Biomarker & Intervention Discovery

Cystic Fibrosis

This page lists 11 druggable gene targets, 42 approved or trial-tested drug candidates and 15 recent clinical trials for Cystic Fibrosis, with each agent ranked by the strength of its evidence (clinical, mechanistic or correlative). Candidates include existing drugs that could be repurposed. Sources are linked so you can check every claim.

Plain-language summary → New Treatments for Cystic Fibrosis (2026): Drugs in Trials

US DALYs744.9KWHO GHE 2021 · all ages
Global DALYs8.99MWHO GHE 2021 · worldwide
US mortality33.5K deaths/yrWHO GHE 2021
Global mortality333.9K deaths/yrWHO GHE 2021 · worldwide
NIH funding$86MFY2025 · RCDC estimate
Funding levelUnderfunded
Spontaneous remissionProgressive lung function decline; no spontaneous remission; historically median survival 30 years; now >50 years with…
Best-intervention remissionElexacaftor-tezacaftor-ivacaftor (ETI, Trikafta): FEV1 improvement 14-15 percentage points, sweat chloride…
Gap sizeNear-zero biological gap for CFTR-modulator-eligible patients (10-15% have non-eligible mutations); very large access…
Primary barrieraccess
Full remission profile →
Research Tracker Chronic Disease Interventions Cystic Fibrosis

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.

ANO1 200CFTR 222CTSC 159CXCR2 224DNASE1 7ELANE 207MUC5B 0SCNN1A 47SCNN1B 9SCNN1G 10SLC26A9 2

ANO1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CROFELEMER ↓ inhibits Mechanistic DGIdb
INS(3,4,5,6)P4 ↓ inhibits Mechanistic DGIdb
DCDPC ↓ inhibits Mechanistic DGIdb
PAM_16A ↑ activates Mechanistic DGIdb
MIBEFRADIL ↓ inhibits Mechanistic DGIdb
TMINH-23 ↓ inhibits Mechanistic DGIdb
NPPB ↓ inhibits Mechanistic DGIdb
NIFLUMIC ACID ↓ inhibits Mechanistic DGIdb
MONNA ↓ inhibits Mechanistic DGIdb
T16AINH-A01 ↓ inhibits Mechanistic DGIdb
TANNIC ACID ↓ inhibits Mechanistic DGIdb
SITS ↓ inhibits Mechanistic DGIdb
CA2+ ↑ activates Mechanistic DGIdb
ANI9 ↓ inhibits Mechanistic DGIdb
CACCINH-A01 ↓ inhibits Mechanistic DGIdb
EACT ↑ activates Mechanistic DGIdb
FLUOXETINE HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
FLUFENAMIC ACID ↓ inhibits Mechanistic DGIdb
DIDS ↓ inhibits Mechanistic DGIdb
9-ANTHROIC ACID ↓ inhibits Mechanistic DGIdb
CHEMBL2046972 ↓ inhibits Mechanistic 28700.0 nM (IC50) ChEMBL
CHEMBL2046973 ↓ inhibits Mechanistic 5900.0 nM (IC50) ChEMBL
CHEMBL2046974 ↓ inhibits Mechanistic 16300.0 nM (IC50) ChEMBL
CHEMBL2047075 ↓ inhibits Mechanistic 29200.0 nM (IC50) ChEMBL
CHEMBL2047076 ↓ inhibits Mechanistic 27000.0 nM (IC50) ChEMBL
CHEMBL2047078 ↓ inhibits Mechanistic 23500.0 nM (IC50) ChEMBL
CHEMBL2047079 ↓ inhibits Mechanistic 21200.0 nM (IC50) ChEMBL
CHEMBL2047080 ↓ inhibits Mechanistic 11200.0 nM (IC50) ChEMBL
CHEMBL2047081 ↓ inhibits Mechanistic 25900.0 nM (IC50) ChEMBL
CHEMBL2046450 ↓ inhibits Mechanistic 15600.0 nM (IC50) ChEMBL
CHEMBL2047082 ↓ inhibits Mechanistic 10800.0 nM (IC50) ChEMBL
CHEMBL2047083 ↓ inhibits Mechanistic 3300.0 nM (IC50) ChEMBL
CHEMBL2047084 ↓ inhibits Mechanistic 13300.0 nM (IC50) ChEMBL
CHEMBL2047085 ↓ inhibits Mechanistic 23000.0 nM (IC50) ChEMBL
CHEMBL2047086 ↓ inhibits Mechanistic 13100.0 nM (IC50) ChEMBL
CHEMBL2047087 ↓ inhibits Mechanistic 9000.0 nM (IC50) ChEMBL
CHEMBL2047088 ↓ inhibits Mechanistic 17300.0 nM (IC50) ChEMBL
CHEMBL2047089 ↓ inhibits Mechanistic 6300.0 nM (IC50) ChEMBL
CHEMBL2047090 ↓ inhibits Mechanistic 16300.0 nM (IC50) ChEMBL
CHEMBL2047091 ↓ inhibits Mechanistic 3800.0 nM (IC50) ChEMBL

