Biomarker & Intervention Discovery

Pulmonary Arterial Hypertension (PAH)

This page lists 11 druggable gene targets, 54 approved or trial-tested drug candidates and 15 recent clinical trials for Pulmonary Arterial Hypertension (PAH), 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.

NIH funding$377MFY2025 · RCDC estimate
Funding levelSeverely Underfunded
Spontaneous remissionWithout treatment, median survival from diagnosis 2.8 years (pre-modern-therapy era); with current therapy 5-year…
Best-intervention remissionTriple upfront combination therapy (ambrisentan + tadalafil + selexipag): 41% reduction in clinical failure events…
Gap sizeLarge — most patients still receive sequential monotherapy or dual therapy rather than upfront triple therapy
Primary barrierpractice/access
Full remission profile →
Research Tracker Chronic Disease Interventions Pulmonary Arterial Hypertension (PAH)

Remission & chronicity

Spontaneous remission
Without treatment, median survival from diagnosis 2.8 years (pre-modern-therapy era); with current therapy 5-year survival ~50-60%; spontaneous remission essentially unknown
Best-intervention remission
Triple upfront combination therapy (ambrisentan + tadalafil + selexipag): 41% reduction in clinical failure events (TRITON trial); macitentan + tadalafil initial combination: 41% risk reduction (AMBITION); lung transplantation: 50-60% 5-year survival
Gap size
Large — most patients still receive sequential monotherapy or dual therapy rather than upfront triple therapy
Primary barrier
practice/access
Upfront combination therapy evidence is robust but drug costs (selexipag alone ~$150K/year) limit access; PAH is often missed or delayed in diagnosis (mean 2-year diagnostic delay); most patients are not treated at PAH expert centres where combination protocols are standard

Biomarker Targets & Therapeutic Agents

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

ACVR2A 47ACVRL1 180BMPR2 70EDNRA 226EDNRB 208GUCY1A1 207GUCY1B1 207KCNK3 199PDE5A 220PDGFRB 269PTGIR 189

ACVR2A

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
BIMAGRUMAB ? unclear Mechanistic DGIdb
DIBOTERMIN ALFA ↑ activates Mechanistic DGIdb
SOTATERCEPT ? unclear Mechanistic DGIdb
DASATINIB ↓ inhibits Mechanistic 210.0 nM (Kd) ChEMBL
JNJ-7706621 ↓ inhibits Mechanistic 2900.0 nM (Kd) ChEMBL
LESTAURTINIB ↓ inhibits Mechanistic 2500.0 nM (Kd) ChEMBL
TAE-684 ↓ inhibits Mechanistic 2200.0 nM (Kd) ChEMBL
KW-2449 ↓ inhibits Mechanistic 320.0 nM (Kd) ChEMBL
CHEMBL1241674 ↓ inhibits Mechanistic 18.0 nM (Kd) ChEMBL
ALVOCIDIB ↓ inhibits Mechanistic 3100.0 nM (Kd) ChEMBL
STAUROSPORINE ↓ inhibits Mechanistic 8900.0 nM (Kd) ChEMBL
CHEMBL386051 ↓ inhibits Mechanistic 10.0 nM (Kd) ChEMBL
CHEMBL4283638 ↓ inhibits Mechanistic 2370.0 nM (IC50) ChEMBL
CHEMBL4792978 ↓ inhibits Mechanistic 180.0 nM (IC50) ChEMBL
CHEMBL4747912 ↓ inhibits Mechanistic 20000.0 nM (IC50) ChEMBL
CHEMBL4783244 ↓ inhibits Mechanistic 1300.0 nM (IC50) ChEMBL
CHEMBL4795223 ↓ inhibits Mechanistic 1100.0 nM (IC50) ChEMBL
CHEMBL4779579 ↓ inhibits Mechanistic 1300.0 nM (IC50) ChEMBL
CHEMBL4741913 ↓ inhibits Mechanistic 190.0 nM (IC50) ChEMBL
CHEMBL4741185 ↓ inhibits Mechanistic 700.0 nM (IC50) ChEMBL
CHEMBL4744988 ↓ inhibits Mechanistic 630.0 nM (IC50) ChEMBL
CHEMBL4752199 ↓ inhibits Mechanistic 310.0 nM (IC50) ChEMBL
CHEMBL4750258 ↓ inhibits Mechanistic 1200.0 nM (IC50) ChEMBL
CHEMBL4777071 ↓ inhibits Mechanistic 3000.0 nM (IC50) ChEMBL
CHEMBL4798505 ↓ inhibits Mechanistic 1200.0 nM (IC50) ChEMBL
CHEMBL4763122 ↓ inhibits Mechanistic 960.0 nM (IC50) ChEMBL
CHEMBL4748097 ↓ inhibits Mechanistic 3800.0 nM (IC50) ChEMBL
CHEMBL4764416 ↓ inhibits Mechanistic 2700.0 nM (IC50) ChEMBL
CHEMBL4782729 ↓ inhibits Mechanistic 2900.0 nM (IC50) ChEMBL
CHEMBL4779273 ↓ inhibits Mechanistic 1900.0 nM (IC50) ChEMBL
CHEMBL4792464 ↓ inhibits Mechanistic 4100.0 nM (IC50) ChEMBL
CHEMBL4749061 ↓ inhibits Mechanistic 5300.0 nM (IC50) ChEMBL
CHEMBL4763627 ↓ inhibits Mechanistic 3000.0 nM (IC50) ChEMBL
CHEMBL4749011 ↓ inhibits Mechanistic 530.0 nM (IC50) ChEMBL
CHEMBL4758580 ↓ inhibits Mechanistic 2300.0 nM (IC50) ChEMBL
CHEMBL4747422 ↓ inhibits Mechanistic 800.0 nM (IC50) ChEMBL
CHEMBL4744618 ↓ inhibits Mechanistic 860.0 nM (IC50) ChEMBL
CHEMBL4787891 ↓ inhibits Mechanistic 2400.0 nM (IC50) ChEMBL
CHEMBL4791414 ↓ inhibits Mechanistic 2300.0 nM (IC50) ChEMBL
CHEMBL4751832 ↓ inhibits Mechanistic 3000.0 nM (IC50) ChEMBL

Showing top 40 of 47 agents ranked by evidence tier.

