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Velicept Therapeutics, Inc. is a privately held, clinical development company focused on advancing best-in-class compounds with the potential to fill unmet medical needs.
Ambys Medicines is a biotechnology company focused on discovering and developing regenerative and restorative therapies for people with advanced liver disease. Founded by world-renowned experts in liver disease and regenerative medicine, Ambys is pioneering the application of novel modalities including cell and gene therapy, and gain-of-function drug therapy, to meet the urgent need for treatments that have the potential to restore liver function and prevent the progression to liver failure across multiple liver diseases that are untreatable or poorly treated today. Ambys was launched in 2018 by Third Rock Ventures and Takeda Pharmaceuticals with $140M in starting capital and is headquartered in South San Francisco, Calif.
jCyte is a clinical-stage company focused on the application of progenitor cell-based technology in retinal diseases. The Company`s allogeneic product candidate, jCell, is delivered by simple intravitreal injection without the need for surgery or immune suppression. jCyte has completed a phase 1/2a, phase 1/2a extension and phase 2b clinical trials testing jCell in patients with Retinitis Pigmentosa.
NovoBiotic Pharmaceuticals is a Cambridge, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
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.