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

Conditions

• Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD

Eligibility Criteria

Sex

MALE

Age

2 Years - 7 Years

Healthy Volunteers

Not accepted

Interventions

• PBGENE-DMD (IV) — BIOLOGICAL
  Participants will receive a single dose of PBGENE-DMD

Study Details

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.

Key Dates

Start date

Apr 24, 2026

Status verified

Apr 2026

Primary completion

Nov 30, 2029

Completion

Dec 31, 2029

Study Design

Enrollment

18 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

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

Primary Outcome Measure

Incidence, severity, and causality of treatment-emergent adverse events and serious adverse events [ Time Frame: From Dosing through Week 104 ]

Central Contacts

• Precision BioSciences Clin Ops
  (800) 593-0346
  [email protected]

Locations (1)

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