Eyeing accelerated approval for Duchenne gene therapy, Sarepta hopes to bring in $1B in bid for profitability

The Duchenne muscular dystrophy-focused biotech is seeking $1 billion ahead of a potential accelerated approval for its gene therapy, which it posted a generally positive readout for in July — though it did report a new case of myocarditis. Following that readout, Sarepta said it was planning to submit for accelerated approval with the FDA, and analysts said an adcomm was a “near certainty.”