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

NCT07429240 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: PBGENE-DMD-01
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this Phase 1/2a trial is to evaluate the safety, tolerability, and preliminary efficacy of PBGENE-DMD in patients with DMD harboring mutations amenable to excision of exons 45-55. Given the limitations of existing therapeutic strategies, PBGENE-DMD represents a novel, innovative approach with the potential for a one-time, durable correction of the underlying genetic defect in the largest molecular subset of patients with DMD.

Conditions

Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD

Keywords

Duchenne muscular dystrophy; DMD; X-linked Muscle wasting disease; Muscular dystrophy; Progressive muscle weakness; Gene editing; AAV serotype 9; AAV-9; Exon 45-55 excision

Outcome Measures

Primary Outcomes (1)

• Incidence, severity, and causality of treatment-emergent adverse events and serious adverse events

  Adverse events and serious adverse events that occur or worsen after initiation of the investigational treatment

  From Dosing through Week 104

Secondary Outcomes (1)

• Dystrophin expression in skeletal muscle

  Week 12, Week 52

Other Outcomes (4)

• Developmental Motor Function 1

  Week 52, Week 104

• Developmental Motor Function 2

  Week 52, Week 104

• Developmental Motor Function 3

  Week 52, Week 104

Study Arms (1)

Experimental- Part 1 (Initial Safety) & Part 2 (Expansion) cohort

EXPERIMENTAL

The trial is planned to enroll participants into 2 parts as follows: * Part 1 (Initial Safety) A total of up to 6 participants may be enrolled. * Part 2 (Expansion) Up to 12 participants

Biological: PBGENE-DMD (IV)

Interventions

PBGENE-DMD (IV) BIOLOGICAL

Participants will receive a single dose of PBGENE-DMD

Experimental- Part 1 (Initial Safety) & Part 2 (Expansion) cohort

Eligibility Criteria

• Age: 2 Years - 7 Years
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Males, 2 to 7 years of age, inclusive, at the time of informed consent/assent
• Molecular confirmed DMD diagnosis (DMD mutation fully contained between exons 45 to 55 \[inclusive\])
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Precision BioSciences, Inc.: lead

Study Sites (1)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Muscular Dystrophies

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Precision BioSciences Clin Ops

CONTACT

(800) 593-0346; function-DMD@precisionbiosciences.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Masking Details: Open label
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: A single dose of PBGENE-DMD administered Intravenously (IV) following screening and pretreatment. Total duration of trial intervention for each participant: approximately 130 weeks

Study Record Dates

• First Submitted: February 17, 2026
• First Posted: February 24, 2026
• Study Start: April 24, 2026
• Primary Completion (Estimated): November 1, 2029
• Study Completion (Estimated): December 1, 2029
• Last Updated: April 30, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)