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.
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