Table 1.

Summary table of clinical trials currently active for selected small interfering RNA (siRNA) and antisense therapeutics (adapted from [10]).

Drug	Target	Condition	Phase	Status
RNAi
DCR-MYC	Myc	Solid tumors Hepatocellular carcinoma	I	Recruiting
			I/II	Recruiting
ALN-TTRSC (Revusiran)	Transthyretin	TTR-mediated familial amyloidotic cardiomyopathy	I	Completed
			II	Active
			III	Active
ALN-CC5	Complement component C5	Paroxysmal nocturnal Hemoglobinuria	I/II	Active
ALN-AS1	ALAS-1	Acute intermittent porphyria	I	Recruiting
ALN-PCSSC	PCSK9	Hypercholesterolemia	I	Completed
ALN-TTR02 (Patisiran)	Transthyretin	TTR-mediated amyloidosis	II	Active
			III	Active
ALN-AT3SC	Antithrombin	Hemophilia A/B	I	Recruiting
TKM-080301	Polo-like kinase 1	Hepatocellular carcinoma Neuroendocrine tumors	I	Active
			I/II	Recruiting
TKM-100802	Ebola genome	Ebola virus	I/II	Terminated
QPI-1007	Caspase 2	Primary angle-closure glaucoma	II	Completed
RXI-109	CTGF	Hypertrophic scar keloid excision surgery	II	Completed
			II	Completed
Atu027	PKN3	Pancreatic ductal carcinoma	I/II	Completed
SYL040012	Β2-adrenergic receptor	Ocular hypertension	II	Completed
Antisense
Mipomersen *	ApoB 100	Heterozygous familial hypercholesterolemia atherosclerosis	III	Completed
			III	Completed
ISIS-TTRRX	Transthyretin	Familial amyloid polyneuropathy	III	Active
ISIS-ApoC-IIIRX	ApoCIII	Hypertriglyceridemia	II	Completed
ISIS-DMPKRX	DMPK	Myotonic dystrophy type 1	I/II	Recruiting
ISIS APO(a)-LRX	Apoliprotein (a)	Elevated lipoprotein (a)	I	Completed
			II	Completed
Curtirsen	Clusterin	Non-small cell lung cancer Prostate cancer	III	Recruiting
			III	Active

* Already approved by the U.S. FDA under the commercial designation Kynamro^®.