With new data, Regenxbio advances plan for Duchenne gene therapy

Regenxbio said Thursday that its experimental gene therapy for Duchenne muscular dystrophy was administered to two more boys and produced high levels of a miniaturized version of the protein needed for muscles to function properly.

The updated results from an early study suggest the Regenxbio therapy, called RGX-202, could become the second genetic medicine for Duchenne to reach the market, following the Food and Drug Administration’s approval of Sarepta Therapeutics’ Elevidys in June.

A third biotech, called Solid Biosciences, is also developing its own Duchenne gene therapy, with early patient dosing underway.

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