Research Overview

Duchenne Therapeutic Pipeline

cd-funded-icon

CureDuchenne Funded

Dystrophin Gene Therapy


Mechanism

Company

Drug

Status
cd-funded-icon
Genome editingVertex (aquired Exonics)CRISPR/Cas9 Preclinicalcd-funded-square
Gene TherapyPfizerPF-06939926clinical Clinicalcd-funded-square
Gene TherapySareptaSRP-9001clinical Clinical
Gene TherapySarepta & GenethonrAAV2/8 micro-dystrophinclinical Clinical
Gene TherapySolid BioscienceSGT-001clinical Clinical
Gene TherapyRegenxBioRGX-202 Preclinical

Dystrophin Exon Skipping


Mechanism

Company

Drug

Status
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Exon Skipping – PMOSareptaExondys51 | Exon 51approved Accelerated Approvalcd-funded-square
Exon Skipping – PMOSareptaVyondys53 | Exon 53approved Accelerated Approval
Exon Skipping – PMOSareptaAmondys45 | Exon 45approved Accelerated Approval
Exon Skipping – PMOSareptaExon 52 Preclinical
Exon Skipping – PPMOSareptaSRP-5051 | Exon 51clinical Clinical
Exon Skipping – PPMOSareptaMultiple Preclinical
Exon Skipping – PMONS PharmaViltepso | Exon 53approved Accelerated Approval
Exon Skipping – PMONS PharmaNS-089/NCP-02 | Exon 44clinical Clinical
Exon Skipping (AAV-U7)Nationwide Children’sExon 2 duplicationclinical Clinicalcd-funded-square
Exon Skipping (AAV-U7)Astellas Gene TherapiesAT702 | Exon 2 duplication Preclinicalcd-funded-square
Exon Skipping (AAV-U7)Astellas Gene TherapiesAT751 | Exon 51 Preclinical
Exon Skipping (AAV-U7)Astellas Gene TherapiesAT753 | Exon 53 Preclinical
Stereopure PN chemistry ASOWave Life SciencesWVE-N531 | Exon 53clinical Clinical
Exon skipping (antibody-ASO)Avidity BiosciencesMultiple Preclinicalcd-funded-square
Exon skipping (antibody-ASO)Dyne TherapeuticsMultiple Preclinicalcd-funded-square
Exon Skipping – PPMOPepGenPGN-ED051 | Exon 51 Preclinical
Exon SkippingBioMarinDrisapersen | Exon 51discontinued Discontinued*
Exon Skipping (Stereopure-ASO)Wave Life SciencesSuvodirsen | Exon 51discontinued Discontinued*

Dystrophin Stop Codon Read-Through


Mechanism

Company

Drug

Status
cd-funded-icon
Nonsense mutationPTC TherapeuticsAtalurenapproved Conditional Approval in EUcd-funded-square

Compensatory Mechanisms


Mechanism

Company

Drug

Status
cd-funded-icon
GALGT2 gene therapySarepta & Nationwide Children’s HospitalGalGT2clinical Clinical
Utrophin upregulationSummitSMT C1100discontinued Discontinued*cd-funded-square

Anti-Inflammatory Therapy


Mechanism

Company

Drug

Status
cd-funded-icon
CorticosteroidPTC TherapeuticsEmflazaapproved Approved
Non-hormonal steroidReveragenVamoroloneclinical Clinicalcd-funded-square
NHE-1 InhibitorEspeRareRimeporideclinical Clinical
CD49d expression inhibitorAntisense TherapeuticsATL1102clinical Clinical
Estrogen receptor inhibitorUniversity Hospital in BaselTamoxifenclinical Clinical
Anti-IL1beta antibodyChildren’s Research InstituteIlarisclinical Clinical
Nitric oxide donator Ca+ modulatorMyotherixMRX-1043 Preclinicalcd-funded-square
Steroid alternativeMallinckrodtCosyntropindiscontinued Discontinued*
Non-steroid anti-inflammatoryCatabasisEdasalonexentdiscontinued Discontinued*

Anti-Fibrosis Therapy


Mechanism

Company

Drug

Status
cd-funded-icon
Anti-CTGF antibodyFibroGenPamrevlumabclinical Clinical
HDAC inhibitorItalfarmacoGivinostatclinical Clinical
Renin-Angiotensin System AgonistsBioPhytisBIO101 Preclinicalcd-funded-square
Anti-fibroticAkashiHT100discontinued Discontinued*cd-funded-square

Mitochondrial


Mechanism

Company

Drug

Status
cd-funded-icon
Mitochondria biogenesisAstellasASP0367clinical Clinical
Mitochondria biogenesisEpirium(+)-Epicatechinclinical Clinical
Coenzyme Q10 analogSantheraIdebenonediscontinued Discontinued*cd-funded-square

Cardiac Function


Mechanism

Company

Drug

Status
cd-funded-icon
Cardiac derived stem cellsCapricorCAP-1002clinical Clinicalcd-funded-square
Thromboxane receptor inhibitorCumberlandIfetrobanclinical Clinical
Membrane sealantPhrixusP188clinical Clinical

Muscle Regeneration


Mechanism

Company

Drug

Status
cd-funded-icon
Follistatin gene therapyMilo BiotechnologyrAAV1-Follistatindiscontinued Discontinued*
Myostatin AntibodyRocheRO7239361discontinued Discontinued*
Myostatin AntibodyPfizerPF-06252616discontinued Discontinued*

Muscle Stabilization


Mechanism

Company

Drug

Status
cd-funded-icon
Muscle StabilizerEdgewise TherapeuticsEDG-5506clinical Clinicalcd-funded-square

Cell Therapy


Mechanism

Company

Drug

Status
cd-funded-icon
Gene editing/Stem cellsAnagenesis BiotherapiesCRISPR/Cas9 edited stem cells Preclinical
Allogenic iPSCsVita TherapeuticsVTA-1100 Preclinical

*Discontinued means Discontinued at the clinical stage

Last Updated 6/21/2021

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