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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.
Metrohm USA Inc is a Riverview, FL-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Benitec is a biotechnology company developing a proprietary therapeutic technology platform that combines RNA interference (RNAi) with gene therapy with a goal of providing sustained, long-lasting silencing of disease-causing genes from a single administration.
ADMA Biologics, Inc. is a late-stage biopharmaceutical company that develops, manufactures and intends to market specialty plasma-based biologics for the treatment and prevention of infectious diseases. The Company is engaged in the development and com...
BioDigital is a New York-based biomedical visualization company that is often referred to as being "Google Earth for the Human Body".