Showing top 40 of 200 agents ranked by evidence tier.

CFTR

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
NPPB ? unclear Mechanistic DGIdb
LUMACAFTOR ~ modulates Mechanistic DGIdb
TEZACAFTOR ↑ activates Mechanistic DGIdb
SULFONAMIDE-01 ↑ activates Mechanistic DGIdb
SCRIPTAID ? unclear Mechanistic DGIdb
APIGENIN ↑ activates Mechanistic DGIdb
TEZACAFTOR AND IVACAFTOR ? unclear Mechanistic DGIdb
ROFLUMILAST ? unclear Mechanistic DGIdb
CROFELEMER ↓ inhibits Mechanistic DGIdb
UCCF-029 ↑ activates Mechanistic DGIdb
CURCUMIN ? unclear Mechanistic DGIdb
CAPSAICIN ↑ activates Mechanistic DGIdb
IVACAFTOR ↑ activates Mechanistic DGIdb
(R)-BPO-27 ↓ inhibits Mechanistic DGIdb
ICENTICAFTOR ↑ activates Mechanistic DGIdb
BAMOCAFTOR ? unclear Mechanistic DGIdb
UCCF-339 ↑ activates Mechanistic DGIdb
CFTRINH-172 ↓ inhibits Mechanistic DGIdb
NIMODIPINE ↑ activates Mechanistic DGIdb
CYSTEAMINE HYDROCHLORIDE ? unclear Mechanistic DGIdb
PHENYLGLYCINE-01 ↑ activates Mechanistic DGIdb
GLPG2737 ↑ activates Mechanistic DGIdb
GLYH-101 ↓ inhibits Mechanistic DGIdb
IOWH-032 ↓ inhibits Mechanistic DGIdb
OLACAFTOR ~ modulates Mechanistic DGIdb
ABBV-3221 ↑ activates Mechanistic DGIdb
DEUTIVACAFTOR ↑ activates Mechanistic DGIdb
NAVOCAFTOR ~ modulates Mechanistic DGIdb
CBIQ ↑ activates Mechanistic DGIdb
VITAMIN A COMPOUND ? unclear Mechanistic DGIdb
GATX1 ↓ inhibits Mechanistic DGIdb
GLPG-1837 ? unclear Mechanistic DGIdb
FELODIPINE ↑ activates Mechanistic DGIdb
ELEXACAFTOR ~ modulates Mechanistic DGIdb
ENTINOSTAT ? unclear Mechanistic DGIdb
VANZACAFTOR ↑ activates Mechanistic DGIdb
GLPG1837 ↑ activates Mechanistic DGIdb
GALICAFTOR ~ modulates Mechanistic DGIdb
NS004 ↑ activates Mechanistic DGIdb
GLPG-2737 ? unclear Mechanistic DGIdb

Showing top 40 of 222 agents ranked by evidence tier.

