Last Update: Aug 6, 2023
Purchase the complete DMD database for trial participant numbers, clinical trial sites (countries & number of sites), primary outcomes for clinical trials, clinical trial status, notes, other non-oligonucleotide & non-gene therapy drugs in the pipeline for DMD (30 rows), discontinued programs (20 rows), approved therapies, full quarter and annual reported net product sales of Translarna, Exondys 51, Emflaza, Vyondys 53, Viltepso, Amondys 45, & Elevidys, the DMD global market (incidence, prevalence, mutation frequencies) and a bonus overview of SRP-9001 (Elevidys).
| Company | Name | MOA | Administration | Indication | Stage | Trial | Trial Initiation |
|---|---|---|---|---|---|---|---|
| Sarepta Therapeutics | SRP-5051 | Peptide-PMO conjugate | IV infusion every 4 weeks | DMD exon 51 | II | NCT04004065 | Jun 2019 |
| Nippon Shinyaku | NS-089/NCNP-02 | ASO | IV infusion 1x weekly (40 or 80 mg/kg) | DMD exon 44 | II | NCT05135663 | Jun 2021 |
| Dyne Therapeutics | DYNE-251 | Antibody-conjugated PMO | IV infusion every 4 weeks | DMD exon 51 | I/II | NCT05524883 | Aug 2022 |
| Wave Life Sciences | WVE-N531 | ASO (stereopure oligonucleotide) | IV infusion every 2 weeks | DMD exon 53 | I/II | NCT04906460 | Sep 2021 |
| Daiichi Sankyo | DS-5141b | ASO | SC 1x weekly | DMD exon 45 | I/II | NCT02667483 | Oct 2015 |
| PepGen | PGN-EDO51 | Peptide-PMO conjugate | IV infusion | DMD exon 51 | II | Canada | 2H23 |
| Entrada Therapeutics | ENTR-601-44 | Peptide-oligonucleotide conjugate | IV infusion | DMD exon 44 | I | UK | Sep 2023 |
| BioMarin Pharmaceutical | BMN-351 | ASO (phosphorothioate oligonucleotide) | IV infusion | DMD exon 51 | Preclin | - | - |
| Avidity Biosciences | AOC 1044 | Antibody-oligonucleotide conjugate | IV infusion | DMD exon 44 | IND cleared | - | - |
| Sarepta Therapeutics | - | Naked PMO | IV infusion | DMD exon 52 | Preclin | - | - |
| Sarepta Therapeutics | SRP-5053 | Peptide-PMO conjugate | IV infusion | DMD exon 53 | Preclin | - | - |
| Sarepta Therapeutics | SRP-5045 | Peptide-PMO conjugate | IV infusion | DMD exon 45 | Preclin | - | - |
| Sarepta Therapeutics | SRP-5052 | Peptide-PMO conjugate | IV infusion | DMD exon 52 | Preclin | - | - |
| Sarepta Therapeutics | SRP-5044 | Peptide-PMO conjugate | IV infusion | DMD exon 44 | Preclin | - | - |
| Sarepta Therapeutics | SRP-5050 | Peptide-PMO conjugate | IV infusion | DMD exon 50 | Preclin | - | - |
| PepGen | PGN-EDO53 | Peptide-PMO conjugate | IV infusion | DMD exon 53 | Disc | - | - |
| PepGen | PGN-EDO45 | Peptide-PMO conjugate | IV infusion | DMD exon 45 | Disc | - | - |
| PepGen | PGN-EDO44 | Peptide-PMO conjugate | IV infusion | DMD exon 44 | Disc | - | - |
| Avidity Biosciences | - | Antibody-oligonucleotide conjugate | IV infusion | DMD exon 51 | Disc | - | - |
| Avidity Biosciences | - | Antibody-oligonucleotide conjugate | IV infusion | DMD exon 45 | Disc | - | - |
| Sutura Therapeutics | SUT-001 | Peptide-PMO conjugate (NG2-PMO) | IV infusion | - | Disc | - | - |
| Orna Therapeutics | - | Non-viral RNA encoding dystrophin | - | DMD | Disc | - | - |
