Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
mRNA-3704
- Development stage
- Discontinued
- Lead developer
- Moderna
- Modality
- mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
mRNA-3704 is an investigational mRNA-based therapy developed to treat isolated methylmalonic acidemia (MMA) due to methylmalonyl-CoA mutase (MUT) deficiency. The drug consists of messenger RNA encoding the human MUT enzyme, encapsulated within Moderna’s proprietary lipid nanoparticle (LNP) delivery system. By delivering functional MUT mRNA, the therapy aims to restore production of the deficient mitochondrial enzyme in patients with MMA, thereby addressing the underlying cause of this rare and life-threatening metabolic disorder. Preclinical studies demonstrated that systemic administration enabled liver expression of functional hMUT and improved survival in mouse models. The drug is administered intravenously and was evaluated in a Phase 1/2 clinical trial for safety, pharmacodynamics, and pharmacokinetics in pediatric patients with MMA due to MUT deficiency[1][3][5][6].
- Other names
- modified mRNA encoding human methylmalonyl-CoA mutase (polymorphism at position 671)mRNA-3704 (hMUT-H671R)
02
Targets
MMUT (Methylmalonyl-coenzyme A mutase)
Beyond the preview
Go deeper on mRNA-3704.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on mRNA-3704.