Table 4.
GalNAc-conjugated RNA drugs.
| On the market | ||||||
|---|---|---|---|---|---|---|
| RNA type | Product | Target | Indications | Company | Study start year | |
| siRNA | Givlaari (givosiran) | ALAS1 | Acute hepatic porphyrias | Alnylam Pharmaceuticals | 2019 | |
| siRNA | OXLUMO (lumasiran) | Glycolate oxidase | Primary hyperoxaluria type 1 | Alnylam Pharmaceuticals | 2020 | |
| siRNA | Inclisiran(Leqvio) | PCSK9 | Hypercholesterolemia | Novartis/Alnylam Pharmaceuticals | 2020 | |
| In clinical trials | ||||||
| RNA type | Drug | Target | Conditions | Phase | Identifier | Start-Complete |
| siRNA | SLN124 | TMPRSS6 | Non-transfusion-dependent Thalassemia, Low Risk Myelodysplastic Syndrome | I | NCT04176653 | August 20, 2019-October 14, 2021 |
| siRNA | Lumasiran (ALN-GO1) |
HAO1 | Primary Hyperoxaluria Type 1 | II | NCT03350451 | April 4, 2018-June 2023 |
| siRNA | ALN-PCSSC | PCSK9 | Homozygous Familial Hypercholesterolemia | II | NCT02963311 | December 13, 2016-October 8, 2018 |
| siRNA | ALNAT3SC (Fitusiran, SAR439774) | AT | Hemophilia A; Hemophilia B | I/II | NCT02554773 | September 18, 2015-August 2023 |
| siRNA | ALN-AGT01 | AGT | Hypertension | II | NCT04936035 | June 25, 2021-December 2024 |
| ASO | Pelacarsen (TQJ230) |
Apo(a) | Cardiovascular Disease and Lipoprotein(a) | III | NCT04023552 | December 12, 2019-June 27, 2024 |
| ASO | IONIS-FB-LRx | Factor B | Primary IgA Nephropathy | II | NCT03313778 | December 4, 2019-December 2023 |
| ASO | ApoC-III | Familial Chylomicronemia Syndrome | III | NCT04568434 | November 18, 2020-June 2023 | |
All the information from https://clinicaltrials.gov/.
Abbreviation: TMPRSS6: transmembrane serine protease 6, HAO1: hydroxyacid oxidase 1, AT: antithrombin.