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Aeglea BioTherapeutics is a clinical-stage biotechnology company redefining the potential of human enzyme therapeutics to benefit people with rare and devastating metabolic diseases with limited treatment options. Aeglea`s lead product candidate, pegzilarginase, is in a pivotal Phase 3 trial for the treatment of Arginase 1 Deficiency and has received both Rare Pediatric Disease and Breakthrough Therapy Designation. In the second quarter of 2020, the Company initiated a Phase 1/2 clinical trial of AGLE-177 for the treatment of Homocystinuria. AGLE-177 has also been granted Rare Pediatric Disease Designation. Aeglea has an active discovery platform focused on engineering small changes in human enzymes to have big impact on the lives of patients and their families.
HudsonAlpha Institute seeks to expand the science of human genomics and hasten its movement into the marketplace where discoveries can benefit society.
NeuroVision is an integrated neuroscience company focused on delivering scientifically validated, diagnostic tests & biomarkers for early detection and monitoring of amyloid pathology related to Alzheimer’s Disease. The company has developed a novel retinal imaging technology, that is incorporated into a number of clinical trials globally.
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
At Ironwood Pharmaceuticals, we are committed to advancing the treatment of GI diseases and redefining the standard of care for GI patients. As a pioneer and champion in the development of a market-leading therapy for IBS-C and CIC, we are singularly focused on advancing the treatment of GI diseases and continue to challenge what has been done before to shape what the future holds. So, we named ourselves after the Ironwood tree, which can live for thousands of years in the harshest desert, creating a shaded microclimate in which life thrives. Iron, like science, is undeniable and strong. Wood, like artistry and humanity, is flexible, resilient and versatile. The two words together represent our passion for making a positive impact on the health – and lives- of GI patients, their families and communities.