Biomarker & Intervention Discovery

Parkinson's Disease

This page lists 7 druggable gene targets and 15 recent clinical trials for Parkinson's Disease, with each agent ranked by the strength of its evidence (clinical, mechanistic or correlative). Sources are linked so you can check every claim.

Disease Overview

Spontaneous remission
Not established
Best-intervention remission
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Gap size
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Primary barrier
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Biomarker Targets & Therapeutic Agents

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

DRD2 427GBA 156LRRK2 206MAOB 218SLC6A3 176SNCA 221TH 29
Therapeutic Agent Effect Direction Evidence Tier Potency Source
ASENAPINE ? unclear Mechanistic
MEPHENTERMINE ? unclear Mechanistic
HALOPERIDOL LACTATE ↓ inhibits Mechanistic
BREXPIPRAZOLE ↑ activates Mechanistic
ANALGESIC AGENT ? unclear Mechanistic
AXITINIB ↓ inhibits Mechanistic
RG-1530 ↓ inhibits Mechanistic
LOXAPINE SUCCINATE ↓ inhibits Mechanistic
QUETIAPINE FUMARATE ↓ inhibits Mechanistic
PROCHLORPERAZINE ↓ inhibits Mechanistic
APOMORPHINE ↑ activates Mechanistic
PRAMIPEXOLE ? unclear Mechanistic
THIETHYLPERAZINE MALEATE ↓ inhibits Mechanistic
IBCASERTIB ↓ inhibits Mechanistic
FAMITINIB ↓ inhibits Mechanistic
SOXATALTINIB ↓ inhibits Mechanistic
SUMANIROLE ↑ activates Mechanistic 9.0 nM (Ki)
DOCARPAMINE ? unclear Mechanistic
PALIPERIDONE ↓ inhibits Mechanistic
PRIDOPIDINE ~ modulates Mechanistic
OLANZAPINE ↓ inhibits Mechanistic 20.0 nM (Ki)
PEGDINETANIB ↓ inhibits Mechanistic
RISPERIDONE ↓ inhibits Mechanistic
PERGOLIDE MESYLATE ↑ activates Mechanistic
LISURIDE ? unclear Mechanistic 0.5 nM (Ki)
ZICRONAPINE ? unclear Mechanistic
CLEBOPRIDE ↓ inhibits Mechanistic
LAZERTINIB ↓ inhibits Mechanistic
REMOXIPRIDE HYDROCHLORIDE ↓ inhibits Mechanistic
CABERGOLINE ↑ activates Mechanistic
JNJ-37822681 ↓ inhibits Mechanistic
ERGOT ALKALOIDS ? unclear Mechanistic
(-)-STEPHOLIDINE ? unclear Mechanistic
SARIZOTAN HYDROCHLORIDE ↑ activates Mechanistic
5-OH-DPAT ? unclear Mechanistic
LITHIUM ? unclear Mechanistic
DIHYDROERGOTOXINE ? unclear Mechanistic
REMOXIPRIDE ↓ inhibits Mechanistic
BROMPERIDOL ↓ inhibits Mechanistic
PHENTOLAMINE ? unclear Mechanistic

Showing top 40 of 427 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
NCGC607 ? unclear Mechanistic
AFEGOSTAT ? unclear Mechanistic 60.0 nM (IC50)
GENTIAN VIOLET CATION ? unclear Mechanistic
VELAGLUCERASE ALFA ? unclear Mechanistic
RALOXIFENE HYDROCHLORIDE ? unclear Mechanistic
CIRSIMARITIN ? unclear Mechanistic
DOMIPHEN BROMIDE ? unclear Mechanistic
CHEMBL:CHEMBL1169388 ? unclear Mechanistic
2,3-DIHYDROXY-6,7-DICHLOROQUINOXALINE ? unclear Mechanistic
CHEMBL:CHEMBL579837 ? unclear Mechanistic
METITEPINE ? unclear Mechanistic
TALIGLUCERASE ALFA ? unclear Mechanistic
APTIGANEL HYDROCHLORIDE ? unclear Mechanistic
CHEMBL:CHEMBL588327 ? unclear Mechanistic
MIGALASTAT ? unclear Mechanistic
CHLORHEXIDINE GLUCONATE ? unclear Mechanistic
CHEMBL:CHEMBL528181 ? unclear Mechanistic
CHEMBL:CHEMBL1379900 ? unclear Mechanistic
L 755507 ? unclear Mechanistic
CHEMBL:CHEMBL73711 ? unclear Mechanistic
DNQX ? unclear Mechanistic
CHEMBL:CHEMBL408500 ? unclear Mechanistic
CASTANOSPERMINE ? unclear Mechanistic
SULFANITRAN ? unclear Mechanistic
AMBROXOL ? unclear Mechanistic
CHEMBL:CHEMBL578928 ? unclear Mechanistic
PHLORETIN ? unclear Mechanistic
GENISTEIN ? unclear Mechanistic
CHEMBL:CHEMBL603154 ? unclear Mechanistic
AFEGOSTAT TARTRATE ~ modulates Mechanistic
CHEMBL:CHEMBL529939 ? unclear Mechanistic
ISOLIQUIRITIGENIN ? unclear Mechanistic
TRICHOSTATIN A ? unclear Mechanistic
EBSELEN ? unclear Mechanistic
CHEMBL3350088 ↓ inhibits Mechanistic 150.0 nM (Ki)
CHEMBL3350093 ↓ inhibits Mechanistic 550.0 nM (Ki)
CHEMBL3350086 ↓ inhibits Mechanistic 25.1 nM (Ki)
CHEMBL3350089 ↓ inhibits Mechanistic 100.0 nM (Ki)
CHEMBL3350092 ↓ inhibits Mechanistic 1.2 nM (Ki)
CHEMBL469844 ↓ inhibits Mechanistic 200.0 nM (Ki)

