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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.
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases.
Concept Medical Inc. (CMI) is establishment of USA with the vision of `Inspiring Innovation`. CMI was founded in year 2008, having the mission of `converting concepts into reality` in the field of drug delivering medical devices industry.
BD Biosciences - Canada is a Mississauga, ON-based company in the Healthcare, Pharmaceuticals, & Biotech sector.
Novartis Consumer Health Inc is a Big Rapids, MI-based company in the Healthcare, Pharmaceuticals, and Biotech sector.