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DNAH5 mRNA therapy
- Development stage
- Preclinical
- Lead developer
- ReCode Therapeutics
- Modality
- mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Inhalation
01
Overview
DNAH5 mRNA therapy is an investigational inhaled mRNA-based treatment being developed by ReCode Therapeutics for the treatment of primary ciliary dyskinesia (PCD). The program specifically targets PCD caused by mutations in the DNAH5 gene, which encodes the dynein axonemal heavy chain 5 protein, a vital component of the outer dynein arms required for ciliary movement. The therapy utilizes ReCode's proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform to deliver functional DNAH5 mRNA directly to the respiratory epithelium via inhalation. Once delivered, the mRNA is translated into functional protein, aiming to restore ciliary motility and improve mucociliary clearance in the lungs. The program is currently in the discovery phase of development.
- Other names
- PCD DNAH5 program
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