CURRENT STATUS
CFTR modulators (Trikafta) transforming cystic fibrosis care. CRISPR correction of CFTR mutations in development. AI-driven drug discovery for IPF and COPD advancing. mRNA therapies for lung delivery entering trials.
KEY BREAKTHROUGHS
Trikafta — CFTR modulator restoring ~90% of lung function in eligible CF patients
Inhaled mRNA therapy delivering CFTR to airway cells in Phase I trials
AI identifying novel anti-fibrotic targets for IPF from single-cell lung atlases
CRISPR correction of ΔF508 CFTR mutation in patient-derived organoids
AI-COMPRESSED PIPELINE
AI TOOLS ACCELERATING CURES
Lung Imaging AIInhaled mRNA DesignCRISPR Airway DeliveryFibrosis Prediction Models
KEY ORGANIZATIONS
Vertex PharmaceuticalsArctus TherapeuticsFlagship PioneeringBoehringer IngelheimCFF
TIMELINE ESTIMATE
CF gene cure: 3–5 years. IPF disease-modifying therapy: 4–6 years. COPD regenerative: 6–10 years.