Sarepta’s small gene therapy study takes big steps

Four boys, from 4 to 7 years old, stood up from a chair, stepped up and down from an elevated box, and hopped on each leg after being treated with an experimental gene therapy. They have Duchenne muscular dystrophy, so those are meaningful improvements in muscle strength, movement, and function. And they're strong, if preliminary, results for Sarepta Therapeutics. As STAT’s Adam Feuerstein reports, the company hopes to launch a larger trial by the end of the year or early in 2019.

Sarepta’s gene therapy is designed to build up a crucial muscle protein normally missing in patients diagnosed with Duchenne. Because the gene in question is too big for a virus to ferry it into cells where it is needed, Sarepta deploys a smaller, “micro-dystrophin” version containing only what's needed to make functional protein. It looked promising in June, and the data reported yesterday were consistent across a series of tests, the company said.