08/31/2026
Precision BioSciences has commenced dosing in the Phase 1/2 FUNCTION‑DMD study, with the first patient receiving PBGENE‑DMD at Arkansas Children’s Hospital.
This milestone marks the first clinical gene‑editing program for Duchenne muscular dystrophy and advances our wholly owned in vivo gene editing approach into the clinic.
PBGENE‑DMD is designed to permanently edit a patient’s own dystrophin gene, with the aim of restoring production of a near full‑length, functional dystrophin protein. The study is enrolling ambulatory boys ages 2–7 with mutations between exons 45 and 55, a key hotspot region representing up to 60% of boys living with Duchenne.
The Precision team is grateful to the patient, their family, and the clinical team for their commitment to advancing this important work. Initial safety data are expected by year-end 2026.
Read the full release, https://loom.ly/QIQX9EA