CTSC

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
BRENSOCATIB ↓ inhibits Mechanistic 2.512 nM (Kd) DGIdb
PROGESTERONE ? unclear Mechanistic DGIdb
ICATCXPZ-01 ↓ inhibits Mechanistic DGIdb
PENTOSAN POLYSULFATE ? unclear Mechanistic DGIdb
GSK2793660 ↓ inhibits Mechanistic DGIdb
COMPOUND 3D [PMID: 3941405] ↓ inhibits Mechanistic DGIdb
HSK31858 ↓ inhibits Mechanistic DGIdb
CALCIUM GLUCONATE ? unclear Mechanistic DGIdb
CHEMBL12954 ↓ inhibits Mechanistic 2700.0 nM (Ki) ChEMBL
CHEMBL212514 ↓ inhibits Mechanistic 9637.0 nM (IC50) ChEMBL
CHEMBL377768 ↓ inhibits Mechanistic 6296.0 nM (IC50) ChEMBL
CHEMBL212521 ↓ inhibits Mechanistic 13.0 nM (IC50) ChEMBL
CHEMBL209998 ↓ inhibits Mechanistic 96.0 nM (IC50) ChEMBL
CHEMBL211388 ↓ inhibits Mechanistic 137.0 nM (IC50) ChEMBL
CHEMBL212523 ↓ inhibits Mechanistic 822.0 nM (IC50) ChEMBL
CHEMBL439145 ↓ inhibits Mechanistic 172.0 nM (IC50) ChEMBL
CHEMBL379542 ↓ inhibits Mechanistic 4787.0 nM (IC50) ChEMBL
CHEMBL230473 ↓ inhibits Mechanistic 1727.0 nM (IC50) ChEMBL
CHEMBL230478 ↓ inhibits Mechanistic 191.0 nM (IC50) ChEMBL
CHEMBL390475 ↓ inhibits Mechanistic 403.0 nM (IC50) ChEMBL
CHEMBL230474 ↓ inhibits Mechanistic 29.0 nM (IC50) ChEMBL
CHEMBL390643 ↓ inhibits Mechanistic 606.0 nM (IC50) ChEMBL
CHEMBL390474 ↓ inhibits Mechanistic 18.0 nM (IC50) ChEMBL
CHEMBL448143 ↓ inhibits Mechanistic 51.0 nM (IC50) ChEMBL
CHEMBL516292 ↓ inhibits Mechanistic 14.0 nM (IC50) ChEMBL
E-64 ↓ inhibits Mechanistic 770.0 nM (IC50) ChEMBL
CHEMBL518284 ↓ inhibits Mechanistic 450.0 nM (IC50) ChEMBL
CHEMBL450997 ↓ inhibits Mechanistic 1.0 nM (IC50) ChEMBL
CHEMBL1162478 ↓ inhibits Mechanistic 790.0 nM (IC50) ChEMBL
CHEMBL461619 ↓ inhibits Mechanistic 10000.0 nM (Ki) ChEMBL
CHEMBL514348 ↓ inhibits Mechanistic 70.0 nM (IC50) ChEMBL
CHEMBL486232 ↓ inhibits Mechanistic 2600.0 nM (IC50) ChEMBL
CHEMBL567341 ↓ inhibits Mechanistic 34.67 nM (IC50) ChEMBL
CHEMBL578159 ↓ inhibits Mechanistic 6918.31 nM (IC50) ChEMBL
CHEMBL567134 ↓ inhibits Mechanistic 588.84 nM (IC50) ChEMBL
GRASSYSTATIN A ↓ inhibits Mechanistic 10000.0 nM (IC50) ChEMBL
CHEMBL1081329 ↓ inhibits Mechanistic 200.0 nM (IC50) ChEMBL
CHEMBL1082054 ↓ inhibits Mechanistic 630.0 nM (IC50) ChEMBL
CHEMBL1079350 ↓ inhibits Mechanistic 1580.0 nM (IC50) ChEMBL
CHEMBL1079351 ↓ inhibits Mechanistic 330.0 nM (IC50) ChEMBL

Showing top 40 of 159 agents ranked by evidence tier.

