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Duchenne Therapeutic Pipeline
CureDuchenne Funded
Dystrophin Gene Therapy
Mechanism | Company | Drug | Status | |
|---|---|---|---|---|
| Micro-dystrophin Gene Replacement | Sarepta Therapeutics | Elevidys/SRP-9001 |  Approved | |
| Micro-dystrophin Gene Replacement | Pfizer | PF-06939926 |  Discontinued |  |
| Micro-dystrophin Gene Replacement | Genethon (Sarepta) | GNT-0004 |  Clinical | |
| Micro-dystrophin Gene Replacement | RegenxBio | RGX-202 |  Clinical | |
| Micro-dystrophin Gene Replacement | Solid Biosciences | SGT-001 | Clinical | |
| Micro-dystrophin Gene Replacement | Solid Biosciences | SGT-003 | Clinical | |
| Micro-dystrophin Gene Replacement | Insmed | INS1201 | Clinical | |
| Micro-dystrophin Gene Replacement | Ultragenyx | UX810 |  Preclinical |
Dystrophin Gene Editing
Mechanism | Company | Drug | Status | |
|---|---|---|---|---|
| Genome Editing (Exon 45-55) | MyoGene Bio | CRISPR/Cas9 | Preclinical | |
| Genome Editing (Exon 45-55) | Precision BioSciences | ARCUS-Mediated Excision | Preclinical |
Dystrophin Exon Skipping
Mechanism | Company | Drug | Status | |
|---|---|---|---|---|
| PMO | Sarepta Therapeutics | Exondys 51 (Exon 51) | Accelerated Approval | |
| PMO | Sarepta Therapeutics | Vyondys 53 (Exon 53) | Accelerated Approval | |
| PMO | Sarepta Therapeutics | Amondys 45 (Exon 45) | Accelerated Approval | |
| PPMO | Sarepta Therapeutics | SRP-5051 (Exon 51) | Discontinued | |
| PMO | NS Pharma | Viltepso (Exon 53) | Accelerated Approval | |
| PMO | NS Pharma | NS-089/NCNP-02 (Exon 44) | Clinical | |
| PMO | NS Pharma | NS‐050/NCNP‐03 (Exon 50) | Clinical | |
| PMO | NS Pharma | Exon 51, 55, 45 | Preclinical | |
| AAV-U7 snRNA | Nationwide Children’s | Exon 2 | Clinical | |
| Stereopure-PN-ASO | Wave Life Sciences | WVE-N531 (Exon 53) | Clinical | |
| Stereopure-PN-ASO | Wave Life Sciences | Exon 44, 45, 51, 52 | Preclinical | |
| Antibody-PMO | Avidity Biosciences | AOC 1044 (Exon 44) | Clinical | |
| Antibody-PMO | Avidity Biosciences | Exon 45, 51 | Preclinical | |
| Antibody-PMO | Dyne Therapeutics | DYNE-251 (Exon 51) | Clinical | |
| Antibody-PMO | Dyne Therapeutics | Exon 44, 45, 53 | Preclinical | |
| PPMO (EDO-CPP) | PepGen | PGN-EDO51 (Exon 51) | Discontinued | |
| PPMO (EDO-CPP) | PepGen | Exon 44, 45, 53 | Discontinued | |
| PPMO (Endosomal Escape) | Entrada Therapeutics | ENTR-601-44 (Exon 44) | Clinical | |
| PPMO (Endosomal Escape) | Entrada Therapeutics | ENTR-601-45 (Exon 45) | Clinical | |
| PPMO (Endosomal Escape) | Entrada Therapeutics | ENTR-601-50 (Exon 50), ENTR-601-51 (Exon 51) | Preclinical | |
| PS-ASO | BioMarin Pharmaceutical | BMN 351 (Exon 51) | Clinical |
Dystrophin Stop Codon Read-Through
Mechanism | Company | Drug | Status | |
|---|---|---|---|---|
| Stop Codon Read-through | PTC Therapeutics | Ataluren | Conditional Approval in EU | |
| Stop Codon Read-through | Tevard Biosciences | tRNA-based approach | Preclinical |
Anti-Inflammatory
Mechanism | Company | Drug | Status | |
|---|---|---|---|---|
| Corticosteroid | PTC Therapeutics | Emflaza | Approved | |
| Corticosteroid | Catalyst/Santhera/ReveraGen | Vamorolone/AGAMREE | Approved | |
OTHERS
Mechanism | Company | Drug | Status | |
|---|---|---|---|---|
| HDAC Inhibitor | Italfarmaco | Givinostat/Duvyzat | Approved | |
| Cardiac-derived Exosomes | Capricor Therapeutics | CAP-1002 | Clinical | |
| Muscle Stabilizer | Edgewise Therapeutics | EDG-5506 | Clinical | |
| CD49d Expression Inhibitor | Percheron Therapeutics | ATL1102 | Discontinued | |
| Anti-IL1beta Antibody | Children’s Research Institute | Ilaris | Clinical | |
| PGD synthase Inhibitor | Taiho Pharmaceutical | TAS-205 | Clinical | |
| IGF-1 | Ipsen | Increlex | Clinical | |
| Thromboxane Receptor Inhibitor | Cumberland Pharmaceuticals | Ifetroban | Clinical |
Last Updated June 2025