Biomarker & Intervention Discovery

Asthma

Pharmacologically actionable gene targets, therapeutic agents ranked by evidence tier, and active clinical trials.

US DALYs1.44MWHO GHE 2021 · all ages
Global DALYs21.99MWHO GHE 2021 · worldwide
US mortality3,646 deaths/yrWHO GHE 2021
Global mortality425.7K deaths/yrWHO GHE 2021 · worldwide
NIH funding$242MFY2025 · RCDC estimate
Funding levelUnderfunded
Spontaneous remission26% of childhood-onset asthma in clinical remission by adulthood; <20% adult-onset achieves sustained remission; 65%…
Best-intervention remissionBiologics (anti-IL-5, anti-IL-4/13, anti-IgE) achieve near-zero exacerbation rates in eligible severe patients…
Gap sizeModerate; highly severity-dependent; large practice gap for AIT
Primary barrierbiological (severe phenotype) + practice (AIT underutilized)
Full remission profile →

Remission & chronicity

Spontaneous remission
26% of childhood-onset asthma in clinical remission by adulthood; <20% adult-onset achieves sustained remission; 65% long-term remission in mild-moderate vs 16% in severe
Best-intervention remission
Biologics (anti-IL-5, anti-IL-4/13, anti-IgE) achieve near-zero exacerbation rates in eligible severe patients; allergen immunotherapy (AIT) achieves sustained drug-free remission in allergic asthma in ~30-40% of treated patients
Gap size
Moderate; highly severity-dependent; large practice gap for AIT
Primary barrier
biological (severe phenotype) + practice (AIT underutilized)
Allergen immunotherapy is the only disease-modifying treatment for allergic asthma and is underused relative to guidelines; biological phenotyping (T2-high vs T2-low) drives outcomes more than treatment intensity alone
Biomarker Targets & Therapeutic Agents

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

ADRB2 148ALOX5 181CYSLTR1 189FCER1A 17GATA3 19IL13 11IL13RA1 2IL1RL1 1IL33 6IL4 4IL4R 6IL5 9IL5RA 1MS4A2 31TSLP 2
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CARVEDILOL inhibits Mechanistic
CHEMBL:CHEMBL1257092 ? unclear Mechanistic
SODIUM CHLORIDE ? unclear Mechanistic
PENBUTOLOL SULFATE inhibits Mechanistic
DESIPRAMINE ? unclear Mechanistic
METIPRANOLOL ? unclear Mechanistic
CHEMBL:CHEMBL531322 ? unclear Mechanistic
NADOLOL inhibits Mechanistic
PIRBUTEROL ? unclear Mechanistic
FENOTEROL activates Mechanistic
TIMOLOL inhibits Mechanistic
ABEDITEROL activates Mechanistic
PINDOLOL activates Mechanistic
BRONCHODILATOR ? unclear Mechanistic
TULOBUTEROL activates Mechanistic
PHENYLEPHRINE HYDROCHLORIDE ? unclear Mechanistic
GSK159802 activates Mechanistic
PRILOCAINE HYDROCHLORIDE ? unclear Mechanistic
CLENBUTEROL activates Mechanistic
PROPRANOLOL HYDROCHLORIDE inhibits Mechanistic
INDACATEROL MALEATE activates Mechanistic
VILANTEROL ? unclear Mechanistic
TERTATOLOL ? unclear Mechanistic
ARFORMOTEROL activates Mechanistic
INDACATEROL ? unclear Mechanistic
FORMOTEROL FUMARATE activates Mechanistic
CHEMBL:CHEMBL111201 ? unclear Mechanistic
RITODRINE ? unclear Mechanistic
RISPERIDONE ? unclear Mechanistic
CHEMBL:CHEMBL599896 ? unclear Mechanistic
VILANTEROL TRIFENATATE activates Mechanistic
RITODRINE HYDROCHLORIDE activates Mechanistic
DISULFIRAM ? unclear Mechanistic
BUCINDOLOL HYDROCHLORIDE inhibits Mechanistic
LEVALBUTEROL TARTRATE activates Mechanistic
LAS190792 activates Mechanistic
ALPRENOLOL ? unclear Mechanistic
CELIPROLOL activates Mechanistic
ALBUTEROL SULFATE activates Mechanistic
CARTEOLOL ? unclear Mechanistic

