TABLE 2.

Investigational antisense drugs.

Drug candidate NCT ID	Chemistry	Mechanism of action	Target/organ	Indication	Route	Company
Tominersen (RG6042)	2′-O-MOE-PS	RNase H1	HTT mRNA/brain	Huntington’s disease	ITH	Ionis
Casimersen	PMO	Exon skipping	DMD exon 45/muscle	DMD	IV	Sarepta
AKCEA-TTR-LRx 04136171	siRNA GalNAc	RNAi	TTR mRNA/liver	ATTR with cardiomyopathy	SC	Ionis
QPI-1002	siRNA	RNAi	p53 mRNA/kidney	Cardiac surgery/kidney delayed graft function/acute kidney injury	IV	Quark
Fitusiran/ALN-AT3	siRNA GalNAc	RNAi	Anti-thrombin mRNA/liver	Haemophilia A and B	SC	Genzyme
Volanesorsen	2′-O-MOE-PS	RNase H1	ApoC-III mRN/liver	FCS/Lipoprotein Lipase Deficiency/Hyperlipo proteinemia type 1	SC	Ionis
Alicaforsen	PS	RNase H1	ICAM1 mRNA/colon	Pouchitis	E	Atlantic
Tivanisiran	siRNA	RNAi	TRPV1/eye	Dry eye syndrome	IVT	Sylentis
IONIS-TTR Rx	2′-O-MOE-PS	RNAse H1	TTR mRNA/liver	FAP/CS	SC	Ionis

Antisense drugs that showed promise in phase III, clinical trials 2′-O- MOE—2′-O-methoxyethyl, ALS—amyotrophic lateral sclerosis, ATTR—Transthyretin amyloidosis, DMD—Duchenne muscular dystrophy, E—Enema, Apo—Apolipoprotein, FAP—familial amyloid poly neuropathy, FCS—Familial chylomicronemia syndrome, GAlNAc—N-acetylgalactosamine, HTT—Huntingtin, ICAM—Intercellular adhesion molecule, ITH—Intrathecal, IV—Intravenous, IVT—intravitreal, PMO, Phosphorodiamidate morpholino; PS, Phosphorothioate, SC—Subcutaneous, siRNA—Small interfering RNA, SOD1 —Superoxide dismutase, TTR—Transthyretin, p53—Tumor protein, TRPV1—Transient Receptor Potential Vanilloid 1.