ACVRL1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
DALANTERCEPT ↓ inhibits Mechanistic DGIdb
COMPOUND 13R [PMID: 23639540] ↓ inhibits Mechanistic DGIdb
ASCRINVACUMAB ? unclear Mechanistic DGIdb
HESPERADIN ↓ inhibits Mechanistic DGIdb
LDN-214117 ↓ inhibits Mechanistic DGIdb
PANULISIB ↓ inhibits Mechanistic DGIdb
ZILURGISERTIB ↓ inhibits Mechanistic DGIdb
ML347 ↓ inhibits Mechanistic DGIdb
COMPOUND 13D [PMID: 23639540] ↓ inhibits Mechanistic DGIdb
COMPOUND 13A [PMID: 23639540] ↓ inhibits Mechanistic DGIdb
DASATINIB ↓ inhibits Mechanistic 460.0 nM (Kd) ChEMBL
JNJ-7706621 ↓ inhibits Mechanistic 2900.0 nM (Kd) ChEMBL
VANDETANIB ↓ inhibits Mechanistic 470.0 nM (Kd) ChEMBL
LESTAURTINIB ↓ inhibits Mechanistic 2200.0 nM (Kd) ChEMBL
CHEMBL1908395 ↓ inhibits Mechanistic 1900.0 nM (Kd) ChEMBL
TAE-684 ↓ inhibits Mechanistic 57.0 nM (Kd) ChEMBL
NINTEDANIB ↓ inhibits Mechanistic 4200.0 nM (Kd) ChEMBL
FEDRATINIB ↓ inhibits Mechanistic 2400.0 nM (Kd) ChEMBL
CHEMBL1241674 ↓ inhibits Mechanistic 2.9 nM (Kd) ChEMBL
ALVOCIDIB ↓ inhibits Mechanistic 4500.0 nM (Kd) ChEMBL
TOZASERTIB ↓ inhibits Mechanistic 9500.0 nM (Kd) ChEMBL
CHEMBL386051 ↓ inhibits Mechanistic 69.0 nM (Kd) ChEMBL
DORSOMORPHIN ↓ inhibits Mechanistic 106.3 nM (IC50) ChEMBL
CHEMBL2385586 ↓ inhibits Mechanistic 55.0 nM (IC50) ChEMBL
CHEMBL2385600 ↓ inhibits Mechanistic 52.4 nM (IC50) ChEMBL
CHEMBL502351 ↓ inhibits Mechanistic 46.0 nM (IC50) ChEMBL
CHEMBL2385579 ↓ inhibits Mechanistic 42.1 nM (IC50) ChEMBL
CHEMBL2385597 ↓ inhibits Mechanistic 27.0 nM (IC50) ChEMBL
CHEMBL511563 ↓ inhibits Mechanistic 26.5 nM (IC50) ChEMBL
CHEMBL2385591 ↓ inhibits Mechanistic 14.4 nM (IC50) ChEMBL
CHEMBL513147 ↓ inhibits Mechanistic 13.3 nM (IC50) ChEMBL
STAUROSPORINE ↓ inhibits Mechanistic 850.0 nM (IC50) ChEMBL
CHEMBL3341789 ↓ inhibits Mechanistic 27.0 nM (IC50) ChEMBL
CHEMBL3671311 ↓ inhibits Mechanistic 4.701 nM (IC50) ChEMBL
CHEMBL3671321 ↓ inhibits Mechanistic 231.3 nM (IC50) ChEMBL
CHEMBL3671317 ↓ inhibits Mechanistic 38.86 nM (IC50) ChEMBL
CHEMBL3671312 ↓ inhibits Mechanistic 15.11 nM (IC50) ChEMBL
CHEMBL3671315 ↓ inhibits Mechanistic 174.4 nM (IC50) ChEMBL
CHEMBL3671318 ↓ inhibits Mechanistic 9.157 nM (IC50) ChEMBL
CHEMBL3671309 ↓ inhibits Mechanistic 9.812 nM (IC50) ChEMBL

Showing top 40 of 180 agents ranked by evidence tier.

BMPR2

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
EPTOTERMIN ALFA ↑ activates Mechanistic DGIdb
COMPOUND 13A [PMID: 23639540] ↓ inhibits Mechanistic DGIdb
DIBOTERMIN ALFA ↑ activates Mechanistic DGIdb
DEUCRAVACITINIB ↓ inhibits Mechanistic 193.0 nM (IC50) DGIdb
LINIFANIB ↓ inhibits Mechanistic 7800.0 nM (Kd) ChEMBL
DOVITINIB ↓ inhibits Mechanistic 3100.0 nM (Kd) ChEMBL
JNJ-7706621 ↓ inhibits Mechanistic 880.0 nM (Kd) ChEMBL
SU-014813 ↓ inhibits Mechanistic 200.0 nM (Kd) ChEMBL
SUNITINIB ↓ inhibits Mechanistic 570.0 nM (Kd) ChEMBL
TOZASERTIB ↓ inhibits Mechanistic 7100.0 nM (Kd) ChEMBL
LESTAURTINIB ↓ inhibits Mechanistic 1500.0 nM (Kd) ChEMBL
PHA-665752 ↓ inhibits Mechanistic 6200.0 nM (Kd) ChEMBL
RUXOLITINIB ↓ inhibits Mechanistic 930.0 nM (Kd) ChEMBL
TAE-684 ↓ inhibits Mechanistic 3900.0 nM (Kd) ChEMBL
NINTEDANIB ↓ inhibits Mechanistic 56.0 nM (Kd) ChEMBL
CHEMBL1908395 ↓ inhibits Mechanistic 310.0 nM (Kd) ChEMBL
FEDRATINIB ↓ inhibits Mechanistic 2900.0 nM (Kd) ChEMBL
KW-2449 ↓ inhibits Mechanistic 370.0 nM (Kd) ChEMBL
CHEMBL1241674 ↓ inhibits Mechanistic 89.0 nM (Kd) ChEMBL
BOSUTINIB ↓ inhibits Mechanistic 580.0 nM (Kd) ChEMBL
STAUROSPORINE ↓ inhibits Mechanistic 2300.0 nM (Kd) ChEMBL
CHEMBL513147 ↓ inhibits Mechanistic 3845.0 nM (IC50) ChEMBL
CHEMBL2385582 ↓ inhibits Mechanistic 3640.0 nM (IC50) ChEMBL
CHEMBL2385596 ↓ inhibits Mechanistic 2360.0 nM (IC50) ChEMBL
CHEMBL2385591 ↓ inhibits Mechanistic 695.0 nM (IC50) ChEMBL
DORSOMORPHIN ↓ inhibits Mechanistic 74.0 nM (IC50) ChEMBL
CHEMBL2385586 ↓ inhibits Mechanistic 59.0 nM (IC50) ChEMBL
CHEMBL2385600 ↓ inhibits Mechanistic 24.0 nM (IC50) ChEMBL
CHEMBL2385579 ↓ inhibits Mechanistic 15.9 nM (IC50) ChEMBL
CYC-116 ↓ inhibits Mechanistic 14377.0 nM (Kd) ChEMBL
PF-03814735 ↓ inhibits Mechanistic 8.0 nM (Kd) ChEMBL
Cerdulatinib Hydrochloride ↓ inhibits Mechanistic 4861.0 nM (Kd) ChEMBL
SILMITASERTIB ↓ inhibits Mechanistic 1056.0 nM (Kd) ChEMBL
ORANTINIB ↓ inhibits Mechanistic 19.0 nM (Kd) ChEMBL
AT-9283 ↓ inhibits Mechanistic 43.0 nM (Kd) ChEMBL
CHEMBL400402 ↓ inhibits Mechanistic 3.0 nM (Kd) ChEMBL
OSI-027 ↓ inhibits Mechanistic 2097.0 nM (Kd) ChEMBL
RGB-286638 ↓ inhibits Mechanistic 153.0 nM (Kd) ChEMBL
TP-030-1 ↓ inhibits Mechanistic 1000.0 nM (IC50) ChEMBL
TP-030-2 ↓ inhibits Mechanistic 1000.0 nM (IC50) ChEMBL

