Precision BioSciences, Inc. today announced the dosing of the first patient in the Phase 1/2 FUNCTION-DMD clinical trial, evaluating the safety, tolerability, and efficacy of PBGENE-DMD for the treatment of Duchenne. PBGENE-DMD is Precision’s investigational in vivo gene editing program designed to improve function by restoring near full-length dystrophin.
PBGENE-DMD uses a single adeno-associated virus (AAV) to deliver two gene-editing tools that remove a specific section of the dystrophin gene (exons 45–55) creating a large in-frame deletion, with the goal of allowing the body to produce a near full-length, functional dystrophin protein. This approach could potentially apply to up to 60% of people with Duchenne whose variants fall within this region.
“Seeing PBGENE-DMD move from research into the clinic turns the possibility of gene editing for Duchenne into reality — a novel approach that could address some of the limitations of currently available therapies,” said PPMD’s Founding President Pat Furlong. “Families have been waiting for options like this, and PPMD is encouraged to see this program advance. We look forward to learning more as the study progresses and continuing collaboration on behalf of all our Duchenne families.”
The FUNCTION-DMD study is currently enrolling ambulatory Duchenne patients between the ages of 2 and 7 with mutations between exons 45 and 55 across multiple U.S. clinical trial sites. The study is actively recruiting patients at specialized Duchenne care centers, and Precision Biosciences reports that it expects initial safety data by year-end 2026.
Read Precision Biosciences’ press release here.
Read the company’s community letter below:
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 patientadvocacy@precisionbiosciences.com.
Sincerely,
Sam Collins, MBBS, PhD
Medical Director / Senior Vice President, Clinical Development
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