CXCR2

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CANOCTAKIN ? unclear Mechanistic DGIdb
[125I]CXCL8 (HUMAN) ↑ activates Mechanistic DGIdb
REPARIXIN ~ modulates Mechanistic DGIdb
CXCL5 ↑ activates Mechanistic DGIdb
MECHLORETHAMINE HYDROCHLORIDE ? unclear Mechanistic DGIdb
[125I]CXCL5 (HUMAN) ↑ activates Mechanistic DGIdb
SX-682 ↓ inhibits Mechanistic DGIdb
VIMNERIXIN ? unclear Mechanistic DGIdb
[125I]CXCL7 (HUMAN) ↑ activates Mechanistic DGIdb
VCXCL1 ↑ activates Mechanistic DGIdb
HIV-1 MATRIX PROTEIN P17 ↑ activates Mechanistic DGIdb
NAVARIXIN ↓ inhibits Mechanistic DGIdb
AZD5069 ↓ inhibits Mechanistic DGIdb
DANIRIXIN ↓ inhibits Mechanistic DGIdb
CXCL8 ↑ activates Mechanistic DGIdb
D-ALPHA-TOCOPHEROL ? unclear Mechanistic DGIdb
CXCL2 ↑ activates Mechanistic DGIdb
CYCLOPHOSPHAMIDE ANHYDROUS ? unclear Mechanistic DGIdb
GENISTEIN ? unclear Mechanistic DGIdb
AZD-5122 ? unclear Mechanistic DGIdb
CLOTRIMAZOLE ? unclear Mechanistic DGIdb
NSC 157449 ↓ inhibits Mechanistic DGIdb
IBUPROFEN, SODIUM SALT ? unclear Mechanistic DGIdb
[125I]CXCL1 (HUMAN) ↑ activates Mechanistic DGIdb
SX-517 ↓ inhibits Mechanistic DGIdb
SB 225002 ↓ inhibits Mechanistic DGIdb
CXCL6 ↑ activates Mechanistic DGIdb
CXCL3 ↑ activates Mechanistic DGIdb
CXCL1 ↑ activates Mechanistic DGIdb
ACETYLCYSTEINE ? unclear Mechanistic DGIdb
LADARIXIN ~ modulates Mechanistic DGIdb
BEVACIZUMAB ? unclear Mechanistic DGIdb
CXCL7 ↑ activates Mechanistic DGIdb
ELUBRIXIN ↓ inhibits Mechanistic DGIdb
NAVARIXIN ANHYDROUS ? unclear Mechanistic 2.6 nM (IC50) Open Targets
CHEMBL25573 ↓ inhibits Mechanistic 200.0 nM (IC50) ChEMBL
CHEMBL27863 ↓ inhibits Mechanistic 25.0 nM (IC50) ChEMBL
CHEMBL283736 ↓ inhibits Mechanistic 860.0 nM (IC50) ChEMBL
CHEMBL280711 ↓ inhibits Mechanistic 320.0 nM (IC50) ChEMBL
CHEMBL27446 ↓ inhibits Mechanistic 22.0 nM (IC50) ChEMBL

Showing top 40 of 224 agents ranked by evidence tier.

DNASE1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
GENTIAN VIOLET ↓ inhibits Mechanistic 350.0 nM (IC50) ChEMBL
CHEMBL3617197 ↓ inhibits Mechanistic 30200.0 nM (IC50) ChEMBL
CHEMBL3617198 ↓ inhibits Mechanistic 27900.0 nM (IC50) ChEMBL
CHEMBL3617201 ↓ inhibits Mechanistic 87000.0 nM (IC50) ChEMBL
CHEMBL3617205 ↓ inhibits Mechanistic 67600.0 nM (IC50) ChEMBL
CHEMBL3617282 ↓ inhibits Mechanistic 62600.0 nM (IC50) ChEMBL
CHEMBL3617285 ↓ inhibits Mechanistic 58900.0 nM (IC50) ChEMBL

