Table 2.
Representative clinical trials of lipid nanoparticle–mRNA therapeutics against infections, cancer and genetic disorders
| Name | Disease | Encoded protein | Administration route | ClinicalTrials.gov identifier | Phase |
|---|---|---|---|---|---|
| Infections | |||||
| mRNA-1944 | Chikungunya virus | Antibody against chikungunya virus | i.v. | NCT03829384 | I |
| Cancer | |||||
| mRNA 2416 | Solid tumours | OX40L | Intratumour | NCT03323398 | II |
| mRNA-2752 | Solid tumours | OX40L, IL-23 and IL-36γ | Intratumour | NCT03739931 | I |
| MEDI1191 | Solid tumours | IL-12 | Intratumour | NCT03946800 | I |
| SAR441000 | Solid tumours | IL-12sc, IL-15sushi, IFNα and GM-CSF | Intratumour | NCT03871348 | I |
| Genetic disorders | |||||
| mRNA-3704 | Methylmalonic acidaemia | Methylmalonyl-CoA mutase | i.v. | NCT03810690 | I/II |
| mRNA-3927 | Propionic acidaemia | Propionyl-CoA carboxylase | i.v. | NCT04159103 | I/II |
| MRT5201 | Ornithine transcarbamylase deficiency | Ornithine transcarbamylase | i.v. | NCT03767270 | I/II |
| MRT5005 | Cystic fibrosis | Cystic fibrosis transmembrane conductance regulator | Inhalation | NCT03375047 | I/II |
| NTLA-2001 | Transthyretin amyloidosis with polyneuropathy | CRISPR–Cas9 gene editing system | i.v. | NCT04601051 | I |
CoA, coenzyme A; CRISPR–Cas9, clustered regularly interspaced short palindromic repeats (CRISPR)–CRISPR-associated protein 9; GM-CSF, granulocyte–macrophage colony-stimulating factor; IFN, interferon; IL, interleukin; i.v., intravenous.