Research Overview

Duchenne Therapeutic Pipeline

cd-funded-icon

CureDuchenne Funded

Dystrophin Gene Therapy


Mechanism

Company

Drug

Status
cd-funded-icon
Micro-dystrophin Gene ReplacementSarepta TherapeuticsElevidys/SRP-9001approved Accelerated Approval
Micro-dystrophin Gene ReplacementPfizerPF-06939926clinical Clinicalcd-funded-square
Micro-dystrophin Gene ReplacementGenethon (Sarepta)GNT-0004clinical Clinical
Micro-dystrophin Gene ReplacementRegenxBioRGX-202clinical Clinical
Micro-dystrophin Gene ReplacementSolid BiosciencesSGT-001clinical Clinical
Micro-dystrophin Gene ReplacementSolid BiosciencesSGT-003clinical Clinical
Micro-dystrophin Gene ReplacementUltragenyxUX810 Preclinical
Micro-dystrophin Gene ReplacementInsmedINS1201 Preclinicalcd-funded-square

Dystrophin Gene Editing


Mechanism

Company

Drug

Status
cd-funded-icon
Genome EditingVertex PharmaceuticalsCRISPR/Cas9 Preclinicalcd-funded-square
Genome Editing (Exon 45-55)MyoGene BioCRISPR/Cas9 Preclinicalcd-funded-square
Genome Editing (Exon 45-55)Precision BioSciencesARCUS-Mediated Excision Preclinical

Dystrophin Exon Skipping


Mechanism

Company

Drug

Status
cd-funded-icon
PMOSarepta TherapeuticsExondys 51 (Exon 51)approved Accelerated Approvalcd-funded-square
PMOSarepta TherapeuticsVyondys 53 (Exon 53)approved Accelerated Approval
PMOSarepta TherapeuticsAmondys 45 (Exon 45)approved Accelerated Approval
PPMOSarepta TherapeuticsSRP-5051 (Exon 51)clinical Clinical
PPMOSarepta TherapeuticsExon 44, 45, 50, 52, 53 Preclinical
PMONS PharmaViltepso (Exon 53)approved Accelerated Approval
PMONS PharmaNS-089/NCNP-02 (Exon 44)clinical Clinical
PMONS PharmaNS‐050/NCNP‐03 (Exon 50)clinical Clinical
PMONS PharmaExon 51, 55, 45 Preclinical
AAV-U7 snRNANationwide Children’sExon 2clinical Clinicalcd-funded-square
Stereopure-PN-ASOWave Life SciencesWVE-N531 (Exon 53)clinical Clinical
Stereopure-PN-ASOWave Life SciencesExon 44, 45, 51, 52 Preclinical
Antibody-PMOAvidity BiosciencesAOC 1044 (Exon 44)clinical Clinicalcd-funded-square
Antibody-PMOAvidity BiosciencesExon 45, 51 Preclinicalcd-funded-square
Antibody-PMODyne TherapeuticsDYNE-251 (Exon 51)clinical Clinicalcd-funded-square
Antibody-PMODyne TherapeuticsExon 44, 45, 53 Preclinicalcd-funded-square
PPMO (EDO-CPP)PepGenPGN-EDO51 (Exon 51)clinical Clinicalcd-funded-square
PPMO (EDO-CPP)PepGenExon 44, 45, 53 Preclinicalcd-funded-square
PPMO (Endosomal Escape)Entrada TherapeuticsExon 44clinical Clinicalcd-funded-square
PPMO (Endosomal Escape)Entrada TherapeuticsExon 45, 50, 51 Preclinical
PS-ASOBioMarinBMN 351 (Exon 51) Preclinicalcd-funded-square

Dystrophin Stop Codon Read-Through


Mechanism

Company

Drug

Status
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Stop Codon Read-throughPTC TherapeuticsAtalurenapproved Conditional Approval in EUcd-funded-square
Stop Codon Read-throughhC BiosciencetRNA-based approach Preclinicalcd-funded-square
Stop Codon Read-throughTevard BiosciencestRNA-based approach Preclinical

Anti-Inflammatory


Mechanism

Company

Drug

Status
cd-funded-icon
CorticosteroidPTC TherapeuticsEmflazaapproved Approved
CorticosteroidCatalyst/Santhera/ReveraGenVamorolone/AGAMREEapproved Approvedcd-funded-square

OTHERS


Mechanism

Company

Drug

Status
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Cardiac-derived ExosomesCapricor TherapeuticsCAP-1002clinical Clinicalcd-funded-square
Muscle StabilizerEdgewise TherapeuticsEDG-5506clinical Clinicalcd-funded-square
HDAC InhibitorItalfarmacoGivinostatclinical Clinical
CD49d Expression InhibitorAntisense TherapeuticsATL1102clinical Clinical
Anti-IL1beta AntibodyChildren’s Research InstituteIlarisclinical Clinical
PGD synthase InhibitorTaiho PharmaceuticalTAS-205clinical Clinical
IGF-1IpsenIncrelex clinical Clinical
Thromboxane Receptor InhibitorCumberland PharmaceuticalsIfetrobanclinical Clinical

Last Updated 01/23/2024

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