SAREPTA

Sarepta Therapeutics is a commercial-stage biopharmaceutical company at the forefront of precision genetic medicine, with a primary focus on developing treatments for rare, inherited diseases. Originally founded as AVI BioPharma, the company rebranded in 2012 to concentrate on RNA-targeted therapeutics. Its mission is to rescue lives devastated by rare diseases, particularly Duchenne muscular dystrophy (DMD), for which it has multiple FDA-approved treatments. Sarepta's development pipeline is built on its diverse and proprietary RNA-based phosphorodiamidate morpholino oligomer (PMO) platform and is also expanding into gene therapy and gene editing technologies to treat a range of neuromuscular and other rare conditions.