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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
Gene editing is changing the world. Founded by pioneers in the CRISPR/CAS revolution, Metagenomi is discovering the next generation of gene-editing systems for use in treating genetic diseases.
Garuda Therapeutics seeks to create a world which eliminates the dependency on donor or patient cells for blood stem cell transplants. Our platform technology for generating off-the-shelf, self-renewing blood stem cells is poised to provide patients with rapid and broad access to consistent, durable, HLA-compatible transgene-free blood stem cell therapies. Like bone marrow transplants, our technology could provide potentially curative therapies for more than 70 diseases.
Armata is a clinical-stage biotechnology company focused on the development of precisely targeted bacteriophage therapeutics for the treatment of antibiotic-resistant and difficult-to-treat bacterial infections using its proprietary bacteriophage-based technology.
XyloCor Therapeutics is a biopharmaceutical company focused on the development of novel gene therapy for unmet needs in advanced coronary artery disease. In the United States, coronary artery disease is a leading cause of death and disability. The Company`s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for which there are no treatment options. XyloCor also has a secondary product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. Co founded by Ronald Crystal, MD, and Todd Rosengart, MD, XyloCor has a licensing agreement with Weill Cornell Medicine for the worldwide rights to develop, manufacture and commercialize XC001.