Biomarker & Intervention Discovery

Type 1 Diabetes

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

US DALYs5.06MWHO GHE 2021 · all ages
Global DALYs80.11MWHO GHE 2021 · worldwide
US mortality61.1K deaths/yrWHO GHE 2021
Global mortality1.61M deaths/yrWHO GHE 2021 · worldwide
NIH funding$1.10BFY2025 · RCDC estimate
Funding levelUnderfunded
Spontaneous remissionEssentially zero for established T1DM (honeymoon phase occurs in 50-60% after diagnosis but lasts 3-12 months then…
Best-intervention remissionClosed-loop insulin delivery (artificial pancreas systems — Control-IQ, Omnipod 5) achieves 70-80% time-in-range vs…
Gap sizeModerate-large — closed-loop approaches are increasingly standard but true insulin independence is rare with any…
Primary barrierbiological + access
Full remission profile →

Remission & chronicity

Spontaneous remission
Essentially zero for established T1DM (honeymoon phase occurs in 50-60% after diagnosis but lasts 3-12 months then ends); the disease is permanent autoimmune destruction of beta cells
Best-intervention remission
Closed-loop insulin delivery (artificial pancreas systems — Control-IQ, Omnipod 5) achieves 70-80% time-in-range vs 60% with standard therapy; teplizumab (anti-CD3 mAb) delays T1DM onset by 2+ years in at-risk individuals; islet cell transplantation achieves insulin independence in ~44% at 5 years (CITR data)
Gap size
Moderate-large — closed-loop approaches are increasingly standard but true insulin independence is rare with any available therapy
Primary barrier
biological + access
Beta cell regeneration and transplantation face immune rejection barriers (all islet recipients require chronic immunosuppression); closed-loop systems dramatically improve quality of life and glycemic control but require intensive engagement and are expensive; teplizumab (FDA approved 2022) for delay is not a cure
Biomarker Targets & Therapeutic Agents

