What drugs target Dystrophin gene?

3+ drugs target Dystrophin gene, including .

What drug classes act on Dystrophin gene?

Drug classes acting on Dystrophin gene include Gene therapy.

Dystrophin gene — Drug Target

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

3 drugs 2 in Phase 3

All drugs that target Dystrophin gene — marketed and clinical-stage. Includes 1 drug classes acting on this target.

Drug classes

Gene therapy

Phase 3 pipeline (2)

SRP-4045 · Sarepta Therapeutics, Inc. · Gene therapy · Muscular dystrophy
SRP-4045 is a micro-dystrophin gene therapy that aims to treat Duchenne muscular dystrophy by providing a functional copy of the dystrophin gene.
SGT-003 · Solid Biosciences Inc. · Gene therapy · Muscular dystrophy
SGT-003 is a gene therapy that aims to deliver a functional dystrophin gene to muscle cells.

Phase 2 pipeline (1)

CX501 · Tigenix S.A.U. · Muscular Dystrophy
CX501 is a gene therapy that aims to increase the production of dystrophin in patients with Duchenne muscular dystrophy.