Table 1.
Representative ongoing nanoparticle-based mRNA clinical trials
| Trial ID | Phase | Route of administration | Therapy name | Cargo | Indication | Sponsor |
|---|---|---|---|---|---|---|
| NCT04917861 | 2 | IM | mRNA-1893 | Viral antigen | Zika virus | Moderna |
| NCT05397223 | 1 | IM | mRNAs 1345, 1647, 1272, and 1010 | Viral antigens | COVID/RSV/Flu/CMV | Moderna |
| NCT03313778 | 1 | IM | mRNA-4157 | Personalized neoantigen | Solid tumors | Moderna |
| NCT05398029 | 1 | IV | VERVE-101 | Base editor + sgRNA | Hypercholesterolemia and atherosclerosis | Verve Therapeutics |
| NCT04601051 | 1 | IV | NTLA-2001 | Cas9 + sgRNA | Transthyretin amyloidosis | Intellia Therapeutics |
| NCT05120830 | 1/2 | IV | NTLA-2002 | Cas9 + sgRNA | Hereditary angioedema | Intellia Therapeutics |
| NCT04159103 | 1/2 | IV | mRNA-3927 | PCCA, PCCB mRNA | Propionic acidemia | Moderna |
| NCT05659264 | 1 | IV | mRNA-0814 | Relaxin | Heart failure | Moderna |
| NCT04486378 | 2 | IV | RO7198457 | Personalized neoantigen | Colorectal cancer | BioNTech |
| NCT03946800 | 1 | IT | MEDI1191 | IL-12 | Solid tumors | MedImmune/Moderna |
| NCT05668741 | 1 | Nebulized | VX-522 | CFTR | CF | Vertex Pharma/Moderna |
| NCT05712538 | 1 | Nebulized | ARCT-032 | CFTR | CF | Arcturus Therapeutics |
| NCT05737485 | 1 | Nebulized | RCT1100 | DNAi1 | Primary ciliary dyskinesia | ReCode Therapeutics |
IM: intramuscular administration. IV: intravenous administration. IT: intratumoral administration. RSV: respiratory syncytial virus. “Flu” refers to seasonal influenza. PCCA and PCCB: subunits of propionyl-CoA carboxylase, mutations in which cause propionic acidemia (122).