Table 1.
Injected nanoparticles for organ-targeted drug delivery studied in clinical trials
| Drug name | Cargo | Delivery target | Application | Clinical trial |
|---|---|---|---|---|
| Patisiran (Onpattro) | TTR siRNA | Liver hepatocytes | Silencing of the TTR gene to treat transthyretin-mediated amyloidosis | NCT04201418 |
| NCT03997383 | ||||
| NCT03862807 | ||||
| NCT03759379 | ||||
| NCT02939820 | ||||
| NCT02510261 | ||||
| NCT02053454 | ||||
| NCT01961921 | ||||
| NCT01960348 | ||||
| NCT01617967 | ||||
| NCT01559077 | ||||
| BMS-986263 | HSP47 siRNA | Hepatic stellate cells | Silencing of HSP47 expression to inhibit collagen secretion in hepatic fibrosis | NCT01858935 |
| NCT02227459 | ||||
| NCT03420768 | ||||
| NCT03241264 | ||||
| NCT03142165 | ||||
| NCT04267393 | ||||
| NCT04225936 | ||||
| mRNA-3927 | PCC subunit-α mRNA, PCC subunit-β mRNA | Liver hepatocytes | Replacement of the PCC enzyme to treat propionic acidaemia | NCT04159103 |
| mRNA-1944 | CHKV-24 light chain mRNA, CHKV-24 heavy chain mRNA | Liver hepatocytes | Systemic secretion of an anti-chikungunya virus antibody for passive immunization | NCT03829384 |
| ARCT-810 | OTC mRNA | Liver hepatocytes | Replacement of the OTC enzyme to treat ornithine transcarbamylase deficiency | NCT04442347 |
| NCT04416126 | ||||
| NTLA-2001 | Cas9 mRNA, TTR sgRNA | Liver hepatocytes | Gene editing of the TTR locus to treat transthyretin-mediated amyloidosis | NCT04601051 |
| NTLA-2002 | Cas9 mRNA, KLKB1 sgRNA | Liver hepatocytes | Gene editing of the KLKB1 locus to treat hereditary angioedema | NCT05120830 |
| VERVE-101 | ABE mRNA, PCSK9 sgRNA | Liver hepatocytes | Gene editing of the PCSK9 locus to lower LDL cholesterol levels and prevent cardiovascular disease | NCT05398029 |
| mRNA-3705 | MCM mRNA | Liver hepatocytes | Replacement of the MCM enzyme to treat methylmalonic acidemia | NCT04899310 |
| Lipo-MERIT | Melanoma-associated antigens mRNA | Dendritic cells | Expression of a cocktail of tumour-associated antigens to stimulate an immune response against advanced melanoma | NCT02410733 |
| CLDN6 RNA-LPX | CLDN6 mRNA | Dendritic cells | Expression of CLDN6 to amplify the activity of CAR-T cells directed against solid tumours | NCT04503278 |
| Autogene cevumeran | Individualized neoantigen mRNA | Dendritic cells | Expression of personalized tumour neoantigens as an individualized cancer vaccine | NCT03289962 |
ABE, adenine base editor; CAR-T, chimeric antigen receptor T; Cas9, CRISPR-associated 9; CHKV, Chikungunya virus; CLDN, Claudin; HSP47, heat shock protein 47; LDL, low-density lipoprotein; MCM, methylmalonyl-CoA mutase; mRNA, messenger RNA; OTC, ornithine transcarbamylase; PCC, propionyl-CoA carboxylase; sgRNA, single guide RNA; siRNA, small interfering RNA.