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AUF1 mRNA LNP (local)
- Development stage
- Preclinical
- Lead developer
- Regerna Therapeutics
- Modality
- mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intramuscular
01
Overview
AUF1 mRNA LNP (local) is an mRNA-based therapeutic candidate being developed by Regerna Therapeutics for the treatment of muscular dystrophy and other muscle-wasting conditions. The therapy utilizes lipid nanoparticles (LNPs) to deliver mRNA encoding AU-rich element RNA-binding protein 1 (AUF1), a critical regulator of muscle regeneration. AUF1 controls the stability and translation of specific mRNAs involved in satellite cell activation and myofiber maturation; its deficiency is notably associated with limb-girdle muscular dystrophy. By restoring AUF1 levels locally via intramuscular injection, the therapy aims to accelerate muscle repair, enhance myofiber maturation, and reduce muscle atrophy. Preclinical studies have demonstrated that this approach can significantly reduce regeneration time following injury. The program is currently in the IND-enabling stage, with plans for clinical evaluation in single and multiple ascending dose (SAD/MAD) studies.
- Other names
- AUF1 LNP (Local Administration)AUF1 mRNA LNPAUF-1 mRNA LNPAUF 1 mRNA LNP
02
Targets
ARE (Adenylate-uridylate-rich element)
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