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.
