Table II.
Lipid nanoparticle-based formulations of selected RNA therapeutics currently under clinical investigation.
| Disease condition | Formulation and mRNA dose (when known) | Company/entity | Clinical trial phase | Clinical trial registry |
|---|---|---|---|---|
| SARS-CoV-2 | BNT162b2 (30 g) | Pfizer/BioNTech SE | EUA/CMA | NCT04368728 |
| SARS-CoV-2 | mRNA-1273 (100 g) | ModernaTX, Inc., BARDA, NIAID | EUA/CMA | NCT04470427 |
| SARS-CoV-2 | CVnCoV (12 g) | CureVac AG | III | NCT04652102 |
| SARS-CoV-2 | ARCT-021 (7.5 g or 2 5 g) | Arcturus Therapeutics, Inc. | II | NCT04728347 |
| SARS-CoV-2 | DS-5670a (10, 30, 60 or 100 g) | Daiichi Sankyo Co., Ltd. | I/II | NCT04821674 |
| SARS-CoV-2 | LNP-nCoVsaRNA (2.5, 5 or 10 g) | Imperial College London | I | ISRCTN17072692/2020-001209-22 |
| SARS-CoV-2 | MRT5500 (up to 13.5 g) | TranslateBio, Sanofi | I/II (abandoned) | NCT04798027 |
| Zika virus | mRNA-1893 (30 or 100 g) | ModernaTX, Inc. | II | NCT04917861 |
| Chikungunya virus | VAL-181388/mRNA-1388 (25, 50 or 100 g) | ModernaTX, Inc., DARPA | I | NCT03325075 |
| H10N8 (Influenza) | VAL-506440/mRNA-1440 (25 or 100 g) | ModernaTX, Inc. | I | NCT03076385 |
| H7N9 (Influenza) | VAL-339851/mRNA-1851 (10, 25 or 50 g) | ModernaTX, Inc. | I | NCT03345043 |
| Rabies | CV7202 (2 1 g) | CureVac AG | I | NCT03713086 |
| Heart failure | AZD8601 (3 mg or 30 mg) | AstraZeneca | II | NCT03370887 |
| Transthyretin-related (ATTR) familial amyloid polyneuropathy, cardiomyopathy, wild-type TTR cardiac amyloidosis | NTLA-2001 (0.1, 0.3, 0.7 or 1.0 mg/kg) | Intellia Therapeutics | I | NCT04601051 |
| TTR-mediated amyloidosis, amyloidosis, hereditary, transthyretin-related | ALN-TTR02 (siRNA dose: 0.3 mg/kg, every 3 weeks for 18 mo.) | Alnylam Pharmaceuticals | FDA-approved (Onpattro/patisiran) | NCT01960348 |
| Melanoma, colon, gastrointestinal, genitourinary or hepatocellular cancer | NCI-4650 (40, 130 or 390 g) | National Cancer Institute (NCI) | I/II | NCT03480152 |
| Melanoma | FixVac (BNT111) (14.4, 50 or 100 g) | BioNTech SE | I | NCT02410733 |
| Non-small-cell lung cancer (NSCLC) | mRNA-5671/V941 (dose unknown) | Merck Sharp & Dohme Corp. | I | NCT03948763 |
| Triple-negative breast cancer (TNBC) | IVAC_W_bre1_ulD or IVAC_M_ulD (dose unknown) | BioNTech SE, Seventh Framework Program | I/II | NCT02316457 |
| HPV-positive cancers (squamous cell carcinoma, head & neck neoplasm, cervical neoplasm, etc.) | HARE-40 (dose unknown) | University of Southampton, BioNTech SE | I/II | NCT03418480 |
| Ovarian cancer | W_ova1 Vaccine (50 g, followed by 100 g total) | University Medical Center Groningen, BioNTech SE | I | NCT04163094 |
| Solid tumors, cancer | MEDI1191 (dose unknown) | MedImmune LLC | I | NCT03946800 |
SARS-CoV-2 severe acute respiratory syndrome coronavirus-2, FDA United States Food and Drug Administration, EUA emergency use authorization, CMA conditional marketing authorization.