| ASO |
Pelacarsen (AKCEA-APO(a)-LRx) |
Ionis, Akcea, and Novartis |
Gen 2+ (LICA) |
Hyperlipoproteinemia(a) and cardiovascular disease |
Phase 2/3 |
NCT03070782 |
| ASO |
AKCEA-TTR-LRx |
Ionis and Akcea |
Gen 2+ (LICA) |
Hereditary transthyretin-mediated amyloid polyneuropathy (ATTR) |
Phase 3 |
NCT04136184 |
| ASO |
Vupanorsen |
Ionis, Akcea, and Pfizer |
Gen 2+ (LICA) (3 GalNAcs are attached on the 5′ end of modified ASO) |
Cardiovascular disease |
Phase 2 |
NCT04459767 |
| ASO |
AKCEA-APOCIII-LRx |
Ionis and Akcea |
Gen 2+ (LICA) |
Cardiovascular disease/familial chylomicronemia syndrome |
Phase 2 |
NCT03385239 |
| ASO |
IONIS-FB-LRx |
Ionis and Roche |
Gen 2+ (LICA) |
IgA nephropathy |
Phase 2 |
NCT04014335 |
| ASO |
IONIS-AGT-LRx |
Ionis |
Gen 2+ (LICA) |
Hypertension |
Phase 2 |
NCT04083222 |
| ASO |
IONIS-FXI-LRx |
Ionis and Bayer |
Gen 2+ (LICA) |
Clotting disorders |
Phase 2 |
NCT03582462 |
| ASO |
IONIS-GHR-LRx |
Ionis |
Gen 2+ (LICA) |
Acromegaly |
Phase 2 |
NCT03548415 |
| ASO |
IONIS-PKK-LRx |
Ionis |
Gen 2+ (LICA) |
Hereditary angiodema |
Phase 2 |
NCT04030598 |
| ASO |
IONIS-TMPRSS6-LRx |
Ionis |
Gen 2+ (LICA) |
β-Thalassemia |
Phase 2 |
NCT04059406 |
| ASO |
IONIS-GCGRRx |
Ionis and Suzhou-Ribo |
Gen 2+ (modified ASO) |
Diabetes |
Phase 2 |
No results |
| ASO |
Tofersen |
Ionis and Biogen |
Gen 2+ (modified ASO) |
Amyotrophic lateral sclerosis (ALS) |
Phase 3 |
NCT02623699 |
| ASO |
IONIS-C9Rx (BIIB078) |
Ionis and Biogen |
Gen 2+ (modified ASO) |
Amyotrophic lateral sclerosis (ALS) |
Phase 1/2 |
NCT03626012 |
| ASO |
IONIS-MAPTRx (BIIB080) |
Ionis and Biogen |
Gen 2+ (modified ASO) |
Alzheimer’s disease and frontotemporal degeneration |
Phase 2 |
NCT03186989 |
| ASO |
ION859 (BIIB094) |
Ionis and Biogen |
Gen 2+ (modified ASO) |
Parkinson’s disease |
Phase 1/2 |
NCT03976349 |
| ASO |
ION464 (BIIB101) |
Ionis and Biogen |
Gen 2+ (modified ASO) |
Multiple system atrophy and Parkinson’s disease |
Phase 1/2 |
NCT04165486 |
| ASO |
IONIS-HBVRx |
Ionis and GSK |
Gen 2+ (modified ASO) |
Hepatitis B |
Phase 2 |
NCT02981602 |
| ASO |
Tominersen (RO7234292) |
Hoffmann-La Roche and Ionis |
Gen 2+ (modified ASO) |
Huntington’s disease |
Phase 3 halted |
NCT03761849 |
| ASO |
IONIS-DNM2-2.5Rx |
Ionis and Dynacure |
Gen 2.5 (modified ASO) |
Centronuclear myopathy |
Phase 2 |
No results |
| ASO |
IONIS-AR-2.5Rx |
Ionis and Suzhou-Ribo |
Gen 2.5 (modified ASO) |
Prostate cancer |
Phase 2 |
NCT02144051 |
| ASO |
Danvatirsen |
Ionis and AstraZeneca |
Gen 2.5 (modified ASO) |
Cancer |
Phase 2 |
NCT02983578 |
| ASO |
ION357/QR1123 |
Ionis and ProQR Therapeutics |
Gen 2.5 (modified ASO) |
Autosomal dominant retinitis pigmentosa |
Phase 1/2 |
NCT04123626 |
| ASO (AON) |
QR-313 |
Wings Therapeutics |
Hydrogel |
Epidermolysis bullosa dystrophica |
Phase 1/2 |
NCT03605069 |
| ASO |
BP1001 |
Bio-Path Holdings Inc. |
Liposome (DOPC-based, neutrally charged) |
Acute myeloid leukemia |
Phase 2 |
NCT02781883 |
| ASO |
Sepofarsen (QR-110) |
ProQR Therapeutics |
— |
Leber’s congenital amaurosis |
Phase 2/3 |
NCT03913143 |
| ASO |
GTX-102 |
GeneTX Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc |
— |
Angelman syndrome |
Phase 1/2 |
NCT04259281 |
| ASO |
QR-010 |
ProQR Therapeutics |
— |
Cystic fibrosis |
Phase 1/2 |
NCT02532764 |
| ASO |
WVE-120102 |
Wave Life Sciences Ltd. |
— |
Huntington’s disease |
Phase 1/2 |
NCT03225846 |
| ASO |
WVE-120101 |
Wave Life Sciences Ltd. |
— |
Huntington’s disease |
Phase 1/2 |
NCT03225833 |
| ASO |
Inotersen-IONIS-TTRRX/ISIS-420915 |
Ionis, Brigham, and Women’s Hospital |
