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Table 2 Current clinical trials involving RNA delivery

From: Advances in the delivery of RNA therapeutics: from concept to clinical reality

Name Treatment Genetic/protein target Delivery vehicle Administration method Disease ClinicalTrials.gov identifier Phase
siRNA-EphA2-DOPC siRNA EphA2 Lipid nanoparticle Intravenous infusion Solid cancer NCT01591356 I
TD101 siRNA K6a Naked (unmodified) Intralesional injection Pachyonychia congenita NCT00716014 I
Atu027 siRNA PKN3 Lipid nanoparticle Intravenous infusion Solid cancer NCT00938574 I
ND-L02-s0201 siRNA HSP47 Lipid nanoparticle Intravenous infusion Liver fibrosis NCT01858935, NCT02227459 I
DCR-PH1 siRNA Glycolate oxidase Lipid nanoparticle Intravenous infusion Primary hyperoxaluria type 1 NCT02795325 I
STP705 siRNA TGF-1β and Cox-2 Polymer nanoparticle Intradermal injection Hypertrophic scarring NCT02956317 I/II
ALN-GO1 siRNA Glycolate oxidase Conjugate (GalNAc) Subcutaneous injection Primary hyperoxaluria type 1 NCT02706886 I/II
Fitusiran (ALN-AT3SC) siRNA Plasma antithrombin Conjugate (GalNAc) Subcutaneous injection Severe hemophilia A or B NCT02554773 I/II
ALN-CC5 siRNA Complement component C5 Conjugate (GalNAc) Subcutaneous injection Paroxysmal nocturnal hemoglobinuria NCT02352493 I/II
ALN-AS1 siRNA ALAS-1 Conjugate (GalNAc) Subcutaneous injection Acute intermittent porphyria NCT02949830 I/II
DCR-MYC siRNA MYC Lipid nanoparticle Intravenous infusion Solid cancer NCT02110563 NCT02314052 II/II
TKM 080301 siRNA PLK1 Lipid nanoparticle Liver injection Intravenous infusion Intravenous infusion Liver cancer
Liver cancer Adrenocortical carcinoma
NCT01437007 NCT02191878 NCT01262235 I
I/II
I/II
siG12D-LODER siRNA KRASG12D Degradable polymer Local implantation Pancreatic cancer NCT01676259 II
Inclisiran (ALN-PCSSC) siRNA PCSK9 Conjugate (GalNAc) Subcutaneous injection Hypercholesterolemia NCT03060577 II
PF-655 siRNA RTP801 Naked (modified) Intravitreal injection Diabetic macular edema NCT01445899 II
SYL1001 siRNA TRPV1 Naked (modified) Eye drops Dry eye syndrome NCT02455999 II
Bamosiran (SYL040012) siRNA β-2 adrenergic receptor Naked (modified) Eye drops Glaucoma NCT02250612 II
QPI-1007 siRNA Caspase 2 Naked (modified) Intravitreal injection Acute nonarteritic anterior ischemic optic neuropathy NCT02341560 II/III
QPI-1002 siRNA p53 Naked (modified) Intravenous infusion Prevention of acute kidney injury
Delayed graft function in kidney transplant recipients
NCT02610283
NCT02610296
II
III
Patisiran (ALN-TTR02) siRNA TTR Lipid nanoparticle Intravenous infusion Familial amyloid polyneuropathy NCT01960348 III
ISTH0036 ASO TGF-β2 Naked (modified) Intravitreal injection Glaucoma NCT02406833 I
EZN-2968 (RO7070179) ASO HIF1A Naked (modified) Intravenous infusion Liver cancer NCT02564614 I
LErafAON-ETU ASO C-raf Lipid nanoparticle Intravenous infusion Advanced cancer NCT00100672 I
AKCEA-APOCIII-LRx ASO ApoCIII Conjugate (GalNAc) Subcutaneous injection Elevated triglycerides NCT02900027 I
BIIB067 (IONIS-SOD1Rx) ASO SOD1 Naked (modified) Intrathecal injection Amyotrophic lateral sclerosis NCT02623699 I
AZD5312 ASO Androgen receptor Naked (modified) Intravenous infusion Prostate cancer NCT02144051 I
Cenersen ASO p53 Naked (modified) Intravenous infusion Myelodysplastic syndrome NCT02243124 I
IONIS-HTT Rx ASO Huntingtin Naked (modified) Intrathecal injection Huntington's disease NCT02519036 I/II
IONIS ANGPTL3-LRx ASO ANGPTL3 Conjugate (GalNAc) Subcutaneous injection Elevated triglycerides/familial hypercholesterolemia NCT02709850 I/II
AZD9150 ASO STAT3 Naked (modified) Intravenous infusion Solid cancer NCT01563302 I/II
QR-010 ASO CFTR (causes base insertion) Naked (modified) Nebulization (inhaled) Cystic fibrosis NCT02532764 I/II
SB012 ASO GATA-3 Naked (modified) Enema Ulcerative colitis NCT02129439 I/II
