About the Duchenne Drug Development Pipeline
This page shows investigational Duchenne programs as a horizontal product pipeline. Each row represents a drug or therapy with its sponsoring company and therapeutic approach, positioned by the most advanced clinical phase recorded in the catalog.
Columns run from Early Phase 1 through Phase 4. The filled bar shows how far each program has progressed. Programs are sorted with the most advanced developments at the top and Early Phase 1 programs at the bottom.
Observational studies and records labeled only as interventional are excluded so the chart focuses on interventional drug development phases. Click a program name to open its full trial page with eligibility, locations, and registry links.
Explore related views in Home, Active DMD Clinical Studies, and DMD Clinical Trial Statistics. The catalog was last updated September 1, 2026; the product pipeline changes when new trials are added or phases are revised.
- Phase 4
Prednisolone
Reducing Inflammation
Nationwide Children's Hospital
- Phase 4
ELEVIDYS (ENHANCE)
Gene Therapy
Sarepta Therapeutics
- Phase 3
AOC 1044 (SAFARI44)
Exon Skipping
Avidity Biosciences
- Phase 3
Deramiocel (HOPE-3)
Improving Muscle Growth & Protection
Capricor
- Phase 3
DYNE-251 (FORZETTO)
Exon Skipping
Dyne Therapeutics
- Phase 3
GNT0004
Gene Therapy
Genethon
- Phase 3
ELEVIDYS (ENVISION)
Gene Therapy
Sarepta Therapeutics
- Phase 3
SGT-003
Gene Therapy
Solid Biosciences
- Phase 2/3
RGX-202
Gene Therapy
REGENXBIO
- Phase 2
AOC 1044 (EXPLORE44)
Exon Skipping
Avidity Biosciences
- Phase 2
BMN 351
Exon Skipping
BioMarin Pharmaceutical
- Phase 2
Deramiocel (HOPE-2-OLE)
Improving Muscle Growth & Protection
Capricor
- Phase 2
Sevasemten (EDG-5506)
Improving Muscle Growth & Protection
Edgewise Therapeutics
- Phase 2
ELEVATE-LTE
Exon Skipping
Entrada Therapeutics
- Phase 2
GNR-097
Gene Therapy
GENERIUM
- Phase 2
ELEVIDYS (ENVOL)
Gene Therapy
Hoffmann-La Roche
- Phase 2
Satralizumab
Enhancing Bone Health
Hoffmann-La Roche
- Phase 2
KER-065
Other Therapeutic Approaches
Keros Therapeutics
- Phase 2
UC-MSC
Other Therapeutic Approaches
MED Institute Inc.
- Phase 2
Brogidirsen (NS-089/NCNP-02-201)
Exon Skipping
NS Pharma
- Phase 2
SAT-3247
Improving Muscle Growth & Protection
Satellos Bioscience
- Phase 2
WVE-N531 (FORWARD-53)
Exon Skipping
Wave Life Sciences
- Phase 1/2
DYNE-251 (DELIVER)
Exon Skipping
Dyne Therapeutics
- Phase 1/2
DT-DEC01
Improving Muscle Growth & Protection
Dystrogen Therapeutics
- Phase 1/2
ENTR-601-44 (ELEVATE-44)
Exon Skipping
Entrada Therapeutics
- Phase 1/2
ENTR-601-45 (ELEVATE-45)
Exon Skipping
Entrada Therapeutics
- Phase 1/2
NS-050/NCNP-03 (Meteor50)
Exon Skipping
NS Pharma
- Phase 1/2
PBGENE-DMD
Gene Editing
Precision BioSciences
- Phase 1/2
SGT-001
Gene Therapy
Solid Biosciences
- Phase 1
INS1201
Gene Therapy
Insmed Gene Therapy LLC
- Phase 1
MyoPAXon
Improving Muscle Growth & Protection
Myogenica
- Phase 1
SRD-001
Improving Heart Function
Sardocor
- Phase 1
ELEVIDYS
Gene Therapy
Sarepta Therapeutics
- Early Phase 1
GEN6050X
Exon Skipping
GenAssist Therapeutics
- Early Phase 1
RAG-18
mRNA / tRNA / saRNA Therapies
Peking Union Medical College Hospital
- Early Phase 1
BBM-D101
Gene Therapy
Shanghai Jiao Tong University School of Medicine
- Early Phase 1
LE051
Exon Skipping
Shanghai Jiao Tong University School of Medicine
- Early Phase 1
SPOT-mRNA03 (SPOT-03)
Gene Therapy
Shanghai Siponuoyin Biotechnology Co Ltd