ELANE

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
LONODELESTAT ? unclear Mechanistic DGIdb
POSTSTATIN ↓ inhibits Mechanistic DGIdb
SIVELESTAT SODIUM ? unclear Mechanistic DGIdb
EPIGALLOCATECHIN GALLATE ? unclear Mechanistic DGIdb
DEPELESTAT ? unclear Mechanistic DGIdb
CHF6333 ↓ inhibits Mechanistic DGIdb
COMPOUND 10L [PMID: 24556381] ↓ inhibits Mechanistic DGIdb
ALPHA 1-ANTITRYPSIN ↓ inhibits Mechanistic DGIdb
NICOTINE POLACRILEX ? unclear Mechanistic DGIdb
COMPOUND 10F [PMID: 24556381] ↓ inhibits Mechanistic DGIdb
TIPRELESTAT ? unclear Mechanistic DGIdb
BAY-678 ↓ inhibits Mechanistic DGIdb
SYMPLOSTATIN 5 ? unclear Mechanistic DGIdb
COMPOUND 4G [PMID: 22595175] ↓ inhibits Mechanistic DGIdb
NIFEDIPINE ? unclear Mechanistic DGIdb
ELAFIN ? unclear Mechanistic DGIdb
ALVELESTAT ↓ inhibits Mechanistic DGIdb
ERDOSTEINE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL310871 ? unclear Mechanistic DGIdb
.ALPHA.1-PROTEINASE INHIBITOR HUMAN ↓ inhibits Mechanistic DGIdb
SIVELESTAT ↓ inhibits Mechanistic DGIdb
CHEMBL314218 ↓ inhibits Mechanistic 0.03 nM (Ki) ChEMBL
CHEMBL274085 ↓ inhibits Mechanistic 2.7 nM (Ki) ChEMBL
CHEMBL329376 ↓ inhibits Mechanistic 31.0 nM (Ki) ChEMBL
CHEMBL543416 ↓ inhibits Mechanistic 0.014 nM (Ki) ChEMBL
CHEMBL51377 ↓ inhibits Mechanistic 102.0 nM (Ki) ChEMBL
CHEMBL432049 ↓ inhibits Mechanistic 0.17 nM (Ki) ChEMBL
CHEMBL89299 ↓ inhibits Mechanistic 8.0 nM (Ki) ChEMBL
CHEMBL95813 ↓ inhibits Mechanistic 300.0 nM (IC50) ChEMBL
CHEMBL330647 ↓ inhibits Mechanistic 25000.0 nM (IC50) ChEMBL
CHEMBL91944 ↓ inhibits Mechanistic 0.06 nM (Ki) ChEMBL
CHEMBL318052 ↓ inhibits Mechanistic 36000.0 nM (IC50) ChEMBL
CHEMBL319173 ↓ inhibits Mechanistic 1200.0 nM (IC50) ChEMBL
CHEMBL2368616 ↓ inhibits Mechanistic 0.31 nM (Ki) ChEMBL
CHEMBL554013 ↓ inhibits Mechanistic 1.73 nM (Ki) ChEMBL
CHEMBL90730 ↓ inhibits Mechanistic 26.0 nM (Ki) ChEMBL
CHEMBL421162 ↓ inhibits Mechanistic 1.0 nM (Ki) ChEMBL
CHEMBL88117 ↓ inhibits Mechanistic 8.0 nM (Ki) ChEMBL
CHEMBL542949 ↓ inhibits Mechanistic 0.16 nM (Ki) ChEMBL
CHEMBL99558 ↓ inhibits Mechanistic 3000.0 nM (IC50) ChEMBL

Showing top 40 of 207 agents ranked by evidence tier.

MUC5B

DGIdb Open Targets

Pipeline results not yet available for this target. Run the pipeline to populate.

