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Hanford Pharmaceuticals is a Syracuse, NY-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Kindred Hospitals LP is a Louisville, KY-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
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
Astra Zeneca Neuroscience is a Cambridge, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Regulus is focused on the discovery and development of microRNA therapeutics. microRNAs are members of a large class of non-coding RNAs of approximately 22 nucleotides in length that regulate most genes in the genome. A single microRNA can target and regulate up to hundreds of genes, and these genes are involved in biological networks or pathways. Dysregulated microRNA expression is involved in the initiation of many complex multi-factoral diseases, including cancer, inflammatory disease, fibrosis, and metabolic disease. microRNA therapeutics are oligonucleotide medicines that modulate the function of microRNAs, correcting the imbalance of gene expression and associated cellular pathways to treat human disease. Regulus was formed in September 2007 by Alnylam Pharmaceuticals (NASDAQ:ALNY) and Isis Pharmaceuticals (NASDAQ:ISIS). Regulus benefits substantially from the collective therapeutic RNA expertise of its founding companies, including established proprietary oligonucleotide-based technologies, and broad and dominant intellectual property estates specific to chemically modified oligonucleotides. Regulus has access to over 900 patents and patent applications pertaining to oligonucleotide technologies useful for targeting microRNA therapeutics, and over 170 patents and patent applications directed to microRNA specific technologies. Regulus is located in La Jolla, California and is led by a seasoned executive team and board of directors, with proven expertise in corporate management, business operations, drug discovery and drug development. Regulus’ scientific advisory board consists of world-class scientists and several of the foremost authorities in the field of microRNA research.