Biomarker & Intervention Discovery

Sickle Cell Disease

This page lists 10 druggable gene targets, 51 approved or trial-tested drug candidates and 15 recent clinical trials for Sickle Cell Disease, 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 Sickle Cell Disease (2026)

US DALYs46.6KWHO GHE 2021 · all ages
Global DALYs4.74MWHO GHE 2021 · worldwide
US mortality720 deaths/yrWHO GHE 2021
Global mortality33.3K deaths/yrWHO GHE 2021 · worldwide
NIH funding$155MFY2025 · RCDC estimate
Funding levelWell-Funded
Spontaneous remissionNo spontaneous remission; progressive organ damage; median survival historically 40-60 years but improving…
Best-intervention remissionHaematopoietic stem cell transplant (HSCT): curative in 90-95% if matched sibling donor available; hydroxyurea reduces…
Gap sizeVery large — HSCT is curative but only 20% have matched donors; gene therapy is curative but costs $2-3M; hydroxyurea…
Primary barrieraccess
Full remission profile →
Research Tracker Chronic Disease Interventions Sickle Cell Disease

Remission & chronicity

Spontaneous remission
No spontaneous remission; progressive organ damage; median survival historically 40-60 years but improving; vaso-occlusive crises (VOC) in 1-4 per year average
Best-intervention remission
Haematopoietic stem cell transplant (HSCT): curative in 90-95% if matched sibling donor available; hydroxyurea reduces VOC frequency 50%, ACS risk 50%; gene therapy (exagamglogene autotemcel [exa-cel]/Casgevy): 30/31 patients free from severe VOC through 24 months; betibeglogene (Zynteglo): 90% reduction in severe VOC
Gap size
Very large — HSCT is curative but only 20% have matched donors; gene therapy is curative but costs $2-3M; hydroxyurea is dramatically underprescribed
Primary barrier
access
Hydroxyurea is inexpensive, highly effective, and generic but prescribed to only 25-35% of eligible SCD patients (racial and healthcare access disparities prominent); gene therapies (Casgevy, Zynteglo) approved December 2023 represent first curative options without matched donor — but $2-3M price tag makes access essentially impossible without negotiation

Biomarker Targets & Therapeutic Agents

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

BCL11A 1HBA1 2HBB 176HBG1 1HBG2 2HMOX1 33NOS3 0PDE9A 168PKLR 119SELP 143

BCL11A

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

HBA1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
VOXELOTOR ~ modulates Mechanistic DGIdb
EFAPROXIRAL ~ modulates Mechanistic DGIdb