Showing top 40 of 148 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
BALSALAZIDE ? unclear Mechanistic
MASOPROCOL ? unclear Mechanistic
DARBUFELONE ? unclear Mechanistic
MECLOFENAMATE ? unclear Mechanistic
CGP 47656 activates Mechanistic
TEPOXALIN inhibits Mechanistic
MESALAMINE inhibits Mechanistic
CHEMBL:CHEMBL416038 ? unclear Mechanistic
PF-4191834 inhibits Mechanistic
PTC299 ? unclear Mechanistic
MELATONIN ? unclear Mechanistic
MECLOFENAMATE SODIUM inhibits Mechanistic
THERAPEUTIC GLUCOCORTICOID ? unclear Mechanistic
MONTELUKAST SODIUM ? unclear Mechanistic
BALSALAZIDE DISODIUM inhibits Mechanistic
LESOGABERAN activates Mechanistic
ATRELEUTON ? unclear Mechanistic
RILOPIROX ? unclear Mechanistic
6BIO ? unclear Mechanistic
ZILEUTON inhibits Mechanistic 3300.0 nM (IC50)
CHEMBL:CHEMBL502496 ? unclear Mechanistic
TENIDAP ? unclear Mechanistic
SULFASALAZINE inhibits Mechanistic
DIETHYLCARBAMAZINE ? unclear Mechanistic
SELENIUM ? unclear Mechanistic
OLSALAZINE SODIUM inhibits Mechanistic
ACACETIN ? unclear Mechanistic
LICOFELONE ? unclear Mechanistic
TIPELUKAST ? unclear Mechanistic
5-OXO-ETE ? unclear Mechanistic
METHOTREXATE ? unclear Mechanistic
SILYMARIN ? unclear Mechanistic
ANACARDIC ACID ? unclear Mechanistic
MECLOFENAMIC ACID ? unclear Mechanistic
OLSALAZINE ? unclear Mechanistic
CHEMBL26083 inhibits Mechanistic 2500.0 nM (IC50)
CHEMBL25790 inhibits Mechanistic 6000.0 nM (IC50)
CHEMBL38172 inhibits Mechanistic 90.0 nM (IC50)
CHEMBL444315 inhibits Mechanistic 30.0 nM (IC50)
CHEMBL29874 inhibits Mechanistic 39000.0 nM (IC50)

Showing top 40 of 181 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
PRANLUKAST inhibits Mechanistic 0.8 nM (IC50)
NEDOCROMIL ? unclear Mechanistic
CR-3465 inhibits Mechanistic
CINALUKAST ? unclear Mechanistic
CHEMBL:CHEMBL314115 ? unclear Mechanistic
RECOMBINANT TRANSFORMING GROWTH FACTOR-BETA 1 ? unclear Mechanistic
MASILUKAST ? unclear Mechanistic
LY-2300559 ? unclear Mechanistic
ZAFIRLUKAST inhibits Mechanistic 1.731 nM (IC50)
MONTELUKAST SODIUM inhibits Mechanistic
IRALUKAST ? unclear Mechanistic
MONTELUKAST ? unclear Mechanistic 0.5 nM (IC50)
SR-2640 inhibits Mechanistic 23.0 nM (IC50)
CHEMBL89340 inhibits Mechanistic 3.1 nM (IC50)
ABLUKAST inhibits Mechanistic 4000.0 nM (IC50)
FPL-55712 inhibits Mechanistic 4000.0 nM (IC50)
CHEMBL89768 inhibits Mechanistic 3.1 nM (IC50)
VERLUKAST inhibits Mechanistic 8.0 nM (IC50)
POBILUKAST inhibits Mechanistic 10.0 nM (Ki)
CHEMBL97356 inhibits Mechanistic 11.0 nM (Ki)
CHEMBL313177 inhibits Mechanistic 6.42 nM (IC50)
CHEMBL316170 inhibits Mechanistic 1.06 nM (IC50)
CHEMBL82790 inhibits Mechanistic 4.7 nM (IC50)
CHEMBL84707 inhibits Mechanistic 1.91 nM (IC50)
CHEMBL86264 inhibits Mechanistic 8.49 nM (IC50)
CHEMBL87528 inhibits Mechanistic 3.36 nM (IC50)
CHEMBL314340 inhibits Mechanistic 1.16 nM (IC50)
CHEMBL314237 inhibits Mechanistic 0.76 nM (IC50)
CHEMBL83233 inhibits Mechanistic 15.7 nM (IC50)
CHEMBL86063 inhibits Mechanistic 0.72 nM (IC50)
CHEMBL86302 inhibits Mechanistic 1.63 nM (IC50)
CHEMBL313195 inhibits Mechanistic 0.47 nM (IC50)
CHEMBL85572 inhibits Mechanistic 2.43 nM (IC50)
CHEMBL85633 inhibits Mechanistic 2.01 nM (IC50)
CHEMBL84841 inhibits Mechanistic 8.67 nM (IC50)
CHEMBL313187 inhibits Mechanistic 0.38 nM (IC50)
CHEMBL414885 inhibits Mechanistic 0.65 nM (IC50)
CHEMBL87696 inhibits Mechanistic 2.16 nM (IC50)
CHEMBL87337 inhibits Mechanistic 0.55 nM (IC50)
CHEMBL315973 inhibits Mechanistic 1.05 nM (IC50)

