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Long-term extension study of mRNA-3705 in isolated methylmalonic acidemia (MUT deficiency)
2022-501997-20-00Phase 2Authorised
- Sponsor
- Moderna Inc
- Start
- Sep 2023
- Modality
- Gene Therapies, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Countries
- 6
01
Overview
Phase 2, open-label, single-arm trial of mRNA-3705 in Methylmalonic acidemia mutase type (MMA), sponsored by Moderna Inc. Primary endpoint: Incidence and severity of TEAEs, including study drug-related and not related TEAEs, AESIs, SAEs, and TEAEs leading to treatment discontinuation. Planned start Sep 2023; status: authorised.
02
Drugs
mRNA-3705
03
Conditions
Methylmalonic acidemia mutase type (MMA)
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