TABLE 2.

Oligonucleotides in Phase 3 clinical trials (http//www.ClinicalTrials.gov).

Disease area	Compound	Indication	Target	Chemical class	NCT ID
Neurological	Tominersen	Huntingtons’ disease	Huntingtin protein (HTT)	ASO	NCT03761849
	Tofersen	Amyotrophic lateral sclerosis (ALS)	Superoxide dismutase type-1 (SOD1)	ASO	NCT02623699
Cardiovascular	Pelacarsen	Elevated lipoprotein (Lp)	Apolipoprotein(a)	ASO	NCT04023552
	AMG 890	Elevated lipoprotein (Lp)	Apolipoprotein(a)	siRNA	NCT03626662
	AKCEA– transthyretin (TTR)-LRX	TTR amyloidosis	Transthyretin (TTTR) protein	ASO	NCT04136184
	Vutrisiran	TTR amyloidosis	TTR protein	siRNA	NCT03759379
	CGT003	Cornory artery diseases	Transcription factor E2F	Decoy	NCT00041925, NCT00042081
Hepatic	Nidosiran	Primary hyperoxaluria type 1, 2 and 3	Lactate dehydrogenase (LDHA)	siRNA	NCT04042402
	JNJ-3989	Hepatitis B (HBV)	RNAi	siRNA	NCT04129554
Hematologic	Fitusiran	Hemophilia and rare bleeding disorder (RBD)	Antithrombin	siRNA	NCT03417245
	Cemdisiran	Peroxysmal Nocturnal Hemoglobinuria	C5	ASO	NCT05070858, NCT05133531, NCT05131204
Metabolic	Alicaforsen	Crohn’s disease	Intracellular adhesion molecules (ICAM-1)	ASO	NCT00048295
Ophthalmic	Sepofarsen	Leber congenital amaurosis 10	CEP290 gene, p. Cys998X mutation	ASO	NCT03913143
	QR-421a	Retinitis pigmentosa	Usher syndrome (USH2A gene)	ASO	NCT05176717
		Dry eye disease
	Ultevursen	Retinitis pigmentosa	USH2A gene	ASO	NCT05158296
	Tivanisiran	Sjögren’s Syndrome	Transient Receptor Potential Vanilloid 1 (TRPV-1)	siRNA	NCT04819269,NCT03108664
	Cosdosiran (QPI-1007)	Caspase-2	Non-arteritic anterior ischemic optic neuropathy (NAION)	siRNA	NCT01965106
Dermatological	Tilsotolimod	Anti-programmed cell death protein 1 (PD-1) refractory metastatic melanoma	Toll-like receptor 9 (TLR9)	ASO	NCT03865082
Renal	Teprasiran (QPI-1002)	Acute kidney injury	p53	siRNA	NCT02610296, NCT03510897
Cancer	Oblimersen	Melanoma	Bcl-2	ASO	NCT00016263, NCT00543205, NCT00030641