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Research Overview

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Preclinical Clinical Approved

Anti-Fibrosis Therapy

	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

Anti-Inflammatory Therapy

	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

Cardiac Function

	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

Cell Therapy

	Research	Company	U7 Exon Skipping	Status
	Gene editing/Stem cells	Anagenesis Biotherapies	CRISPR/Cas9 edited stem cells	Preclinical

Exon Skipping

	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

Gene Correction

	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

Mitochondrial

	Research	Company	U7 Exon Skipping	Status
	Coenzyme Q10 analog	Santhera	Catena Idebenone	Clinical
	Mediated gene regulation	Mitobridge	MTB-1	Preclinical

Muscle Regeneration

	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

Protein Replacement

	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

Stop Codon Read-Through

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