– CureDuchenne Funded   – CureDuchenne Cares Partner

 Preclinical    Clinical   Approved

ResearchCompanyDrug NameStatus
Renin-Angiotensin System AgonistsRASRxMMX1902 Preclinical
Ang(1-7)/MasTarixTXA127 Preclinical
Anti-fibroticAkashiHT100 Clinical – hold
Anti-fibroticFibroGenFG-3019; CTGF Ab Clinical
ResearchCompanyDrug Name Status
Nitric oxide donator Ca+ modulatorMyotherixMRX-1043Preclinical
Anti inflammatory, Anti fibroticNicoxHCT 1026Preclinical

Non-hormonal steroid

ReveragenVBP15Clinical
Ryanodine modulationArmgoS48168Clinical
Non Steroid Anti-inflammatoryCatabasisEdasalonexent (previously CAT-1004)Clinical
NHE-1 InhibitorEspeRareEMD 87 580 RimeporidePreclinical
SteroidMarathon

EMFLAZA

Approved
ResearchCompanyDrug Name Status
Cardiac derived stem cellsCapricorCAP-1002Clinical
VIP AgonistPhaseBioPB1046 Vasomera Preclinical
Membrane sealantPhrixusP188 Preclinical
ResearchCompanyDrug Name Status
Gene editing/Stem cellsAnagenesis BiotherapiesCRISPR/Cas9 edited stem cells Preclinical
ResearchCompanyDrug Name Status
Exon SkippingBioMarinDrisapersenClinical – Halted
Duplication Exon SkippingK Flanigan Nationwide OhiorAAV.U7 snRNA exon 2 Preclinical
Exon SkippingSarepta

Exondys51


Accelerated Approval
Exon SkippingSareptaSRP-4053Clinical
Exon SkippingSareptaSRP-4045Clinical
Exon SkippingSynthenaTricyclo-LNA Preclinical
Stereopure antisense oligonucleotide therapeuticWaveExon 44 Skipping Preclinical
Stereopure antisense oligonucleotide therapeuticWaveExon 45 Skipping Preclinical
Stereopure antisense oligonucleotide therapeuticWaveWVE-210201 (Exon 51 Skipping)Clinical
Stereopure antisense oligonucleotide therapeuticWaveExon 53 Skipping Preclinical
Exon SkippingGenethonrAAV.U7 snRNA exon 53 Preclinical

Exon Skipping

NS PharmaExon 53Clinical
ResearchCompanyDrug Name Status
Genome editing

Exonics

CRISPR/Cas9 Preclinical
Gene TherapyBamboo

BMB-D001

 Preclinical
Gene TherapySolid Bioscience

SGT-001

Clinical
Gene Therapy

Nationwide Children’s Hospital

microdystrophin/AAV

 Preclinical
ResearchCompanyDrug Name Status
Coenzyme Q10 analogSantheraCatena IdebenoneClinical

Mediated gene regulation

Mitobridge

MTB-1

 Preclinical
ResearchCompanyDrug Name Status
Myostatin AntibodyRoche/GenentechBMS-986089Clinical
Follistatin gene therapyMilo BiotechnologyrAAV1-FollistatinClinical
Myostatin AntibodyPfizer

PF-06252616

Clinical
ResearchCompanyDrug Name Status
Compensatory protein P Martin Nationwide OhioGalGT2
Preclinical
Utrophin upregulationSummitSMT C1100Clinical
Compensatory proteinTivorsan

Biglycan


Preclinical
ResearchCompanyDrug Name Status
Stop Codon readthroughEloxx PharmaAminoglycoside derivatives
Preclinical
Nonsense mutationPTC TherapeuticsAtaluren
Conditional Approval in EU