Remission & chronicity

Spontaneous remission

Progressive lung function decline; no spontaneous remission; historically median survival 30 years; now >50 years with CFTR modulators for eligible genotypes

Best-intervention remission

Elexacaftor-tezacaftor-ivacaftor (ETI, Trikafta): FEV1 improvement 14-15 percentage points, sweat chloride normalisation in F508del homozygous patients (AURORA trial); 80-90% of CF patients in US have eligible genotypes; 'functional cure' in many patients — near-normal lung function in those starting young

Gap size

Near-zero biological gap for CFTR-modulator-eligible patients (10-15% have non-eligible mutations); very large access gap globally

Primary barrier

access

Trikafta is among the most expensive drugs ever approved (~$330K/year); most middle-income countries have no access; 10-15% of CF patients (class I nonsense mutations) are not eligible for CFTR modulators and remain without disease-modifying therapy; expanded mRNA/gene therapy approach needed for ineligible patients

Biomarker Targets & Therapeutic Agents

Gene targets queried against DGIdb, Open Targets, ChEMBL, PubMed, and Europe PMC. Agents ranked: ● Clinical > ● Mechanistic > ● Correlative.

Pipeline results not yet available. Run: python -m biomarker_pipeline.run_for_diseases --skip-llm

Clinical Trials

Recruiting and recently completed trials from ClinicalTrials.gov. Data retrieved 2026-07-06.

No recent clinical trials found on ClinicalTrials.gov for this condition.

Agents Found By Disease-Level Search

Searched directly from “Cystic Fibrosis” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 0-gene target panel above. This surfaces agents whose mechanism doesn't route through a curated gene (combination therapies, standard-of-care drugs, targets outside the panel).

Not yet run. python -m biomarker_pipeline.run_disease_agent_discovery --disease "..."

Cystic Fibrosis

Remission & chronicity

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