Two of the first efforts to treat Duchenne muscular dystrophy with CRISPR gene editing are getting off the ground in China, even as projects in the U.S. have seemingly stalled.
Trials for the fatal muscle-wasting disease were started in the last couple of months by two different Chinese companies. One, GenAssist, has already dosed one young boy. Huidagene, the other biotech, expects to dose the first of three boys soonand report data by Q1 next year.
The efforts are notable, as scientists and advocates have long hoped CRISPR could unlock a more powerful treatment for Duchenne than the technology used in Elevidys, Sarepta’s much-debated gene therapy. The need for such a treatment only grew last October, when trial results confirmed that Elevidys, while potentially useful, is far from a cure.
Continue to STAT+ to read the full story…
Two of the first efforts to treat Duchenne muscular dystrophy with CRISPR gene editing are getting off the ground in China, even as projects in the U.S. have seemingly stalled.
Trials for the fatal muscle-wasting disease were started in the last couple of months by two different Chinese companies. One, GenAssist, has already dosed one young boy. Huidagene, the other biotech, expects to dose the first of three boys soonand report data by Q1 next year.
The efforts are notable, as scientists and advocates have long hoped CRISPR could unlock a more powerful treatment for Duchenne than the technology used in Elevidys, Sarepta’s much-debated gene therapy. The need for such a treatment only grew last October, when trial results confirmed that Elevidys, while potentially useful, is far from a cure.
Continue to STAT+ to read the full story…
