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Vertex Pharmaceuticals Incorporated is a pioneering biotechnology company dedicated to advancing therapies for cystic fibrosis (CF) and other serious diseases. Established in 1989 and headquartered in Boston, Massachusetts, Vertex specializes in developing and commercializing innovative treatments that target specific mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The company's current portfolio includes SYMDEKO/SYMKEVI, ORKAMBI, KALYDECO, and TRIKAFTA, which are tailored to address the underlying genetic causes of CF in patients with specific mutations.
In addition to its marketed therapies, Vertex Pharmaceuticals has a robust pipeline of investigational treatments. These include VX-864 for alpha-1 antitrypsin deficiency in Phase 2 clinical trials, VX-147 for APOL1-mediated focal segmental glomerulosclerosis (FSGS) and other kidney diseases in Phase 2, VX-880 for Type 1 Diabetes in Phase 1/2, VX-548, a NaV1.8 inhibitor for acute and chronic pain in Phase 2, and CTX001 for severe sickle cell disease (SCD) and transfusion-dependent thalassemia (TDT) in Phase 3. This diverse pipeline underscores Vertex's commitment to addressing unmet medical needs across various therapeutic areas.
Vertex Pharmaceuticals collaborates extensively with leading biotech and pharmaceutical companies, including Affinia Therapeutics, CRISPR Therapeutics, Moderna, and others. These partnerships facilitate the development of cutting-edge therapies and leverage Vertex's expertise in genetic research and precision medicine. Through its innovative approach and strategic collaborations, Vertex Pharmaceuticals continues to redefine treatment standards and improve outcomes for patients worldwide.
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