PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)

Inclusion Criteria

• •1. Males, 2 to 7 years of age, inclusive, at the time of informed consent/assent
• •2. Molecular confirmed DMD diagnosis (DMD mutation fully contained between exons 45 to 55 \[inclusive\])
• •3. Clinical phenotype consistent with DMD in the opinion of the Investigator
• •4. Ability to complete age-appropriate motor testing assessments requirements.
• •Participants aged 2 to \< 4 years at the time of screening must:
• •1. Be able to walk at least 10 meters independently (without assistive devices).
• •2. 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:
• •3. Be able to walk at least 100 meters independently (without assistive devices).
• •4. Have an NSAA total score between 16 and 29, inclusive.
• •5. Participant has received age-appropriate routine childhood immunizations per the local country's national immunization schedule.
• •6. 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.
• •7. The participant and their parent(s)/LAR(s) are willing to participate in a LTFU study after the completion of this trial.