Precision BioSciences Initiates Dosing in Phase 1/2 FUNCTION-DMD Trial for Duchenne Muscular Dystrophy
DTIL has more than doubled off its 52-week low of $3.53.
Summary
Precision BioSciences announced the first patient has been dosed in its Phase 1/2 FUNCTION-DMD trial for PBGENE-DMD, a gene-editing therapy for Duchenne muscular dystrophy. Initial safety data is expected by year-end 2026.
Key Events · Product Development and Regulatory · DTIL
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First Patient Dosed in FUNCTION-DMD Trial
The first patient has been dosed with PBGENE-DMD at Arkansas Children's Hospital, marking the first clinical gene-editing program for Duchenne muscular dystrophy.
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PBGENE-DMD Targets Up to 60% of DMD Patients
The therapy uses two ARCUS nucleases in a single AAV to excise exons 45-55, aiming to restore near full-length functional dystrophin in patients with mutations in this hot spot region.
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Initial Safety Data Expected by Year-End 2026
The Phase 1/2 study is enrolling ambulatory DMD patients ages 2-7 across multiple U.S. sites, with initial safety data anticipated by year-end 2026.
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Regulatory Designations Support Program
PBGENE-DMD holds Orphan Drug Designation (July 2025), Fast Track designation (February 2026), and is eligible for a Rare Pediatric Disease Priority Review Voucher.
Analysis · DTIL · Life Sciences
The first patient has been dosed in the Phase 1/2 FUNCTION-DMD trial evaluating PBGENE-DMD, Precision BioSciences' wholly owned in vivo gene editing therapy for Duchenne muscular dystrophy. This marks the first clinical gene-editing program for DMD, targeting up to 60% of patients with mutations in the exon 45-55 hot spot region. The milestone validates the ARCUS platform's ability to deliver two complementary nucleases in a single AAV to excise exons 45-55 and restore near full-length dystrophin. Initial safety data is expected by year-end 2026, providing a near-term readout for this high-unmet-need indication.
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At the time of this filing, DTIL was trading at $8.95 on NASDAQ in the Life Sciences sector, with a market capitalization of approximately $244.3M. The 52-week trading range was $3.53 to $9.62. This filing was assessed with positive market sentiment and an importance score of 7 out of 10.