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Graphite Bio is a next-generation gene editing company focused on the development of first-in-class therapies for patients suffering from life-threatening genetic diseases. The company`s platform harnesses the natural cellular process of homology directed repair (HDR) and targeted DNA integration to precisely repair genetic defects at their source. Graphite Bio is leveraging its differentiated platform to advance a portfolio of transformative genetic treatments with potential for saving and dramatically improving patients` lives
Prothena Corporation plc (Nasdaq: PRTA) Prothena is a global biotechnology company seeking to fundamentally alter the course of progressive diseases. We are a late-stage clinical company focused on the discovery, development, and commercialization of novel protein immunotherapies for the treatment of diseases that involve protein misfolding and inflammatory cell adhesion disorders. Our team has a recognized track record both for profound scientific discoveries and for the development of therapeutics that have become leading commercial products in their respective therapeutic categories. Prothena`s pipeline focuses on therapeutic monoclonal antibodies directed specifically to disease-causing proteins and our antibody-based product candidates target a number of potential indications, including AL amyloidosis (NEOD001), Parkinson’s disease and other related synucleinopathies (PRX002), ATTR amyloidosis (PRX004), and novel cell adhesion targets involved in psoriasis, psoriatic arthritis and other inflammatory diseases (PRX003). Prior to December 2012, Prothena’s business operated as part of Elan Corporation, plc. Prothena’s business consists of a substantial portion of Elan’s former drug discovery business platform. After the separation from Elan and the related distribution of our ordinary shares to Elan’s stockholders, our ordinary shares began trading on The Nasdaq Global Market under the symbol “PRTA” on December 21, 2012.
Spring Bank Pharmaceuticals (NASDAQ: SBPH) is a clinical-stage biopharmaceutical company engaged in the discovery and development of a novel class of therapeutics using our proprietary small molecule nucleic acid hybrid (SMNH) chemistry platform. SMNH compounds are small segments of nucleic acids that the company designs to selectively target and modulate the activity of specific proteins implicated in various disease states. Spring Bank is developing its most advanced SMNH product candidate, inarigivir soproxil (formerly referred to as SB 9200), for the treatment of viral diseases, including hepatitis B virus (HBV), and other SMNH product candidates, including SB 11285, the company`s lead immunotherapeutic agent for the treatment of selected cancers through the activation of the STimulator of Interferon Genes, or STING, pathway.
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
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