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 | Ultragenyx | UX810 |  Preclinical |
Micro-dystrophin Gene Replacement | Insmed | INS1201 | 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 |
PPMO | Sarepta Therapeutics | Exon 44, 45, 50, 52, 53 | Preclinical |
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 | scAAV9.U7.ACCA (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) | Clinical |
PPMO (EDO-CPP) | PepGen | Exon 44, 45, 53 | Preclinical |
PPMO (Endosomal Escape) | Entrada Therapeutics | ENTR-601-44 (Exon 44) | Clinical |
PPMO (Endosomal Escape) | Entrada Therapeutics | Exon 45, 50, 51 | Preclinical |
PS-ASO | BioMarin Pharmaceutical | BMN 351 (Exon 51) | Clinical |
Dystrophin Gene Editing
Mechanism | Company | Drug | Status |
|---|---|---|---|
Genome Editing | Vertex Pharmaceuticals | CRISPR/Cas9 | Preclinical |
Genome Editing (Exon 45-55) | MyoGene Bio | CRISPR/Cas9 | Preclinical |
Genome Editing (Exon 45-55) | Precision BioSciences | ARCUS-Mediated Excision | Preclinical |
Genome Editing (Exon 50) | GenAssist | GEN6050X | 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 | hC Bioscience | tRNA-based approach | Preclinical |
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 |
Other Treatments
Mechanism | Company | Drug | Status |
|---|---|---|---|
Oral Molecule Drug | Satellos | SAT-3247 | Clinical |
HDAC Inhibitor | Italfarmaco | Givinostat/Duvyzat | Approved |
Cardiac-derived Exosomes | Capricor Therapeutics | CAP-1002 | Clinical |
Muscle Stabilizer | Edgewise Therapeutics | EDG-5506 | Clinical |
CD49d Expression Inhibitor | Antisense Therapeutics | ATL1102 | Clinical |
Anti-IL1beta Antibody | Children’s Research Institute | Ilaris | Clinical |
PGD synthase Inhibitor | Taiho Pharmaceutical | TAS-205 | Clinical |
Thromboxane Receptor Inhibitor | Cumberland Pharmaceuticals | Ifetroban | Clinical |
