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GTB-CF-CRISPR
- Development stage
- Preclinical
- Lead developer
- GeneToBe
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
01
Overview
GTB-CF-CRISPR is an experimental CRISPR-based gene editing therapy being developed by GeneToBe for the treatment of cystic fibrosis. The program focuses on correcting mutations within the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene, which encodes a protein responsible for regulating the flow of salt and fluids in and out of cells. By utilizing CRISPR technology to repair or bypass pathogenic CFTR variants, the therapy aims to restore functional protein expression and improve clinical outcomes for patients with cystic fibrosis. The program is currently in the late discovery stage.
- Other names
- Cystic fibrosis CRISPR program
02
Targets
CFTR (Cystic fibrosis transmembrane conductance regulator)
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