Showing top 40 of 70 agents ranked by evidence tier.

EDNRA

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
MACITENTAN ↓ inhibits Mechanistic DGIdb
ZIBOTENTAN ↓ inhibits Mechanistic DGIdb
FR139317 ↓ inhibits Mechanistic DGIdb
DARUSENTAN ↓ inhibits Mechanistic DGIdb
BMS-193884 ↓ inhibits Mechanistic DGIdb
[125I]PD151242 ↓ inhibits Mechanistic DGIdb
DIOSUXENTAN ↓ inhibits Mechanistic DGIdb
AMBRISENTAN ↓ inhibits Mechanistic DGIdb
SITAXENTAN ↓ inhibits Mechanistic 1.4 nM (IC50) DGIdb
CLAZOSENTAN ↓ inhibits Mechanistic DGIdb
ATRASENTAN ↓ inhibits Mechanistic 0.31 nM (IC50) DGIdb
[3H]S0139 ↓ inhibits Mechanistic DGIdb
GETAGOZUMAB ? unclear Mechanistic DGIdb
[3H]BQ123 ↓ inhibits Mechanistic DGIdb
EDONENTAN ANHYDROUS ↓ inhibits Mechanistic DGIdb
BOSENTAN ANHYDROUS ↓ inhibits Mechanistic DGIdb
SOVATELTIDE ? unclear Mechanistic DGIdb
PD-156707 ↓ inhibits Mechanistic DGIdb
[125I]SARAFOTOXIN S6B ↑ activates Mechanistic DGIdb
ENDOTHELIN-2 ↑ activates Mechanistic DGIdb
AVOSENTAN ↓ inhibits Mechanistic DGIdb
[18F]ET-1 (HUMAN, MOUSE, RAT) ↑ activates Mechanistic DGIdb
SPARSENTAN ↓ inhibits Mechanistic DGIdb
ENRASENTAN ↓ inhibits Mechanistic 1.1 nM (Ki) DGIdb
APROCITENTAN ↓ inhibits Mechanistic DGIdb
NEBENTAN ↓ inhibits Mechanistic DGIdb
ENDOTHELIN-1 ↑ activates Mechanistic DGIdb
PD164333 ↓ inhibits Mechanistic DGIdb
[125I]PD164333 ↓ inhibits Mechanistic DGIdb
SITAXENTAN SODIUM ↓ inhibits Mechanistic DGIdb
[125I]ET-2 (HUMAN) ↑ activates Mechanistic DGIdb
BQ-123 ? unclear Mechanistic 22.0 nM (IC50) DGIdb
SB234551 ↓ inhibits Mechanistic DGIdb
ATRASENTAN HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
TBC-3711 ? unclear Mechanistic DGIdb
BQ123 ↓ inhibits Mechanistic DGIdb
TEZOSENTAN ↓ inhibits Mechanistic 23.0 nM (IC50) DGIdb
PREDNISONE ? unclear Mechanistic DGIdb
[125I]ET-1 (HUMAN, MOUSE, RAT) ↑ activates Mechanistic DGIdb
SARAFOTOXIN S6B ↑ activates Mechanistic DGIdb

Showing top 40 of 226 agents ranked by evidence tier.

