| hereditary transthyretin-mediated amyloidosis |
transthyretin |
siRNA-lipid nano particle (Patisiran) |
approved |
(65, 192, and 208) |
| acute hepatic porphyria |
targets amino levulinate synthase I (ALAS-1) |
siRNA-GalNAc
conjugate (Givosiran) |
approved |
(94, 95, and 209) |
| primary hyperoxaluria type 1 (PH1) |
hydroxy acid oxidase I |
chemically stabilized siRNA (Lumasiran) |
approved |
(210−212) |
| transthyretin-mediated amyloidosis |
TTR |
siRNA-GalNAc conjugate (Vutrisiran) |
approved |
(33 and 213) |
| heterozygous familial hypercholesterolemia |
PCSK9 |
siRNA-GalNAc conjugate (Inclisiran) |
approved |
(97 and 213) |
| primary hyperoxaluria |
PHI |
siRNA-GalNAc conjugate (Nedosiran) |
phase III |
(211 and 214) |
| hemophilia A and B |
antithrombin (AT) |
siRNA-GalNAc conjugate (fitusiran) |
phase III |
(215 and 216) |
| acute kidney injury |
p53 |
chemically stabilized-siRNA (teprasian) |
phase III |
(217) |
| nonarteritic anterior ischemic optic neurotherapy and primary
angle glaucoma |
CASP2 |
naked siRNA (cosdorian) |
phase III |
(211) |
| ocular pain and dry eye disease |
TRPV1 |
naked siRNA (tivanisiran) |
phase III |
(211 and 218) |
| multiple cancers |
PLK1 |
lipid nanoparticle |
phases II and III |
(192 and 219) |
| solid tumor |
KSP and VEGF |
lipid nanoparticle |
phases II and III |
(192 and 220) |
| AMD/CNV |
VEGFR |
naked siRNA |
phase II |
(192 and 221) |
| AMD/DME |
RTP801 |
naked siRNA |
phase II |
(192 and 222) |
| RSV
virus infection |
RSV nucleocapsid |
naked
siRNA |
phase II |
(192 and 223) |
| hypercholesterolemia |
PCSK9 |
lipid
nanoparticle |
phase II |
(192 and 224) |
| diabetic AMD |
VEGF |
naked
siRNA |
phase II |
(192 and 222) |
| advanced solid tumors |
PKN3 |
lipid
nanoparticle |
phase II |
(192 and 225) |
| HCC, multiple melanoma |
c-Myc |
lipid nanoparticle |
phase II |
(192 and 226) |
| Ebola
virus infection |
VP24, VP35, Zaire Ebola Lpolymerase |
lipid nanoparticle |
phase II |
(192 and 227) |
| glaucoma ocular hypertension |
ADRB2 |
naked siRNA |
phases I and II |
(192) |
| delayed graft function kidney transplant |
P53 |
naked siRNA |
phases I and II |
(228) |
| advanced cancers |
EphA2 |
lipid nanoparticle |
phase I |
(192 and 229) |
| chronic
optic nerve atrophy |
caspase 2 |
naked siRNA |
phase I |
(192 and 230) |
| metastatic melanoma |
LMP2, LMP7, MECL1 |
LODER polymer |
phase I |
(140 and 192) |
| prostate cancer treatment |
polo-like
kinase gene |
siRNA-peptide based |
clinical
trials |
(231) |
| targeted stem cell therapy |
OCT4 transcript |
dendrimer |
pre-clinical
trials |
(232 and 233) |
| idiopathic pulmonary fibrosis (IPF) |
Interleukin-11 (IL-11) |
polymeric nanoparticle |
clinical trials |
(234) |
| chronic myeloid leukemia (CML) |
BCR-ABL
fusion oncogene |
lipid nanoparticle |
preclinical
studies |
(235 and 236) |
