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Drug
Delandistrogene moxeparvovec
In clinical developmentClassDystrophin exogenous geneEmerging researchEarly-to-mid development
Also known as Elevidys, rAAVrh74.MHCK7.micro-dystrophin.
1
Research papers
1
Active clinical trials
Dystrophin
Primary target
Latest activity
betaRecent clinical, regulatory, research and industry developments relating to this drug.
Clinical trial2026-06-24Results expected Q4 2027 · ClinicalTrials.gov
Clinical trial2025-10-10Terminated · ClinicalTrials.gov
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial.
Research2024-10-09Nature medicine
Profile
Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.
Canonical name
Delandistrogene moxeparvovec
Aliases & brands
ElevidysrAAVrh74.MHCK7.micro-dystrophin
RxNorm CUI
2641675
ChEMBL ID
CHEMBL4650241
ATC codes
M09AX15
UNII
2P6QV2ZE52
Primary mechanism
Dystrophin exogenous gene
Pharmacology & targets
Known molecular targets and mechanisms supported by curated pharmacology databases.
Clinical trials
The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.
Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY2
registered trials across all phases
1
Active studies
1
Recruiting
1
Discontinued
PHASE DISTRIBUTIONn = 2
Phase 12
Research activity
Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.
Most influential
Major research themes4
Dependovirus1Gene Therapy Agents1Genetic Therapy1Muscular Dystrophy, Duchenne1
Journals, researchers & institutions
Top journals
- Nature medicine1
Leading researchers
- Asher DR1
- Ciafaloni E1
- Darton E1
- Elkins JS1
- Fontoura P1
- Guridi M1
- Komaki H1
- Leon-Astudillo C1
Leading institutions
free-text, unnormalised
- Center for Gene Therapy1
- Center for Neuromuscular Disorders in Children and Adolescents1
- Children's Hospital of the King's Daughters1
- Dubowitz Neuromuscular Centre1
- Hospital Sant Joan de Déu1
- Pediatric Neurology Institute1
References & data sources
- RxNorm (U.S. National Library of Medicine) — drug identity
- ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
- Europe PMC — research literature
- ClinicalTrials.gov — clinical trials