Sarepta is best known for its RNA technology platform, which has led to two approved — though also controversial — drugs for Duchenne muscular dystrophy.
Yet, Sarepta is also deeply invested in gene therapy, having developed an extensive list of more than two dozen experimental treatments, six of which have reached human testing.
Rare diseases have been an early target in this rapidly growing field. The two gene therapies approved in the U.S., Roche’s Luxturna and Novartis’ Zolgensma, are respectively used to treat an uncommon form of blindness and a muscle disease that occurs in about 1 in every 10,000 births.
