Table 5.

Representative clinical trials of RNAi therapeutics in the past 10 years.

Year	Therapeutic Name	Disease	Delivery Route	Phase Stage	Target	NCT ID	Ref.
2020	ALN-HSD	NASH	Subcutaneous	I	HSD17B13	NCT04565717	[153]
2020	DCR-PHXC	PH3	Subcutaneous	I	LDHA	NCT04555486	[154]
2019	Vutrisiran	ATTR With Cardiomyopathy	Subcutaneous	III	TTR	NCT04153149	[155]
2019	DCR-PHXC	PH1, PH2, Kidney Diseases	Subcutaneous	II	LDHA	NCT03847909	[154]
2018	Lumasiran	PH1	Subcutaneous	II	Glycolate oxidase	NCT03350451	[156]
2018	DCR-HBVS	Chronic Hepatitis B	Subcutaneous	I	HBV transcripts	NCT03772249	[157]
2018	siG12D-LODER	Pancreatic Cancer	Intravenous	II	KRAS G12D, all additional G12X mutations	NCT01676259	[6]
2018	Inclisiran	ACD	Intravenous	III	PCSK9	NCT03705234	[158]
2016	ARC-AAT	AATD	Intravenous	II	Z-AAT	NCT02900183	[159]
2016	ALN-HBV	HBV	Subcutaneous	I	HBV RNA	NCT02826018	[160]
2016	DCR-PH1	PH1	Intravenous	I	GO	NCT02795325	[161]
2016	ALN-TTRSC02	ATTR Amyloidosis	Subcutaneous	I	TTR	NCT02797847	[162]
2015	ARC-520	Chronic Hepatitis B	Intravenous	II	HBV DNA	NCT02349126	[163]
2015	ALN-CC5	PNH	Subcutaneous	I, II	C5	NCT02352493	[164]
2015	Fitusiran	Hemophilia A, B	Subcutaneous	I, II	AT	NCT02554773	[165]
2015	ALN-AS1	AIP	Subcutaneous	I	ALAS1	NCT02452372	[166]
2014	SYL040012	Open Angle Glaucoma	Ocular topical	II	ADRβ2	NCT02250612	[68]
2014	ALN-TTR02	TTR-Mediated Amyloidosis	Intravenous	I	TTR	NCT02053454	[167]
2014	ALN-PCSSC	Hypercholesterolemia	Subcutaneous	I	PCSK9	NCT02314442	[168]
2013	ALN-TTRSC	TTR-Mediated Amyloidosis	Subcutaneous	II	TTR	NCT01981837	[169]
2012	SYL1001	Ocular Pain	Ocular topical	I, II	TRPV1	NCT01776658	[170]
2012	AVI-7100	Influenza	Intravenous	I	Influenza A M1/M2	NCT01747148	[171]

Notes: NASH, nonalcoholic steatohepatitis; PH3, primary hyperoxaluria type 3; ATTR, transthyretin amyloidosis; PH1, primary hyperoxaluria type 1; PH2, primary hyperoxaluria type 2; ACD; atherosclerotic cardiovascular disease; AATD, alpha-1 antitrypsin deficiency; PNH, paroxysmal nocturnal hemoglobinuria; AIP, acute intermittent porphyria.