Table 1.

Clinical trials for RNAi therapy^*

Clinical setting	Drug	Indication(s)	Target(s)	Sponsor	Status
Ocular and retinal disorders	TD101	Pachyonychia congenita	Keratin 6A N171K mutant	Pachyonychia Congenita Project	Completed, Phase I
	QPI-1007	Non-arteritic anterior ischaemic optic neuropathy	Caspase 2	Quark Pharm., Inc.	Active, Phase I
	AGN211745	Age-related macular degeneration; choroidal neovascularization	VEGFR1	Sirna Therapeutics, Inc.	Completed, Phase I, II
	PF-655	Diabetic macular oedema (DME); age-related macular degeneration (AMD)	RTP801	Quark Pharm., Inc.	Active, Phase I
	SYL040012	Glaucoma	β2 adrenergic receptor	Sylentis	Active, Phase I, II
	Bevasiranib	Diabetic macular oedema	VEGF	Opko Health, Inc.	Completed, Phase II
	Bevasiranib	Macular degeneration	VEGF	Opko Health, Inc.	Completed, Phase II
Cancer	CEQ508	Familial adenomatous polyposis	β-catenin	MDRNA, Inc.	Active, Phase I
	ALN-PLK1	Liver tumours	PLK1	Alnyam Pharm.	Active, Phase I
	FANG	Solid tumours	Furin	Gradalis, Inc.	Active, Phase II
	CALAA-01	Solid tumours	RRM2	Calando Pharm.	Active, Phase I
	SPC2996	Chronic myeloid leukemia	BCL-2	Santaris Pharm.	Ongoing, Phase I, II
	ALN-VSP02	Solid tumours	VEGF, kinesin spindle protein	Alnylam Pharm.	Active, Phase I
	NCT00672542	Metastatic melanoma	LMP2, LMP7, and MECL1	Duke University	Active, Phase I
	Atu027	Advanced, recurrent or metastatic solid malignancies	PKN3	Silence Therapeutics	Active, Phase I
Kidney disorders	QPI-1002/I5NP	Acute kidney injury	p53	Quark Pharm., Inc.	Terminated, Phase I
	QPI-1002/I5NP	Delayed graft function kidney transplant	p53	Quark Pharm., Inc.	Active, Phase I, II
	QPI-1002/I5NP	Kidney injury acute renal failure	p53	Quark Pharm., Inc.	Completed, Phase I
LDL lowering	TKM-ApoB	Hypercholesterolaemia	APOB	Tekmira Pharm. Corp.	Terminated, Phase I
	PRO-040,201	Hypercholesterolaemia	APOB	Tekmira Pharm. Corp.	Terminated, Phase I
Antiviral	SPC3649	Hepatitis C virus	miR-122	Santaris Pharm	Active, Phase II
	pHIV7-shI-TAR-CCR5RZ	HIV	HIV Tat protein, HIV TAR RNA, human CCR5	City of Hope Medical Center/Benitec	Active, Phase 0
	ALN-RSV01	RSV in volunteers	RSV nucleocapsid	Alnylam Pharm.	Completed, Phase II
	ALN-RSV01	RSV in lung transplant patients	RSV nucleocapsid	Alnylam Pharm.	Completed, Phase I
	ALN-RSV01	RSV in lung transplant patients	RSV nucleocapsid	Alnylam Pharm.	Active, Phase II
APOB, apolipoprotein B; BCL-2, B-cell CLL/lymphoma 2; CCR5, C-C chemokine receptor type 5; LDL, low-density lipoprotein; LMP2, also known as proteasome subunit beta type 9 (PSMB9); LMP7, also known as proteasome subunit beta type 8 (PSMB8); MECL1, also known as proteasome subunit beta type 10 (PSMB10); Pharm., Pharmaceuticals; PKN3, protein kinase N3; PLK1, polo-like kinase 1; RRM2, ribonucleoside-diphosphate reductase subunit M2; RSV, respiratory syncytial virus; RTP801, also known as DNA damage-inducible transcript 4 protein (DDIT4); VEGF, vascular endothelial growth factor.
*From ClinicalTrials.gov.