CureDuchenne | Funding research to find a cure for Duchenne

Duchenne Therapeutic Pipeline

CureDuchenne Funded

Dystrophin Gene Therapy

Mechanism	Company	Drug	Status
Genome editing	Vertex (acquired Exonics)	CRISPR/Cas9	Preclinical
Gene Therapy	Pfizer	PF-06939926	Clinical
Gene Therapy	Sarepta	SRP-9001	Clinical
Gene Therapy	Sarepta & Genethon	rAAV2/8 micro-dystrophin	Clinical
Gene Therapy	Solid Bioscience	SGT-001	Clinical
Gene Therapy	RegenxBio	RGX-202	Preclinical

Dystrophin Exon Skipping

Mechanism	Company	Drug	Status
Exon Skipping – PMO	Sarepta	Exondys51 \| Exon 51	Accelerated Approval
Exon Skipping – PMO	Sarepta	Vyondys53 \| Exon 53	Accelerated Approval
Exon Skipping – PMO	Sarepta	Amondys45 \| Exon 45	Accelerated Approval
Exon Skipping – PMO	Sarepta	Exon 52	Preclinical
Exon Skipping – PPMO	Sarepta	SRP-5051 \| Exon 51	Clinical
Exon Skipping – PPMO	Sarepta	Multiple	Preclinical
Exon Skipping – PMO	NS Pharma	Viltepso \| Exon 53	Accelerated Approval
Exon Skipping – PMO	NS Pharma	NS-089/NCP-02 \| Exon 44	Clinical
Exon Skipping (AAV-U7)	Nationwide Children’s	Exon 2 duplication	Clinical
Exon Skipping (AAV-U7)	Astellas Gene Therapies	AT702 \| Exon 2 duplication	Preclinical
Exon Skipping (AAV-U7)	Astellas Gene Therapies	AT751 \| Exon 51	Preclinical
Exon Skipping (AAV-U7)	Astellas Gene Therapies	AT753 \| Exon 53	Preclinical
Stereopure PN chemistry ASO	Wave Life Sciences	WVE-N531 \| Exon 53	Clinical
Exon skipping (antibody-ASO)	Avidity Biosciences	Multiple	Preclinical
Exon skipping (antibody-ASO)	Dyne Therapeutics	Multiple	Preclinical
Exon Skipping – PPMO	PepGen	PGN-ED051 \| Exon 51	Preclinical
Exon Skipping	BioMarin	Drisapersen \| Exon 51	Discontinued*
Exon Skipping (Stereopure-ASO)	Wave Life Sciences	Suvodirsen \| Exon 51	Discontinued*

Dystrophin Stop Codon Read-Through

Mechanism	Company	Drug	Status
Nonsense mutation	PTC Therapeutics	Ataluren	Conditional Approval in EU

Compensatory Mechanisms

Mechanism	Company	Drug	Status
GALGT2 gene therapy	Sarepta & Nationwide Children’s Hospital	GalGT2	Clinical
Utrophin upregulation	Summit	SMT C1100	Discontinued*

Anti-Inflammatory Therapy

Mechanism	Company	Drug	Status
Corticosteroid	PTC Therapeutics	Emflaza	Approved
Non-hormonal steroid	Reveragen	Vamorolone	Clinical
NHE-1 Inhibitor	EspeRare	Rimeporide	Clinical
CD49d expression inhibitor	Antisense Therapeutics	ATL1102	Clinical
Estrogen receptor inhibitor	University Hospital in Basel	Tamoxifen	Clinical
Anti-IL1beta antibody	Children’s Research Institute	Ilaris	Clinical
Nitric oxide donator Ca+ modulator	Myotherix	MRX-1043	Preclinical
Steroid alternative	Mallinckrodt	Cosyntropin	Discontinued*
Non-steroid anti-inflammatory	Catabasis	Edasalonexent	Discontinued*

Anti-Fibrosis Therapy

Mechanism	Company	Drug	Status
Anti-CTGF antibody	FibroGen	Pamrevlumab	Clinical
HDAC inhibitor	Italfarmaco	Givinostat	Clinical
Renin-Angiotensin System Agonists	BioPhytis	BIO101	Preclinical
Anti-fibrotic	Akashi	HT100	Discontinued*

Mitochondrial

Mechanism	Company	Drug	Status
Mitochondria biogenesis	Astellas	ASP0367	Clinical
Mitochondria biogenesis	Epirium	(+)-Epicatechin	Clinical
Coenzyme Q10 analog	Santhera	Idebenone	Discontinued*

Cardiac Function

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

Muscle Regeneration

Mechanism	Company	Drug	Status
Follistatin gene therapy	Milo Biotechnology	rAAV1-Follistatin	Discontinued*
Myostatin Antibody	Roche	RO7239361	Discontinued*
Myostatin Antibody	Pfizer	PF-06252616	Discontinued*

Muscle Stabilization

Mechanism	Company	Drug	Status
Muscle Stabilizer	Edgewise Therapeutics	EDG-5506	Clinical

Cell Therapy

Mechanism	Company	Drug	Status
Gene editing/Stem cells	Anagenesis Biotherapies	CRISPR/Cas9 edited stem cells	Preclinical
Allogenic iPSCs	Vita Therapeutics	VTA-1100	Preclinical

*Discontinued means Discontinued at the clinical stage

Last Updated 6/21/2021