transduce specific cells and tissues effectively drives administration of higher doses and resulting safety liabilities. We are addressing these concerns with our proprietary adeno-associated virus (AAV) engineering platform that generates capsids optimized to target specific tissues and cells in the disease organ while limiting transduction of tissues and cells not relevant to the target disease. Through our next-generation AAV platform combined with cargo development and state-of-the-art manufacturing, we are tackling monogenetic and sporadic CNS and non-CNS disorders that have previously been challenging to address.
Bioneer is a Alameda, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Homology is based on groundbreaking science that harnesses the naturally occurring process of homologous recombination. This non-nuclease-based approach offers clear advantages in its precision, efficiency and on-target in vivo editing of genetic mutations. Homology obtained an exclusive worldwide license to this technology platform, which is based on the pioneering research of Saswati Chatterjee, Ph.D., Professor of Virology at the Beckman Research Institute at the City of Hope in California, member of the Recombinant DNA Advisory Committee (RAC) to the Office of the Director, National Institutes of Health (NIH) and former charter member of the Therapeutic Approaches to Genetic Diseases Study Section of the NIH. Dr. Chatterjee and her team led the first adeno-associated virus (AAV) vector-mediated gene transfer studies into human hematopoietic stem cells and subsequently identified and isolated a series of naturally-occurring AAVs from human CD34+ cells.
The mission of REVOLUTION Medicines is redesigning evolution`s products to treat serious diseases. The company discovers and develops new drugs by reconfiguring natural substances that are inherently rich with biological function as a result of natural selection. The company`s first drug candidates are innovative small molecules that exploit and improve upon the properties of amphotericin B, a powerful, broad-spectrum antifungal compound found in nature that has avoided generating significant drug resistance in 50 years of clinical use. Headquartered in Redwood City, Calif. at the intersection of Silicon Valley and the birthplace of biotechnology, REVOLUTION Medicines is a private company financed by top-tier investor Third Rock Ventures.
Asher Bio is a biotechnology company developing precisely targeted immunotherapies for cancer and other diseases.