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Mechanism | Company | Drug | Status | |
---|---|---|---|---|
Genome editing | Vertex (acquired Exonics) | CRISPR/Cas9 | Preclinical | |
Gene Therapy | Pfizer | PF-06939926 | Clinical | |
Gene Therapy | Sarepta | SRP-9001 | Clinical | |
Gene Therapy | Sarepta & Genethon | rAAV2/8 micro-dystrophin | Clinical | |
Gene Therapy | Solid Bioscience | SGT-001 | Clinical | |
Gene Therapy | RegenxBio | RGX-202 | Preclinical |
Mechanism | Company | Drug | Status | |
---|---|---|---|---|
Exon Skipping – PMO | Sarepta | Exondys51 | Exon 51 | Accelerated Approval | |
Exon Skipping – PMO | Sarepta | Vyondys53 | Exon 53 | Accelerated Approval | |
Exon Skipping – PMO | Sarepta | Amondys45 | Exon 45 | Accelerated Approval | |
Exon Skipping – PMO | Sarepta | Exon 52 | Preclinical | |
Exon Skipping – PPMO | Sarepta | SRP-5051 | Exon 51 | Clinical | |
Exon Skipping – PPMO | Sarepta | Multiple | Preclinical | |
Exon Skipping – PMO | NS Pharma | Viltepso | Exon 53 | Accelerated Approval | |
Exon Skipping – PMO | NS Pharma | NS-089/NCP-02 | Exon 44 | Clinical | |
Exon Skipping (AAV-U7) | Nationwide Children’s | Exon 2 duplication | Clinical | |
Exon Skipping (AAV-U7) | Astellas Gene Therapies | AT702 | Exon 2 duplication | Preclinical | |
Exon Skipping (AAV-U7) | Astellas Gene Therapies | AT751 | Exon 51 | Preclinical | |
Exon Skipping (AAV-U7) | Astellas Gene Therapies | AT753 | Exon 53 | Preclinical | |
Stereopure PN chemistry ASO | Wave Life Sciences | WVE-N531 | Exon 53 | Clinical | |
Exon skipping (antibody-ASO) | Avidity Biosciences | Multiple | Preclinical | |
Exon skipping (antibody-ASO) | Dyne Therapeutics | Multiple | Preclinical | |
Exon Skipping – PPMO | PepGen | PGN-ED051 | Exon 51 | Preclinical | |
Exon Skipping | BioMarin | Drisapersen | Exon 51 | Discontinued* | |
Exon Skipping (Stereopure-ASO) | Wave Life Sciences | Suvodirsen | Exon 51 | Discontinued* |
Mechanism | Company | Drug | Status | |
---|---|---|---|---|
Nonsense mutation | PTC Therapeutics | Ataluren | Conditional Approval in EU |
Mechanism | Company | Drug | Status | |
---|---|---|---|---|
GALGT2 gene therapy | Sarepta & Nationwide Children’s Hospital | GalGT2 | Clinical | |
Utrophin upregulation | Summit | SMT C1100 | Discontinued* |
Mechanism | Company | Drug | Status | |
---|---|---|---|---|
Corticosteroid | PTC Therapeutics | Emflaza | Approved | |
Non-hormonal steroid | Reveragen | Vamorolone | Clinical | |
NHE-1 Inhibitor | EspeRare | Rimeporide | Clinical | |
CD49d expression inhibitor | Antisense Therapeutics | ATL1102 | Clinical | |
Estrogen receptor inhibitor | University Hospital in Basel | Tamoxifen | Clinical | |
Anti-IL1beta antibody | Children’s Research Institute | Ilaris | Clinical | |
Nitric oxide donator Ca+ modulator | Myotherix | MRX-1043 | Preclinical | |
Steroid alternative | Mallinckrodt | Cosyntropin | Discontinued* | |
Non-steroid anti-inflammatory | Catabasis | Edasalonexent | Discontinued* |
Mechanism | Company | Drug | Status | |
---|---|---|---|---|
Anti-CTGF antibody | FibroGen | Pamrevlumab | Clinical | |
HDAC inhibitor | Italfarmaco | Givinostat | Clinical | |
Renin-Angiotensin System Agonists | BioPhytis | BIO101 | Preclinical | |
Anti-fibrotic | Akashi | HT100 | Discontinued* |
Mechanism | Company | Drug | Status | |
---|---|---|---|---|
Mitochondria biogenesis | Astellas | ASP0367 | Clinical | |
Mitochondria biogenesis | Epirium | (+)-Epicatechin | Clinical | |
Coenzyme Q10 analog | Santhera | Idebenone | Discontinued* |
Mechanism | Company | Drug | Status | |
---|---|---|---|---|
Cardiac derived stem cells | Capricor | CAP-1002 | Clinical | |
Thromboxane receptor inhibitor | Cumberland | Ifetroban | Clinical | |
Membrane sealant | Phrixus | P188 | Clinical |
Mechanism | Company | Drug | Status | |
---|---|---|---|---|
Follistatin gene therapy | Milo Biotechnology | rAAV1-Follistatin | Discontinued* | |
Myostatin Antibody | Roche | RO7239361 | Discontinued* | |
Myostatin Antibody | Pfizer | PF-06252616 | Discontinued* |
Mechanism | Company | Drug | Status | |
---|---|---|---|---|
Muscle Stabilizer | Edgewise Therapeutics | EDG-5506 | Clinical |
Mechanism | Company | Drug | Status | |
---|---|---|---|---|
Gene editing/Stem cells | Anagenesis Biotherapies | CRISPR/Cas9 edited stem cells | Preclinical | |
Allogenic iPSCs | Vita Therapeutics | VTA-1100 | Preclinical |
*Discontinued means Discontinued at the clinical stage
Last Updated 6/21/2021