TABLE 4.
Selected representative examples of current active siRNA clinical trials
| NCT ID (Phase) | Disease type (indication) | Intervention (Sponsor) | Administration | Delivery System (Detailed) |
|---|---|---|---|---|
| NCT01591356 (Phase 1) | Cancer (advanced malignant solid neoplasm) | EphA2‐targeting DOPC‐encapsulated siRNA (National Cancer Institute) | Intravenous | Liposome |
| NCT03819387 (Phase 1) | Cancer (non‐small cell lung cancer, pancreatic cancer, colorectal cancer) | NBF‐006 (Nitto BioPharma) | Intravenous | LNP |
| NCT01676259 (Phase 2) | Cancer (pancreatic ductal adenocarcinoma, pancreatic cancer) | siG12D‐LODER (Silenseed Ltd) | Implant | Other |
| NCT04995536 (Phase 1) | Cancer (recurrent B‐cell non‐Hodgkin lymphoma) | CpG‐STAT3 siRNA (National Cancer Institute) | Intratumoral | Conjugate (CpG oligodeoxynucleotide) |
| NCT04844840 (Phase 2) | Cosmetic (Keloid) | STP705 (Sirnaomics) | Intradermal | Other (Non Lipid NP) |
| NCT03814187 (Phase 3) | Genetic (atherosclerotic cardiovascular disease, elevated cholesterol, heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia) | Inclisiran (Novartis) | Subcutaneous | Conjugate (GalNAc) |
| NCT04666298 (Phase 2) | Genetic (hypercholesterolemia, heterozygous familial hypercholesterolemia) | Inclisiran (Novartis) | Subcutaneous | Conjugate (GalNAc) |
| NCT03759379 (Phase 3) | Genetic (amyloidosis, hereditary, transthyretin amyloidosis) | Patisiran and Vutrisiran (Alnylam Pharmaceuticals) | Intravenous | Conjugate (GalNAc) |
| NCT04042402 (Phase 3) | Genetic (primary hyperoxaluria type 1, primary hyperoxaluria type 2, kidney diseases, urologic diseases, genetic disease) | DCR‐PHXC (Dicerna Pharmaceuticals) | Subcutaneous | Conjugate (GalNAc) |
| NCT04153149 (Phase 3) | Genetic (transthyretin amyloidosis [ATTR] with cardiomyopathy) | Vutrisiran (Alnylam Pharmaceuticals) | Subcutaneous | Conjugate (GalNAc) |
| NCT02949830 (Phase 1/2) | Genetic (acute intermittent porphyria) | Givosiran (Alnylam Pharmaceuticals) | Subcutaneous | Conjugate (GalNAc) |
| NCT03681184 (Phase 3) | Genetic (primary hyperoxaluria type 1) | Lumasiran (Alnylam Pharmaceuticals) | Subcutaneous | Conjugate (GalNAc) |
| NCT03772249 (Phase 1) | Infectious (hepatitis B, chronic) | DCR‐HBVS (Dicerna Pharmaceuticals) | Subcutaneous | Conjugate (GalNAc) |
| NCT04819269 (Phase 3) | Inflammation (dry eye disease, Sjogren syndrome) | Tivanisiran (Sylentis) | Ocular | Other (Naked) |
| NCT04601844 (Phase 1) | Broad indications | Cemdisiran (Alnylam Pharmaceuticals) | Subcutaneous | Conjugate (GalNAc) |
| NCT03705234 (Phase 3) | Physiological (atherosclerotic cardiovascular disease) | Inclisiran (Novartis) | Subcutaneous | Conjugate (GalNAc) |
| NCT04267393 (Phase 2) | Physiological (nonalcoholic steatohepatitis [NASH]) | BMS‐986263 (Bristol‐Myers Squibb) | Intravenous | LNP |
| NCT03841448 (Phase 2) | Physiological (IgA nephropathy [IgAN], Berger disease, glomerulonephritis) | Cemdisiran (Alnylam Pharmaceuticals) | Subcutaneous | Conjugate (GalNAc) |