and othersExeo Therapeutics Inc. announced Monday that a gene therapy designed to prevent fatal cardiac complications from a genetic disorder called Friedrich’s ataxia has reduced signs of heart disease in a small number of patients.
The data is still early and far from clear, but given recent efforts by Food and Drug Administration officials to advance gene therapies under accelerated approval — particularly its decision to expand approval of Sarepta’s Duchenne muscular dystrophy drug despite two failed trials — the company believes it can chart a path to market after additional studies.
“In my opinion, this fits squarely into the kind of framework that Peter Marks has been promoting,” Lexeo CEO Nolan Townsend said, referring to the head of the agency’s biologics division.
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