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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.
Achieve is a specialty pharmaceutical company committed to advancing cytisine as a widely available treatment option to help people battling nicotine addiction.
The Center for Professional Innovation and Education is a Wayne, PA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Synchem is a Elk Grove Village, IL-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Sienna Biopharmaceuticals is focused on the discovery, clinical development and commercialization of products in aesthetics and dermatology based on scientific breakthroughs that dramatically improve patient outcomes, and physicians’ practice of medicine.