Regenxbio gives glimpse at early Duchenne gene therapy results, plans pivotal study for accelerated approval

Re­genxbio shared ear­ly bio­mark­er re­sults from a 12-year-old boy with Duchenne mus­cu­lar dy­s­tro­phy who re­ceived a high­er dose of its gene ther­a­py, and the biotech said Tues­day it’s now hop­ing to start a piv­otal study lat­er this year that would in­clude a “broad age range” of boys.

Like oth­er gene ther­a­pies for DMD, Re­genxbio’s treat­ment is meant to ex­press a minia­ture ver­sion of the key mus­cle pro­tein dy­s­trophin, though the com­pa­ny said that its treat­ment ex­press­es an ad­di­tion­al com­po­nent of the pro­tein called the C-ter­mi­nal do­main that’s al­so found in nat­u­ral­ly oc­cur­ring dy­s­trophin.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.