EDNRB

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
K-8794 ↓ inhibits Mechanistic DGIdb
AMBRISENTAN ↓ inhibits Mechanistic DGIdb
BOSENTAN ANHYDROUS ↓ inhibits Mechanistic DGIdb
BQ 3020 ↑ activates Mechanistic DGIdb
ENDOTHELIN-1 ? unclear Mechanistic DGIdb
ENRASENTAN ↓ inhibits Mechanistic 110.0 nM (Ki) DGIdb
BMS-193884 ↓ inhibits Mechanistic DGIdb
[125I]ET-1 (HUMAN, MOUSE, RAT) ↑ activates Mechanistic DGIdb
SOVATELTIDE ↑ activates Mechanistic DGIdb
[125I]BQ3020 ↑ activates Mechanistic DGIdb
MACITENTAN ↓ inhibits Mechanistic DGIdb
PACLITAXEL PROTEIN-BOUND ? unclear Mechanistic DGIdb
APROCITENTAN ↓ inhibits Mechanistic DGIdb
ENDOTHELIN-3 ↑ activates Mechanistic DGIdb
IRL 2500 ↓ inhibits Mechanistic DGIdb
A192621 ↓ inhibits Mechanistic DGIdb
SITAXENTAN SODIUM ↓ inhibits Mechanistic DGIdb
SITAXENTAN ↓ inhibits Mechanistic DGIdb
RG-7636 ~ modulates Mechanistic DGIdb
CLAZOSENTAN ↓ inhibits Mechanistic DGIdb
SARAFOTOXIN S6C ↑ activates Mechanistic DGIdb
[125I][ALA1,3,11,15]ET-1 ↑ activates Mechanistic DGIdb
RO 46-8443 ↓ inhibits Mechanistic DGIdb
[ALA1,3,11,15]ET-1 ↑ activates Mechanistic DGIdb
BQ788 ↓ inhibits Mechanistic DGIdb
RES7011 ↓ inhibits Mechanistic DGIdb
TEZOSENTAN ↓ inhibits Mechanistic DGIdb
ATRASENTAN ↓ inhibits Mechanistic DGIdb
DARUSENTAN ↓ inhibits Mechanistic DGIdb
BOSENTAN ? unclear Mechanistic 340.0 nM (Ki) Open Targets
CHEMBL170335 ↓ inhibits Mechanistic 21000.0 nM (IC50) ChEMBL
CHEMBL175228 ↓ inhibits Mechanistic 54000.0 nM (IC50) ChEMBL
CHEMBL177546 ↓ inhibits Mechanistic 48000.0 nM (IC50) ChEMBL
CHEMBL174448 ↓ inhibits Mechanistic 34000.0 nM (IC50) ChEMBL
CHEMBL353502 ↓ inhibits Mechanistic 70000.0 nM (IC50) ChEMBL
CHEMBL368629 ↓ inhibits Mechanistic 79000.0 nM (IC50) ChEMBL
CHEMBL173059 ↓ inhibits Mechanistic 50000.0 nM (IC50) ChEMBL
CHEMBL414605 ↓ inhibits Mechanistic 20000.0 nM (IC50) ChEMBL
CHEMBL175698 ↓ inhibits Mechanistic 90000.0 nM (IC50) ChEMBL
CHEMBL278176 ↓ inhibits Mechanistic 60.0 nM (IC50) ChEMBL

Showing top 40 of 208 agents ranked by evidence tier.

GUCY1A1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
NITROGLYCERIN ↑ activates Mechanistic DGIdb
METHYLENE BLUE ANHYDROUS ↓ inhibits Mechanistic DGIdb
SODIUM NITROPRUSSIDE ↑ activates Mechanistic DGIdb
PENTAERYTHRITOL TETRANITRATE ↑ activates Mechanistic DGIdb
PRALICIGUAT ↑ activates Mechanistic DGIdb
ISOSORBIDE MONONITRATE ↑ activates Mechanistic DGIdb
RIOCIGUAT ~ modulates Mechanistic DGIdb
VERICIGUAT ↑ activates Mechanistic DGIdb
RUNCACIGUAT ↑ activates Mechanistic DGIdb
ISOSORBIDE DINITRATE ↑ activates Mechanistic DGIdb
NITRIC OXIDE ↑ activates Mechanistic DGIdb
OLINCIGUAT ~ modulates Mechanistic DGIdb
CINACIGUAT ↑ activates Mechanistic DGIdb
ATACIGUAT ↑ activates Mechanistic DGIdb
NITROPRUSSIDE ? unclear Mechanistic Open Targets
BENZYDAMINE ↑ activates Mechanistic 1020.0 nM (EC50) ChEMBL
CHEMBL202402 ↑ activates Mechanistic 21900.0 nM (EC50) ChEMBL
CHEMBL3604521 ↓ inhibits Mechanistic 37000.0 nM (IC50) ChEMBL
CHEMBL3808502 ↑ activates Mechanistic 1500.0 nM (EC50) ChEMBL
CHEMBL3809082 ↑ activates Mechanistic 530.0 nM (EC50) ChEMBL
CHEMBL3808494 ↑ activates Mechanistic 280.0 nM (EC50) ChEMBL
CHEMBL3810301 ↑ activates Mechanistic 240.0 nM (EC50) ChEMBL
CHEMBL3808807 ↑ activates Mechanistic 380.0 nM (EC50) ChEMBL
CHEMBL3810321 ↑ activates Mechanistic 370.0 nM (EC50) ChEMBL
CHEMBL3809131 ↑ activates Mechanistic 880.0 nM (EC50) ChEMBL
CHEMBL3810028 ↑ activates Mechanistic 180.0 nM (EC50) ChEMBL
CHEMBL3809867 ↑ activates Mechanistic 1500.0 nM (EC50) ChEMBL
CHEMBL3810365 ↑ activates Mechanistic 320.0 nM (EC50) ChEMBL
CHEMBL3809227 ↑ activates Mechanistic 730.0 nM (EC50) ChEMBL
CHEMBL3810113 ↑ activates Mechanistic 160.0 nM (EC50) ChEMBL
CHEMBL3810075 ↑ activates Mechanistic 350.0 nM (EC50) ChEMBL
CHEMBL3809557 ↑ activates Mechanistic 900.0 nM (EC50) ChEMBL
CHEMBL3808998 ↑ activates Mechanistic 590.0 nM (EC50) ChEMBL
CHEMBL3809479 ↑ activates Mechanistic 190.0 nM (EC50) ChEMBL
CHEMBL3809730 ↑ activates Mechanistic 290.0 nM (EC50) ChEMBL
CHEMBL3809566 ↑ activates Mechanistic 500.0 nM (EC50) ChEMBL
CHEMBL3809864 ↑ activates Mechanistic 240.0 nM (EC50) ChEMBL
CHEMBL3929069 ↑ activates Mechanistic 5500.0 nM (EC50) ChEMBL
CHEMBL3951365 ↑ activates Mechanistic 2600.0 nM (EC50) ChEMBL
CHEMBL3923657 ↑ activates Mechanistic 720.0 nM (EC50) ChEMBL

Showing top 40 of 207 agents ranked by evidence tier.