HBB

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
MERCAPTOPURINE ? unclear Mechanistic DGIdb
THIOGUANINE ? unclear Mechanistic DGIdb
5-FLUOROURIDINE ? unclear Mechanistic DGIdb
CAMPTOTHECIN ? unclear Mechanistic DGIdb
HYDROXYCAMPTOTHECIN ? unclear Mechanistic DGIdb
VOXELOTOR ~ modulates Mechanistic DGIdb
LOVOTIBEGLOGENE AUTOTEMCEL ? unclear Mechanistic DGIdb
TOPOTECAN HYDROCHLORIDE ? unclear Mechanistic DGIdb
PHENYLMERCURIC ACETATE ? unclear Mechanistic DGIdb
8-AZAGUANINE ? unclear Mechanistic DGIdb
CRIZANLIZUMAB ? unclear Mechanistic DGIdb
THIOINOSINE ? unclear Mechanistic DGIdb
AZATHIOPRINE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL374632 ? unclear Mechanistic DGIdb
AES-103 ? unclear Mechanistic DGIdb
NEBULARINE ? unclear Mechanistic DGIdb
FLUOROURACIL ? unclear Mechanistic DGIdb
MERCURIC CHLORIDE ? unclear Mechanistic DGIdb
LUSPATERCEPT ? unclear Mechanistic DGIdb
PHENAZOPYRIDINE HYDROCHLORIDE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL458765 ? unclear Mechanistic DGIdb
EFAPROXIRAL ~ modulates Mechanistic DGIdb
BETIBEGLOGENE AUTOTEMCEL ? unclear Mechanistic DGIdb
(3Z)-N,N-DIMETHYL-2-OXO-3-(4,5,6,7-TETRAHYDRO-1H-INDOL-2-YLMETHYLIDENE)-2,3-DIHYDRO-1H-INDOLE-5-SULFONAMIDE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL515505 ? unclear Mechanistic DGIdb
QUINACRINE DIHYDROCHLORIDE ? unclear Mechanistic DGIdb
AZACITIDINE ? unclear Mechanistic DGIdb
VITAMIN K3 ? unclear Mechanistic DGIdb
ELLAGIC ACID ? unclear Mechanistic DGIdb
ELLIPTECINE ? unclear Mechanistic DGIdb
CHEMBL1514486 ~ modulates Mechanistic ChEMBL
BAICALEIN ~ modulates Mechanistic ChEMBL
CHEMBL1450895 ~ modulates Mechanistic ChEMBL
ISOGINKGETIN ~ modulates Mechanistic ChEMBL
CHEMBL2069121 ~ modulates Mechanistic ChEMBL
TRICETIN ~ modulates Mechanistic ChEMBL
CHEMBL1355522 ~ modulates Mechanistic ChEMBL
MERCAPTOPURINE ANHYDROUS ~ modulates Mechanistic ChEMBL
CURCUMIN ~ modulates Mechanistic ChEMBL
CHEMBL1572280 ~ modulates Mechanistic ChEMBL

Showing top 40 of 176 agents ranked by evidence tier.

HBG1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CHEMBL5653589 ↓ inhibits Mechanistic 330.13 nM (Kd) ChEMBL

HBG2

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
HYDROXYUREA ? unclear Mechanistic DGIdb
CHEMBL5653589 ↓ inhibits Mechanistic 1856.4 nM (Kd) ChEMBL

HMOX1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
SORAFENIB ? unclear Mechanistic DGIdb
SELENIUM ? unclear Mechanistic DGIdb
SUNITINIB ? unclear Mechanistic DGIdb
COMPOUND 1 [PMID: 16821802] ↓ inhibits Mechanistic DGIdb
ASPIRIN ? unclear Mechanistic DGIdb
RECOMBINANT NEUROTROPHIC FACTOR ? unclear Mechanistic DGIdb
STANNSOPORFIN ? unclear Mechanistic DGIdb
D VITAMIN ? unclear Mechanistic DGIdb
ZINC CHLORIDE ? unclear Mechanistic DGIdb
THERAPEUTIC HORMONE ? unclear Mechanistic DGIdb
CHEMBL536056 ↓ inhibits Mechanistic 1500.0 nM (IC50) ChEMBL
CHEMBL3621940 ↓ inhibits Mechanistic 2600.0 nM (IC50) ChEMBL
CHEMBL539857 ↓ inhibits Mechanistic 1900.0 nM (IC50) ChEMBL
CHEMBL4161975 ↓ inhibits Mechanistic 60.0 nM (IC50) ChEMBL
CHEMBL4456149 ↓ inhibits Mechanistic 250.0 nM (IC50) ChEMBL
CHEMBL4541767 ↓ inhibits Mechanistic 900.0 nM (IC50) ChEMBL
CHEMBL4442308 ↓ inhibits Mechanistic 54000.0 nM (IC50) ChEMBL
CHEMBL5614205 ↓ inhibits Mechanistic 648.0 nM (Kd) ChEMBL
CHEMBL5612877 ↓ inhibits Mechanistic 5100.0 nM (Kd) ChEMBL
CHEMBL5613041 ↓ inhibits Mechanistic 141.0 nM (Kd) ChEMBL
CHEMBL5612022 ↓ inhibits Mechanistic 1420.0 nM (Kd) ChEMBL
CHEMBL5612456 ↓ inhibits Mechanistic 11000.0 nM (Kd) ChEMBL
CHEMBL5613017 ↓ inhibits Mechanistic 7590.0 nM (Kd) ChEMBL
CHEMBL5613326 ↓ inhibits Mechanistic 9290.0 nM (Kd) ChEMBL
CHEMBL5613267 ↓ inhibits Mechanistic 794.0 nM (Kd) ChEMBL
CHEMBL5614291 ↓ inhibits Mechanistic 7930.0 nM (Kd) ChEMBL
CHEMBL5613955 ↓ inhibits Mechanistic 1840.0 nM (Kd) ChEMBL
CHEMBL5613612 ↓ inhibits Mechanistic 703.0 nM (Kd) ChEMBL
CHEMBL5613604 ↓ inhibits Mechanistic 692.0 nM (Kd) ChEMBL
CHEMBL5614120 ↓ inhibits Mechanistic 6920.0 nM (Kd) ChEMBL
CHEMBL5613507 ↓ inhibits Mechanistic 17100.0 nM (Kd) ChEMBL
CHEMBL5613257 ↓ inhibits Mechanistic 3790.0 nM (Kd) ChEMBL
CHEMBL5612106 ↓ inhibits Mechanistic 19800.0 nM (Kd) ChEMBL