SCNN1A

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
AMILORIDE HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
TRIAMTERENE ↓ inhibits Mechanistic DGIdb
552-02 ? unclear Mechanistic DGIdb
IDREVLORIDE ↓ inhibits Mechanistic DGIdb
AMILORIDE ? unclear Mechanistic 776.0 nM (IC50) DGIdb
COFIRASERSEN ? unclear Mechanistic Open Targets
CHEMBL211072 ↓ inhibits Mechanistic 42.0 nM (IC50) ChEMBL
CHEMBL380393 ↓ inhibits Mechanistic 73.0 nM (IC50) ChEMBL
CHEMBL208889 ↓ inhibits Mechanistic 31.0 nM (IC50) ChEMBL
CHEMBL211600 ↓ inhibits Mechanistic 17.0 nM (IC50) ChEMBL
CHEMBL379433 ↓ inhibits Mechanistic 20.0 nM (IC50) ChEMBL
CHEMBL209017 ↓ inhibits Mechanistic 48.0 nM (IC50) ChEMBL
CHEMBL211658 ↓ inhibits Mechanistic 28.0 nM (IC50) ChEMBL
CHEMBL212534 ↓ inhibits Mechanistic 26.0 nM (IC50) ChEMBL
CHEMBL212531 ↓ inhibits Mechanistic 7.0 nM (IC50) ChEMBL
CHEMBL209303 ↓ inhibits Mechanistic 9.0 nM (IC50) ChEMBL
552-02 FREE BASE ↓ inhibits Mechanistic 8.0 nM (IC50) ChEMBL
CHEMBL212431 ↓ inhibits Mechanistic 7.0 nM (IC50) ChEMBL
CHEMBL211161 ↓ inhibits Mechanistic 9.0 nM (IC50) ChEMBL
CHEMBL211773 ↓ inhibits Mechanistic 41.0 nM (IC50) ChEMBL
CHEMBL211946 ↓ inhibits Mechanistic 32.0 nM (IC50) ChEMBL
CHEMBL214239 ↓ inhibits Mechanistic 14.0 nM (IC50) ChEMBL
CHEMBL386222 ↓ inhibits Mechanistic 21.0 nM (IC50) ChEMBL
CHEMBL213175 ↓ inhibits Mechanistic 51.0 nM (IC50) ChEMBL
CHEMBL214220 ↓ inhibits Mechanistic 25.0 nM (IC50) ChEMBL
CHEMBL209104 ↓ inhibits Mechanistic 77.0 nM (IC50) ChEMBL
CHEMBL379756 ↓ inhibits Mechanistic 22.0 nM (IC50) ChEMBL
CHEMBL377986 ↓ inhibits Mechanistic 15.0 nM (IC50) ChEMBL
CHEMBL212410 ↓ inhibits Mechanistic 21.0 nM (IC50) ChEMBL
CHEMBL379066 ↓ inhibits Mechanistic 30.0 nM (IC50) ChEMBL
CHEMBL380011 ↓ inhibits Mechanistic 20.0 nM (IC50) ChEMBL
CHEMBL211221 ↓ inhibits Mechanistic 64.0 nM (IC50) ChEMBL
CHEMBL210876 ↓ inhibits Mechanistic 17.0 nM (IC50) ChEMBL
CHEMBL210967 ↓ inhibits Mechanistic 124.0 nM (IC50) ChEMBL
CHEMBL211793 ↓ inhibits Mechanistic 88.0 nM (IC50) ChEMBL
CHEMBL209977 ↓ inhibits Mechanistic 62.0 nM (IC50) ChEMBL
CHEMBL208620 ↓ inhibits Mechanistic 58.0 nM (IC50) ChEMBL
CHEMBL210866 ↓ inhibits Mechanistic 477.0 nM (IC50) ChEMBL
CHEMBL377239 ↓ inhibits Mechanistic 68.0 nM (IC50) ChEMBL
CHEMBL209659 ↓ inhibits Mechanistic 29.0 nM (IC50) ChEMBL

Showing top 40 of 47 agents ranked by evidence tier.

SCNN1B

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
FARGLITAZAR ? unclear Mechanistic DGIdb
ALDOSTERONE ? unclear Mechanistic DGIdb
IDREVLORIDE ↓ inhibits Mechanistic DGIdb
TRIAMTERENE ↓ inhibits Mechanistic DGIdb
AMILORIDE HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
552-02 ? unclear Mechanistic DGIdb
GLYBURIDE ? unclear Mechanistic DGIdb
AMILORIDE ? unclear Mechanistic DGIdb
COFIRASERSEN ? unclear Mechanistic Open Targets

SCNN1G

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
BUMETANIDE ? unclear Mechanistic DGIdb
TRIAMTERENE ↓ inhibits Mechanistic DGIdb
FUROSEMIDE ? unclear Mechanistic DGIdb
AMILORIDE HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
TORSEMIDE ? unclear Mechanistic DGIdb
AMILORIDE ? unclear Mechanistic DGIdb
IDREVLORIDE ↓ inhibits Mechanistic DGIdb
HYDROCHLOROTHIAZIDE ? unclear Mechanistic DGIdb
552-02 ? unclear Mechanistic DGIdb
COFIRASERSEN ? unclear Mechanistic Open Targets

SLC26A9

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
OLANZAPINE ? unclear Mechanistic DGIdb
IVACAFTOR ? unclear Mechanistic DGIdb

Clinical Trials

Drug, biologic and supplement trials (treatment or prevention) from ClinicalTrials.gov whose main condition is Cystic Fibrosis: recruiting, active or completed, most recently updated first. Data retrieved 2026-10-28.

