Research Overview

Duchenne Therapeutic Pipeline

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CureDuchenne Funded

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CureDuchenne Cares Partner

Dystrophin Gene Therapy


Mechanism

Company

Drug

Status
cd-funded-iconcd-partner-icon
Genome editingVertex (aquired Exonics)CRISPR/Cas9 Preclinicalcd-funded-square
Gene TherapyPfizerPF-06939926clinical Clinicalcd-funded-squarecd-partner-square
Gene TherapySareptaSRP-9001clinical Clinicalcd-partner-square
Gene TherapySarepta & GenethonrAAV2/8 micro-dystrophin Preclinicalcd-partner-square
Gene TherapySolid BioscienceSGT-001clinical Clinicalcd-partner-square

Dystrophin Exon Skipping


Mechanism

Company

Drug

Status
cd-funded-iconcd-partner-icon
Exon Skipping – PMOSareptaExondys51 | Exon 51approved Accelerated Approvalcd-funded-squarecd-partner-square
Exon Skipping – PMOSareptaVyondys53 | Exon 53approved Accelerated Approvalcd-partner-square
Exon Skipping – PMOSareptaCasimersen | Exon 45clinical Clinicalcd-partner-square
Exon Skipping – PMOSareptaExon 52 Preclinicalcd-partner-square
Exon Skipping – PPMOSareptaSRP-5051 | Exon 51clinical Clinicalcd-partner-square
Exon Skipping – PPMOSareptaExon 44 Preclinicalcd-partner-square
Exon Skipping – PPMOSareptaExon 45 Preclinicalcd-partner-square
Exon Skipping – PPMOSareptaExon 50 Preclinicalcd-partner-square
Exon Skipping – PPMOSareptaExon 52 Preclinicalcd-partner-square
Exon Skipping – PPMOSareptaExon 53 Preclinicalcd-partner-square
Exon Skipping – PPMONS PharmaViltepso | Exon 53approved Accelerated Approvalcd-partner-square
Exon Skipping – PPMONS PharmaNS-089/NCP-02 | Exon 44clinical Clinicalcd-partner-square
Exon Skipping (AAV-U7)Nationwide Children’sExon 2 duplicationclinical Clinical
Exon Skipping (AAV-U7)AudentesAT702 | Exon 2 duplication Preclinicalcd-funded-squarecd-partner-square
Exon Skipping (AAV-U7)AudentesAT751 | Exon 51 Preclinicalcd-partner-square
Exon Skipping (AAV-U7)AudentesAT753 | Exon 53 Preclinicalcd-partner-square
Exon skipping (antibody-ASO)Avidity BiosciencesMultiple Preclinicalcd-funded-squarecd-partner-square
Exon skipping (antibody-ASO)Dyne TherapeuticsMultiple Preclinicalcd-partner-square
Exon SkippingGenethonrAAV.U7 snRNA | Exon 53 Preclinical
Exon SkippingBioMarinDrisapersen | Exon 51discontinued Discontinued*cd-partner-square
Stereopure ASOWaveSuvodirsen | Exon 51discontinued Discontinued*cd-partner-square

Dystrophin Stop Codon Read-Through


Mechanism

Company

Drug

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

Compensatory Mechanisms


Mechanism

Company

Drug

Status
cd-funded-iconcd-partner-icon
GALGT2 gene theraySarepta & Nationwide Children’s HospitalGalGT2clinical Clinicalcd-partner-square
Utrophin upregulationSummitSMT C1100discontinued Discontinued*cd-funded-squarecd-partner-square

Anti-Inflammatory Therapy


Mechanism

Company

Drug

Status
cd-funded-iconcd-partner-icon
CorticosteroidPTC TherapeuticsEmflazaapproved Approvedcd-partner-square
Non-hormonal steroidReveragenVamoroloneclinical Clinicalcd-funded-square
Non-steroid anti-inflammatoryCatabasisEdasalonexentclinical Clinicalcd-partner-square
NHE-1 InhibitorEspeRareRimeporideclinical Clinical
CD49d espression 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*cd-partner-square

Anti-Fibrosis Therapy


Mechanism

Company

Drug

Status
cd-funded-iconcd-partner-icon
Anti-CTGF antibodyFibroGenPamrevlumabclinical Clinicalcd-partner-square
HDAC inhibitorItalfarmacoGivinostatclinical Clinicalcd-partner-square
Renin-Angiotensin System AgonistsRASRxRASRx1902 Preclinicalcd-funded-square
Renin-Angiotensin System AgonistsBioPhytisBIO101 Preclinicalcd-funded-squarecd-partner-square
Anti-fibroticAkashiHT100discontinued Discontinued*cd-funded-square

Mitochondrial


Mechanism

Company

Drug

Status
cd-funded-iconcd-partner-icon
Coenzyme Q10 analogSantheraIdebenoneclinical Clinicalcd-funded-squarecd-partner-square
Mitochondria biogenesisAstellasASP0367clinical Clinicalcd-partner-square
Mitochondria biogenesisEpiriumEPM-01clinical Clinical

Cardiac Function


Mechanism

Company

Drug

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

Muscle Regeneration


Mechanism

Company

Drug

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

Cell Therapy


Mechanism

Company

Drug

Status
cd-funded-iconcd-partner-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 09/15/2020

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