GUCY1B1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
RUNCACIGUAT ↑ activates Mechanistic DGIdb
VERICIGUAT ↑ activates Mechanistic DGIdb
RIOCIGUAT ~ modulates Mechanistic DGIdb
PRALICIGUAT ↑ activates Mechanistic DGIdb
ATACIGUAT ↑ activates Mechanistic DGIdb
ISOSORBIDE MONONITRATE ↑ activates Mechanistic DGIdb
PENTAERYTHRITOL TETRANITRATE ↑ activates Mechanistic DGIdb
ISOSORBIDE DINITRATE ↑ activates Mechanistic DGIdb
CINACIGUAT ↑ activates Mechanistic DGIdb
OLINCIGUAT ~ modulates Mechanistic DGIdb
SODIUM NITROPRUSSIDE ↑ activates Mechanistic DGIdb
NITROGLYCERIN ↑ activates Mechanistic DGIdb
METHYLENE BLUE ANHYDROUS ↓ inhibits Mechanistic DGIdb
NITRIC OXIDE ↑ activates Mechanistic DGIdb
NITROPRUSSIDE ? unclear Mechanistic Open Targets
BENZYDAMINE ↑ activates Mechanistic 1020.0 nM (EC50) ChEMBL
CHEMBL202402 ↑ activates Mechanistic 21900.0 nM (EC50) ChEMBL
CHEMBL3604521 ↓ inhibits Mechanistic 37000.0 nM (IC50) ChEMBL
CHEMBL3808502 ↑ activates Mechanistic 1500.0 nM (EC50) ChEMBL
CHEMBL3809082 ↑ activates Mechanistic 530.0 nM (EC50) ChEMBL
CHEMBL3808494 ↑ activates Mechanistic 280.0 nM (EC50) ChEMBL
CHEMBL3810301 ↑ activates Mechanistic 240.0 nM (EC50) ChEMBL
CHEMBL3808807 ↑ activates Mechanistic 380.0 nM (EC50) ChEMBL
CHEMBL3810321 ↑ activates Mechanistic 370.0 nM (EC50) ChEMBL
CHEMBL3809131 ↑ activates Mechanistic 880.0 nM (EC50) ChEMBL
CHEMBL3810028 ↑ activates Mechanistic 180.0 nM (EC50) ChEMBL
CHEMBL3809867 ↑ activates Mechanistic 1500.0 nM (EC50) ChEMBL
CHEMBL3810365 ↑ activates Mechanistic 320.0 nM (EC50) ChEMBL
CHEMBL3809227 ↑ activates Mechanistic 730.0 nM (EC50) ChEMBL
CHEMBL3810113 ↑ activates Mechanistic 160.0 nM (EC50) ChEMBL
CHEMBL3810075 ↑ activates Mechanistic 350.0 nM (EC50) ChEMBL
CHEMBL3809557 ↑ activates Mechanistic 900.0 nM (EC50) ChEMBL
CHEMBL3808998 ↑ activates Mechanistic 590.0 nM (EC50) ChEMBL
CHEMBL3809479 ↑ activates Mechanistic 190.0 nM (EC50) ChEMBL
CHEMBL3809730 ↑ activates Mechanistic 290.0 nM (EC50) ChEMBL
CHEMBL3809566 ↑ activates Mechanistic 500.0 nM (EC50) ChEMBL
CHEMBL3809864 ↑ activates Mechanistic 240.0 nM (EC50) ChEMBL
CHEMBL3929069 ↑ activates Mechanistic 5500.0 nM (EC50) ChEMBL
CHEMBL3951365 ↑ activates Mechanistic 2600.0 nM (EC50) ChEMBL
CHEMBL3923657 ↑ activates Mechanistic 720.0 nM (EC50) ChEMBL

Showing top 40 of 207 agents ranked by evidence tier.

KCNK3

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
KU124 ↓ inhibits Mechanistic DGIdb
DOXAPRAM HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
ISOFLURANE ↑ activates Mechanistic DGIdb
CANDESARTAN CILEXETIL ? unclear Mechanistic DGIdb
AVE1231 ↓ inhibits Mechanistic DGIdb
HALOTHANE ↑ activates Mechanistic DGIdb
ENFLURANE ↑ activates Mechanistic DGIdb
ARACHIDONIC ACID ↓ inhibits Mechanistic DGIdb
R-(+)-METHANANDAMIDE ↓ inhibits Mechanistic DGIdb
SEVOFLURANE ↑ activates Mechanistic DGIdb
BA2+ ↓ inhibits Mechanistic DGIdb
DESFLURANE ↑ activates Mechanistic DGIdb
ANANDAMIDE ↓ inhibits Mechanistic DGIdb
DOXAPRAM ? unclear Mechanistic Open Targets
CHEMBL2324344 ↓ inhibits Mechanistic 300.0 nM (IC50) ChEMBL
CHEMBL148342 ↓ inhibits Mechanistic 7.0 nM (IC50) ChEMBL
CHEMBL1310568 ↓ inhibits Mechanistic 27.0 nM (IC50) ChEMBL
CHEMBL3186207 ↓ inhibits Mechanistic 100.0 nM (IC50) ChEMBL
CHEMBL3186489 ↓ inhibits Mechanistic 21.0 nM (IC50) ChEMBL
CHEMBL3188343 ↓ inhibits Mechanistic 220.0 nM (IC50) ChEMBL
CHEMBL3183938 ↓ inhibits Mechanistic 330.0 nM (IC50) ChEMBL
CHEMBL1532914 ↓ inhibits Mechanistic 45.0 nM (IC50) ChEMBL
CHEMBL3184075 ↓ inhibits Mechanistic 310.0 nM (IC50) ChEMBL
CHEMBL3182809 ↓ inhibits Mechanistic 500.0 nM (IC50) ChEMBL
CHEMBL3184966 ↓ inhibits Mechanistic 52.0 nM (IC50) ChEMBL
CHEMBL3186206 ↓ inhibits Mechanistic 130.0 nM (IC50) ChEMBL
CHEMBL3186946 ↓ inhibits Mechanistic 160.0 nM (IC50) ChEMBL
CHEMBL3184653 ↓ inhibits Mechanistic 260.0 nM (IC50) ChEMBL
CHEMBL1716797 ↓ inhibits Mechanistic 37.0 nM (IC50) ChEMBL
CHEMBL3188024 ↓ inhibits Mechanistic 800.0 nM (IC50) ChEMBL
CHEMBL3182777 ↓ inhibits Mechanistic 29.0 nM (IC50) ChEMBL
CHEMBL3183945 ↓ inhibits Mechanistic 160.0 nM (IC50) ChEMBL
CHEMBL3182370 ↓ inhibits Mechanistic 86.0 nM (IC50) ChEMBL
CHEMBL1541525 ↓ inhibits Mechanistic 1290.0 nM (IC50) ChEMBL
CHEMBL3188245 ↓ inhibits Mechanistic 250.0 nM (IC50) ChEMBL
CHEMBL3184095 ↓ inhibits Mechanistic 79.0 nM (IC50) ChEMBL
CHEMBL3181939 ↓ inhibits Mechanistic 91.0 nM (IC50) ChEMBL
CHEMBL3185736 ↓ inhibits Mechanistic 30.0 nM (IC50) ChEMBL
CHEMBL3184438 ↓ inhibits Mechanistic 330.0 nM (IC50) ChEMBL
CHEMBL3181946 ↓ inhibits Mechanistic 190.0 nM (IC50) ChEMBL

Showing top 40 of 199 agents ranked by evidence tier.

