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, leading to progressive weakness, loss of ambulation, respiratory insufficiency, and cardiac failure leading to premature death.

Current DMD treatments have limitations and do not provide durable or significant functional improvements for patients with DMD.

~15k

DMD Patients in the United States

~550 births
per year in the US

~300-400k

DMD Patients Globally

> 20K births
per year globally

~60% of patients

have mutations in exons 45-55, a critical hotspot where disrupted reading frame prevents dystrophin expression

PBGENE-DMD:

PBGENE-DMD is designed to permanently correct 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.

Reasons to Believe in Precision’s Approach to DM

The first ARCUS-based therapeutic approach designed to permanently correct the dystrophin gene.
Novel mechanism results in naturally-produced functional dystrophin protein retaining the vast majority of near full-length dystrophin protein domains.
Designed to provide long-term therapeutic benefit, as supported by mouse studies showing levels of naturally-produced functional dystrophin out to 9 months after dosing.
In vivo gene editing offers a novel approach for DMD patients and a path towards meaningful functional benefits.

Clinical trial expected to initiate in 2026