Vertex Pharmaceuticals Incorporated

Vertex Pharmaceuticals Incorporated is a leading global biotechnology company focused on developing and commercializing therapies for treating cystic fibrosis and other rare and life-threatening diseases. Vertex was founded in 1989 and has since become one of the world’s largest biopharmaceutical companies with a market capitalization of more than $55 billion. The company’s products are approved in many countries and its treatments are used to treat patients in more than 80 countries around the world.

Vertex’s primary focus is on the development and commercialization of treatments for cystic fibrosis. Its product portfolio includes SYMDEKO/SYMKEVI, ORKAMBI, and KALYDECO that are marketed to treat patients with cystic fibrosis who have specific mutations in their cystic fibrosis transmembrane conductance regulator gene. Additionally, the company markets TRIKAFTA for the treatment of patients with CF who are 6 years of age or older who have at least one F508del mutation.

Vertex is also investing heavily in research and development of new treatments. Its pipeline includes several potential treatments such as VX-864, a potential treatment for AAT deficiency, which is currently in Phase 2 clinical trial; VX-147, a potential treatment for APOL1-mediated focal segmental glomerulosclerosis, or FSGS, which is currently in Phase 2 clinical trial; VX-880, a potential treatment for Type 1 diabetes which is currently in Phase 1/2 clinical trial; VX-548, a NaV1.8 inhibitor for treatments of acute, neuropathic, musculoskeletal pain which is in Phase 2 clinical trial; and CTX001 for the treatment of severe SCD and TDT which is in Phase 3 clinical trial.

The company also has collaborations with a number of different organizations to drive innovation in drug development. Vertex collaborates with Affinia Therapeutics, Inc., Arbor Biotechnologies, Inc., CRISPR Therapeutics AG., Kymera Therapeutics, Inc., Mammoth Biosciences, Inc., Moderna, Inc., Obsidian Therapeutics, Inc., Skyhawk Therapeutics, Inc., Ribometrix, Inc., Genomics plc, Merck KGaA, Darmstadt, Germany, and X-Chem, Inc.

Vertex markets its products primarily to specialty pharmacy and specialty distributors in the United States and specialty distributors, retail chains, hospitals and clinics internationally. The company also engages in an extensive research and development program. Its R&D efforts are focused on the development of new treatments and therapies for treatments of rare and serious diseases. Vertex has spent more than $1.2 billion in R&D spending in 2020, reflecting the company’s commitment to finding new treatments for serious diseases.