Precision BioSciences has dosed the first patient at Arkansas Children’s Hospital, a PPMD Certified Duchenne Care Center and designated MDA Care Center, in the Phase 1/2 Function-DMD clinical trial evaluating PBGENE-DMD, the first clinical gene-editing program for Duchenne muscular dystrophy. The study aims to evaluate the safety, tolerability, and efficacy of PBGENE-DMD in patients ages 2 to 7 living with DMD. For more information, read Precision BioSciences’ community letter below or view the company’s press release.
August 24, 2026
Dear Duchenne Community
At Precision BioSciences, we’re honored to be part of the Duchenne community’s ongoing efforts to advance research and explore new treatment possibilities. We are deeply grateful for the trust, support, and engagement families, advocates, and community members continue to share with us.
Today, we’re pleased to share an important milestone for the FUNCTION-DMD clinical trial: the first participant has received our investigational study treatment, PBGENE-DMD. This is a meaningful moment for everyone involved in the study and represents the beginning of an important phase of clinical research. It reflects the commitment of participating families, investigators, study site teams, and the broader Duchenne community, whose partnership makes this work possible.
The FUNCTION-DMD clinical trial is evaluating PBGENE-DMD, our investigational gene editing program designed to address the underlying cause of Duchenne muscular dystrophy. As the study continues, we will work closely with participating families and study sites to better understand the safety profile and potential effects of this approach.
While this milestone is an exciting step forward, we recognize that progress in clinical research takes time. We are especially thankful to the families who choose to participate in research and contribute to advancing knowledge for the Duchenne community.
We also understand how important clear and timely communication is. As enrollment continues and the study progresses, we remain committed to sharing updates and communicating transparently about what we learn along the way.
Our team remains focused on advancing this research carefully and responsibly, with the goal of helping improve the future for individuals living with Duchenne and their families. We look forward to keeping you informed as the study moves forward.
If you have questions about the FUNCTION-DMD trial, please contact our Patient Advocacy team at [email protected].
Sincerely,
Sam Collins, MBBS, PhD
Medical Director / Senior Vice President, Clinical Development