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REGENXBIO is a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno‐associated virus (AAV) gene therapy. REGENXBIO`s NAV® Technology Platform, a proprietary AAV gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO`s mission is to transform the lives of patients suffering from severe diseases with significant unmet medical need by developing and commercializing in vivo gene therapy products based on REGENXBIO`s NAV Technology Platform. REGENXBIO seeks to accomplish this mission through a combination of internal development efforts and third‐party NAV Technology Platform licensees.
Laureate Pharma L.P. is a Princeton, NJ-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Aptinyx Inc. (formerly Naurex Inc.) is a biopharmaceutical company focused on discovery and development of transformative therapies for challenging disorders of the brain and nervous system. Aptinyx has a proven platform for discovering compounds that enhance synaptic plasticity, or strengthen the network for neural cell communication. Molecules discovered by Aptinyx achieve this through a novel mechanism that modulates NMDA receptors, resulting in drugs that are both highly effective and well tolerated. This mechanism has applicability across a number of brain and nervous system disorders. Aptinyx is rapidly advancing promising drug candidates into clinical development for indications with high unmet medical need.
Terumo Interventional Systems is a Somerset, NJ-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Abeona Therapeutics (Nasdaq: ABEO) is a fully-integrated gene and cell therapy company at the forefront of the rapidly-advancing field of genetic medicine. The Company`s multi-platform expertise across the manufacture, delivery, development, and discovery of novel gene and cell therapies has it uniquely positioned for success. Underpinning the Company`s robust pipeline is its fully-operational manufacturing facility producing therapies and vectors for preclinical and clinical studies. Abeona is also developing the AIM™ Vector Platform: 100+ next-generation AAV capsids for delivering gene therapies targeting a wide range of organs and multiple routes of delivery. A robust and diverse pipeline is led by a novel gene-corrected cell therapy poised to enter Phase 3 in mid-2019 and complemented by one-time gene therapy candidates across four lysosomal storage diseases. Several preclinical discoveries are led by an emerging program in cystic fibrosis that uses the AIM vector platform and a capsid that has shown potential across inherited and acquired retinal diseases.