Microcap gene therapy player says three children suffered serious side effects in PhI/II study
A small biotech looking to develop a gene therapy for a rare condition that can cause color or legal blindness ran into some significant safety trouble in a Phase I/II dose-escalation study.
Applied Genetic Technologies Corporation, also known as AGTC, reported Thursday afternoon that three of five children in the study’s highest dose cohort experienced severe inflammation one month after dosing. The events were concerning enough to be categorized as suspected unexpected serious adverse reactions, or SUSARs.
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