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STAT·Aug 19·1 min read
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Experts split over Huidagene’s gene-editing approach after boy’s death in Duchenne trial

Aug 19, 2026·read at STAT ↗Negative

A young boy died while participating in Huidagene’s gene‑editing trial for Duchenne muscular dystrophy, raising serious questions about the company’s experimental strategy.

STAT+ reviewed video of a presentation given by Huidagene’s former CEO at a major gene‑therapy conference three months before the fatal incident. Several gene‑editing and muscle‑disorder experts were asked to assess the data and methodology shown.

The experts were sharply divided. Some said the approach was not unreasonable to try, while others described the data as poor quality and expressed dismay at the trial’s execution.

source

This writeup was produced by pharmadog from original reporting by STAT.

Original headline: “STAT+: In wake of boy’s death, gene editing experts dissect Huidagene’s decisions”

read at STAT ↗
450 words · retrieved Aug 19
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companies & drugs in this story

companies
Ultragenyx Pharmaceutical Inc.3
drugs
Rare Disease2(aeruginascin + baeocystin + psilocybin)MYCO-003Oral PsilocybinPsilocybin (Microneedle Patch)psilocybin
topics
CRISPR/Cas96Duchenne Muscular Dystrophy3Rare Disease3Cell TherapyEndocrinology & MetabolismGene Therapy (AAV)ObesityOphthalmology

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