| DTx Pharma | - | Lipid-siRNA conjugate | IV infusion | Muscle disorder | Disc | - | - |
| UGISense AG | - | Peptide nucleic acids; exon skipping | - | DMD | Disc | - | - |
| Sarepta & Institute of Myology | - | Peptide-PMO conjugate | - | - | R&D | - | - |
| Alpha Anomeric | - | 7′,5′-alpha-bicyclo-DNA; exon skipping | - | - | R&D | - | - |
| Consortium.AI | - | Small molecule exon-skipper | - | - | R&D | - | - |
| OliPass | - | Peptide nucleic acids; exon skipping | - | - | R&D | - | - |
| Company | Name | MOA | Administration | Indication | Stage | Trial | Trial Initiation |
|---|---|---|---|---|---|---|---|
| Sarepta & Roche | SRP-9001 | AAVrh74 delivering micro-dystrophin | Single IV infusion | DMD | III | NCT05881408 | June 2023 |
| Pfizer | PF-06939926 | AAV9 delivering mini-dystrophin | Single IV infusion | DMD | III | NCT04281485 | Nov 2020 |
| Genethon/Sarepta Therapeutics | GNT 0004 | AAV8 delivering micro-dystrophin | Single IV infusion | DMD | I/II/III | 2020-002093-27 | Apr 2021 |
| Solid Biosciences | SGT-001 | AAV9 delivering micro-dystrophin | Single IV infusion | DMD | II | NCT03368742 | Dec 2017 |
| REGENXBIO | RGX-202 | NAV AAV8 delivering micro-dystrophin | Single IV infusion | DMD | I/II | NCT05693142 | Jan 2023 |
| Sarepta Therapeutics | GALGT2 gene therapy | AAVrh74 carrying GALGT2 gene | Single intravascular limb infusion | DMD | I | NCT03333590 | Nov 2017 |
| Cure Rare Disease | CRD-TMH-001 | AAV9 delivering dCas9-VP64 | Single IV infusion | DMD exon 1 | I | NCT05514249 | Aug 2022 |
| Insmed | INS1201 | AAV | Intrathecal delivery | DMD | Preclin | - | - |
| Solid Biosciences | SGT-003 | AAV-based vector delivering microdystrophin | Single IV infusion | DMD | IND-enabling | - | - |
| Vertex Pharmaceuticals | - | AAV9 delivering CRISPR/Cas9 | Single IV infusion | DMD | Preclin | - | - |
| Ultragenyx | UX810 | AAV delivering microdystrophin | Single IV infusion | DMD | Preclin | - | - |
| MyoGene Bio | MyoDys 45-55 | CRISPR/Cas9 gene editing | - | DMD exons 45-55 | Preclin | - | - |
| GenAssist Therapeutics | GEN6050 | sgRNA guided AID-mediated mutagenesis base editing | - | DMD exon 50 | Preclin | - | - |
| GenAssist Therapeutics | GEN6045 | sgRNA guided AID-mediated mutagenesis base editing | - | DMD exon 45 | Preclin | - | - |
| GenAssist Therapeutics | GEN6051 | sgRNA guided AID-mediated mutagenesis base editing | - | DMD exon 51 | Disc | - | - |
| Sarepta Therapeutics & Duke | - | CRISPR/Cas9 | Single IV infusion | DMD | R&D | - | - |
| Sarepta & Harvard | - | Gene editing | - | DMD | R&D | - | - |
| Code Biotherapeutics | - | Non-viral '3DNA' scaffold | - | DMD | R&D | - | - |
| Sarepta & Institute of Myology | - | Gene therapy | - | DMD | R&D | - | - |
| Eli Lilly & Precision BioSciences | PBGENE-DMD | Gene editing, AAV | - | DMD | R&D | - | - |
| AAVogen | AVGN7 | AAV6 delivering SMAD7 | - | DMD | R&D | - | - |
| CANBridge Pharmaceuticals | - | - | - | DMD | R&D | - | - |
| Myosana Therapeutics | - | Non-viral gene delivery | - | DMD | R&D | - | - |
| LambdaGen Therapeutics | - | Ex-vivo gene therapy | - | DMD | R&D | - | - |