Showing top 40 of 156 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
LRRK2 INHIBITOR 6 [PMID: 40353293] ↓ inhibits Mechanistic
LIRADASERTIB ↓ inhibits Mechanistic
ADAVOSERTIB ? unclear Mechanistic
PF-562271 ? unclear Mechanistic
NIK INHIBITOR 12F ↓ inhibits Mechanistic
DCLK1-IN-1 ↓ inhibits Mechanistic
DACTOLISIB ? unclear Mechanistic
VANDETANIB ? unclear Mechanistic 1995.26 nM (Ki)
LRRK2-IN-1 ↓ inhibits Mechanistic
DOVITINIB ? unclear Mechanistic 1500.0 nM (Kd)
BOS172722 ↓ inhibits Mechanistic
CHEMBL:CHEMBL2204495 ? unclear Mechanistic
TOZASERTIB ? unclear Mechanistic 630.96 nM (Ki)
MLI-2 ↓ inhibits Mechanistic
PALBOCICLIB ? unclear Mechanistic 2511.89 nM (Ki)
ENTRECTINIB ? unclear Mechanistic 2511.89 nM (Ki)
HG-10-102-01 ↓ inhibits Mechanistic
RG-1530 ? unclear Mechanistic
CHEMBL:CHEMBL379975 ? unclear Mechanistic
GSK2646264 ↓ inhibits Mechanistic
PD166285 ? unclear Mechanistic
GNE-9605 ↓ inhibits Mechanistic
PELERASERTIB ↓ inhibits Mechanistic
ALISERTIB ? unclear Mechanistic 794.33 nM (Ki)
AST-487 ? unclear Mechanistic 950.0 nM (Kd)
PF-06447475 ↓ inhibits Mechanistic
URMC-099 ↓ inhibits Mechanistic
PICTILISIB ? unclear Mechanistic
NVP-TAE684 ? unclear Mechanistic
XMD-12 ↓ inhibits Mechanistic
EB-42486 ↓ inhibits Mechanistic
PF-06454589 ↓ inhibits Mechanistic
NIK SMI1 ↓ inhibits Mechanistic
JAK3 INHIBITOR VI ↓ inhibits Mechanistic
TTT-3002 ↓ inhibits Mechanistic
XL01126 ↓ inhibits Mechanistic
COMPOUND 8 [PMID: 17480064] ? unclear Mechanistic
ILORASERTIB ? unclear Mechanistic
CENISERTIB ? unclear Mechanistic 0.631 nM (Ki)
FASUDIL ? unclear Mechanistic 1995.26 nM (Ki)