NOS3

UniProt Ensembl DGIdb Open Targets

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

PDE9A

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
PENTOXIFYLLINE ↓ inhibits Mechanistic DGIdb
TOVINONTRINE ↓ inhibits Mechanistic DGIdb
SCH51866 ↓ inhibits Mechanistic DGIdb
EDELINONTRINE ↓ inhibits Mechanistic 8.3 nM (IC50) DGIdb
IRSENONTRINE ↓ inhibits Mechanistic DGIdb
DIPYRIDAMOLE ↓ inhibits Mechanistic DGIdb
ZAPRINAST ↓ inhibits Mechanistic 35000.0 nM (Ki) DGIdb
OSORESNONTRINE ↓ inhibits Mechanistic DGIdb
SILDENAFIL ↓ inhibits Mechanistic 3400.0 nM (IC50) ChEMBL
VARDENAFIL ↓ inhibits Mechanistic 460.0 nM (IC50) ChEMBL
CHEMBL466474 ↓ inhibits Mechanistic 530.0 nM (IC50) ChEMBL
CHEMBL513129 ↓ inhibits Mechanistic 87.0 nM (IC50) ChEMBL
CHEMBL511564 ↓ inhibits Mechanistic 82.0 nM (IC50) ChEMBL
CHEMBL467282 ↓ inhibits Mechanistic 56.0 nM (IC50) ChEMBL
CHEMBL466480 ↓ inhibits Mechanistic 46.0 nM (IC50) ChEMBL
CHEMBL466068 ↓ inhibits Mechanistic 41.0 nM (IC50) ChEMBL
CHEMBL466475 ↓ inhibits Mechanistic 36.0 nM (IC50) ChEMBL
CHEMBL466481 ↓ inhibits Mechanistic 35.0 nM (IC50) ChEMBL
CHEMBL513304 ↓ inhibits Mechanistic 23.0 nM (IC50) ChEMBL
CHEMBL466680 ↓ inhibits Mechanistic 19.0 nM (IC50) ChEMBL
CHEMBL512790 ↓ inhibits Mechanistic 10.0 nM (IC50) ChEMBL
CHEMBL466280 ↓ inhibits Mechanistic 7.0 nM (IC50) ChEMBL
CHEMBL572934 ↓ inhibits Mechanistic 2.089 nM (IC50) ChEMBL
CHEMBL575745 ↓ inhibits Mechanistic 38.02 nM (IC50) ChEMBL
CHEMBL574449 ↓ inhibits Mechanistic 15.85 nM (IC50) ChEMBL
CHEMBL572517 ↓ inhibits Mechanistic 66.07 nM (IC50) ChEMBL
CHEMBL583064 ↓ inhibits Mechanistic 10.72 nM (IC50) ChEMBL
CHEMBL583914 ↓ inhibits Mechanistic 1.778 nM (IC50) ChEMBL
CHEMBL576952 ↓ inhibits Mechanistic 154.88 nM (IC50) ChEMBL
CHEMBL578033 ↓ inhibits Mechanistic 10.96 nM (IC50) ChEMBL
CHEMBL566300 ↓ inhibits Mechanistic 12.3 nM (IC50) ChEMBL
CHEMBL582856 ↓ inhibits Mechanistic 194.98 nM (IC50) ChEMBL
CHEMBL572747 ↓ inhibits Mechanistic 63.1 nM (IC50) ChEMBL
CHEMBL565667 ↓ inhibits Mechanistic 1.905 nM (IC50) ChEMBL
CHEMBL575791 ↓ inhibits Mechanistic 11.22 nM (IC50) ChEMBL
CHEMBL575775 ↓ inhibits Mechanistic 28.84 nM (IC50) ChEMBL
CHEMBL575790 ↓ inhibits Mechanistic 14.79 nM (IC50) ChEMBL
CHEMBL1094374 ↓ inhibits Mechanistic 88.0 nM (IC50) ChEMBL
CHEMBL1098370 ↓ inhibits Mechanistic 22.0 nM (IC50) ChEMBL
CHEMBL2178122 ↓ inhibits Mechanistic 7470.0 nM (IC50) ChEMBL

