Precision BioSciences, Inc. activated Arkansas Children’s Hospital as the inaugural site for its Phase 1/2 FUNCTION‑DMD study, marking the first patient‑enrollment phase for the company’s PBGENE‑DMD gene‑editing therapy targeting Duchenne muscular dystrophy (DMD). The activation, announced on April 29 2026, enables the company to begin screening and enrolling ambulatory DMD patients whose mutations fall between exons 45 and 55, a genetic subset that represents roughly 60 % of the DMD population.
PBGENE‑DMD has already secured key regulatory designations that accelerate its development path: an Orphan Drug designation granted in July 2025, Fast Track status awarded in February 2026, and eligibility for a Rare Pediatric Disease Priority Review Voucher. These designations reduce the time and cost required to bring the therapy to market, positioning Precision BioSciences to potentially secure a first‑to‑market advantage in a highly competitive gene‑editing landscape.
The therapy is a first‑in‑class in‑vivo gene‑editing approach that excises exons 45‑55 from the dystrophin gene, restoring the production of a near full‑length functional dystrophin protein. By targeting the most common mutation class in DMD, PBGENE‑DMD could provide durable functional improvement for a large segment of patients who currently have limited treatment options.
"The activation of Arkansas Children’s Hospital marks a critical step in advancing PBGENE‑DMD into the clinic and expanding access for patients and families who are looking for novel options to address this devastating genetic disease," said Michael Amoroso, Chief Executive Officer of Precision BioSciences. "Dr. Aravindhan Veerapandiyan and the team at Arkansas Children’s Hospital bring deep experience in running clinical trials and research in Duchenne. As the inaugural site for the FUNCTION‑DMD study, we are pleased to open screening and enrollment at this center while we continue to work to activate other trial sites for inclusion in the FUNCTION‑DMD study."
Precision BioSciences has maintained a robust cash runway, with a current ratio of 13.32 and a net income of $20.1 million reported in Q4 2025, a turnaround from prior losses. The company’s focus on funding its pipeline, particularly the PBGENE‑DMD program, underscores its strategy to leverage its proprietary ARCUS platform across multiple therapeutic areas while preserving financial flexibility for future clinical milestones.
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