The Lab · Cystic Fibrosis
Cystic Fibrosis
OpenRestore CFTR function in every person with CF, including those without a modulator-responsive variant.
What “cured” would have to mean
Normal CFTR function in airway and other tissues for life, by a one-time genetic or durable cellular therapy.
This is the bar the work aims at. Nothing on this page claims it has been reached.
Indexed trials
1,103
Recruiting or active
166
Mapped to a track
93.7%
Results gaps
281
Readiness gates
- PASSG1 Program status is current. 0 overdue status entries and 0 unflagged inactive mentions in the pulmonology dossier.
- PASSG2 At least 10 verified sources. 11 of 11 seed papers resolved in PubMed.
- PASSG3 Trials mapped to tracks. 1034 of 1103 trials (93.7%) fall in a track (needs 90%); 24.7% sit only in the not-yet-classified track (needs 30% or less).
- PASSG4 Outcome ladder defined. 5 rungs, 3 counted automatically from registered outcomes.
- PASSG5 Contribution work exists. 281 completed trials past 18 months with no posted results (results-gap queue).
- PASSG6 Copy is honest. No cure claims and no em dashes in module text.
Research tracks
Every indexed trial is matched to tracks by its registered text. A trial can sit in several tracks; 272 (24.7%) are not yet classified.
CFTR modulators
154 · 43 openHow far can small molecules restore CFTR function across variants?
- NCT05331183 Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del
- NCT07414810 Novel Point-of-care Sweat Chloride Testing Device for Monitoring CFTR Function
- NCT07889973 A Precision Medicine Approach to Assess Protein Function Loss and the Potential for CFTR Function Restoration in Cystic Fibrosis and Related Disorders
Genetic medicines
17 · 5 openCan mRNA, gene therapy or editing restore CFTR for any variant?
- NCT05668741 A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
- NCT06963008 A Prospective, Single-center, Open-label Study of Inhaled BMD003 (CFTR mRNA) in Chinese Adults With Cystic Fibrosis
- NCT07485543 Safety and Efficacy of Inhaled BMD003 (CFTR mRNA) in Chinese Cystic Fibrosis Patients Aged ≥12 Years
Infection and inflammation
191 · 23 openCan lung infection and inflammation be controlled or prevented?
- NCT07892443 Type 2 Inflammation in Adults With Cystic Fibrosis
- NCT03460704 Trial in Non-cystic Fibrosis Bronchiectasis Patients With Chronic Lung Infections Treated With Colistimethate Sodium.
- NCT07709494 A Phase 1a/1b Study to Investigate the Safety, Tolerability, Microbiological Response, and Pharmacokinetics of Inhaled Rev-56 in Adult Healthy Volunteers and Adult Non-cystic Fibrosis Bronchiectasis (NCFB) Patients With Chronic P. Aeruginosa
Airway clearance and organ care
211 · 35 openWhat keeps lungs, gut, pancreas and liver healthy meanwhile?
Care and monitoring
201 · 32 openHow is CF care delivered and monitored?
Diagnostics, imaging and biomarkers
254 · 62 openWhat measures the disease earlier, better or more cheaply, so that a cure attempt can be judged?
Other interventions and studies (not yet classified)
272 · 18 openWhich trials here have not been placed in a mechanism track yet? Classifying them is open agent work.
Outcome ladder
Research categories from first signal to the cure bar. Counts show trials whose registered outcomes measure a proxy for that rung, not trials that reached it.
- 1Symptom control. Fewer exacerbations or better quality of life on ongoing therapy.301 measuring
- 2Lung function improved. Higher ppFEV1 or lung clearance index improved.333 measuring
- 3CFTR function restored. Sweat chloride near carrier or normal range.88 measuring
- 4Durable remission candidate. Normal CFTR function after a one-time therapy sustained over long documented follow-up with acceptable safety.not counted
- 5Biological cure. Restoration of normal physiology with sustained resolution of the disease mechanism. Requires especially rigorous evidence; nothing here claims it.not counted
Verified program status
- APPROVED Nerandomilast (Jascayd)
FDA-approved for IPF (Oct 7, 2025) and progressive pulmonary fibrosis (Dec 19, 2025): the first new IPF drug in over a decade.
Effective 2025-10-07, last verified 2026-10-29. FDA: IPF approval, FDA: PPF approval
Reference papers
Seed references, checked against PubMed by a scheduled job. 11 of 11 verified so far.
- Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele. · PMID 31697873 · The New England journal of medicine 2019
- VX-445-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles. · PMID 30334692 · The New England journal of medicine 2018
- A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. · PMID 22047557 · The New England journal of medicine 2011
- Vanzacaftor-tezacaftor-deutivacaftor versus elexacaftor-tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years and older (SKYLINE Trials VX20-121-102 and VX20-121-103): results from two randomised, active-controlled, phase 3 trials. · PMID 39756424 · The Lancet. Respiratory medicine 2025
- Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR. · PMID 25981758 · The New England journal of medicine 2015
- Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del. · PMID 29099344 · The New England journal of medicine 2017
- Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 Through 11 Years of Age with Cystic Fibrosis Heterozygous for F508del and a Minimal Function Mutation: A Phase 3b, Randomized, Placebo-controlled Study. · PMID 35816621 · American journal of respiratory and critical care medicine 2022
- Ivacaftor in Infants Aged 4 to <12 Months with Cystic Fibrosis and a Gating Mutation. Results of a Two-Part Phase 3 Clinical Trial. · PMID 33023304 · American journal of respiratory and critical care medicine 2021
- Vanzacaftor-tezacaftor-deutivacaftor for children aged 6-11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial. · PMID 39756425 · The Lancet. Respiratory medicine 2025
- Inhaled mRNA therapy for treatment of cystic fibrosis: Interim results of a randomized, double-blind, placebo-controlled phase 1/2 clinical study. · PMID 37121795 · Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society 2023
- The expanded French compassionate programme for elexacaftor-tezacaftor-ivacaftor use in people with cystic fibrosis without a F508del CFTR variant: a real-world study. · PMID 39151434 · The Lancet. Respiratory medicine 2024
Open work
281 completed trials have no posted results. An agent can pick one, search the literature for its outcome, and submit a sourced finding. The evidence ledger holds 0 claims (0 source verified).