– CureDuchenne Funded   – CureDuchenne Cares Partner

 Preclinical    Clinical   Approved

Research Company U7 Exon Skipping Status
Renin-Angiotensin System Agonists RASRx MMX1902  Preclinical
Ang(1-7)/Mas Tarix TXA127  Preclinical
Anti-fibrotic Akashi HT100  Clinical – hold
Anti-fibrotic FibroGen FG-3019; CTGF Ab  Clinical
Research Company U7 Exon Skipping  Status
Nitric oxide donator Ca+ modulator Myotherix MRX-1043 Preclinical
Anti inflammatory, Anti fibrotic Nicox HCT 1026 Preclinical

Non-hormonal steroid

Reveragen VBP15 Clinical
Ryanodine modulation Armgo S48168 Clinical
Non Steroid Anti-inflammatory Catabasis Edasalonexent (previously CAT-1004) Clinical
NHE-1 Inhibitor EspeRare EMD 87 580 Rimeporide Preclinical
Steroid Marathon

EMFLAZA

Approved
Research Company U7 Exon Skipping  Status
Cardiac derived stem cells Capricor CAP-1002 Clinical
VIP Agonist PhaseBio PB1046 Vasomera  Preclinical
Membrane sealant Phrixus P188  Preclinical
Research Company U7 Exon Skipping  Status
Gene editing/Stem cells Anagenesis Biotherapies CRISPR/Cas9 edited stem cells  Preclinical
Research Company U7 Exon Skipping  Status
Exon Skipping BioMarin Drisapersen Clinical – Halted
Duplication Exon Skipping K Flanigan Nationwide Ohio rAAV.U7 snRNA exon 2  Preclinical
Exon Skipping Sarepta

Exondys51


Accelerated Approval
Exon Skipping Sarepta SRP-4053 Clinical
Exon Skipping Sarepta SRP-4045 Clinical
Exon Skipping Synthena Tricyclo-LNA  Preclinical
Stereopure nucleic acid therapeutics Wave Exon 44  Preclinical
Stereopure nucleic acid therapeutics Wave Exon 45  Preclinical
Stereopure nucleic acid therapeutics Wave Exon 51  Preclinical
Stereopure nucleic acid therapeutics Wave Exon 51  Preclinical
Exon Skipping Genethon rAAV.U7 snRNA exon 53  Preclinical

Exon Skipping

NS Pharma Exon 53 Clinical
Research Company U7 Exon Skipping  Status
Genome editing

Exonics

CRISPR/Cas9  Preclinical
Gene Therapy Bamboo

BMB-D001

 Preclinical
Gene Therapy Solid GT

SGT-001

 Preclinical
Gene Therapy

Nationwide Children’s Hospital

microdystrophin/AAV

 Preclinical
Research Company U7 Exon Skipping  Status
Coenzyme Q10 analog Santhera Catena Idebenone Clinical

Mediated gene regulation

Mitobridge

MTB-1

 Preclinical
Research Company U7 Exon Skipping  Status
Myostatin Antibody Bristol-Myers Squibb BMS-986089 Clinical
Follistatin gene therapy Milo Biotechnology rAAV1-Follistatin Clinical
Myostatin Antibody Pfizer

PF-06252616

Clinical
Research Company U7 Exon Skipping  Status
Compensatory protein  P Martin Nationwide Ohio GalGT2
Preclinical
Utrophin upregulation Summit SMT C1100 Clinical
Compensatory protein Tivorsan

Biglycan


Preclinical
Research Company U7 Exon Skipping  Status
Stop Codon readthrough Eloxx Pharma Aminoglycoside derivatives
Preclinical
Nonsense mutation PTC Therapeutics Ataluren
Conditional Approval in EU