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Korro Bio is an RNA editing company focused on the discovery and development of a new class of precision genetic medicines for both rare and common diseases. The company`s proprietary and modular platform, OPERA™ (Oligonucleotide promoted editing of RNA), builds on a deep understanding of ADAR biology, and combines data-driven design, oligo discovery and chemistry with clinically validated delivery vehicles, to achieve highly selective RNA editing. This unique technology enables the development of RNA editing therapeutics that deliver the functional benefits of gene therapy with a reversible, transient, titratable and specific treatment regimen, offering the potential to propel genetic medicine beyond rare genetic diseases into larger patient populations with common diseases. Korro is based in Cambridge, Mass.
Celgene Corporation is an integrated global pharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through gene and protein regulation. Our talent, scientific knowledge and research capabilities help us to deliver access to innovative, disease-altering therapies to those who need them most. Celgene puts patients first with industry-leading programs that provide information, support and access to our innovative therapies. Celgene is headquartered in Summit, New Jersey, and employs more than 7,000 people located in more than 60 countries.
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
Axial Biotherapeutics is a biopharmaceutical company harnessing the link between the human gut microbiome and Central Nervous System to develop a new class of therapeutics to improve the quality of life for people with CNS diseases and disorders
Essential Group is a Gurnee, IL-based company in the Healthcare, Pharmaceuticals, and Biotech sector.