Showing top 40 of 189 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
BENZYLPENICILLOYL POLYLYSINE ? unclear Mechanistic
COMPOUND 27D [PMID: 28231433] inhibits Mechanistic
OPAGANIB inhibits Mechanistic
PF-543 inhibits Mechanistic
SKI II inhibits Mechanistic
ROME inhibits Mechanistic
MP-A08 inhibits Mechanistic
COMPOUND 55 [PMID: 30889352] inhibits Mechanistic
COMPOUND 49 [PMID: 30889352] inhibits Mechanistic
OMALIZUMAB ? unclear Mechanistic
SLC4101431 inhibits Mechanistic
COMPOUND 60 [PMID: 30889352] inhibits Mechanistic
SLM6071469 inhibits Mechanistic
COMPOUND 59 [PMID: 30889352] inhibits Mechanistic
CHEMBL275575 inhibits Mechanistic 71000.0 nM (IC50)
CHEMBL412391 inhibits Mechanistic 25000.0 nM (IC50)
CHEMBL267118 inhibits Mechanistic 82000.0 nM (IC50)
Therapeutic Agent Effect Direction Evidence Tier Potency Source
PREDNISOLONE ? unclear Mechanistic
DEXAMETHASONE ? unclear Mechanistic
METHOTREXATE ? unclear Mechanistic
ASPARAGINASE ? unclear Mechanistic
RIBAVIRIN inhibits Mechanistic
MYCOPHENOLATE MOFETIL inhibits Mechanistic
PEGASPARGASE ? unclear Mechanistic
MYCOPHENOLATE inhibits Mechanistic
CYCLOPHOSPHAMIDE ANHYDROUS ? unclear Mechanistic
VINCRISTINE ? unclear Mechanistic
CYTARABINE ? unclear Mechanistic
PREDNISONE ? unclear Mechanistic
LEUCOVORIN CALCIUM ? unclear Mechanistic
DOXORUBICIN HYDROCHLORIDE ? unclear Mechanistic
THIOGUANINE inhibits Mechanistic
MERIMEPODIB inhibits Mechanistic
GLUCARPIDASE ? unclear Mechanistic
DAUNORUBICIN LIPOSOMAL ? unclear Mechanistic
MERCAPTOPURINE ? unclear Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
DECTREKUMAB ? unclear Mechanistic
TRALOKINUMAB inhibits Mechanistic
QAX-576 inhibits Mechanistic
ROMILKIMAB inhibits Mechanistic
CENDAKIMAB inhibits Mechanistic
ADALIMUMAB ? unclear Mechanistic
TNX-650 inhibits Mechanistic
USTEKINUMAB ? unclear Mechanistic
LEBRIKIZUMAB inhibits Mechanistic
CNTO-5825 inhibits Mechanistic
ANRUKINZUMAB inhibits Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CINTREDEKIN BESUDOTOX ~ modulates Mechanistic
EBLASAKIMAB ? unclear Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
ASTEGOLIMAB inhibits Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
ASTEGOLIMAB ? unclear Mechanistic
TORUDOKIMAB inhibits Mechanistic
ETOKIMAB ~ modulates Mechanistic
ITEPEKIMAB inhibits Mechanistic
CHEMBL5270750 inhibits Mechanistic 43000.0 nM (IC50)
CHEMBL5276831 inhibits Mechanistic 42000.0 nM (IC50)
Therapeutic Agent Effect Direction Evidence Tier Potency Source
PASCOLIZUMAB inhibits Mechanistic
TEPOXALIN ? unclear Mechanistic
ROMILKIMAB inhibits Mechanistic
ASPIRIN ? unclear Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
SUNITINIB ? unclear Mechanistic
CINTREDEKIN BESUDOTOX ~ modulates Mechanistic
PITRAKINRA ? unclear Mechanistic
DUPILUMAB inhibits Mechanistic
BIZAXOFUSP ? unclear Mechanistic
ROMILKIMAB ? unclear Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
METHYLPREDNISOLONE ? unclear Mechanistic
ADENOVIRUS VECTOR ? unclear Mechanistic
BENRALIZUMAB ? unclear Mechanistic
RESLIZUMAB inhibits Mechanistic
DEPEMOKIMAB inhibits Mechanistic
CYCLOPHOSPHAMIDE ANHYDROUS ? unclear Mechanistic
INTERLEUKIN-3 ? unclear Mechanistic
CYCLOSPORINE ? unclear Mechanistic
MEPOLIZUMAB inhibits Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
BENRALIZUMAB inhibits Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
TIRBANIBULIN inhibits Mechanistic
CCT196969 inhibits Mechanistic
N-(5-METHYL-1H-PYRAZOL-3-YL)-2-PHENYLQUINAZOLIN-4-AMINE inhibits Mechanistic
KX2-361 inhibits Mechanistic
(3Z)-N,N-DIMETHYL-2-OXO-3-(4,5,6,7-TETRAHYDRO-1H-INDOL-2-YLMETHYLIDENE)-2,3-DIHYDRO-1H-INDOLE-5-SULFONAMIDE inhibits Mechanistic
PD166285 inhibits Mechanistic
COMPOUND 36 [PMID: 21958547] inhibits Mechanistic
GEMCITABINE ? unclear Mechanistic
COMPOUND 25 [PMID: 31260299] inhibits Mechanistic
ELZOVANTINIB inhibits Mechanistic
SYK INHIBITOR III inhibits Mechanistic
COMPOUND 8H [PMID: 22765894] inhibits Mechanistic
MAX-40279 inhibits Mechanistic
ACALABRUTINIB inhibits Mechanistic
SI306 inhibits Mechanistic
COMPOUND 19A [PMID: 30503936] inhibits Mechanistic
NG-25 inhibits Mechanistic
DASATINIB ANHYDROUS inhibits Mechanistic
ECF506 inhibits Mechanistic
CCT241161 inhibits Mechanistic
COMPOUND 2 [PMID: 15546730] inhibits Mechanistic
PP-121 inhibits Mechanistic
SARACATINIB inhibits Mechanistic
BOSUTINIB inhibits Mechanistic
DORSOMORPHIN inhibits Mechanistic
BALAMAPIMOD inhibits Mechanistic
IBRUTINIB inhibits Mechanistic
TAK-020 inhibits Mechanistic
ENMD-2076 inhibits Mechanistic
WH-4-023 inhibits Mechanistic
OMALIZUMAB ? unclear Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
TEZEPELUMAB inhibits Mechanistic
AMG-157 inhibits Mechanistic

