Precision BioSciences has developed PBGENE-DMD, an ARCUS gene editor designed to excise the ‘mutation hot spot’ region spanning exons 45 through 55 of the dystrophin gene with the goal of permanently and safely restoring muscle function.

PBGENE-DMD
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FUNCTION-DMD is a phase I/II trial for ambulatory boys aged 2 to 7 with confirmed mutation(s) within exons 45 through 55.

ClinicalTrials.gov Identifier

NCT07429240

To connect with one of our study sites, please contact our Patient Advocacy Team at:  
patientadvocacy@precisionbiosciences.com

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DMD Currently Lacks a Curative Treatment

DMD is a genetic disorder resulting in progressive muscle degeneration and early death. It affects skeletal, cardiac, and respiratory muscles, resulting in progressive weakness, loss of ambulation, respiratory insufficiency, and cardiac failure leading to premature death.

Current DMD treatments have limitations, including their inability to provide durable functional improvement for patients.

∼15K

DMD patients in the United States1

∼550 births
per year in the US

∼300-400K

DMD patients globally1

>20K births
per year globally

PBGENE-DMD Target Population

∼60% of patients

have mutations in exons 45-55, a critical hot spot where a disrupted reading frame prevents dystrophin expression.2

PBGENE-DMD

PBGENE-DMD is designed to permanently address the root genetic cause of DMD by removing a frequently mutated region of the dystrophin gene in ∼60% of patients, restoring the correct reading frame and enabling natural production of near full-length functional dystrophin.

Learn about DMD and PBGENE-DMD





PBGENE-DMD (Muscle Targeted Excision Program)
Validated preclinical studies demonstrate durable improvements in muscle function, supporting ARCUS gene editing as a potential approach to restore near full-length dystrophin and improve long-term functional outcomes for a broad population of patients living with DMD by addressing the root cause of the disease.

Reasons to Believe in Precision's Approach to DMD

The first ARCUS-based therapeutic approach designed to excise the 'hot spot' region (exons 45-55) of the dystrophin gene with the goal of permanently and safely restoring muscle function for the majority of patients living with DMD
The protein produced by PBGENE-DMD has known function in humans
Designed for durable improvements in muscle function independent of the persistence of AAV transgene; durable functional improvements maintained for up to 9 months in mouse models
Manufacturing process produces high-quality AAV with >90% full capsids for enhanced potency and safety

PBGENE-DMD is an investigational therapy that has not been approved by the U.S. Food and Drug Administration (FDA) or any other regulatory authority.
Although the safety and efficacy profiles are currently unknown, it is being assessed in the FUNCTION-DMD clinical trial.

Explore the science behind our approach and the research guiding our work.

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Clinical Trial Now Enrolling

  1. Prevalence and incidence based on CureDuchenne and Orphanet Journal of Rare Diseases.
  2. Béroud C, et al. Hum Mutat. 2007;28(2):196-202.