– CureDuchenne Funded – CureDuchenne Cares Partner

preclinical Preclinical clinical Clinical approved Approved discontinued Discontinued

Anti-Fibrosis Therapy

Research Company Drug Name Status
Renin-Angiotensin System Agonists RASRx RASRx1902 Preclinical
Renin-Angiotensin System Agonists BioPhytis BIO101 Preclinical
Anti-fibrotic Akashi HT100 discontinued Discontinued
Anti-CTGF antibody FibroGen Pamrevlumab Clinical
HDAC inhibitor Italfarmaco Givinostat Clinical

Anti-Inflammatory Therapy

Research Company Drug Name Status
Nitric oxide donator Ca+ modulator Myotherix MRX-1043 Preclinical

Non-hormonal steroid

 Reveragen Vamorolone Clinical
Non Steroid Anti-inflammatory Catabasis Edasalonexent Clinical
NHE-1 Inhibitor EspeRare Rimeporide Clinical
CD49d espression inhibitor Antisense Therapeutics ATL1102 approved Clinical
Estrogen receptor inhibitor University Hospital in Basel Tamoxifen approved Clinical
Sterioid alternative Mallinckrodt Cosyntropin discontinued Discontinued
Corticosteroid PTC Therapeutics

EMFLAZA

 approved Approved

Cardiac Function

Research Company Drug Name Status
Cardiac derived stem cells Capricor CAP-1002 Clinical
Thromboxane receptor inhibitor Cumberland Ifetroban Clinical
Membrane sealant Phrixus P188 Clinical

Cell Therapy

Research Company Drug Name Status
Gene editing/Stem cells Anagenesis Biotherapies CRISPR/Cas9 edited stem cells Preclinical

Exon Skipping

Research Company Drug Name Status
cureduchenne cares supporter Exon Skipping BioMarin Drisapersen
Exon 51		 discontinued Discontinued
funded by cureduchennecureduchenne cares supporter Exon Skipping – PMO Sarepta Exondys51
Exon 51		 approved Accelerated Approval
cureduchenne cares supporter Exon Skipping – PMO Sarepta Vyondys53
Exon 53		 approved Accelerated Approval
cureduchenne cares supporter Exon Skipping – PMO Sarepta Casimersen
Exon 45		 status Clinical
cureduchenne cares supporter Exon Skipping – PMO Sarepta Exon 52 status Preclinical
cureduchenne cares supporter Exon Skipping – PPMO Sarepta SRP-5051
Exon 51		 status Clinical
cureduchenne cares supporter Exon Skipping – PPMO Sarepta Exon 44 status Preclinical
cureduchenne cares supporter Exon Skipping – PPMO Sarepta Exon 45 status Preclinical
cureduchenne cares supporter Exon Skipping – PPMO Sarepta Exon 50 status Preclinical
cureduchenne cares supporter Exon Skipping – PPMO Sarepta Exon 52 status Preclinical
cureduchenne cares supporter Exon Skipping – PPMO Sarepta Exon 53 status Preclinical
cureduchenne cares supporter Stereopure antisense oligonucleotide Wave Suvodirsen
Exon 51		 discontinued Discontinued
cureduchenne cares supporter Stereopure antisense oligonucleotide Wave WVE-N531
Exon 53		 discontinued Discontinued
cureduchenne cares supporter Exon Skipping – PPMO NS Pharma Vitolarsen
Exon 53		 status Clinical
cureduchenne cares supporter Exon Skipping – PPMO NS Pharma NS-089/NCP-02
Exon 44		 status Clinical
funded by cureduchennecureduchenne cares supporter Exon Skipping (AAV-ASO) Nationwide Children’s
& Audentes		 AT702
Exon 2 duplication, Exon 1-5		 status Preclinical
cureduchenne cares supporter Exon Skipping (AAV-ASO) Audentes AT751
Exon 51		 status Preclinical
cureduchenne cares supporter Exon Skipping (AAV-ASO) Audentes AT753
Exon 53		 status Preclinical
Exon Skipping Genethon rAAV.U7 snRNA
Exon 53		 status Preclinical

Gene Correction

Research Company Drug Name Status
Genome editing Vertex (aquired Exonics) CRISPR/Cas9 Preclinical
Gene Therapy Pfizer PF-06939926 Clinical
Gene Therapy Sarepta SRP-9001 Clinical
Gene Therapy Sarepta & Genethon rAAV2/8
micro-dystrophin		 Preclinical
Gene Therapy Solid Bioscience SGT-001 Clinical

Mitochondrial

Research Company Drug Name Status
Coenzyme Q10 analog Santhera Idebenone Clinical
Mitochondria biogenesis Astellas ASP0367 Clinical
Mitochondria biogenesis Cardero Epicatechin Clinical

Muscle Regeneration

Research Company Drug Name Status
Follistatin gene therapy Milo Biotechnology rAAV1-Follistatin discontinued Discontinued
Myostatin Antibody Roche RO7239361 discontinued Discontinued
Myostatin Antibody Pfizer PF-06252616 discontinued Discontinued

Protein Replacement

Research Company Drug Name Status
GALGT2 gene theray Sarepta
& Nationwide Children’s Hospital		 GalGT2 Clinical
Utrophin upregulation Summit SMT C1100 discontinued Discontinued

Stop Codon Read-Through

Research Company Drug Name Status
Nonsense mutation PTC Therapeutics Ataluren approved Conditional Approval in EU

Chart updated on 1/16/20