Sarepta Therapeutics and Roche have announced that they will be working in partnership to develop Sarepta’s potential gene therapy for Duchenne muscular dystrophy outside the US.
Sarepta will continue to organise the clinical trials of the potential gene therapy (called SRP-9001) in Duchenne muscular dystrophy. The trials are currently in an early phase, but following encouraging early results, the company is planning to start bigger trials.
By combining these activities with Roche’s global reach and regulatory expertise, the companies hope to more quickly bring the potential treatment to patients outside the United States.
The agreement focuses on SRP-9001, a potential treatment that aims to use a virus to deliver a small, but functional dystrophin (called a micro-dystrophin) gene to the muscles.
It’s important to note that the gene therapy is still in clinical trials, and further trials will be required to test the effectiveness of the potential treatment. If those trials are successful, we hope that this agreement will help those living with Duchenne in the UK gain access to the treatment more quickly.
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