Precision BioSciences PBGENE-DMD Phase 1/2a Clinical Trial for Duchenne muscular dystrophy

Actively RecruitingPhase 1/2Gene Editing

Age: 2–7 years
Phase: Phase 1/2
Sponsor: Precision BioSciences
Therapeutic Approach: Gene Editing
Variant Requirement: Molecular confirmed DMD diagnosis (DMD mutation fully contained between exons 45 to 55 [inclusive])
Eligible Sex: Male
Ambulation: Ambulatory
Study Start (Actual): 2026-04-24
Primary Completion (Estimated): 2029-11
Study Completion (Estimated): 2029-12
Enrollment (Estimated): 18
Countries: United States

Males, 2 to 7 years of age, inclusive, at the time of informed consent/assent
Clinical phenotype consistent with DMD in the opinion of the Investigator
Ability to complete age-appropriate motor testing assessments requirements.
Participants aged 2 to < 4 years at the time of screening must:
Be able to walk at least 10 meters independently (without assistive devices).
Be able to rise from the floor without physical assistance (use of a Gowers' maneuver is acceptable).
Participants aged 4 to 7 years at the time of screening must:
Be able to walk at least 100 meters independently (without assistive devices).
Have an NSAA total score between 16 and 29, inclusive.
Participant has received age-appropriate routine childhood immunizations per the local country's national immunization schedule.
The participant's parent(s)/LAR(s) are willing and able to provide written informed consent prior to the initiation of any trial-specific procedures; where applicable, the participant must provide written or verbal assent in accordance with local regulations.
The participant and their parent(s)/LAR(s) are willing to participate in a LTFU study after the completion of this trial.

Prior treatment with any gene therapy, gene editing therapy, or cell-based therapy at any time.
Receipt of any investigational medication or experimental therapy within 6 months prior to Day 1.
Prior or ongoing use of any product designed to increase dystrophin expression, investigational, or otherwise, including exon-skipping therapies, within 6 months of the scheduled Day 1 dose or inability or unwillingness to refrain from initiating or resuming these therapies for at least 5 years following gene therapy administration.
Prior ongoing use of any product designed to increase dystrophin expression, investigational, or otherwise, including exon-skipping therapies, within 6 months of the scheduled Day 1 dose.
Concurrent enrollment in another clinical trial, unless it is observational (non-interventional).
A positive test for antibodies to AAV9
A participant has any condition that would contraindicate treatment with immunosuppression.
Participants with pathogenic mutations in exons 1-44 and/or exons 56-79.
Evidence of cardiomyopathy or clinically significant left ventricular dysfunction, defined as LVEF <50% on screening echocardiogram.

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NCT ID

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Study Requirements and Criteria