Sarep­ta touts clean da­ta for com­mer­cial grade Duchenne gene ther­a­py, clear­ing key hur­dle to PhI­II

Af­ter a tri­al flop and an un­ex­pect­ed de­tour, Sarep­ta says its gene ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy looks ready to get back on the Phase III track.

A re­view of pre­lim­i­nary safe­ty and ex­pres­sion da­ta from 11 pa­tients sug­gests that the ma­te­r­i­al man­u­fac­tured with their up­dat­ed com­mer­cial process spurred con­sis­tent pro­duc­tion of dy­s­trophin, the biotech re­port­ed, while main­tain­ing a clean side ef­fect pro­file. The study, SRP-9001-103, was the first to test Sarep­ta’s com­mer­cial grade process in pa­tients.

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