Lexeo Therapeutics said Monday that a gene therapy designed to prevent fatal cardiac complications of a genetic disorder called Friedrich’s ataxia reduced signs of heart disease in a handful of patients.
The data are very early and less-than-clear-cut. But given a top Food and Drug Administration official’s recent efforts to advance gene therapies under accelerated approval — most notably, with the decision to expand approval for Sarepta’s Duchenne muscular dystrophy treatment, despite two failed trials— the company thinks it can chart a path to market, after an additional study.
“In my opinion, it sits squarely in the type of framework that Peter Marks has been sort of driving,” said Lexeo CEO Nolan Townsend, referring to the director of the agency’s biologics division.
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