A Phase 1 Relative Bioavailability Study of a VX-581 Tablet Formulation Active Not Recruiting
NCT07813039 Phase: 1 Started: 2026-09-23

The purpose of this study is to evaluate the relative bioavailability of VX-581 tablet formulation compared to a suspension formulation along with its safety and tolerability.

A Phase II Study of Ensifentrine in Non-Cystic Fibrosis Bronchiectasis Recruiting
NCT06559150 Phase: 2 Started: 2024-09-11

This study is a randomized, double-blind, placebo-controlled study designed to assess the efficacy and safety of ensifentrine inhalation suspension (3 mg) delivered twice daily via standard jet nebulizer up to 52 weeks, compared to placebo, in participants with non-cystic fibrosis bronchiectasis (NC...

A Study of 24 to 52 Weeks Treatment to Evaluate The Efficacy And Safety of Galvokimig in Adult Study Participants With Non-Cystic Fibrosis Bronchiectasis Recruiting
NCT07720375 Phase: 2 Started: 2026-08-05

The purpose of the study is to investigate the efficacy of galvokimig versus placebo on the time to the first pulmonary exacerbation in study participants with non-cystic fibrosis bronchiectasis (NCFB)

Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del Active Not Recruiting
NCT05331183 Phase: 3 Started: 2022-11-23

This study will evaluate the long-term safety, efficacy and pharmacodynamics of ELX/TEZ/IVA in participants with cystic fibrosis (CF) with at least 1 non-F508del ELX/TEZ/IVA-responsive CF transmembrane conductance regulator (CFTR) gene mutation.

Dose Range Finding, Efficacy, and Safety Study of Nebulized CSL787 in Adults With Non-cystic Fibrosis Bronchiectasis (NCFB) Recruiting
NCT07048262 Phase: 2 Started: 2025-09-03

This study is a proof-of-concept phase 2b, multicenter, randomized, double-blind, placebo-controlled, parallel-group, dose selection study designed to explore the efficacy, safety, and tolerability of 2 active treatment regimens of CSL787 (human normal immunoglobulin G \[IgG\] inhalation solution) c...

Evaluation of VX-581 in Healthy Participants and in Participants With Cystic Fibrosis Recruiting
NCT07283770 Phase: 1 Started: 2025-12-09

The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics of single dose of VX-581 and multiple ascending doses of VX-581 alone and in combination with tezacaftor (TEZ)/deutivacaftor (D-IVA) or D-IVA in healthy participants. The study will also evaluate the safety, tole...

Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of Age Completed
NCT05882357 Phase: 3 Started: 2023-06-27

The study evaluates the pharmacokinetics (PK), safety, tolerability, pharmacodynamics (PD), and efficacy of ELX/TEZ/IVA in CF participants 2 to less than (\<) 24 months of age.

A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy Active Not Recruiting
NCT05444257 Phase: 3 Started: 2022-11-08

The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in participants with cystic fibrosis.

A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF) Completed
NCT05668741 Phase: 1/2 Started: 2023-02-27

The purpose of this study is to evaluate the safety, and tolerability and efficacy of VX-522 in participants 18 years of age and older with cystic fibrosis and a cystic fibrosis transmembrane conductance regulator (CFTR) genotype not responsive to CFTR modulator therapy.

Bacteriophages for Adults With Cystic Fibrosis and Chronic Achromobacter Lung Infection Recruiting
NCT07275905 Phase: 1 Started: 2026-08-20

The goal of this clinical trial is to learn if a new treatment called AchromoPhage is safe and well tolerated in adults with cystic fibrosis (CF) who have long-term lung infections caused by Achromobacter bacteria. AchromoPhage is a mixture of four naturally occurring viruses, called phages, that ar...

Safety and Efficacy of Inhaled BMD003 (CFTR mRNA) in Chinese Cystic Fibrosis Patients Aged ≥12 Years Recruiting
NCT07485543 Phase: Early 1 Started: 2026-04-03

This is a prospective, single-center, open-label clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of inhaled BMD003 (CFTR mRNA) in Chinese patients aged 12 years and above with cystic fibrosis (CF) following multiple administrations. Additionally, the study aims...

