Pa­tient death in piv­otal gene ther­a­py tri­al rat­tles Sarep­ta part­ner al­ready fac­ing clin­i­cal hold

A child with mu­copolysac­cha­ri­do­sis type II­IA (MPS II­IA) has died in Lyso­gene’s piv­otal tri­al for its lead gene ther­a­py, four months af­ter the FDA im­posed a clin­i­cal hold on the study for pos­si­ble safe­ty con­cerns.

The study was sup­posed to pave the path to­ward com­mer­cial­iza­tion by Sarep­ta, which inked a $125 mil­lion deal in 2018 to mar­ket the drug, LYS-SAF302, in the US and world­wide ex­cept Eu­rope.

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