Showing top 40 of 206 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
SAFINAMIDE ↓ inhibits Mechanistic 450.0 nM (Ki)
LEVODOPA ? unclear Mechanistic
CHEMBL:CHEMBL1927670 ? unclear Mechanistic
MOCLOBEMIDE ↓ inhibits Mechanistic 100000.0 nM (Ki)
CHEMBL:CHEMBL1927684 ? unclear Mechanistic
SELEGILINE ↓ inhibits Mechanistic 970.0 nM (Ki)
BIFEMELANE ↓ inhibits Mechanistic
MEBANAZINE ↓ inhibits Mechanistic
MELATONIN ? unclear Mechanistic
PARGYLINE HYDROCHLORIDE ↓ inhibits Mechanistic
CHEMBL:CHEMBL602260 ? unclear Mechanistic
ISOCARBOXAZID ↓ inhibits Mechanistic
SEROTONIN ? unclear Mechanistic
[18F]FLUORORASAGILINE ? unclear Mechanistic
RASAGILINE ↓ inhibits Mechanistic 700.0 nM (Ki)
CHEMBL:CHEMBL1642672 ? unclear Mechanistic
PHENOXYPROPAZINE ↓ inhibits Mechanistic
LAZABEMIDE ↓ inhibits Mechanistic 84.0 nM (Ki)
NIALAMIDE ↓ inhibits Mechanistic
CHEMBL:CHEMBL1813518 ? unclear Mechanistic
SAFINAMIDE MESYLATE ↓ inhibits Mechanistic
COBALAMIN ? unclear Mechanistic
PHENELZINE ↓ inhibits Mechanistic
RASAGILINE TARTRATE ↓ inhibits Mechanistic
RALFINAMIDE ↓ inhibits Mechanistic
SELEGILINE HYDROCHLORIDE ↓ inhibits Mechanistic
VITAMIN K3 ? unclear Mechanistic
CHEMBL:CHEMBL2088303 ? unclear Mechanistic
TRANYLCYPROMINE ↓ inhibits Mechanistic
PARGYLINE ? unclear Mechanistic 1800.0 nM (Ki)
ANTIDEPRESSANT AGENT ? unclear Mechanistic
MOFEGILINE ? unclear Mechanistic
INDANTADOL ? unclear Mechanistic
NAPHTHOQUINONE ? unclear Mechanistic
METHAMPHETAMINE ? unclear Mechanistic
HALOPERIDOL DECANOATE ? unclear Mechanistic
PHENELZINE SULFATE ↓ inhibits Mechanistic
IPRONIAZID ↓ inhibits Mechanistic 7540.0 nM (IC50)
LINEZOLID ↓ inhibits Mechanistic
CHEMBL:CHEMBL602466 ? unclear Mechanistic

Showing top 40 of 218 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
ALTROPANE I-123 ? unclear Mechanistic
BUPROPION HYDROCHLORIDE ↓ inhibits Mechanistic
DEXMETHYLPHENIDATE ↓ inhibits Mechanistic
AMPHETAMINE ADIPATE ? unclear Mechanistic
DEXTROAMPHETAMINE SACCHARATE ? unclear Mechanistic
LIAFENSINE ↓ inhibits Mechanistic
LISDEXAMFETAMINE DIMESYLATE ? unclear Mechanistic
METHAMPHETAMINE ↓ inhibits Mechanistic
SIBUTRAMINE ? unclear Mechanistic
METHYLPHENIDATE HYDROCHLORIDE ↓ inhibits Mechanistic
3,4-METHYLENEDIOXYMETHAMPHETAMINE ? unclear Mechanistic
LEVODOPA ? unclear Mechanistic
DEXTROAMPHETAMINE ADIPATE ? unclear Mechanistic
METYROSINE ? unclear Mechanistic
ANESTHETIC AGENT ? unclear Mechanistic
SERTRALINE HYDROCHLORIDE ? unclear Mechanistic
BENZTROPINE ? unclear Mechanistic 312.0 nM (IC50)
BICIFADINE ↓ inhibits Mechanistic
ALTROPANE ? unclear Mechanistic
DASOTRALINE ↓ inhibits Mechanistic
DISULFIRAM ? unclear Mechanistic
AMITIFADINE HYDROCHLORIDE ? unclear Mechanistic
AMPHETAMINE ASPARTATE ? unclear Mechanistic
NICOTINE POLACRILEX ? unclear Mechanistic
SPIROGLUMIDE ? unclear Mechanistic
HINOKININ ? unclear Mechanistic
DIETHYLPROPION HYDROCHLORIDE ↓ inhibits Mechanistic
SERDEXMETHYLPHENIDATE CHLORIDE ↓ inhibits Mechanistic
PHENTERMINE RESIN ? unclear Mechanistic
PSEUDOEPHEDRINE ? unclear Mechanistic
PHENMETRAZINE ? unclear Mechanistic
CENTANAFADINE ↓ inhibits Mechanistic
BUPRENORPHINE ? unclear Mechanistic
PYROVALERONE HYDROCHLORIDE ↓ inhibits Mechanistic
SERDEXMETHYLPHENIDATE ↓ inhibits Mechanistic
PHENMETRAZINE HYDROCHLORIDE ↓ inhibits Mechanistic
FENCAMFAMIN ? unclear Mechanistic
IOFLUPANE I-123 ? unclear Mechanistic
PHENDIMETRAZINE ↓ inhibits Mechanistic
MAZINDOL ↓ inhibits Mechanistic 42.6 nM (IC50)

