STAT+: Young boy dies in trial for Pfizer Duchenne gene therapy

A young boy died in a trial for Pfizer’s experimental gene therapy for Duchenne muscular dystrophy, the company told patient advocates Tuesday.

The boy was enrolled in Daylight, a trial studying the treatment in boys aged 2 or 3. The boy had received the therapy early last year, Pfizer told the advocates in a note posted online by Parent Project Muscular Dystrophy.

Pfizer said it had not yet determined precisely what happened or how the boy died. But it is pausing crossing over patients in its randomized phase 3 trial of the treatment in older boys — meaning it will, for now, stop dosing patients who initially received a placebo with the gene therapy after a year. Pfizer is expected to announce data from that study in the coming weeks.

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