Showing top 40 of 168 agents ranked by evidence tier.

PKLR

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
MITAPIVAT ↑ activates Mechanistic DGIdb
METFORMIN ? unclear Mechanistic DGIdb
MITAPIVAT SULFATE ↑ activates Mechanistic DGIdb
ETAVOPIVAT ↑ activates Mechanistic DGIdb
CHEMBL1315893 ~ modulates Mechanistic ChEMBL
CHEMBL1601132 ~ modulates Mechanistic ChEMBL
CHEMBL1435361 ~ modulates Mechanistic ChEMBL
CHEMBL1356205 ~ modulates Mechanistic ChEMBL
CHEMBL3392411 ~ modulates Mechanistic ChEMBL
CHEMBL3392401 ~ modulates Mechanistic ChEMBL
CHEMBL1368101 ~ modulates Mechanistic ChEMBL
CHEMBL1082815 ~ modulates Mechanistic ChEMBL
CHEMBL1938904 ~ modulates Mechanistic ChEMBL
CHEMBL1329865 ~ modulates Mechanistic ChEMBL
CHEMBL1603814 ~ modulates Mechanistic ChEMBL
CHEMBL1438727 ~ modulates Mechanistic ChEMBL
CHEMBL1553485 ~ modulates Mechanistic ChEMBL
CHEMBL1565443 ~ modulates Mechanistic ChEMBL
CHEMBL1939081 ~ modulates Mechanistic ChEMBL
CHEMBL1557530 ~ modulates Mechanistic ChEMBL
CHEMBL1084625 ~ modulates Mechanistic ChEMBL
CHEMBL1410384 ~ modulates Mechanistic ChEMBL
CHEMBL1370593 ~ modulates Mechanistic ChEMBL
CHEMBL1402938 ~ modulates Mechanistic ChEMBL
CHEMBL1481562 ~ modulates Mechanistic ChEMBL
CHEMBL1317577 ~ modulates Mechanistic ChEMBL
CHEMBL1090777 ~ modulates Mechanistic ChEMBL
CHEMBL1373546 ~ modulates Mechanistic ChEMBL
CHEMBL1091501 ~ modulates Mechanistic ChEMBL
CHEMBL1088747 ~ modulates Mechanistic ChEMBL
CHEMBL1092462 ~ modulates Mechanistic ChEMBL
CHEMBL1088762 ~ modulates Mechanistic ChEMBL
CHEMBL1092463 ~ modulates Mechanistic ChEMBL
CHEMBL1089364 ~ modulates Mechanistic ChEMBL
CHEMBL1093061 ~ modulates Mechanistic ChEMBL
CHEMBL1092297 ~ modulates Mechanistic ChEMBL
CHEMBL1090775 ~ modulates Mechanistic ChEMBL
CHEMBL1091457 ~ modulates Mechanistic ChEMBL
CHEMBL1090774 ~ modulates Mechanistic ChEMBL
CHEMBL1093361 ~ modulates Mechanistic ChEMBL

