Duchenne Muscular Dystrophy (DMD) | Oligonucleotide, RNA & Gene Therapy Global Pipeline

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).

Oligonucleotide & RNA Therapies

Company Name MOA Administration Indication Stage Trial Trial Initiation
Sarepta TherapeuticsSRP-5051Peptide-PMO conjugateIV infusion every 4 weeksDMD exon 51IINCT04004065Jun 2019
Nippon ShinyakuNS-089/NCNP-02ASOIV infusion 1x weekly (40 or 80 mg/kg)DMD exon 44IINCT05135663Jun 2021
Dyne TherapeuticsDYNE-251Antibody-conjugated PMOIV infusion every 4 weeksDMD exon 51I/IINCT05524883Aug 2022
Wave Life SciencesWVE-N531ASO (stereopure oligonucleotide)IV infusion every 2 weeksDMD exon 53I/IINCT04906460Sep 2021
Daiichi SankyoDS-5141bASOSC 1x weeklyDMD exon 45I/IINCT02667483Oct 2015
PepGenPGN-EDO51Peptide-PMO conjugateIV infusionDMD exon 51IICanada2H23
Entrada TherapeuticsENTR-601-44Peptide-oligonucleotide conjugateIV infusionDMD exon 44IUKSep 2023
BioMarin PharmaceuticalBMN-351ASO (phosphorothioate oligonucleotide)IV infusionDMD exon 51Preclin--
Avidity BiosciencesAOC 1044Antibody-oligonucleotide conjugateIV infusionDMD exon 44IND cleared--
Sarepta Therapeutics-Naked PMOIV infusionDMD exon 52Preclin--
Sarepta TherapeuticsSRP-5053Peptide-PMO conjugateIV infusionDMD exon 53Preclin--
Sarepta TherapeuticsSRP-5045Peptide-PMO conjugateIV infusionDMD exon 45Preclin--
Sarepta TherapeuticsSRP-5052Peptide-PMO conjugateIV infusionDMD exon 52Preclin--
Sarepta TherapeuticsSRP-5044Peptide-PMO conjugateIV infusionDMD exon 44Preclin--
Sarepta TherapeuticsSRP-5050Peptide-PMO conjugateIV infusionDMD exon 50Preclin--
PepGenPGN-EDO53Peptide-PMO conjugateIV infusionDMD exon 53Disc--
PepGenPGN-EDO45Peptide-PMO conjugateIV infusionDMD exon 45Disc--
PepGenPGN-EDO44Peptide-PMO conjugateIV infusionDMD exon 44Disc--
Avidity Biosciences-Antibody-oligonucleotide conjugateIV infusionDMD exon 51Disc--
Avidity Biosciences-Antibody-oligonucleotide conjugateIV infusionDMD exon 45Disc--
Sutura TherapeuticsSUT-001Peptide-PMO conjugate (NG2-PMO)IV infusion-Disc--
Orna Therapeutics-Non-viral RNA encoding dystrophin-DMDDisc--
DTx Pharma-Lipid-siRNA conjugateIV infusionMuscle disorderDisc--
UGISense AG-Peptide nucleic acids; exon skipping-DMDDisc--
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--

Gene Therapies

Company Name MOA Administration Indication Stage Trial Trial Initiation
Sarepta & RocheSRP-9001AAVrh74 delivering micro-dystrophinSingle IV infusionDMD IIINCT05881408June 2023
PfizerPF-06939926AAV9 delivering mini-dystrophinSingle IV infusionDMD IIINCT04281485Nov 2020
Genethon/Sarepta TherapeuticsGNT 0004AAV8 delivering micro-dystrophinSingle IV infusionDMD I/II/III2020-002093-27Apr 2021
Solid BiosciencesSGT-001AAV9 delivering micro-dystrophinSingle IV infusionDMDIINCT03368742Dec 2017
REGENXBIORGX-202NAV AAV8 delivering micro-dystrophinSingle IV infusionDMDI/II NCT05693142Jan 2023
Sarepta TherapeuticsGALGT2 gene therapyAAVrh74 carrying GALGT2 geneSingle intravascular limb infusionDMD INCT03333590Nov 2017
Cure Rare DiseaseCRD-TMH-001AAV9 delivering dCas9-VP64Single IV infusionDMD exon 1INCT05514249Aug 2022
InsmedINS1201AAVIntrathecal deliveryDMDPreclin--
Solid BiosciencesSGT-003AAV-based vector delivering microdystrophinSingle IV infusionDMDIND-enabling--
Vertex Pharmaceuticals-AAV9 delivering CRISPR/Cas9Single IV infusionDMDPreclin--
Ultragenyx UX810AAV delivering microdystrophinSingle IV infusionDMDPreclin--
MyoGene BioMyoDys 45-55CRISPR/Cas9 gene editing-DMD exons 45-55Preclin--
GenAssist TherapeuticsGEN6050sgRNA guided AID-mediated mutagenesis base editing-DMD exon 50Preclin--
GenAssist TherapeuticsGEN6045sgRNA guided AID-mediated mutagenesis base editing-DMD exon 45Preclin--
GenAssist TherapeuticsGEN6051sgRNA guided AID-mediated mutagenesis base editing-DMD exon 51Disc--
Sarepta Therapeutics & Duke-CRISPR/Cas9Single IV infusionDMDR&D--
Sarepta & Harvard-Gene editing-DMDR&D--
Code Biotherapeutics-Non-viral '3DNA' scaffold-DMDR&D--
Sarepta & Institute of Myology-Gene therapy-DMDR&D--
Eli Lilly & Precision BioSciencesPBGENE-DMDGene editing, AAV-DMDR&D--
AAVogenAVGN7AAV6 delivering SMAD7-DMDR&D--
CANBridge Pharmaceuticals---DMDR&D--
Myosana Therapeutics-Non-viral gene delivery-DMDR&D--
LambdaGen Therapeutics-Ex-vivo gene therapy-DMDR&D--