Showing top 40 of 176 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
CHEMBL:CHEMBL1521156 ? unclear Mechanistic
CHEMBL:CHEMBL599100 ? unclear Mechanistic
DOXORUBICIN HYDROCHLORIDE ? unclear Mechanistic
LEVOKETOCONAZOLE ? unclear Mechanistic
CHEMBL:CHEMBL450802 ? unclear Mechanistic
CALCDPWW ? unclear Mechanistic
CHEMBL:CHEMBL580727 ? unclear Mechanistic
PRASINEZUMAB ? unclear Mechanistic
CHEMBL:CHEMBL505670 ? unclear Mechanistic
CONGO RED FREE ACID ? unclear Mechanistic
CHEMBL:CHEMBL548540 ? unclear Mechanistic
GARDENIN ? unclear Mechanistic
6-AZAURIDINE ? unclear Mechanistic
PARAROSANILINE ? unclear Mechanistic
CHEMBL:CHEMBL534353 ? unclear Mechanistic
CHEMBL:CHEMBL2094833 ? unclear Mechanistic
GENTIAN VIOLET CATION ? unclear Mechanistic
CHEMBL:CHEMBL2094567 ? unclear Mechanistic
2-CHLOROADENOSINE-5-TRIPHOSPHATE ? unclear Mechanistic
CHEMBL:CHEMBL442906 ? unclear Mechanistic
CHEMBL:CHEMBL1534035 ? unclear Mechanistic
CHEMBL:CHEMBL1526167 ? unclear Mechanistic
PP2 ? unclear Mechanistic
MYCOPHENOLATE ? unclear Mechanistic
ALTENUSIN ? unclear Mechanistic
GW305074X ? unclear Mechanistic
INDENEBART ? unclear Mechanistic
ABT702 ? unclear Mechanistic
CINPANEMAB ↓ inhibits Mechanistic
LICARIN A ? unclear Mechanistic
LITHOCHOLIC ACID ? unclear Mechanistic
CAFFEIC ACID PHENETHYL ESTER ? unclear Mechanistic
CHEMBL1594514 ~ modulates Mechanistic
CHEMBL1411318 ~ modulates Mechanistic
CHEMBL1379677 ~ modulates Mechanistic
CHEMBL1476741 ~ modulates Mechanistic
CHEMBL1565212 ~ modulates Mechanistic
CHEMBL1331245 ~ modulates Mechanistic
CHEMBL1411120 ~ modulates Mechanistic
CHEMBL1459532 ~ modulates Mechanistic

Showing top 40 of 221 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
METYROSINE ↓ inhibits Mechanistic
STREPTOZOCIN ? unclear Mechanistic
SAPONIN ? unclear Mechanistic
PROSAVIN ? unclear Mechanistic
OPIATE ? unclear Mechanistic
FLUTAMIDE ? unclear Mechanistic
3-IODOBENZYLGUANIDINE ? unclear Mechanistic
PRASTERONE ? unclear Mechanistic
METHYLPHENIDATE ? unclear Mechanistic
RECOMBINANT INTERFERON ? unclear Mechanistic
SERTRALINE HYDROCHLORIDE ? unclear Mechanistic
INDOMETHACIN ? unclear Mechanistic
3-IODOTYROSINE ↓ inhibits Mechanistic
3-CHLOROTYROSINE ↓ inhibits Mechanistic
SAPROPTERIN ? unclear Mechanistic
DOXYCYCLINE ANHYDROUS ? unclear Mechanistic
TRIAMTERENE ? unclear Mechanistic
ALPHA-TOCOPHEROL ? unclear Mechanistic
Α-PROPYLDOPACETAMIDE ↓ inhibits Mechanistic
TYROSINE ? unclear Mechanistic
PHENYLALANINE ? unclear Mechanistic
METHADONE HYDROCHLORIDE ? unclear Mechanistic
ANTIESTROGEN ? unclear Mechanistic
EPOETIN ALFA ? unclear Mechanistic
CHEMBL27559 ↓ inhibits Mechanistic 42000.0 nM (IC50)
CHEMBL330274 ↓ inhibits Mechanistic 1000.0 nM (IC50)
R-N-PROPYLNORAPOMORPHINE ↓ inhibits Mechanistic 300.0 nM (IC50)
CHEMBL283489 ↓ inhibits Mechanistic 87000.0 nM (IC50)
IOTYROSINE ↓ inhibits Mechanistic 720.0 nM (IC50)

Clinical Trials

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

NCT07055087 Phase: 2 Started: 2026-10

This is a proof-of-concept trial to investigate the efficacy of prasinezumab to slow or prevent cognitive decline in people with Parkinson's disease carrying a severe mutation in the GBA (glucocerebrosidase) gene. The duration of the intervention per patient will be 104 weeks with monthly infusions....