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 “Asthma” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 15-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).

60 agents shown (approved or with trial evidence) — 57 not found by the gene-target search above.

Mometasone furoateNOT in gene-target list

Approved · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT00835094 NCT00835094

Fluticasone propionateNOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01159912

Fluticasone furoateNOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01159912

SpirivaNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02489981

Fixed combination of budesonide and formoterolNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01676987

BudesonideNOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01676987

Formoterol fumerateNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00460577

fenoterol/ipratropium bromideNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00460577

budesonide/formoterol TurbuhalerNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00235911

CM310NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05761028

AMAZE™ ApplicationNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04868500

AMAZE™ DashboardNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04868500

CHF5993 pMDI with HFA-152aNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07301736

CHF5993 pMDI with HFA-134aNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07301736

CHF718 pMDI with HFA-134aNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07301736

IBI3002NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06213844

SB010NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01470911

Educational Video and Question Prompt ListNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02498834

SHARP programNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01625364

Open Airways for SchoolsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01625364

OXIMAXNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00975741

ASMANEX TWISTHALERNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00975741

IgE adsorberNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02096237

Administration of inhaled tiotropium bromideNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00557700

Administration of placeboNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00557700

Formoterol Fumarate

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00215371

HEPA Air CleanerNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00006565

Placebo Filtration UnitNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00006565

Single Inhalation techniqueNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05428956

Tidal inhalation techniqueNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05428956

Postcard and IVR Reminder GroupNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01852656

Postcard Only Reminder GroupNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01852656

IVR Only Reminder GroupNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01852656

AlfacalcidolNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02747381

AER 001NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00535028

Smart inhalerNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04810169

PEF Interventional SessionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06612294

Control SessionsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06612294

Control BoosterNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06612294

Active boosterNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06612294

Clinical Center Reference Endotoxin (CCRE)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03623022

Normal salineNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03623022

SHR-1703 InjectionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06441812

BENRALIZUMAB

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT04742504

QVM149NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03158311

Salmeterol/fluticasone plus tiotropiumNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03158311

Ginger ExtractNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03705832

Resistive Flexibility and Strength TrainingNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02370004

Halotherapy (Salt Therapy)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06250452

Prick testsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07402200

Portable sensorsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07402200

FP 100NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01156792

GSK2190915 100NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01156792

GSK2190915 300NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01156792

montelukast

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01156792

FP/SAL 100/50NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01156792

Use of smart inhaler to enhance asthma inhaler proficiencyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06783101

Residential mechanical ventilation system (Exhaust)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06098287

Residential mechanical ventilation system (CFIS) and air filtration upgradesNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06098287

Residential mechanical ventilation system (ERV) and air filtration upgradesNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06098287

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