Showing top 40 of 119 agents ranked by evidence tier.

SELP

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
BIMOSIAMOSE ↓ inhibits Mechanistic 95000.0 nM (IC50) DGIdb
PSI-697 ? unclear Mechanistic DGIdb
RPSGL-IG ? unclear Mechanistic DGIdb
INCLACUMAB ↓ inhibits Mechanistic DGIdb
CRIZANLIZUMAB ? unclear Mechanistic DGIdb
RG-1512 ↓ inhibits Mechanistic DGIdb
RIVIPANSEL ↓ inhibits Mechanistic DGIdb
SELECTIN P LIGAND ? unclear Mechanistic DGIdb
CY-1503 ? unclear Mechanistic DGIdb
CHEMBL2303881 ↓ inhibits Mechanistic 70000.0 nM (IC50) ChEMBL
CHEMBL305902 ↓ inhibits Mechanistic 10600.0 nM (IC50) ChEMBL
CHEMBL3215517 ↓ inhibits Mechanistic 2570.0 nM (IC50) ChEMBL
CHEMBL3215516 ↓ inhibits Mechanistic 450.0 nM (IC50) ChEMBL
CHEMBL175597 ↓ inhibits Mechanistic 44000.0 nM (IC50) ChEMBL
CHEMBL2303775 ↓ inhibits Mechanistic 2.0 nM (IC50) ChEMBL
CHEMBL7864 ↓ inhibits Mechanistic 500.0 nM (IC50) ChEMBL
CHEMBL3085443 ↓ inhibits Mechanistic 100000.0 nM (IC50) ChEMBL
CHEMBL310195 ↓ inhibits Mechanistic 85000.0 nM (IC50) ChEMBL
CHEMBL74108 ↓ inhibits Mechanistic 5000.0 nM (IC50) ChEMBL
CHEMBL78221 ↓ inhibits Mechanistic 39000.0 nM (IC50) ChEMBL
CHEMBL302139 ↓ inhibits Mechanistic 290.0 nM (IC50) ChEMBL
CHEMBL305599 ↓ inhibits Mechanistic 34000.0 nM (IC50) ChEMBL
CHEMBL63898 ↓ inhibits Mechanistic 3000.0 nM (IC50) ChEMBL
CHEMBL421697 ↓ inhibits Mechanistic 87000.0 nM (IC50) ChEMBL
CHEMBL292851 ↓ inhibits Mechanistic 100000.0 nM (IC50) ChEMBL
CHEMBL293171 ↓ inhibits Mechanistic 17000.0 nM (IC50) ChEMBL
CHEMBL307807 ↓ inhibits Mechanistic 76000.0 nM (IC50) ChEMBL
CHEMBL62853 ↓ inhibits Mechanistic 130.0 nM (IC50) ChEMBL
CHEMBL77654 ↓ inhibits Mechanistic 34000.0 nM (IC50) ChEMBL
CHEMBL308039 ↓ inhibits Mechanistic 41000.0 nM (IC50) ChEMBL
CHEMBL77658 ↓ inhibits Mechanistic 7000.0 nM (IC50) ChEMBL
CHEMBL306986 ↓ inhibits Mechanistic 18000.0 nM (IC50) ChEMBL
CHEMBL65395 ↓ inhibits Mechanistic 16000.0 nM (IC50) ChEMBL
CHEMBL77488 ↓ inhibits Mechanistic 15000.0 nM (IC50) ChEMBL
CHEMBL77834 ↓ inhibits Mechanistic 20000.0 nM (IC50) ChEMBL
CHEMBL305870 ↓ inhibits Mechanistic 28000.0 nM (IC50) ChEMBL
CHEMBL293833 ↓ inhibits Mechanistic 21000.0 nM (IC50) ChEMBL
CHEMBL306480 ↓ inhibits Mechanistic 32000.0 nM (IC50) ChEMBL
CHEMBL77499 ↓ inhibits Mechanistic 28000.0 nM (IC50) ChEMBL
CHEMBL2029056 ↓ inhibits Mechanistic 100000.0 nM (IC50) ChEMBL

