Sarepta licenses next-gen AAV tech from Broad Institute in deal covering five indications — including Duchenne
Sarepta’s research agreement with the Broad Institute is taking the next step Monday.
The biotech will license a new group of adeno-associated viruses from the institute for an undisclosed upfront payment and milestone promises, the pair announced Monday morning. Under the agreement, Sarepta will have the rights to five neuromuscular and cardiac indications, including Duchenne muscular dystrophy, where it’s already well-versed.
According to Sarepta, the deal covers the MyoAAV program that aims to deliver more efficient gene therapies using modified capsids. Endpoints News has also reached out to the Broad Institute and will update this story accordingly.
The group of viruses, per Sarepta:
- Delivered 25-50 times greater gene expression in multiple skeletal muscles and 10-15 times greater gene expression in cardiac muscle;
- Demonstrated reduced delivery to the liver by 50 percent and showed lower accumulation in the liver;
- Can be used at up to a log lower dose than traditional AAV vectors, due to increased efficiency.
Full research for MyoAAV in a Duchenne mouse model was published in Cell in 2021, the pair added. Sarepta CEO Doug Ingram said in a statement that the viruses could “substantially reduce viral load,” while CSO Louise Rodino-Klapac touted the platform’s “broad applicability.”
That Sarepta says it’s planning to continue pursuing next-generation Duchenne therapies suggests the biotech has no intention of resting on its laurels. Execs shepherded the first Duchenne gene therapy across the FDA finish line back in 2016 and have since won two more approvals. They’re seeking a fourth later this year.
But those green lights have not come without controversy. Almost five years before the Biogen brouhaha with Aduhelm, Sarepta won an accelerated OK despite outside experts recommending against approval due to a lack of data clarity. Though the FDA is not bound by law to follow its panel’s advice, they typically do so in about four out of every five cases.
Sarepta’s next two approvals also followed the accelerated path, and the upcoming application will also seek a quick OK. A key part of this pathway involves a study to confirm a therapy’s benefit in order to convert the accelerated approval to a full endorsement, but Sarepta has still not completed any of the three confirmatory trials.
Analysts expect another adcomm for Sarepta’s newest pitch, and the panel is likely to discuss heart inflammation associated with the company’s latest trial. Though the efficacy data proved largely positive, a new occurrence of myocarditis in a separate cohort of older patients (and classified as a serious adverse event) clouded the readout.