Sarepta and Roche have signed a deal providing Roche exclusive commercial rights to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy (DMD), outside the US.

Under the deal, Sarepta will receive $1.15 billion in an upfront payment and an equity investment; up to $1.7 billion in regulatory and sales milestones; and royalties on net sales, anticipated to be in the mid-teens.

In addition, Roche and Sarepta will equally share global development expenses, the firms revealed.

Sarepta retains all rights to SRP-9001 in the United States.

DMD is an X-linked rare degenerative neuromuscular disorder causing severe progressive muscle loss and premature death.

SRP-9001, currently in clinical development for DMD, is designed to deliver the micro-dystrophin-encoding gene directly to the muscle tissue for the targeted production of the micro-dystrophin protein.

“This collaboration will not only increase the speed with which SRP-9001 could benefit DMD patients outside the United States, but will also greatly expand the scope of territories within which we could potentially launch SRP-9001 and improve and save lives,” said Doug Ingram, president and chief executive officer, Sarepta.