— |
TTR Amyloidosis |
Phase 2/3 |
NCT03702829 |
| ASO |
QR-421a |
ProQR Therapeutics |
— |
Retinitis Pigmentosa, Usher syndrome type 2 |
Phase 1/2 |
NCT03780257 |
| ASO |
STK-001 |
Stoke Therapeutics |
— |
Dravet syndrome |
Phase 1/2 |
NCT04442295 |
| siRNA |
ARO-AAT |
Arrowhead Pharmaceuticals |
TRiM (GalNAc-siRNA conjugates) |
Alpha 1 liver disease |
Phase 2/3 |
NCT03945292 |
| siRNA |
ARO-APOC3 |
Arrowhead Pharmaceuticals |
TRiM (GalNAc-siRNA conjugates) |
Hypertrglyceridemia, Familial chylomicronemia |
Phase 1/2 |
NCT03783377 |
| siRNA |
ARO-ANG3 |
Arrowhead Pharmaceuticals |
TRiM (GalNAc-siRNA conjugates) |
Dyslipidemia |
Phase 1/2 |
NCT03747224 |
| siRNA |
ARO-HSD |
Arrowhead Pharmaceuticals |
(TRiM)-ligand conjugate |
Non-alcoholic steatohepatitis (NASH) |
Phase 1/2 |
NCT04202354 |
| siRNA |
Tivanisiran (SYL1001) |
Sylentis |
Self-delivered (Hybridized single strand complementary RNA) |
Dry eye disease |
Phase 3 |
NCT03108664 |
| siRNA |
BMT101 |
Hugel |
Self-delivered |
Hypertrophic scar |
Phase 2 |
NCT04012099 |
| siRNA |
Inclisiran |
Novartis Pharmaceuticals |
GalNAc conjugate (Modified siRNA conjugated to triantennary N-acetylgalactosamine carbohydrates) |
Homozygous familial hypercholesterolemia |
Phase 3 |
NCT03851705 |
| siRNA |
Lumasiran |
Alnylam Pharmaceuticals |
GalNAc conjugate (ESC) |
Primary hyperoxaluria type 1 |
Phase 3 (commercial) |
NCT03681184 |
| siRNA |
DCR-PHXC |
Dicerna Pharmaceuticals |
GalNAc conjugate (GalXC) |
Primary hyperoxaluria type 1 and 2 |
Phase 2 |
NCT03847909 |
| siRNA |
Fitusiran |
Genzyme (Sanofi) and Alnylam |
GalNAc conjugate (N-acetylgalactosamine conjugation) |
Hemophelia A, B; rare bleeding diseases |
Phase 3 |
NCT03417245 |
| siRNA |
Vutrisiran |
Alnylam Pharmaceuticals |
GalNAc conjugate (ESC) |
Transthyretin amyloidosis (ATTR) |
Phase 3 |
NCT03759379 |
| siRNA |
Cemdisiran |
Alnylam Pharmaceuticals |
GalNAc conjugate (N-acetylgalactosamine conjugation) |
IgA nephropathy (IgAN), Berger disease, Glomerulonephritis, IgA |
Phase 2 |
NCT03841448 |
| siRNA |
siG12D LODER |
Silenseed Ltd. |
Polymer matrix (poly (lactic-co-glycolic) acid (PLGA)) |
Locally advanced pancreatic cancer |
Phase 2 |
NCT01676259 |
| mRNA |
mRNA-1273 |
Moderna |
Ionizable LNP (SM-102, PEG-2000-DMG, DSPC, cholesterol) |
SARS-COV-2 |
Phase 3 |
NCT04405076 |
| mRNA |
mRNA-4157 |
Moderna |
Ionizable LNP |
Melanoma |
Phase 2 |
NCT03897881 |
| mRNA |
mRNA-1647 |
Moderna |
Ionizable LNP |
Cytomegalovirus |
Phase 2 |
NCT04232280 |
| mRNA |
mRNA-3704 |
Moderna |
Ionizable LNP |
Methylmalonic acidemia |
Phase 1/2 |
NCT03810690 |
| mRNA |
mRNA 2416 |
Moderna |
Ionizable LNP |
Relapsed/refractory solid tumor malignancies, lymphoma |
Phase 1/2 |
NCT03323398 |
| mRNA |
BI 1361849 (CV9202) |
CureVac, Boehringer Ingelheim, Ludwig Cancer Research |
Combination of modified mRNA and protamine complexed mRNA |
Metastatic non-small cell lung cancer |
Phase1/2 |
NCT03164772 |
| mRNA |
W_pro1 |
BioNTech RNA Pharmaceuticals |
Lipoplex |
Metastatic castration resistant prostate cancer |
Phase1/2 |
NCT04382898 |
| mRNA |
PF-07302048 (BNT162) |
Pfizer and BioNTech |
LNP (ALC-0315, ALC-0159, DSPC, and cholesterol) |
SARS-CoV-2 |
Phase 2/3 |
NCT04368728 |
| DNA |
AD26.COV2.S |
Johnson & Johnson |
Adenovirus serotype 26 vector |
SARS-CoV-2 |
Phase 3 |
NCT04614948 |
| DNA |
AZD1222(ChAdOx1 nCoV-19) |
AstraZeneca, Oxford University |
Adenovirus vector |
SARS-CoV-2 |
Phase 3 |
NCT04516746 |
| DNA |
Ad5-nCoV |
CanSino Biologics |
Adenovirus type-5 vector |
SARS-CoV-2 |
Phase 3 |
NCT04540419 |