Table 2.
Name | Developer | Type | Clinical phase | Target | Route of administration |
---|---|---|---|---|---|
SAFE-T-HBV | Lion TCR Pte. Ltd. | mRNA HBV/TCR T-cells | Phase 1 | Hepatocellular carcinoma | IV infusion |
Amaretto | UTC Therapeutics Inc. | mRNA-engineered anti-mesothelin (MESO) chimeric antigen receptor T-Cell (CAR T-cells) therapy | Phase 1 | Refractory malignant solid neoplasm | IV infusion |
mRNA-3927 | Moderna TX, Inc. | mRNA encoding two proteins that form the deficient enzyme (PCCA [PA type I]/PCCB [PA type II]) | Phase 1/2 | Propionic acidemia | IV infusion |
mRNA-3705 | Moderna TX, Inc. | mRNA encoding MUT enzyme (methylmalonic CoA mutase) | Phase 1/2 | Methylmalonic acidemia | IV |
ARCT-810 | Arcturus Therapeutics, Inc. | mRNA encoding ornithine transcarbamylase (OTC) | Phase 2 | Ornithine transcarbamylase deficiency | IV infusion |
mRNA-3745 | Moderna TX, Inc. | mRNA encoding glucose 6-phosphatase (G6Pase) enzyme | Glycogen storage disease | IV infusion |
IV, intravenous injection.