PDE5A

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
IBUDILAST ↓ inhibits Mechanistic DGIdb
SILDENAFIL ↓ inhibits Mechanistic 8.7 nM (EC50) DGIdb
GISADENAFIL ↓ inhibits Mechanistic DGIdb
AVANAFIL ↓ inhibits Mechanistic DGIdb
COMPOUND 3M [PMID: 32603117] ↓ inhibits Mechanistic DGIdb
DIPYRIDAMOLE ↓ inhibits Mechanistic DGIdb
T0156 ↓ inhibits Mechanistic DGIdb
TADALAFIL ↓ inhibits Mechanistic 6.7 nM (IC50) DGIdb
PF-03049423 ↓ inhibits Mechanistic DGIdb
SCH51866 ↓ inhibits Mechanistic DGIdb
PENTOXIFYLLINE ↓ inhibits Mechanistic DGIdb
MIRODENAFIL ↓ inhibits Mechanistic DGIdb
THEOPHYLLINE ? unclear Mechanistic DGIdb
ZAPRINAST ↓ inhibits Mechanistic 900.0 nM (IC50) DGIdb
PAPAVERINE ? unclear Mechanistic DGIdb
ICARIIN ↓ inhibits Mechanistic DGIdb
ROLIPRAM ? unclear Mechanistic DGIdb
SLX-2101 ? unclear Mechanistic DGIdb
VARDENAFIL ↓ inhibits Mechanistic DGIdb
PF-00489791 ↓ inhibits Mechanistic DGIdb
SILDENAFIL CITRATE ↓ inhibits Mechanistic DGIdb
ENALAPRIL MALEATE ? unclear Mechanistic DGIdb
NITROSOPRODENAFIL ↓ inhibits Mechanistic DGIdb
OSI-461 ? unclear Mechanistic DGIdb
SIMENAFIL ↓ inhibits Mechanistic DGIdb
C1 ESTERASE INHIBITOR (RECOMBINANT) ? unclear Mechanistic DGIdb
VARDENAFIL HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
MILRINONE ↓ inhibits Mechanistic DGIdb
LORNEIC ACID A ? unclear Mechanistic DGIdb
EXISULIND ? unclear Mechanistic DGIdb
UDENAFIL ↓ inhibits Mechanistic DGIdb
COMPOUND 53 [PMID: 19631533] ↓ inhibits Mechanistic DGIdb
TUNODAFIL ↓ inhibits Mechanistic DGIdb
CHEMBL90164 ↓ inhibits Mechanistic 11.0 nM (IC50) ChEMBL
CHEMBL87901 ↓ inhibits Mechanistic 60.0 nM (IC50) ChEMBL
CHEMBL313197 ↓ inhibits Mechanistic 24.0 nM (IC50) ChEMBL
CHEMBL86578 ↓ inhibits Mechanistic 93.0 nM (IC50) ChEMBL
CHEMBL313561 ↓ inhibits Mechanistic 130.0 nM (IC50) ChEMBL
CHEMBL87202 ↓ inhibits Mechanistic 10.0 nM (IC50) ChEMBL
CHEMBL87954 ↓ inhibits Mechanistic 135.0 nM (IC50) ChEMBL

Showing top 40 of 220 agents ranked by evidence tier.

PDGFRB

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
TELATINIB ↓ inhibits Mechanistic DGIdb
RIPRETINIB ? unclear Mechanistic DGIdb
DOVITINIB ↓ inhibits Mechanistic DGIdb
DASATINIB ANHYDROUS ↓ inhibits Mechanistic DGIdb
LINIFANIB ↓ inhibits Mechanistic DGIdb
ROMIPLOSTIM ? unclear Mechanistic DGIdb
IMATINIB ↓ inhibits Mechanistic DGIdb
SORAFENIB ↓ inhibits Mechanistic DGIdb
AMUVATINIB ↓ inhibits Mechanistic DGIdb
SUNITINIB ↓ inhibits Mechanistic 2.0 nM (IC50) DGIdb
SNS-314 ? unclear Mechanistic DGIdb
PP2 ? unclear Mechanistic DGIdb
TAK-593 ↓ inhibits Mechanistic DGIdb
AXITINIB ↓ inhibits Mechanistic DGIdb
SERALUTINIB ↓ inhibits Mechanistic DGIdb
RG-1530 ↓ inhibits Mechanistic DGIdb
GTP-14564 ↓ inhibits Mechanistic DGIdb
CENISERTIB ? unclear Mechanistic DGIdb
BECAPLERMIN ↑ activates Mechanistic DGIdb
XL-820 ↓ inhibits Mechanistic DGIdb
VELZATINIB ↓ inhibits Mechanistic DGIdb
TANDUTINIB ↓ inhibits Mechanistic 26.0 nM (IC50) DGIdb
HESPERADIN ↓ inhibits Mechanistic DGIdb
FAMITINIB ↓ inhibits Mechanistic DGIdb
REGORAFENIB ↓ inhibits Mechanistic DGIdb
IMATINIB MESYLATE ↓ inhibits Mechanistic DGIdb
X-82 ↓ inhibits Mechanistic DGIdb
TAK-632 ↓ inhibits Mechanistic DGIdb
FLUMBATINIB ↓ inhibits Mechanistic DGIdb
MOTESANIB ↓ inhibits Mechanistic DGIdb
TAMATINIB ? unclear Mechanistic DGIdb
LEUCOVORIN CALCIUM ? unclear Mechanistic DGIdb
TG100-801 ↓ inhibits Mechanistic DGIdb
TOCERANIB PHOSPHATE ↓ inhibits Mechanistic DGIdb
ORANTINIB ↓ inhibits Mechanistic 60.0 nM (IC50) DGIdb
VATALANIB ↓ inhibits Mechanistic DGIdb
CEDIRANIB ↓ inhibits Mechanistic DGIdb
ENMD-981693 ↓ inhibits Mechanistic DGIdb
IACS-9439 ↓ inhibits Mechanistic DGIdb
RINUCUMAB ↓ inhibits Mechanistic DGIdb

Showing top 40 of 269 agents ranked by evidence tier.