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

CD3E 36CTLA4 32GAD2 2HLA-DQA1 10HLA-DQB1 15IL2RA 51INS 6PTPRN 0SLC2A2 115
Therapeutic Agent Effect Direction Evidence Tier Potency Source
FORALUMAB ? unclear Mechanistic
FBT-A05 ~ modulates Mechanistic
MUROMONAB-CD3 inhibits Mechanistic
TEPLIZUMAB ? unclear Mechanistic
OTELIXIZUMAB ~ modulates Mechanistic
IPRAGLIFLOZIN inhibits Mechanistic
DAPAGLIFLOZIN PROPANEDIOL inhibits Mechanistic
RESIMMUNE ~ modulates Mechanistic
MOSUNETUZUMAB ~ modulates Mechanistic
BEXAGLIFLOZIN inhibits Mechanistic
IMC-GP100 ~ modulates Mechanistic
VISILIZUMAB inhibits Mechanistic
SERGLIFLOZIN inhibits Mechanistic
EPCORITAMAB ~ modulates Mechanistic
ERTUMAXOMAB ~ modulates Mechanistic
GLOFITAMAB ~ modulates Mechanistic
REMOGLIFLOZIN inhibits Mechanistic
SOTAGLIFLOZIN inhibits Mechanistic
ODRONEXTAMAB ~ modulates Mechanistic
ELRANATAMAB ~ modulates Mechanistic
EMPAGLIFLOZIN inhibits Mechanistic
HOKT3 inhibits Mechanistic
CATUMAXOMAB ~ modulates Mechanistic
SOLITOMAB ~ modulates Mechanistic
ERTUGLIFLOZIN inhibits Mechanistic
LICOGLIFLOZIN inhibits Mechanistic
MEDI-565 ~ modulates Mechanistic
TALQUETAMAB ~ modulates Mechanistic
ANTI-CD3 MONOCLONAL ANTIBODY ? unclear Mechanistic
TECLISTAMAB ~ modulates Mechanistic
ENAVOGLIFLOZIN inhibits Mechanistic
BLINATUMOMAB ~ modulates Mechanistic
CANAGLIFLOZIN inhibits Mechanistic
TOFOGLIFLOZIN ANHYDROUS inhibits Mechanistic
FLOTETUZUMAB ~ modulates Mechanistic
TARLATAMAB ? unclear Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
TREMELIMUMAB inhibits Mechanistic
IPILIMUMAB inhibits Mechanistic
CYCLOSPORINE ? unclear Mechanistic
ATEZOLIZUMAB ? unclear Mechanistic
VISILIZUMAB ? unclear Mechanistic
BLEOMYCIN ? unclear Mechanistic
ANTIBIOTIC ? unclear Mechanistic
METHIMAZOLE ? unclear Mechanistic
RETRO-INVERSO PEPTIDE ? unclear Mechanistic
MELPHALAN ? unclear Mechanistic
RECOMBINANT FAS LIGAND ? unclear Mechanistic
ZALIFRELIMAB inhibits Mechanistic
RIBAVIRIN ? unclear Mechanistic
ANTI-THYMOCYTE GLOBULIN ? unclear Mechanistic
QUAVONLIMAB inhibits Mechanistic
COMPOUND 32 [PMID: 20471253] inhibits Mechanistic
SIROLIMUS ? unclear Mechanistic
BISPECIFIC ANTIBODY ? unclear Mechanistic
CADONILIMAB inhibits Mechanistic
APLITHIANINE A inhibits Mechanistic
PEGINTERFERON ALFA-2A ? unclear Mechanistic
INOSINE 5'-MONOPHOSPHATE DEHYDROGENASE INHIBITOR FF-10501-01 ? unclear Mechanistic
ABATACEPT ? unclear Mechanistic
WORTMANNIN ? unclear Mechanistic
ERFONRILIMAB inhibits Mechanistic
THALIDOMIDE ? unclear Mechanistic
FF-10501-01 ? unclear Mechanistic
PEGINTERFERON ALFA-2B ? unclear Mechanistic
TETRADECANOYLPHORBOL ACETATE ? unclear Mechanistic
DEHYDRATED ALCOHOL ? unclear Mechanistic
PREDNISONE ? unclear Mechanistic
CHEMBL5291170 inhibits Mechanistic 31000.0 nM (Kd)
Therapeutic Agent Effect Direction Evidence Tier Potency Source
S-ALLYLGLYCINE inhibits Mechanistic
METHAMPHETAMINE ? unclear Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
RECOMBINANT LYMPHOKINE ? unclear Mechanistic
INTERFERON BETA-1B ? unclear Mechanistic
FLOXACILLIN ? unclear Mechanistic
INTERFERON BETA-1A ? unclear Mechanistic
PEGASPARGASE ? unclear Mechanistic
MEASLES VACCINE ? unclear Mechanistic
LUMIRACOXIB ? unclear Mechanistic
MERCAPTOPURINE ? unclear Mechanistic
AZATHIOPRINE ? unclear Mechanistic
LAPATINIB ? unclear Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
FLOXACILLIN ? unclear Mechanistic
ASPIRIN ? unclear Mechanistic
FLUPIRTINE ? unclear Mechanistic
LUMIRACOXIB ? unclear Mechanistic
PEGASPARGASE ? unclear Mechanistic
TICLOPIDINE ? unclear Mechanistic
BUCILLAMINE ? unclear Mechanistic
ABACAVIR ? unclear Mechanistic
LAMOTRIGINE ? unclear Mechanistic
NEVIRAPINE ? unclear Mechanistic
CLAVULANIC ACID ? unclear Mechanistic
ACETAMINOPHEN ? unclear Mechanistic
AMOXICILLIN ANHYDROUS ? unclear Mechanistic
INFLIXIMAB ? unclear Mechanistic
CARBAMAZEPINE ? unclear Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CAFFEINE ? unclear Mechanistic
DENILEUKIN DIFTITOX ~ modulates Mechanistic
MEGESTROL ACETATE ? unclear Mechanistic
HUMAN CHORIONIC GONADOTROPIN ? unclear Mechanistic
LENTINAN ? unclear Mechanistic
PROPYLTHIOURACIL ? unclear Mechanistic
STAUROSPORINE ? unclear Mechanistic
ANTISERUM ? unclear Mechanistic
BASILIXIMAB inhibits Mechanistic
ALDESLEUKIN activates Mechanistic
ISOTRETINOIN ? unclear Mechanistic
ATORVASTATIN CALCIUM TRIHYDRATE ? unclear Mechanistic
HEPATITIS B VACCINE ? unclear Mechanistic
CAMIDANLUMAB TESIRINE ~ modulates Mechanistic
CETIRIZINE HYDROCHLORIDE ? unclear Mechanistic
PROGESTERONE ? unclear Mechanistic
FLUOROURACIL ? unclear Mechanistic
VERAPAMIL ? unclear Mechanistic
DINITROCHLOROBENZENE ? unclear Mechanistic
THERAPEUTIC HORMONE ? unclear Mechanistic
CHINESE HERBS ? unclear Mechanistic
ANTITUBERCULAR AGENT ? unclear Mechanistic
DRUGSATFDA.NDA:008378 ? unclear Mechanistic
DHA-PACLITAXEL ? unclear Mechanistic
METHIMAZOLE ? unclear Mechanistic
MONOCLONAL ANTIBODY R24 ? unclear Mechanistic
SHO-SAIKO-TO ? unclear Mechanistic
THEOPHYLLINE ? unclear Mechanistic
BRYOSTATIN 1 ? unclear Mechanistic
RECOMBINANT INTERLEUKIN-2 ? unclear Mechanistic
METHYLPREDNISOLONE ? unclear Mechanistic
BECLOMETHASONE DIPROPIONATE ? unclear Mechanistic
INTERFERON BETA-1B ? unclear Mechanistic
INTERLEUKIN-2 GENE ? unclear Mechanistic
DEXTRAN SULFATE ? unclear Mechanistic
FLUTAMIDE ? unclear Mechanistic
PACLITAXEL ? unclear Mechanistic
DECITABINE ? unclear Mechanistic
MERCAPTOPURINE ? unclear Mechanistic
INOLIMOMAB inhibits Mechanistic

