Table 12. mRNA Therapeutic Products in Clinical Trials62a.
| mRNA drug name | CAS Registry Number | Disease indications | Description | Company |
|---|---|---|---|---|
| A-001; TriMix-MEL; ECL-006; E011-MEL | 2877674-59-2 | Melanoma | A mixture of three mRNAs encoding constitutively activated CLT4, CD40L, and TLR4 plus mRNAs for five melanoma-associated antigens (tyrosinase, gp100, MAGE-A3, MAGE-C2, and PRAME), which activate key immune cells against cancer | eTheRNA Immunotherapies |
| ARCT-810; LUNAR-OTC | 2877704-48-6 | Ornithine trans-carbamylase deficiency | mRNA encoding ornithine transcarbamylase formulated in a lipid nanoparticle to correct the enzyme deficiency | Arcturus Therapeutics |
| AZD-8601 | 2603440-18-0 | Heart failure and ischemic cardiovascular diseases | mRNA encoding vascular endothelial growth factor A to stimulate new vascular blood vessel formation and repair as well as regenerate heart cells | AstraZeneca |
| BD-111 | 2901016-63-3 | Herpetic viral keratitis | Viral-like particle drug delivery system used to transduce cas9 mRNA that directly targets and cuts the viral genome of herpes simplex virus 1 to effectively remove the virus | BD Gene |
| BNT-141 | 2877707-22-5 | Solid tumors | mRNA encoding a monoclonal antibody targeting claudin 18, a protein commonly expressed in multiple cancers | BioNTech |
| BNT-142 | 2877707-34-9 | Solid tumors | mRNA encoding a bispecific antibody targeting CD3, a protein involved in activation of certain types of T cells, and claudin 6 (CLDN6), a protein highly expressed in certain cancers | BioNTech |
| BNT-151 | 2877709-82-3 | Solid tumors | A nucleoside-modified, cationic lipoplexes-loaded mRNA encoding an interleukin-2 (IL-2) variant to stimulate anti-cancer T cells | BioNTech |
| BNT-152 | 2877709-92-5 | Solid tumors | A nucleoside-modified mRNA encoding interleukin-7 to stimulate anti-cancer T cells | BioNTech |
| BNT-153 | 2877709-93-6 | Solid tumors | A nucleoside-modified mRNA encoding interleukin 2 (IL-2) to stimulate anti-cancer T cells | BioNTech |
| LioCynx-M004; Lion TCR | 2901015-92-5 | Hepatitis B virus-related hepatocellular carcinoma | A genetically modified autologous cell therapy derived from T cells transfected with mRNA encoding to express a T-cell receptor that recognizes the hepatitis B surface antigen on the surface of HBV-related cancer cells | Lion TCR |
| MEDI-1191 | 2877712-03-1 | Solid tumors | LNP-encapsulated mRNA encoding IL-12 to increase intratumor production of IL-2 via intratumoral injection | Moderna |
| mRNA-2752 | 2878461-50-6 | Solid tumors | Lipid nanoparticle-encapsulated mRNAs encoding human T cell co-stimulator, OX40L, and proinflammatory cytokines, IL-23 and IL36γ, for intratumoral injection | Moderna |
| mRNA-3705 | 2878470-78-9 | Methylmalonic acidemia | Lipid nanoparticle-encapsulated mRNA encoding the mitochondrial enzyme methyl-CoA mutase that is deficient in methylmalonic acidemia | Moderna |
| mRNA-3745 | 2878574-58-2 | Glycogen storage disease type 1a | Lipid nanoparticle-encapsulated mRNA encoding glucose 6-phosphatase to restore the deficient enzyme responsible for converting glycogen into glucose for treatment of type 1a glycogen storage disease | Moderna |
| mRNA-3927 | 2878577-32-1 | Propionic acidemia | Lipid nanoparticle-encapsulated mRNAs encoding propionyl-CoA carboxylase α subunit and β subunit to restore the deficient enzyme and reduce toxic buildup of some substances in propionic acidemia | Moderna |
| mRNA-6231b | 2878577-39-8 | Autoimmune diseases | Lipid nanoparticle-encapsulated modified mRNA encoding a mutated form of human IL-2 fused to human serum albumin to increase its half-life to restore IL-2- and T-cell-mediated immune homeostasis | Moderna |
| MRT-5005b | 2328142-67-0 | Cystic fibrosis | Inhalable form of engineered mRNA variant encoding fully functional cystic fibrosis transmembrane conductance regulator protein for restoring lung function in cystic fibrosis; Phase I/II clinical trial (NCT03375047) showed no improvement in lung function but did discover that repeated inhaled doses of mRNA are safe63 | Translate Bio (acquired by Sanofi) |
| SAR-441000 | 2879301-17-2 | Solid tumors | Mixture of four mRNAs encoding IL-2 single chain, IL-15 fused to the sushi domain of IL-15Rα, GM-CSF, and interferon α2b, which have been reported as mediators of tumor regression | Sanofi, BioNTech |
| SQZ-eAPC-HPV | 2879306-51-9 | HPV and solid tumors | mRNA-based cell therapeutic agent that delivers five mRNAs for HPV16 protein antigens and immune-stimulating proteins, including CD86 and membrane bound IL-2 and IL-12, into four different types of engineered immune cells (monocytes, T-cells, B-cells, and NK cells) of cancer patients in a single step | SQZ Biotechnologies |
| UX053; LUNAR-GSDIII | 2901003-30-1 | Glycogen storage disease type III | Lipid nanoparticle-encapsulated mRNA therapeutic encoding the glycogen debranching enzyme; replacing the defective AGL gene product allows cells to break down glycogen using normal pathways | Ultragenyx, Arcturus Therapeutics |
| Verve-101 | 2894841-30-4 | Heterozygous familial hypercholesterolemia | mRNA-based lipid nanoparticle therapeutic that targets the liver and base-editing in the PCSK9 gene to disrupt PCSK9 protein production to lower LDL cholesterol and treat cardiovascular disease | Verve Therapeutics |
a
Data from the CAS Content Collection, Clinicaltrials.gov, and Pharmaprojects.
b
Drug no longer in development.