PTGIR

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
AFP-07 ↑ activates Mechanistic DGIdb
BUTAPROST (FREE ACID FORM) ↑ activates Mechanistic DGIdb
[3H]ILOPROST ↑ activates Mechanistic DGIdb
EP 157 ↑ activates Mechanistic DGIdb
TREPROSTINIL ↑ activates Mechanistic DGIdb
TAPROSTENE ↑ activates Mechanistic DGIdb
CAY10441 ↓ inhibits Mechanistic DGIdb
FK-788 ↑ activates Mechanistic DGIdb
RALINEPAG ↑ activates Mechanistic DGIdb
ESUBERAPROST ↑ activates Mechanistic DGIdb
TG6-129 ↓ inhibits Mechanistic DGIdb
TEI-9063 ↑ activates Mechanistic DGIdb
FR181157 ↑ activates Mechanistic DGIdb
TREPROSTINIL DIOLAMINE ↑ activates Mechanistic DGIdb
ASPIRIN ? unclear Mechanistic DGIdb
BERAPROST ↑ activates Mechanistic DGIdb
MRE-269 ↑ activates Mechanistic DGIdb
CARBACYCLIN ↑ activates Mechanistic DGIdb
SELEXIPAG ↑ activates Mechanistic DGIdb
EPOPROSTENOL SODIUM ↑ activates Mechanistic DGIdb
BMY 45778 ↑ activates Mechanistic DGIdb
TREPROSTINIL SODIUM ↑ activates Mechanistic DGIdb
ILOPROST TROMETHAMINE ↑ activates Mechanistic DGIdb
LAROPIPRANT ? unclear Mechanistic 6628.0 nM (Ki) DGIdb
CICAPROST ↑ activates Mechanistic DGIdb
ALPROSTADIL ↑ activates Mechanistic 150.0 nM (Ki) DGIdb
EPOPROSTENOL ↑ activates Mechanistic DGIdb
ILOPROST ↑ activates Mechanistic 6.5 nM (Ki) DGIdb
BAY-73-1449 ↓ inhibits Mechanistic DGIdb
U46619 ↑ activates Mechanistic DGIdb
DINOPROST TROMETHAMINE ? unclear Mechanistic DGIdb
RO3244794 ↓ inhibits Mechanistic DGIdb
MISOPROSTOL ? unclear Mechanistic DGIdb
CHEMBL432522 ↑ activates Mechanistic 37.0 nM (EC50) ChEMBL
CHEMBL64804 ↑ activates Mechanistic 260.0 nM (EC50) ChEMBL
CHEMBL64663 ↓ inhibits Mechanistic 870.0 nM (Ki) ChEMBL
CHEMBL132589 ↓ inhibits Mechanistic 54.0 nM (Ki) ChEMBL
CHEMBL309987 ↓ inhibits Mechanistic 5900.0 nM (IC50) ChEMBL
CHEMBL420398 ↓ inhibits Mechanistic 4500.0 nM (IC50) ChEMBL
CHEMBL435382 ↓ inhibits Mechanistic 3300.0 nM (IC50) ChEMBL

Showing top 40 of 189 agents ranked by evidence tier.

Clinical Trials

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

Clinical Trial of NS-863 in Participants With Pulmonary Arterial Hypertension (PAH) Recruiting
NCT07441200 Phase: 2 Started: 2026-09-29

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Dose Finding Trial to Evaluate the Efficacy and Safety of Orally Administered NS-863 in Participants with Pulmonary Arterial Hypertension (PAH)

Study of REGN13335 in Adult Participants With Pulmonary Arterial Hypertension (PAH) Recruiting
NCT07318597 Phase: 2 Started: 2026-05-06

This study is researching an experimental drug called REGN13335. The study is focused on participants with Pulmonary Arterial Hypertension (PAH). The aim of the study is to see how safe and effective REGN13335 is in participants with PAH who are taking other PAH medicines. The study is looking at se...

A Study of Sotatercept (MK-7962) in People With Pulmonary Arterial Hypertension (PAH) in India (MK-7962-037) Recruiting
NCT07600723 Phase: 4 Started: 2026-10-08

Researchers are looking for other ways to treat people in India with pulmonary arterial hypertension (PAH), also known as Group 1 pulmonary hypertension (PH). In PAH, the blood vessels in the lungs become thick and narrow, which makes it harder for blood to flow to the lungs. This causes high blood ...

An Extension Study of Treprostinil Palmitil Inhalation Powder (TPIP) for Pulmonary Arterial Hypertension (PAH) Active Not Recruiting
NCT05649748 Phase: 2/3 Started: 2023-03-07

The primary purpose of the study is to evaluate the safety and tolerability of the long-term use of TPIP in participants with PAH from studies INS1009-201 (NCT04791514), INS1009-202 (NCT05147805) and other lead-in studies of TPIP in participants with PAH.

Open-label Extension Study of Seralutinib in Adult Subjects With PAH (PROSERA-EXT) Recruiting
NCT06274801 Phase: 3 Started: 2024-09-03

This open-label extension study will evaluate the long-term safety, tolerability and efficacy of orally inhaled seralutinib in subjects who have completed a previous seralutinib study

A Study to Evaluate the Efficacy and Safety of Once Daily Treprostinil Palmitil Inhalation Powder (TPIP) in Participants With Pulmonary Arterial Hypertension (PAH) Recruiting
NCT07481981 Phase: 3 Started: 2026-07-21

The primary objective of this study is to evaluate the effect of 24-weeks of once daily treatment with TPIP compared with placebo on exercise capacity in adults with PAH.

A Clinical Study of Sotatercept (MK-7962) in People With Pulmonary Arterial Hypertension (MK-7962-038) Active Not Recruiting
NCT07218029 Phase: 3 Started: 2021-05-12

Researchers are looking for more ways to treat PAH. In PAH, the blood vessels in the lungs become thick and narrow, which makes it harder for blood to flow. This causes high blood pressure in the lungs and overworks the heart. PAH can make it hard to breathe and be active. Some standard (usual) trea...

