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RCT1100
- Development stage
- Phase 1
- Lead developer
- ReCode Therapeutics
- Modality
- mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Inhalation
01
Overview
RCT1100 is an investigational, disease-modifying mRNA-based therapeutic developed for the treatment of primary ciliary dyskinesia (PCD) caused by pathogenic mutations in the DNAI1 gene. The therapy consists of DNAI1 mRNA packaged within lipid nanoparticles (LNPs) and is administered via inhalation using a nebulizer system. By delivering functional DNAI1 mRNA directly to airway cells, RCT1100 aims to restore production of the Dynein axonemal intermediate chain 1 protein (DNAI1), which is essential for normal ciliary movement and mucus clearance in the respiratory tract. This approach targets a critical unmet need, as there are currently no approved disease-modifying treatments or cures for PCD[1][2][4][5][6][7][8].
- Other names
- DNAI1 mRNADNAI-1 mRNADNAI 1 mRNAinhaled mRNA-based therapy for primary ciliary dyskinesiamRNA-based genetic medicine for primary ciliary dyskinesia
02
Targets
DNAI1 (Dynein axonemal intermediate chain 1)
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