NCT07630545 Phase: 1 Started: 2023-11-30

The goal of this trial is to learn if MTX325 can be developed as a potential disease-modifying treatment for Parkinson's Disease. Parts 1-4 complete, Part 5 (multiple doses in patients with Parkinson's Disease) in progress.

NCT06068465 Phase: 3 Started: 2023-09-27

The purpose of this study is to evaluate the safety and efficacy of 34 mg pimavanserin compared to placebo in patients with Parkinson's disease psychosis (PDP).

NCT06565195 Phase: 1 Started: 2024-08-27

The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics/pharmacodynamics (PK/PD) of LY3962681 in healthy volunteers and patients with Parkinson's disease. The study consists of two parts, the Single Ascending Dose (SAD) study and the Multiple Ascending Dose (MAD) stud...

NCT07174310 Phase: 3 Started: 2025-11-24

The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics (PK) of prasinezumab compared with placebo in participants with early-stage Parkinson's disease (PD) on stable symptomatic monotherapy with levodopa.

NCT07080775 Phase: 1/2 Started: 2025-08-05

This Phase I clinical trial is designed to evaluate the safety, tolerability of a single dose of XS411 (derived dopaminergic neural progenitor cell injection) in patients with primary Parkinson's disease. This Phase II clinical trial is designed to evaluate the safety, tolerability and Efficacy of X...

NCT07216066 Phase: 1 Started: 2025-12-05

This study is researching an experimental drug called ALN-SNCA (called "study drug"). The study is focused on people with early Parkinson's disease, a disorder of the nervous system that affects movement. Parkinson's disease is caused by a gradual loss of nerve cells in the brain, especially those d...

NCT07284784 Phase: 2/3 Started: 2026-01-09

This study will examine the long-term safety of buntanetap in participants with PD. This will be a 36-month open-label safety study. This study will be conducted with two cohorts. Cohort 1 will enroll via invitation only for PD participants who have previously participated in buntanetap clinical tri...

NCT02719496 Phase: 2 Started: 2016-04

The main objective of this study is to evaluate the efficacy of STW5 Iberogast, over a 28-day period, for the treatment of constipation in parkinsonian patients suffering from gastrointestinal disorders.

NCT03100149 Phase: 2 Started: 2017-06-27

This multicenter, randomized, double-blind, placebo-controlled, Phase 2 study will evaluate the efficacy of intravenous prasinezumab (RO7046015/PRX002) versus placebo over 52 weeks in participants with early Parkinson's Disease (PD) who are untreated or treated with monoamine oxidase B (MAO-B) inhib...

NCT07777484 Phase: 1 Started: 2026-07-28

An unblinded, single arm, pilot observational study to test the safety, tolerability, immunogenicity and the biological effects of TRB-001 vaccination in individuals who have previously undergone aSyn immunotherapy

NCT07514858 Phase: 2 Started: 2026-07-28

This trial will evaluate the effects of Lu AF28996 in adults with Parkinson's disease (PD) experiencing motor fluctuations despite optimized non-invasive symptomatic treatment. These are individuals with PD who take medications to control movement symptoms, such as slowness, stiffness, and tremor, b...

NCT04777331 Phase: 2 Started: 2021-05-05

This is a multicenter, randomized, double-blind, placebo-controlled study that will evaluate the efficacy and safety of intravenous (IV) prasinezumab versus placebo in participants with Early Parkinson's Disease (PD) who are on stable symptomatic PD medication.

NCT04506073 Phase: 2 Started: 2020-11-09

The purpose of this study is to select the safest and most effective number of repeat doses of allogeneic bone marrow-derived mesenchymal stem cell (MSC) infusions to slow the progression of Parkinson's disease (PD).

NCT07774312 Phase: N/A Started: 2026-07-27

The goal of this clinical trial is to compare the efficacy and safety of different Intervention Intervals of subthalamic nucleus-targeted transcranial temporal interference stimulation (STN-tTIS) in patients with Parkinson disease. Previous studies suggest that STN-tTIS may improve motor symptoms in...


Agents Found By Disease-Level Search

Searched directly from “Parkinson's Disease” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 7-gene target panel above. This surfaces agents whose mechanism doesn't route through a curated gene (combination therapies, standard-of-care drugs, targets outside the panel).

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