VERTEX PHARMACEUTICALS INC / MA

Vertex Pharmaceuticals Incorporated, a biotechnology company, engages in developing and commercializing therapies for treating cystic fibrosis (CF). It markets TRIKAFTA/KAFTRIO and SYMDEKO/SYMKEVI for people with CF with at least one F508del mutation for 6 years of age or older; ORKAMBI for CF homozygous F508del mutation for CF patients 2 year or older; and KALYDECO for the treatment of patients with 4 months or older who have CF with a mutation that is responsive to ivacaftor, and R117H mutation or one of certain gating mutations. The company's pipeline includes VX-522, a CF mRNA therapeutic designed to treat the underlying cause of CF, which is in Phase 1 clinical trial; VX-548, a non-opioid medicine for the treatment of acute and neuropathic pain which is in Phase 3 clinical trial; Exa-cel, for the treatment of sickle cell disease and transfusion-dependent beta thalassemia which is in Phase 2/3 clinical trial; and VX-864 for treatment of AAT deficiency, which is in Phase 2 clinical trial. In addition, it provides VX-147 for the treatment of APOL1-mediated focal segmental glomerulosclerosis and co-morbidities, such as hypertension which is in single Phase 2/3; VX- 880, treatment for Type 1 Diabetes which is in Phase 1/2 clinical trial; VX-970, which is in Phase 2 clinical trial for the treatment of cancer; and VX-803 and VX-984 for treatment of cancer in Phase 1 clinical trial. Further, it sell the products to specialty pharmacy and specialty distributors in the United States, as well as retail pharmacies or pharmacy chains, hospitals, and clinics. Additionally, the company has collaborations with CRISPR Therapeutics AG.; Moderna, Inc.; Entrada Therapeutics, Inc.; Affinia Therapeutics; Arbor Biotechnologies, Inc.; Kymera Therapeutics, Inc.; Mammoth Biosciences, Inc.; Obsidian Therapeutics, Inc.; Verve Therapeutics; Skyhawk Therapeutics; and Ribometrix, Inc. Vertex Pharmaceuticals Incorporated was founded in 1989 and is headquartered in Boston, Massachusetts.