Dec 23, 2019: Roche and Sarepta Therapeutics announced the signing of a licensing agreement providing Roche exclusive commercial rights to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin), Sarepta’s investigational gene therapy for the Duchenne muscular dystrophy (DMD), outside the United States.
Under the conditions of the agreement, Sarepta will receive a direct payment of $750million in cash and $400million in equity. Sarepta is eligible to obtain regulatory and sales milestones and royalties on net sales.
Roche and Sarepta will even contribute to global development expenses. It combines Roche’s global reach, commercial presence and regulatory expertise with Sarepta’s gene therapy candidate for DMD to accelerate access to SRP-9001 for the patients outside the United States.
DMD is an X-linked rare degenerative neuromuscular disorder causing severe progressive muscle loss and premature death, it affects approximately one in every 3,500 – 5,000 male births worldwide.
SRP-9001, currently in clinical development for the DMD, is designed in order to deliver the microdystrophin-encoding gene directly to the muscle tissue for the targeted production of the microdystrophin protein. https://www.roche.com/media/releases/med-cor-2019-12-23.htm
