SRPT vs VRTX

Sarepta Therapeutics Inc.

Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery and development of RNA-targeted therapeutics, gene therapy, and other genetic therapeutic modalities for the treatment of rare diseases. The company offers EXONDYS 51 injection to treat duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping; and VYONDYS 53 for the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. It also developing AMONDYS 45, a product candidate that uses phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 45 of the dystrophin gene; SRP-5051, a peptide conjugated PMO that binds exon 51 of dystrophin pre-mRNA; SRP-9001, a DMD micro-dystrophin gene therapy program; and SRP-9003, a limb-girdle muscular dystrophies gene therapy program. The company has collaboration agreements with F. Hoffman-La Roche Ltd; Nationwide Children's Hospital; Lysogene; Duke University; Genethon; and StrideBio. It also has a research and option agreement with Codiak BioSciences, Inc. to design and develop engineered exosome therapeutics to deliver gene therapy, gene editing, and RNA technologies for neuromuscular diseases; and research collaboration with Genevant Sciences for lipid nanoparticle-based gene editing therapeutics. Sarepta Therapeutics, Inc. was incorporated in 1980 and is headquartered in Cambridge, Massachusetts.

Vertex Pharmaceuticals Incorporated

Vertex Pharmaceuticals Incorporated engages in developing and commercializing therapies for treating cystic fibrosis. The company markets SYMDEKO/SYMKEVI, ORKAMBI, and KALYDECO to treat patients with cystic fibrosis who have specific mutations in their cystic fibrosis transmembrane conductance regulator gene; and TRIKAFTA for the treatment of patients with CF 12 years of age or older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator, or CFTR, gene. Its pipeline includes Pipeline for Alpha-1 antitrypsin deficiency that is in Phase 2 clinical trial; VX-864, a second investigational small molecule corrector for the treatment of AAT deficiency, which is in Phase 1 clinical trial; and VX-147 that completed a Phase 1 clinical trial for the treatment of APOL1-mediated focal segmental glomerulosclerosis, or FSGS, and other serious kidney diseases. The company sells its products primarily to specialty pharmacy and specialty distributors in the United States, as well as specialty distributors and retail chains, and hospitals and clinics internationally. Vertex Pharmaceuticals Incorporated has collaborations with CRISPR Therapeutics AG; Arbor Biotechnologies, Inc.; Moderna, Inc.; Genomics plc; Merck KGaA; Darmstadt, Germany; X-Chem, Inc.; Janssen Pharmaceuticals, Inc.; Merck KGaA; Kymera Therapeutics; Ribometrix, Inc.; Molecular Templates, Inc.; and Affinia Therapeutics, as well as a strategic research collaboration and licensing agreement with Obsidian Therapeutics, Inc. for the discovery of novel therapies that regulate gene editing for the treatment of serious diseases. The company was founded in 1989 and is headquartered in Boston, Massachusetts.