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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