Table 4.
Examples of ongoing clinical trials for mRNA-based protein replacement therapy and RNA-NP based gene editing applications [19,51,72,73].
| Company/Investigator | Disease target | Type of therapy | RNA | NP carrier | Method of administration | Phase | NCT Number |
|---|---|---|---|---|---|---|---|
| Moderna Therapeutics Inc. | Propionic acidemia | Protein replacement | mRNA 3927 encoding for and subunits of mitochondrial propionyl-CoA carboxylase protein | Lipid NP (Moderna lipid 5, cholesterol, PEG2000, DSPC) | IV infusion, up to 10 doses over 30 weeks | I/II | NCT04159103 |
| Translate Bio Inc. | Cystic fibrosis (CF) | Protein replacement | mRNA MRT 5005 for human CF transmembrane regulator protein | Lipid NP (Poly(β-amino esters, DOPE, PEG2000, cholesterol) | Nebulization | I/II | NCT03375047 |
| Nitto Denko Corporation | Hepatic and pulmonary fibrosis | Gene editing | siRNA for heat shock protein (HSP) 47 | Lipid NP (diretinamide-PEG-diretinamide conjugated to Vitamin A) | IV, every 2 weeks | II | NCT03538301 |
| Arcturus Therapeutics, Inc. | Ornithine Transcarb-amylase (OTC) deficiency | Protein replacement | ARCT-810 mRNA encoding for OTC enzyme | Proprietary lipid NP (LUNAR®) | IV | I | NCT04442347 |
Legend: NP- nanoparticle, PEG-polyethylene glycol, DSPC- 1,2-distearoyl-sn-glycero-3-phosphocholine, IM-intramuscular, ID- intradermal, IV- intravenous, SQ- subcutaneous, DOPE- 1,2-dioleoyl-sn-glycero-3-phosphoethanolamine.