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Table 1 Novel siRNAs –based drugs in clinical trials (adapted and revised with permission from [9, 96])

From: Harnessing RNAi nanomedicine for precision therapy

Candidate name

Disease

Target

Delivery system

Phase

Year

Bevasiranib

AMD

VEGF

Local - intravitreal needle injection.

lll - was interupted in 2009

2004

Cand5

AMD, DME

VEGF

Local - intravitreal needle injection.

ll

2004

ALN-RSV01

RSV infection

Virial RNA

Local - inhalation of unformulated siRNAs.

ll

2005

DGFi

Acute kidney injury, delayed graft function

p53

Systematic - naked siRNA

ll

2007

TD101

Pachyonychia congenita

Mutant keratin (K6a)

Local - intradermal needle injection

l

2008

QPI-2007

Chronic nerve atrophy, nonarteritic ischemic optic neurophaty

Caspace 2

Local - intravitreal needle injection

l

2009

siG12D LODER

Operable pancreatic ductal adenocarcinoma

Mutated KRAS

Local - drug elution

l

2010

CALAA-01

Metastatic solid tumors

RRM2

Systematic - CDP NPs

l

2008

ALN-VSP02

Liver cancer, cancer with liver involvement

VEGF, KSP

Systematic - SNALP liposomes (hypatocytes)

l

2008

Atu027

Advanced solid tumors

PKN3

Systematic - AtuPLEX lipoplex (vascular endothelial cells)

l

2009

TKM-ApoB

Hypercolesterolemia

ApoB

Systematic - SNALP liposomes (hypatocytes)

l

2009

ALN-TTR01

ATTR

TTR

Systematic - SNALP liposomes (hypatocytes)

l

2009

TKM-PLK1

Solids cancers and lymphoma

PLK1

Systematic - SNALP liposomes (solid tumors)

l

2010

ALN-PCS02

Hypercolesterolemia

PCSK9

Systematic - SNALP liposomes (solid tumors)

l

2011

TKM-EBOLA

Ebola infection (biodefence)

Viral RNA polymerase

Systemic - SNALP liposomes (hypotocytes and phagocytes)

l

2011

  1. Abbreviations: ApoB Apolipoprotein B, AMD age-related macular edema, ATTR transthyretin (TTR)-mediated amyloidosis, CDP Cyclodextrin polycation, DME diabetic macular edema, KSP kinesin spindle protein, NP nanoparticle, PCSK9 proprotein convertase subtilisin/kexin type 9, PKN3 protein kinase N3, PLK1 polo-like kinase 1, RRM2 ribonucleotide reductase subunit 2, RSV respiratory syncytial virus, siRNA small interfering RNA, SNALP stable nucleic acid lipid particles, VEGF vascular endothelial growth factor.