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Mechanism | Company | Drug | Status | ![]() |
---|---|---|---|---|
Micro-dystrophin Gene Replacement | Sarepta Therapeutics | Elevidys/SRP-9001 | ![]() | |
Micro-dystrophin Gene Replacement | Pfizer | PF-06939926 | ![]() | ![]() |
Micro-dystrophin Gene Replacement | Genethon (Sarepta) | GNT-0004 | ![]() | |
Micro-dystrophin Gene Replacement | RegenxBio | RGX-202 | ![]() | |
Micro-dystrophin Gene Replacement | Solid Biosciences | SGT-001 | ![]() | |
Micro-dystrophin Gene Replacement | Solid Biosciences | SGT-003 | ![]() | |
Micro-dystrophin Gene Replacement | Ultragenyx | UX810 | ![]() | |
Micro-dystrophin Gene Replacement | Insmed | INS1201 | ![]() | ![]() |
Mechanism | Company | Drug | Status | ![]() |
---|---|---|---|---|
Genome Editing | Vertex Pharmaceuticals | CRISPR/Cas9 | ![]() | ![]() |
Genome Editing (Exon 45-55) | MyoGene Bio | CRISPR/Cas9 | ![]() | ![]() |
Genome Editing (Exon 45-55) | Precision BioSciences | ARCUS-Mediated Excision | ![]() |
Mechanism | Company | Drug | Status | ![]() |
---|---|---|---|---|
PMO | Sarepta Therapeutics | Exondys 51 (Exon 51) | ![]() | ![]() |
PMO | Sarepta Therapeutics | Vyondys 53 (Exon 53) | ![]() | |
PMO | Sarepta Therapeutics | Amondys 45 (Exon 45) | ![]() | |
PPMO | Sarepta Therapeutics | SRP-5051 (Exon 51) | ![]() | |
PPMO | Sarepta Therapeutics | Exon 44, 45, 50, 52, 53 | ![]() | |
PMO | NS Pharma | Viltepso (Exon 53) | ![]() | |
PMO | NS Pharma | NS-089/NCNP-02 (Exon 44) | ![]() | |
PMO | NS Pharma | NS‐050/NCNP‐03 (Exon 50) | ![]() | |
PMO | NS Pharma | Exon 51, 55, 45 | ![]() | |
AAV-U7 snRNA | Nationwide Children’s | Exon 2 | ![]() | ![]() |
Stereopure-PN-ASO | Wave Life Sciences | WVE-N531 (Exon 53) | ![]() | |
Stereopure-PN-ASO | Wave Life Sciences | Exon 44, 45, 51, 52 | ![]() | |
Antibody-PMO | Avidity Biosciences | AOC 1044 (Exon 44) | ![]() | ![]() |
Antibody-PMO | Avidity Biosciences | Exon 45, 51 | ![]() | ![]() |
Antibody-PMO | Dyne Therapeutics | DYNE-251 (Exon 51) | ![]() | ![]() |
Antibody-PMO | Dyne Therapeutics | Exon 44, 45, 53 | ![]() | ![]() |
PPMO (EDO-CPP) | PepGen | PGN-EDO51 (Exon 51) | ![]() | ![]() |
PPMO (EDO-CPP) | PepGen | Exon 44, 45, 53 | ![]() | ![]() |
PPMO (Endosomal Escape) | Entrada Therapeutics | Exon 44 | ![]() | ![]() |
PPMO (Endosomal Escape) | Entrada Therapeutics | Exon 45, 50, 51 | ![]() | |
PS-ASO | BioMarin | BMN 351 (Exon 51) | ![]() | ![]() |
Mechanism | Company | Drug | Status | ![]() |
---|---|---|---|---|
Stop Codon Read-through | PTC Therapeutics | Ataluren | ![]() | ![]() |
Stop Codon Read-through | hC Bioscience | tRNA-based approach | ![]() | ![]() |
Stop Codon Read-through | Tevard Biosciences | tRNA-based approach | ![]() |
Mechanism | Company | Drug | Status | ![]() |
---|---|---|---|---|
Corticosteroid | PTC Therapeutics | Emflaza | ![]() | |
Corticosteroid | Catalyst/Santhera/ReveraGen | Vamorolone/AGAMREE | ![]() | ![]() |
Mechanism | Company | Drug | Status | ![]() |
---|---|---|---|---|
HDAC Inhibitor | Italfarmaco | Givinostat/Duvyzat | ![]() | |
Cardiac-derived Exosomes | Capricor Therapeutics | CAP-1002 | ![]() | ![]() |
Muscle Stabilizer | Edgewise Therapeutics | EDG-5506 | ![]() | ![]() |
CD49d Expression Inhibitor | Antisense Therapeutics | ATL1102 | ![]() | |
Anti-IL1beta Antibody | Children’s Research Institute | Ilaris | ![]() | |
PGD synthase Inhibitor | Taiho Pharmaceutical | TAS-205 | ![]() | |
IGF-1 | Ipsen | Increlex | ![]() | |
Thromboxane Receptor Inhibitor | Cumberland Pharmaceuticals | Ifetroban | ![]() |
Last Updated 03/28/2024