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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