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SGT-003

Gene therapy (AAV vector) · Gene replacement · developed by Solid Biosciences

Phase 3 Most advanced trial

SLDB Developer

1 Registered trials

What is SGT-003?

SGT-003 is an AAV-based gene therapy that delivers a codon-optimized, CpG island-minimized human 5-repeat microdystrophin gene. It is being studied in Duchenne muscular dystrophy.

Summary written with AI from the sources listed at the end of this page.

At a glance

Drug type: Gene therapy (AAV vector)
Mechanism: Gene replacement
Therapy area: Musculoskeletal
Developer: Solid Biosciences Inc. (SLDB)
FDA status: Investigational, not approved in the US. Most advanced trial: Phase 3

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT07160634	Phase 3	Duchenne Muscular Dystrophy	Recruiting	SLDB

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

Duchenne Muscular Dystrophy

More from Solid Biosciences

SGT-212 Phase 1 SGT-501 Phase 1

Sources

ClinicalTrials.gov NCT07160634

Updated Oct 3, 2026. Something wrong or missing? Report it on the SLDB page.

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Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z