DURHAM, N.C. — Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for high unmet need diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to PBGENE-DMD for the treatment of Duchenne muscular dystrophy (DMD). The Company also announced that it will host a virtual key opinion leader (KOL) event on Tuesday, March 17, 2026, at 9:00 AM ET to discuss PBGENE-DMD and the planned Phase 1/2 FUNCTION-DMD clinical study.
“Fast Track designation is an important regulatory milestone for PBGENE-DMD and reflects the significant unmet need in DMD,” said Michael Amoroso, Chief Executive Officer of Precision BioSciences. “We believe this designation, together with our recent IND clearance, supports PBGENE-DMD’s continued momentum towards clinical investigation in boys living with DMD.”
Fast Track designation is intended to facilitate the development of investigational therapies for serious conditions with unmet medical need and may support more efficient interactions with the FDA as development advances. Precision believes this designation further underscores the potential of PBGENE-DMD and the urgent need for new DMD treatment options.
In addition, on March 17, 2026, Precision will host an investor event that will feature key DMD leaders Dr. Aravindhan Veerapandiyan, MD, Pediatric Neurologist and Associate Professor of Pediatrics, Arkansas Children’s Hospital, and Pat Furlong, Founding President of Parent Project Muscular Dystrophy. Parent Project Muscular Dystrophy (PPMD) is the largest nonprofit organization in the United States focused entirely on Duchenne muscular dystrophy and fights to end Duchenne by advancing research, improving care, and expanding access to therapies. The presenters will discuss the unmet need, current treatment landscape and PBGENE-DMD for Duchenne muscular dystrophy (DMD), including an overview of the clinical trial design for the FUNCTION-DMD trial.
About Duchenne Muscular Dystrophy Investor Event
Date and Time: Tuesday, March 17, 2026, at 9:00 AM ET
A live question-and-answer session with investors and analysts will follow the formal presentation. To register for the event, please click here. A replay of the webinar will be accessible in the Investors section of Precision’s website following the event.
About PBGENE-DMD
PBGENE-DMD, a novel first-in-class gene editing therapy, utilizes a gene excision approach that is clearly differentiated from existing microdystrophin and exon skipping treatments. PBGENE-DMD is designed to potentially provide durable functional muscle improvement for DMD patients with mutations in exons 45-55, representing up to 60% of boys with DMD. A single AAV encodes two ARCUS proteins designed to permanently edit a patient’s DNA within the dystrophin gene, resulting in a naturally-expressed, near full-length, functional dystrophin protein. Supported by robust preclinical evidence, PBGENE-DMD is designed to drive functional improvement over time by targeting muscle satellite cells.
In preclinical studies, PBGENE-DMD demonstrated the ability to target key muscle types involved in the progression of DMD and produced significant, durable functional improvements in a humanized DMD mouse model. PBGENE-DMD restored the body’s ability to produce a near full-length functional dystrophin protein across multiple muscles, including cardiac tissue, diaphragm and various key skeletal muscle groups. In addition, PBGENE-DMD edited satellite muscle stem cells, believed to be critical for long-term durability and sustained functional improvement.
About FUNCTION-DMD Trial
The Phase 1/2 FUNCTION-DMD study is expected to enroll ambulatory DMD patients between the age of 2-7 with mutations between exons 45 and 55 representing up to 60% of boys with DMD. The objective of the FUNCTION-DMD study is to evaluate safety, tolerability, and efficacy, including dystrophin protein expression and functional outcomes in patients afflicted with DMD. For more information about this clinical trial and contact information, please visit www.clinicaltrials.gov and search for NCT07429240.
Investor and Media Contact
Naresh Tanna
Vice President of Investor Relations
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