Showing top 40 of 143 agents ranked by evidence tier.

Clinical Trials

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

A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD) Recruiting
NCT07656415 Phase: 3 Started: 2026-07-21

The primary objective of this study is to determine the effect of mitapivat versus placebo on the need for transfusions in subjects with SCD.

A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD) Active Not Recruiting
NCT06924970 Phase: 2 Started: 2025-05-01

The main purpose of this study is to compare the effect of tebapivat versus placebo on anemia and to detect a dose-response for hemoglobin (Hb) response in participants with SCD.

A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease Recruiting
NCT06546670 Phase: 1/2 Started: 2024-08-15

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512. This will be the first evaluation of the potential therapeutic effect of ITU512 in healthy participants a...

A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP) Active Not Recruiting
NCT05031780 Phase: 2/3 Started: 2022-02-11

This clinical trial is a Phase 2/3 study that will determine the recommended dose of mitapivat and evaluate the efficacy and safety of mitapivat in sickle cell disease by testing how well mitapivat works compared to placebo to increase the amount of hemoglobin in the blood and to reduce or prevent t...

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Sickle Cell Disease Recruiting
NCT07708350 Phase: 1 Started: 2026-08-01

A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly deoxyribonucleic acid (DNA)) from the patient to treat his or her own disease. In gene therapy, the investigators introduce new gen...

Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease Recruiting
NCT04774536 Phase: 1/2 Started: 2025-08-22

This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old severe Sickle Cell Disease (SCD). The study will e...

The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease Recruiting
NCT06975865 Phase: 3 Started: 2025-08-12

This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, flexible-adaptive, group-sequential study (Part A), followed by an open-label LTE period (Part B) to investigate the efficacy, and safety of rilzabrutinib in participants with sickle-cell disease (SCD). Study detail...

A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE) Recruiting
NCT06439082 Phase: 3 Started: 2024-10-24

A phase III, multi-center, randomized, placebo-controlled, double-blind study to assess efficacy and safety of crizanlizumab (5 mg/kg) versus placebo, with or without hydroxyurea/hydroxycarbamide therapy, in adolescent and adult Sickle Cell Disease patients with frequent vaso-occlusive crises.

Gene Editing For Sickle Cell Disease Recruiting
NCT06506461 Phase: 1 Started: 2025-03-21

This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin (HbF) and help reduce the sympto...

Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients Active Not Recruiting
NCT03814746 Phase: 3 Started: 2019-07-26

The purpose of this study is to compare the efficacy and safety of 2 doses of crizanlizumab (5.0 mg/kg and 7.5 mg/kg) versus placebo in adolescent and adult sickle cell disease (SCD) patients with history of vaso-occlusive crisis (VOC) leading to healthcare visit.

Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors Recruiting
NCT07599176 Phase: 1/2 Started: 2026-07-08

This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions. The intensity of the transplant is slightly increased from our previous transplant regimens. The goal is to aim for higher percentage of donor cells...

Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis Recruiting
NCT06699849 Phase: 2 Started: 2025-07-10

This is a phase 2, randomized, multiple-dose, placebo-controlled study designed to evaluate the safety, efficacy, and pharmacokinetics (PK) of CSL889 (human hemopexin) when given intravenously (IV) to adults and adolescents with sickle cell disease (SCD) experiencing vaso-occlusive crises (VOC). The...

