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vx-522
- Development stage
- Phase 2
- Lead developer
- Vertex
- Modality
- mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Inhalation
01
Overview
VX-522 is an investigational messenger RNA (mRNA) therapy being developed for the treatment of cystic fibrosis (CF), particularly in patients whose CFTR genotype does not respond to current CFTR modulator therapies. The drug consists of a CFTR mRNA encapsulated in a lipid nanoparticle and is administered via inhalation using a nebulizer. Once delivered to lung cells, the mRNA provides instructions for producing functional copies of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This approach aims to address the underlying cause of CF by restoring proper function to defective or missing CFTR proteins in affected individuals[1][4][5][6][8]. The drug is co-developed by Vertex Pharmaceuticals and Moderna.
- Other names
- vx-522vx522vx 522mRNA-3692mRNA3692mRNA 3692VXc-0522VXc0522VXc 0522VXc-522VXc522VXc 522
02
Targets
CFTR (Cystic fibrosis transmembrane conductance regulator)
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