TABLE 1 | Clinical trials for RNAi therapy*

From the following article:

Current prospects for RNA interference-based therapies

Beverly L. Davidson & Paul B. McCray, Jr

Nature Reviews Genetics 12, 329-340 (May 2011)


Clinical settingDrugIndication(s)Target(s)SponsorStatus
Ocular and retinal disordersTD101Pachyonychia congenitaKeratin 6A N171K mutantPachyonychia Congenita ProjectCompleted, Phase I
QPI-1007Non-arteritic anterior ischaemic optic neuropathyCaspase 2Quark Pharm., Inc.Active, Phase I
AGN211745Age-related macular degeneration; choroidal neovascularizationVEGFR1Sirna Therapeutics, Inc.Completed, Phase I, II
PF-655Diabetic macular oedema (DME); age-related macular degeneration (AMD)RTP801Quark Pharm., Inc.Active, Phase I
SYL040012Glaucomaβ2 adrenergic receptorSylentisActive, Phase I, II
BevasiranibDiabetic macular oedemaVEGFOpko Health, Inc.Completed, Phase II
BevasiranibMacular degenerationVEGFOpko Health, Inc.Completed, Phase II
CancerCEQ508Familial adenomatous polyposisβ-cateninMDRNA, Inc.Active, Phase I
ALN-PLK1Liver tumoursPLK1Alnyam Pharm.Active, Phase I
FANGSolid tumoursFurinGradalis, Inc.Active, Phase II
CALAA-01Solid tumoursRRM2Calando Pharm.Active, Phase I
SPC2996Chronic myeloid leukemiaBCL-2Santaris Pharm.Ongoing, Phase I, II
ALN-VSP02Solid tumoursVEGF, kinesin spindle proteinAlnylam Pharm.Active, Phase I
NCT00672542Metastatic melanomaLMP2, LMP7, and MECL1Duke UniversityActive, Phase I
Atu027Advanced, recurrent or metastatic solid malignanciesPKN3Silence TherapeuticsActive, Phase I
Kidney disordersQPI-1002/I5NPAcute kidney injuryp53Quark Pharm., Inc.Terminated, Phase I
QPI-1002/I5NPDelayed graft function kidney transplantp53Quark Pharm., Inc.Active, Phase I, II
QPI-1002/I5NPKidney injury acute renal failurep53Quark Pharm., Inc.Completed, Phase I
LDL loweringTKM-ApoBHypercholesterolaemiaAPOBTekmira Pharm. Corp.Terminated, Phase I
PRO-040,201HypercholesterolaemiaAPOBTekmira Pharm. Corp.Terminated, Phase I
AntiviralSPC3649Hepatitis C virusmiR-122Santaris PharmActive, Phase II
pHIV7-shI-TAR-CCR5RZHIVHIV Tat protein, HIV TAR RNA, human CCR5City of Hope Medical Center/BenitecActive, Phase 0
ALN-RSV01RSV in volunteersRSV nucleocapsidAlnylam Pharm.Completed, Phase II
ALN-RSV01RSV in lung transplant patientsRSV nucleocapsidAlnylam Pharm.Completed, Phase I
ALN-RSV01RSV in lung transplant patientsRSV nucleocapsidAlnylam 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.

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