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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.
ProteinOne is a Derwood, MD-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Inspiration Biopharmaceuticals is a Cambridge, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
eGenesis is revolutionizing the field of transplantation with an unparalleled, multiplexed gene editing platform for the development of human-compatible organs, tissues and cells. Harnessing the latest gene-editing techniques, eGenesis has the capability to solve the global organ crisis by providing an alternative to allotransplantation. eGenesis is uniquely positioned to reinvigorate the field of xenotransplantation by addressing both the key virology and immunology hurdles that have impeded its advancement to date and provide commercially-viable products to save and enhance the lives of patients in need.
CellCarta provides integrated analytical platforms in immunology, histopathology, proteomics and genomics, as well as digital pathology & AI.