A Study of ROC-101 in Patients With Pulmonary Arterial Hypertension (PAH) and Pulmonary Hypertension Associated With Interstitial Lung Disease (ILD-PH) (ROCSTAR STUDY) Recruiting
NCT07175038 Phase: 2 Started: 2025-10-01

This study evaluates the effect of ROC-101 in adults with either Pulmonary Arterial Hypertension (PAH) or Pulmonary Hypertension Associated with Interstitial Lung Disease (ILD-PH). Each eligible participant will receive standard of care (SOC) plus ROC-101 for a 24-week treatment period, followed by ...

Outcome Study Assessing a 75 Milligrams (mg) Dose of Macitentan in Patients With Pulmonary Arterial Hypertension Active Not Recruiting
NCT04273945 Phase: 3 Started: 2020-06-30

The purpose of this study is to demonstrate superiority of macitentan 75 milligrams (mg) in prolonging the time to the first clinical events committee (CEC)-adjudicated morbidity or mortality (M/M) event in participants with symptomatic pulmonary arterial hypertension (PAH) compared to macitentan 10...

A Study of Sotatercept (MK-7962) in Japanese Children With Pulmonary Arterial Hypertension (PAH) (MK-7962-032) Recruiting
NCT07646860 Phase: 2 Started: 2026-09-17

The goal of this study is to learn about the safety of sotatercept and how well Japanese children tolerate it, when taken along with standard (usual) pulmonary arterial hypertension (PAH) treatment. Researchers also want to learn what happens to it in a person's body over time and whether it lowers ...

A Clinical Study of to Confirm the Doses of Selexipag in Children With Pulmonary Arterial Hypertension Active Not Recruiting
NCT03492177 Phase: 2 Started: 2018-07-23

The purpose of this study to confirm the selexipag starting dose(s), selected based on pharmacokinetic (PK) extrapolation from adults, that leads to similar exposure as adults doses in children from greater than or equal to (\>=) 2 to less than (˂) 18 years of age with Pulmonary Arterial Hypertensio...

The Effect of Additive On-Demand Sildenafil vs. Dextrose Candy on Maximum Exercise Capacity in Pulmonary Arterial Hypertension Recruiting
NCT07824674 Phase: 2/3 Started: 2026-09

The goal of this clinical study is to investigate whether the additional intake of sildenafil 20mg on top of standard medical treatment results in a benefit on maximum exercise workload (Wmax) in patients with pulmonary arterial hypertension (PAH).

A Study of HS235 for Treatment of Pulmonary Arterial Hypertension (PAH) in Adults Recruiting
NCT07143448 Phase: 1/2 Started: 2026-08-19

Phase 1b/2 Study of HS235 Assessing the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of the Activin Signaling Inhibitor HS235 Added to Background Treatment in Adults with Pulmonary Arterial Hypertension (PAH)

An Adaptive Program of IKT-001 in Pulmonary Arterial Hypertension (PAH) Recruiting
NCT07365332 Phase: 3 Started: 2026-04-23

This is an adaptive, 2-part, randomized, multicenter, double-blind, placebo-controlled, parallel-group study designed to evaluate the efficacy and safety of IKT-001 in adult participants with WHO Group 1 PAH.

Empagliflozin to Improve Right Ventricular Function in Pulmonary Arterial Hypertension Recruiting
NCT06992440 Phase: 2 Started: 2025-06-26

Randomized, triple-masked, parallel arm clinical trial of empagliflozin versus placebo in pulmonary arterial hypertension (PAH) participants on stable approved PAH-targeted medical therapy.

Agents Found By Disease-Level Search

Searched directly from “Pulmonary Arterial Hypertension (PAH)” 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).

54 agents shown (approved or with trial evidence) — 47 not found by the gene-target search above.

Sildenafil citrate

Approved · 3 trials · source: ClinicalTrials.gov, Open Targets
NCT00159913 NCT00159913 NCT00159913

BIA 5-1058NOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT04675944 NCT03489005

Sildenafil

Phase 3 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT02951429 NCT01392469

FrespaciguatNOT in gene-target list

Phase 2/3 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT04732221 NCT03744637

Bosentan

Approved · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT00319267 NCT01392469

ClevidipineNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01121458

RosuvastatinNOT in gene-target list

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00984932

HGP1206NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01864863

TraclearNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01864863

NitriteNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01932606

selexipag (Uptravi)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03492177

Iloprost

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01274481

PB1046 Subcutaneous InjectionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03315507

Blood pressureNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05169411

Urine proteinNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05169411

1 dose of sildenafilNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01670136

treprostinil

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT04675944

inhaled Nitric OxideNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00732537

Oxygen (>90% by hood) - standard therapyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00732537

L606 inhalation suspensionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04691154

LCAR-AIO T cellsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06653556

eNOS transfected EPCs will be delivered via a PA lineNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00469027

TPN171HNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05948644

Selexipag Film Coated TabletsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06932198

Uptravi® Film Coated TabletsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06932198

PirfenidoneNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02951429

EXOMIC small RNA sequencingNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07224737

EXOMIC assay (qRT-PCR validation)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07224737

Dual-energy computed tomography investigationNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01829672

Inspiratory muscle trainerNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04919031

CarvedilolNOT in gene-target list

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01586156

Nitric Oxide

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT02734953

High-TouchNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03451630

High-TechNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03451630

Optimal Discharge PlanningNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03451630

CPAP therapyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04769635

DHEA tabletNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03648385

Iloprost (Ventavis, BAYQ 6256)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01894035

Moderate altitude sojournNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02143687

Low altitude sojournNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02143687

OxygenNOT in gene-target list

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT02143687

POC-USNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05332847

Opsumit (macitentan)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02126943

acetazolamideNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00424970

Imatinib mesylate

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00477269

HS235NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07143448

Impedance CardiographyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03876509

levosimendan at a dose of 3 mcg/kgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04599816

levosimendan at a dose of 6 mcg/kgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04599816

levosimendan at a dose of 12 mcg/kgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04599816

Balloon pulmonary angioplastyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04206852

Nebulized nitroglycerinNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07214129

Hymecromone (H01)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05128929

CyclophosphamideNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03630211

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