Drug intelligence / Profile preview

MRT5410

Development stage
Unknown
Lead developer
Sanofi Pasteur
Modality
mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Inhalation
01

Overview

MRT5410 is an inhaled messenger RNA (mRNA) therapeutic candidate designed for the treatment of cystic fibrosis (CF). Developed by Translate Bio (now part of Sanofi), it utilizes a lipid nanoparticle (LNP) delivery system to transport functional CFTR mRNA directly to the lung epithelial cells. Once inside the cells, the mRNA is translated into functional CFTR protein, which restores chloride ion transport across the cell membrane, addressing the underlying cause of the disease regardless of the patient's specific genetic mutation. MRT5410 is a next-generation candidate following MRT5005, optimized for improved potency and delivery efficiency.

Other names
CFTR mRNA therapy
02

Targets

CFTR (Cystic fibrosis transmembrane conductance regulator)

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