A Prospective, Single-center, Open-label Study of Inhaled BMD003 (CFTR mRNA) in Chinese Adults With Cystic Fibrosis Active Not Recruiting
NCT06963008 Phase: Early 1 Started: 2025-06-27

The study uses a prospective, single-center, open-label clinical trial design to evaluate the safety and efficacy of inhaled BMD003 (CFTR mRNA) in adult Chinese patients with cystic fibrosis. This is a single-dose escalation study of nebulized inhaled BMD003 in patients with cystic fibrosis. The tri...

Azithromycin to Modify Bronchiectasis Exacerbation Risk Recruiting
NCT07608328 Phase: 4 Started: 2026-06-29

The Azithromycin to Modify Bronchiectasis Exacerbation Risk (AMBER) trial is a prospective, randomized, double-blind, placebo-controlled, parallel-group clinical trial in adults with clinically and radiologically confirmed non-cystic fibrosis bronchiectasis (NCFB). The trial evaluates whether azithr...

Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-272 in Healthy Participants Recruiting
NCT07437105 Phase: 1 Started: 2026-02-25

The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of single and multiple ascending doses of VX-272 in combination with deutivacaftor (D-IVA) with or without tezacaftor (TEZ) in healthy subjects.

SGLT2 Inhibitor Therapy in Cystic Fibrosis-related Diabetes Active Not Recruiting
NCT06149793 Phase: 2/3 Started: 2024-01-08

This will be a placebo-controlled, randomized, double blind, crossover study with a 4 week wash out period. Collect pilot data on the safety, tolerability, and feasibility of empagliflozin therapy in overweight/obese patients with CFRD to support a future larger randomized controlled trial.

Agents Found By Disease-Level Search

Searched directly from “Cystic Fibrosis” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 11-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).

42 agents shown (approved or with trial evidence) — 40 not found by the gene-target search above.

mannitolNOT in gene-target list

Approved · 4 trials · source: ClinicalTrials.gov, Open Targets
NCT00251056 NCT00251056 NCT00251056

IVANOT in gene-target list

· 3 trials · source: ClinicalTrials.gov
NCT05331183 NCT02951195 NCT04537793

EUR-1008 (APT-1008)NOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00297167 NCT00297167

ELX/TEZ/IVANOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT05331183 NCT04537793

Hyperpolarized Xe129NOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT06339593 NCT04624490

FDL169NOT in gene-target list

Phase 1 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT03424252 NCT02359357

spirometryNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT03839992 NCT05639556

elubrixin (SB656933)NOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00903201 NCT00903201

Ivacaftor

Approved · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT01705145 NCT03783286

interferon gamma-1bNOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00043316

Nasal brushingNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03894657

OrkambiNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03894657

PioglitazoneNOT in gene-target list

Phase 1 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00531882

SimvastatinNOT in gene-target list

Phase 1/2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00531882

IbuprofenNOT in gene-target list

Unknown · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00531882

tobramycinNOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00004829

SPI-1005 Ebselen 200mg Capsule x1NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02819856

SPI-1005 Ebselen 200mg Capsule x2NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02819856

SPI-1005 Ebselen 200mg Capsule x3NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02819856

AtbFinderNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05584982

Creon®NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02137382

Creon NNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02137382

Airway-clearance vestNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06339593

VX-152NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02951195

TEZ/IVANOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02951195

Autogenic DrainageNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07729787

Mechanical PercussionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07729787

Alpha1-Proteinase Inhibitor (Human)

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00486837

VX-121/TEZ/D-IVANOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05444257

Human recombinant growth hormoneNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00005112

PhototherapyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01822197

Docosahexaenoic acid administration: 50 mg/kg/day during 12 monthsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01783613

Placebo to Ciprofloxacin DPI (BAYQ3939)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02661438

ARINA-1NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05495243

LansoprazoleNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00458614

SymdekoNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03587961

mucoid exopolysaccharide P. aeruginosa immune globulin IVNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00004747

SimeoxNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06237348

FenretinideNOT in gene-target list

Phase 1 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT02141958

Tobacco Treatment Specialist (TTS) interventionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04777344

AmikacinNOT in gene-target list

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT02081963

alvelestat (AZD9668)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00757848

Generated by the Open Source Medicine Foundation Biomarker Pipeline — 2026-10-08. Data sources: DGIdb, Open Targets, ChEMBL, PubMed, ClinicalTrials.gov. Not medical advice.