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Adolor Corporation is a Exton, PA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Encoded Therapeutics Inc. is a biotechnology company advancing precision medicines for a broad range of genetic disorders. Our mission is to unlock new opportunities for viral gene therapy through genomics-driven discovery. We focus on delivering life-changing advances for individuals with genetic disorders not addressable with current approaches.
Skyhawk Therapeutics is a drug discovery and development company focused on revolutionizing disease treatment with small molecules that correct RNA expression.
AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe diseases, with an initial focus in ophthalmology. AGTC`s lead product candidates are designed to treat inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments. AGTC`s product pipeline includes six named ophthalmology development programs across five targets (X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP), achromatopsia, wet age-related macular degeneration and blue cone monochromacy), two non-ophthalmology programs (alpha-1 antitrypsin deficiency and adrenoleukodystrophy) and AGTC is continuing to develop early research studies in additional indications. The company is also exploring genetic defects in cells in the inner ear that lead to deafness and expects to advance several product candidates into development within the next few years. AGTC employs a highly targeted approach to selecting and designing its product candidates, choosing to develop therapies for indications having high unmet medical need, clinical feasibility and commercial potential. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as, expertise in the formulation, manufacture and physical delivery of gene therapy products
AuraSense Therapeutics (AST) is a biopharmaceutical company developing first-in-class therapies based upon its proprietary Spherical Nucleic Acid (SNA) gene regulation and immune modulation strategies. AST is leading the translation of SNA constructs as innovative medicines with a core focus on debilitating diseases with limited or no treatment options. AuraSense Therapeutics' uniquely engineered SNA™ constructs possess attractive biocompatibility and versatility as therapeutics and hold great promise for combating the most life-threatening diseases, including many forms of cancer, infectious disease, and skin conditions.