Funding Research to Find a Cure for Duchenne

Duchenne Therapeutic Pipeline

Mechanism	Company	Drug	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

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	Casimersen \| Exon 45	Clinical
Exon Skipping – PMO	Sarepta	Exon 52	Preclinical
Exon Skipping – PPMO	Sarepta	SRP-5051 \| Exon 51	Clinical
Exon Skipping – PPMO	Sarepta	Exon 44	Preclinical
Exon Skipping – PPMO	Sarepta	Exon 45	Preclinical
Exon Skipping – PPMO	Sarepta	Exon 50	Preclinical
Exon Skipping – PPMO	Sarepta	Exon 52	Preclinical
Exon Skipping – PPMO	Sarepta	Exon 53	Preclinical
Exon Skipping – PPMO	NS Pharma	Viltepso \| Exon 53	Accelerated Approval
Exon Skipping – PPMO	NS Pharma	NS-089/NCP-02 \| Exon 44	Clinical
Exon Skipping (AAV-U7)	Nationwide Children’s	Exon 2 duplication	Clinical
Exon Skipping (AAV-U7)	Audentes	AT702 \| Exon 2 duplication	Preclinical
Exon Skipping (AAV-U7)	Audentes	AT751 \| Exon 51	Preclinical
Exon Skipping (AAV-U7)	Audentes	AT753 \| Exon 53	Preclinical
Exon skipping (antibody-ASO)	Avidity Biosciences	Multiple	Preclinical
Exon skipping (antibody-ASO)	Dyne Therapeutics	Multiple	Preclinical
Exon Skipping	Genethon	rAAV.U7 snRNA \| Exon 53	Preclinical
Exon Skipping	BioMarin	Drisapersen \| Exon 51	Discontinued*
Stereopure ASO	Wave	Suvodirsen \| Exon 51	Discontinued*

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

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

Mechanism	Company	Drug	Status
Corticosteroid	PTC Therapeutics	Emflaza	Approved
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	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*

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

Mechanism	Company	Drug	Status
Coenzyme Q10 analog	Santhera	Idebenone	Clinical
Mitochondria biogenesis	Astellas	ASP0367	Clinical
Mitochondria biogenesis	Epirium	EPM-01	Clinical

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

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

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 09/15/2020