Precision BioSciences has received a Study May Proceed notification from the US Food and Drug Administration (FDA), permitting initiation of institutional review board (IRB) activities and site activation for its Phase I/II FUNCTION-DMD trial of PBGENE-DMD in ambulatory Duchenne muscular dystrophy (DMD) patients.
PBGENE-DMD is a first-in-class in vivo gene editing programme developed solely by Precision BioSciences, intended as a permanent gene excision therapy for DMD patients with mutations between exons 45 and 55.
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The clinical trial aims to assess the tolerability, safety, efficacy, functional outcomes, and dystrophin expression in affected patients.
It will enrol ambulatory DMD patients with mutations spanning exons 45-55. The protocol incorporates an immune modulation regimen and safety monitoring programme at specialised DMD centres.
Initial data from several patients are anticipated by year end 2026, with early efficacy measured by the percentage of near full-length dystrophin protein detected in muscle biopsies.
After receiving supportive data from at least ten DMD patients, Precision BioSciences plans to meet with the FDA to discuss regulatory alignment regarding future development steps.
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By GlobalDataPrecision BioSciences CEO Michael Amoroso said: “The Study May Proceed notification for PBGENE-DMD by the FDA represents yet another regulatory achievement for Precision BioSciences as we advance our second wholly owned programme toward the clinic. Despite approved therapies today, boys with DMD are lacking treatments that lead to functional improvements over time.
“We’re excited to bring this novel gene excision approach for DMD to the clinic with the goal of activating the first clinical site in the US in the first half of 2026. The company will continue to work with multiple IRBs and the FDA to initiate clinical site activations.”
In February 2025, Precision BioSciences reported initial outcomes from the ELIMINATE-B trial of PBGENE-HBV, a gene editing programme for treating chronic hepatitis B patients who are HBeAg-negative.
