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Sierra Sciences is a Reno, NV-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Q-State Biosciences is a technology-derived medicines company based in Cambridge, MA, that combines advanced human cellular models, unique measurement engineering and AI / machine learning into a proprietary platform that enables novel discovery of optimized genetically targeted medicines for epilepsy, pain, and other diseases of the CNS.
Peptilogics is a clinical-stage biotechnology company that designs and develops novel peptide therapeutics. Through their computational platform, Peptilogics is radically altering drug discovery by not only uncovering new, previously unexplored chemical design space, but ensuring that each potential therapeutic is biologically viable and scalable to manufacture. Peptilogics` lead clinical stage peptide therapeutic, PLG0206, is a novel, broad-spectrum anti-infective that has been granted FDA Orphan Drug Designation and Qualified Infectious Disease Product Designation for its initial focus on the treatment of prosthetic joint infections, an unmet medical need.
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
AveXis, now a Novartis Company, is a clinical-stage gene therapy company relentlessly focused on bringing gene therapy out of the lab and into the clinical setting for patients and families devastated by rare and life-threatening neurological genetic d...