Sarepta picks up a slate of preclinical gene therapy programs, seeding new StrideBio deal with $48M upfront
Having tasted early success in treating a number of muscular dystrophies with gene therapy, Sarepta is setting itself up to tackle more rare diseases.
The Boston-based biotech has picked StrideBio for a partnership evidently designed to run years, kicking things off with $48 million upfront and four central nervous system targets. The smaller partner is tasked with all R&D work up to the IND stage, coming up with the AAV-based therapies to be tested in Rett syndrome (MECP2), Dravet syndrome (SCN1A), Angelman syndrome (UBE3A) and Niemann-Pick (NPC1).
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