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Mechanism | Company | Drug | Status | ![]() | ![]() |
---|---|---|---|---|---|
Genome editing | Vertex (aquired Exonics) | CRISPR/Cas9 | ![]() | ![]() | |
Gene Therapy | Pfizer | PF-06939926 | ![]() | ![]() | ![]() |
Gene Therapy | Sarepta | SRP-9001 | ![]() | ![]() | |
Gene Therapy | Sarepta & Genethon | rAAV2/8 micro-dystrophin | ![]() | ![]() | |
Gene Therapy | Solid Bioscience | SGT-001 | ![]() | ![]() |
Mechanism | Company | Drug | Status | ![]() | ![]() |
---|---|---|---|---|---|
Exon Skipping – PMO | Sarepta | Exondys51 | Exon 51 | ![]() | ![]() | ![]() |
Exon Skipping – PMO | Sarepta | Vyondys53 | Exon 53 | ![]() | ![]() | |
Exon Skipping – PMO | Sarepta | Casimersen | Exon 45 | ![]() | ![]() | |
Exon Skipping – PMO | Sarepta | Exon 52 | ![]() | ![]() | |
Exon Skipping – PPMO | Sarepta | SRP-5051 | Exon 51 | ![]() | ![]() | |
Exon Skipping – PPMO | Sarepta | Exon 44 | ![]() | ![]() | |
Exon Skipping – PPMO | Sarepta | Exon 45 | ![]() | ![]() | |
Exon Skipping – PPMO | Sarepta | Exon 50 | ![]() | ![]() | |
Exon Skipping – PPMO | Sarepta | Exon 52 | ![]() | ![]() | |
Exon Skipping – PPMO | Sarepta | Exon 53 | ![]() | ![]() | |
Exon Skipping – PPMO | NS Pharma | Viltepso | Exon 53 | ![]() | ![]() | |
Exon Skipping – PPMO | NS Pharma | NS-089/NCP-02 | Exon 44 | ![]() | ![]() | |
Exon Skipping (AAV-U7) | Nationwide Children’s | Exon 2 duplication | ![]() | ||
Exon Skipping (AAV-U7) | Audentes | AT702 | Exon 2 duplication | ![]() | ![]() | ![]() |
Exon Skipping (AAV-U7) | Audentes | AT751 | Exon 51 | ![]() | ![]() | |
Exon Skipping (AAV-U7) | Audentes | AT753 | Exon 53 | ![]() | ![]() | |
Exon skipping (antibody-ASO) | Avidity Biosciences | Multiple | ![]() | ![]() | ![]() |
Exon skipping (antibody-ASO) | Dyne Therapeutics | Multiple | ![]() | ![]() | |
Exon Skipping | Genethon | rAAV.U7 snRNA | Exon 53 | ![]() | ||
Exon Skipping | BioMarin | Drisapersen | Exon 51 | ![]() | ![]() | |
Stereopure ASO | Wave | Suvodirsen | Exon 51 | ![]() | ![]() |
Mechanism | Company | Drug | Status | ![]() | ![]() |
---|---|---|---|---|---|
Nonsense mutation | PTC Therapeutics | Ataluren | ![]() | ![]() | ![]() |
Mechanism | Company | Drug | Status | ![]() | ![]() |
---|---|---|---|---|---|
GALGT2 gene theray | Sarepta & Nationwide Children’s Hospital | GalGT2 | ![]() | ![]() | |
Utrophin upregulation | Summit | SMT C1100 | ![]() | ![]() | ![]() |
Mechanism | Company | Drug | Status | ![]() | ![]() |
---|---|---|---|---|---|
Corticosteroid | PTC Therapeutics | Emflaza | ![]() | ![]() | |
Non-hormonal steroid | Reveragen | Vamorolone | ![]() | ![]() | |
NHE-1 Inhibitor | EspeRare | Rimeporide | ![]() | ||
CD49d espression inhibitor | Antisense Therapeutics | ATL1102 | ![]() | ||
Estrogen receptor inhibitor | University Hospital in Basel | Tamoxifen | ![]() | ||
Anti-IL1beta antibody | Children’s Research Institute | Ilaris | ![]() | ||
Nitric oxide donator Ca+ modulator | Myotherix | MRX-1043 | ![]() | ![]() | |
Steroid alternative | Mallinckrodt | Cosyntropin | ![]() | ![]() | |
Non-steroid anti-inflammatory | Catabasis | Edasalonexent | ![]() | ![]() |
Mechanism | Company | Drug | Status | ![]() | ![]() |
---|---|---|---|---|---|
Anti-CTGF antibody | FibroGen | Pamrevlumab | ![]() | ![]() | |
HDAC inhibitor | Italfarmaco | Givinostat | ![]() | ![]() | |
Renin-Angiotensin System Agonists | RASRx | RASRx1902 | ![]() | ![]() | |
Renin-Angiotensin System Agonists | BioPhytis | BIO101 | ![]() | ![]() | ![]() |
Anti-fibrotic | Akashi | HT100 | ![]() | ![]() |
Mechanism | Company | Drug | Status | ![]() | ![]() |
---|---|---|---|---|---|
Mitochondria biogenesis | Astellas | ASP0367 | ![]() | ![]() | |
Mitochondria biogenesis | Epirium | EPM-01 | ![]() | ||
Coenzyme Q10 analog | Santhera | Idebenone | ![]() | ![]() | ![]() |
Mechanism | Company | Drug | Status | ![]() | ![]() |
---|---|---|---|---|---|
Cardiac derived stem cells | Capricor | CAP-1002 | ![]() | ![]() | ![]() |
Thromboxane receptor inhibitor | Cumberland | Ifetroban | ![]() | ||
Membrane sealant | Phrixus | P188 | ![]() |
Mechanism | Company | Drug | Status | ![]() | ![]() |
---|---|---|---|---|---|
Follistatin gene therapy | Milo Biotechnology | rAAV1-Follistatin | ![]() | ||
Myostatin Antibody | Roche | RO7239361 | ![]() | ![]() | |
Myostatin Antibody | Pfizer | PF-06252616 | ![]() | ![]() |
Mechanism | Company | Drug | Status | ![]() | ![]() |
---|---|---|---|---|---|
Gene editing/Stem cells | Anagenesis Biotherapies | CRISPR/Cas9 edited stem cells | ![]() | ||
Allogenic iPSCs | Vita Therapeutics | VTA-1100 | ![]() |
*Discontinued means Discontinued at the clinical stage
Last Updated 10/27/2020