Dunno, but they are testing a few…
Here is an overview of forthcoming RNA-based drugs, focusing on mRNA, RNA interference (RNAi), and self-amplifying RNA (saRNA) therapies. The field is seeing rapid expansion into new areas like cancer, infectious diseases, and genetic disorders, driven by lessons from the success of COVID-19 mRNA vaccines.
Forthcoming mRNA drugs
Drug Name(s) Company(s) Target Condition(s) Status Notes
mRNA-1083 Moderna Combination flu and COVID vaccine Phase 3 Combines an influenza vaccine and a COVID-19 vaccine to potentially simplify annual immunization against both viruses.
mRNA-1010, mRNA-1018 Moderna Influenza (Seasonal and Pandemic) Phase 3 and 2, respectively Aims to provide more effective protection than traditional flu shots.
Melanoma Cancer Vaccines Moderna (mRNA-4157/V940), BioNTech (BNT111) Melanoma (advanced, resectable) Phase 2 and ongoing, respectively mRNA-4157 is an individualized vaccine designed for each patient, while BNT111 is a "fixed" vaccine targeting common melanoma antigens.
Universal Flu Vaccine Penn Medicine, in collaboration with other entities Influenza Preclinical to Clinical Trials An mRNA-based vaccine designed to protect against all 20 known subtypes of the influenza virus.
Gene Editing (CRISPR) Intellia Therapeutics, Beam Therapeutics, others Various Genetic Disorders Clinical Trials (e.g., Phase 1) Involves delivering mRNA encoding CRISPR-Cas9 components via lipid nanoparticles (LNPs) for in-body gene editing.
Protein Replacement Therapies Multiple companies Genetic Disorders (e.g., cystic fibrosis, ornithine transcarbamylase deficiency) Clinical Trials (early phase) Uses mRNA to produce proteins that are missing or defective in patients.
Forthcoming RNAi drugs (siRNA)
Drug Name(s) Company(s) Target Condition(s) Status Notes
Nedosiran Alnylam Primary hyperoxaluria (PH) Phase 3 A GalNAc-conjugated siRNA for the treatment of PH, a rare genetic disorder.
Cosdosiran, Tivanisiran, Fitusiran, Teprasiran Multiple companies Varied (e.g., genetic disorders, ocular conditions) Phase 3 Several siRNA candidates are progressing through late-stage clinical trials.
Di-siRNAs (Divalent siRNAs) Multiple companies CNS Disorders (e.g., Huntington's disease) Preclinical to Clinical Trials Di-siRNAs are a specialized form that links two siRNAs to allow for prolonged gene silencing, even after a single administration.
Forthcoming self-amplifying RNA (saRNA) drugs
Drug Name(s) Company(s) Target Condition(s) Status Notes
MTL-CEBPA MiNA Therapeutics Hepatocellular Carcinoma and other solid tumors Phase 1 The first saRNA drug in clinical trials. It upregulates the CEBPA transcription factor.
RAG-01 Ractigen Therapeutics Bladder Cancer Investigational New Drug (IND) submission A potential future saRNA treatment for non-muscle invasive bladder cancer.
Forthcoming circular RNA (circRNA) drugs
Drug Name(s) Company(s) Target Condition(s) Status Notes
Undisclosed Programs Multiple companies, including Sail Biomedicines, Merck, and Novo Nordisk Vaccines and Genetic Disorders Preclinical and Research Phase circRNA offers high stability and prolonged protein expression, making it a "next-generation" RNA therapy with significant investment.
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Edited on 30/08/2025 at 23:04:13.
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Almost 5 years since the first mRNA vaccine was approved for human use...[more]