STAT+: Duchenne gene therapy from Solid Biosciences produces promising results in early study
Solid said that the first patients to receive its Duchenne gene therapy all produced high levels of the target protein, an early sign of promise
Solid Biosciences said Tuesday that the first three patients to receive its experimental gene therapy for Duchenne muscular dystrophy all produced high levels of microdystrophin, a miniature version of the protein broken in the fatal disease.
The data are the latest in a high-stakes race to create a better or safer gene therapy than Sarepta Therapeutics’ Elevidys, which was given broad approval last year amid intense debate at the Food and Drug Administration.
Solid is competing primarily with Regenxbio, which has already announced data from several patients, and Kate Biosciences, a Broad Institute spinout purchased last year by Novartis.