A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD) Recruiting
NCT07187973 Phase: 1 Started: 2026-01-06

This is an open-label, multicenter, within-participant dose-escalation study examining up to 3 dose levels of DISC-3405 and will assess the safety, tolerability, PK, and PD of DISC 3405 in participants with sickle cell disease.

Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease Recruiting
NCT06578507 Phase: 2 Started: 2025-01-21

The purpose of this interventional, phase II, national, multicentric, non-randomised, open-label study is to evaluate the pharmacokinetics (PK), efficacy and safety of Hydroxycarbamide Paediatric dispersible tablets with a twice daily dosing regimen in children with Sickle Celle Disease between 9 mo...

A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease Recruiting
NCT04055818 Phase: 1 Started: 2020-01-24

A randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide).

Agents Found By Disease-Level Search

Searched directly from “Sickle Cell Disease” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 10-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).

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

hydroxyurea

Approved · 7 trials · source: ClinicalTrials.gov, Open Targets
NCT02149537 NCT00000602 NCT00004492

Crizanlizumab

Approved · 3 trials · source: ClinicalTrials.gov, Open Targets
NCT03474965 NCT03938454 NCT04662931

fludarabineNOT in gene-target list

Phase 3 · 3 trials · source: ClinicalTrials.gov, Open Targets
NCT03121001 NCT02065596 NCT01877837

osivelotor (GBT021601)NOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT05718687 NCT04983264

cyclophosphamideNOT in gene-target list

Phase 2 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT03121001 NCT02757885

L-arginineNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT02536170 NCT05470998

Codeine (30 mg)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00174538

rivipansel (GMI-1070)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00911495

Arginine hydrochlorideNOT in gene-target list

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00029731

ATGNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03121001

SirolimusNOT in gene-target list

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT03121001

mycophenolate mofetilNOT in gene-target list

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT03121001

peripheral blood stem cell graft that are CD34+ selectedNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02165007

Alemtuzumab, low dose total body irradiation, SirolimusNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03587272

SELFY (Self Management for Youth with SCD)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03069430

25-OH Vitamin D measureNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04694976

TicagrelorNOT in gene-target list

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT02482298

Thiotepa

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT02757885

Fludarabine monophosphateNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02757885

Vitamin ENOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03903133

HBI-002NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06144749

Sapropterin DihydrochlorideNOT in gene-target list

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00445978

N-AcetylcysteineNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01849016

SimvastatinNOT in gene-target list

Phase 1/2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01702246

panobinostatNOT in gene-target list

Phase 1 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01245179

Recombinant-methionyl human stem cell factorNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00005783

L-arginine Loading DoseNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02536170

HydroxycarbamidNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06578507

Nitric OxideNOT in gene-target list

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00001716

NitroglycerinNOT in gene-target list

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00001716

clotrimazoleNOT in gene-target list

Phase 1/2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00004492

NiCordNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01590628

DefibrotideNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03805581

Aspirin 100mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06417411

Intervention: Combined use of a respiratory broad panel multiplex PCR and procalcitoninNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03919266

Control: usual antibiotic treatmentNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03919266

RejuvesolNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02731157

lovo-celNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02140554

AlemtuzumabNOT in gene-target list

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01877837

MelphalanNOT in gene-target list

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01877837

Stem CellsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01877837

olinciguat (IW-1701)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03285178

laying-on-of-handsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01518218

SenicapocNOT in gene-target list

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT07282210

Mitapivat

Phase 2/3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT05031780

COVID-19 VaccineNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05139992

Alendronate SodiumNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06016634

exagamglogene autotemcel (CTX001)NOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov
NCT05477563

tovinontrine (IMR-687)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04053803

PlerixaforNOT in gene-target list

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT03653247

BusulfanNOT in gene-target list

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT03653247

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