AEG35156 ASO XIAP Naked (modified) Intravenous infusion Solid cancer NCT00882869 I/II
DS-5141b ASO Dystrophin (exon skipping) Naked (modified) Subcutaneous injection Duchenne muscular dystrophy NCT02667483 I/II
AKCEA-APO(a)-LRx ASO ApoA Conjugate (GalNAc) Subcutaneous injection Hyperlipoproteinemia(a) NCT03070782 II
Apatorsen (OGX-427) ASO Hsp27 Naked (modified) Intravenous infusion Solid cancer NCT01829113 II
IONIS-HBV Rx ASO HBV surface antigen Naked (modified) Subcutaneous injection Hepatitis B infection NCT02981602 II
IONIS-GCGR Rx ASO GCGR Naked (modified) Subcutaneous injection Type 2 diabetes NCT02824003 II
ASM8 ASO CCR3, β-chain of IL-3, IL-5, and GM-CSF Naked (modified) Nebulization (inhaled) Allergen-induced asthma NCT00822861 II
SB010 ASO GATA-3 Naked (modified) Nebulization (inhaled) Asthma NCT01743768 II
SB011 ASO GATA-3 Naked (modified) Topical Atopic dermatitis NCT02079688 II
G4460 ASO C-myb Naked (modified) Intravenous infusion Liquid cancer NCT00780052 II
Prexigebersen (BP1001) ASO Grb2 Lipid nanoparticle Intravenous infusion Myeloid leukemia NCT02781883 II
IONIS-FXI Rx ASO Factor XI Naked (modified) Subcutaneous injection Clotting disorders NCT02553889 NCT01713361 II
Aganirsen (GS-101) ASO IRS-1 Naked (modified) Eye drops Neovascular glaucoma NCT02947867 II/III
Eteplirsen (AVI-4658) ASO Dystrophin (exon skipping) Naked (modified) Intramuscular injection Duchenne muscular dystrophy NCT02255552 III
Alicaforsen ASO ICAM-1 Naked (modified) Enema Pouchitis NCT02525523 III
Volanesorsen ASO ApoCIII Naked (modified) Subcutaneous injection Familial chylomicronemia syndrome
Familial partial lipodystrophy
NCT02658175
NCT02527343
III
II/III
IONIS-TTR Rx ASO TTR Naked (modified) Subcutaneous injection Familial amyloid polyneuropathy NCT01737398 III
Custirsen (OGX-011) ASO Clusterin Naked (modified) Intravenous infusion Prostate cancer
Non small cell lung cancer
NCT01578655 NCT01630733 III
Lipo-MERIT mRNA Tumor-associated antigens mRNA–Lipoplex Intravenous infusion Melanoma NCT02410733 I
IVAC mutanome/warehouse mRNA Patient-specific tumor antigens Naked mRNA Intra-nodal Triple negative breast cancer
Melanoma
NCT02316457
NCT02035956
I
TNBC-MERIT mRNA Tumor-associated antigens mRNA–Lipoplex Intravenous infusion Triple negative breast cancer NCT02316457 I
CV7201 mRNA Rabies virus glycoprotein Naked mRNA Intramuscular injection Rabies NCT02241135 I
CV8102 mRNA RNA-based adjuvant Naked mRNA Intramuscular injection RSV, HIV, rabies NCT02238756 I
mRNA-1851 mRNA Hemagglutinin 7 (H7) protein ND Intramuscular injection Influenza A ND I
mRNA-1440 mRNA Hemagglutinin 10 (H10) protein ND Intramuscular injection Influenza A ND I
mRNA MRK-1777 mRNA Vaccine ND Intramuscular injection Undisclosed ND I
mRNA AZD-8601 mRNA VEGF-A Naked (modified) Intradermal Cardiovascular disease NCT02935712 I
mRNA-1325 mRNA Viral antigenic proteins Lipid nanoparticle Intramuscular injection Zika NCT03014089 I/II
CV9103 mRNA Tumor-associated antigens Naked mRNA ND Prostate cancer NCT00831467 I/II
  mRNA Tumor-specific antigens Naked mRNA Autologous dendritic cell therapy Prostate cancer patients NCT01197625 I/II
AGS-004 mRNA Vaccine Naked mRNA Autologous dendritic cell therapy HIV infections NCT01069809, NCT02707900 I
II
  mRNA CT7, MAGE-A3, and WT1 Naked mRNA Autologous dendritic cell therapy Multiple myeloma
Acute myeloid leukemia
NCT01995708 NCT01686334 I
II
AGS-003-LNG mRNA Tumor-specific antigens Naked mRNA Autologous dendritic cell therapy Non-small cell lung cancer NCT02662634 II
iHIVARNA-01 mRNA HIV target antigens Naked mRNA Intranodal route HIV infections NCT02888756 II
AGS-003 mRNA Tumor-specific antigens Naked mRNA Autologous dendritic cell therapy Renal cell carcinoma NCT01482949 NCT00678119 NCT01582672 II
III
  1. ASO antisense oligonucleotide, mRNA messenger RNA, siRNA short interfering RNA, ND not disclosed