Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
CRISPR-Vertex cystic fibrosis program
- Development stage
- Preclinical
- Lead developer
- Vertex
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Inhalation
01
Overview
The CRISPR-Vertex cystic fibrosis program is a preclinical-stage gene-editing therapy being developed through a strategic collaboration between CRISPR Therapeutics and Vertex Pharmaceuticals. The program utilizes CRISPR/Cas9 technology to directly target and correct mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene within the lung epithelium. Unlike traditional CFTR modulators that attempt to improve the function of defective proteins, this approach aims to permanently repair the underlying genetic defect, potentially providing a one-time curative treatment for patients with cystic fibrosis, including those with mutations not addressed by current small-molecule therapies. The delivery mechanism typically involves lipid nanoparticles (LNPs) optimized for pulmonary administration.
- Other names
- CRISPR Therapeutics-Vertex CF programVertex-CRISPR cystic fibrosis collaborationCFTR gene editing program
02
Targets
CFTR (Cystic fibrosis transmembrane conductance regulator)
Beyond the preview
Go deeper on CRISPR-Vertex cystic fibrosis program.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on CRISPR-Vertex cystic fibrosis program.