Showing top 40 of 51 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
INSULIN ZINC SUSP RECOMBINANT HUMAN ? unclear Mechanistic
INSULIN SUSP ISOPHANE RECOMBINANT HUMAN ? unclear Mechanistic
INSULIN SUSP PROTAMINE ZINC BEEF/PORK ? unclear Mechanistic
INULIN ? unclear Mechanistic
INSULIN PEGLISPRO ? unclear Mechanistic
INSULIN DEGLUDEC ? unclear Mechanistic

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

Therapeutic Agent Effect Direction Evidence Tier Potency Source
[3H]2-DEOXYGLUCOSE ? unclear Mechanistic
METFORMIN ? unclear Mechanistic
GLUFOSFAMIDE ? unclear Mechanistic
CHEMBL3780459 inhibits Mechanistic 1120.0 nM (IC50)
CHEMBL3781149 inhibits Mechanistic 60.0 nM (IC50)
CHEMBL3781094 inhibits Mechanistic 5990.0 nM (IC50)
CHEMBL3781994 inhibits Mechanistic 1210.0 nM (IC50)
CHEMBL3781827 inhibits Mechanistic 1480.0 nM (IC50)
CHEMBL3780717 inhibits Mechanistic 220.0 nM (IC50)
CHEMBL3780534 inhibits Mechanistic 8700.0 nM (IC50)
CHEMBL3781183 inhibits Mechanistic 1700.0 nM (IC50)
CHEMBL3781331 inhibits Mechanistic 100.0 nM (IC50)
CHEMBL3781654 inhibits Mechanistic 1200.0 nM (IC50)
CHEMBL3780764 inhibits Mechanistic 1360.0 nM (IC50)
CHEMBL3780372 inhibits Mechanistic 510.0 nM (IC50)
CHEMBL3781835 inhibits Mechanistic 950.0 nM (IC50)
CHEMBL3781308 inhibits Mechanistic 1400.0 nM (IC50)
CHEMBL3781633 inhibits Mechanistic 2500.0 nM (IC50)
CHEMBL3781913 inhibits Mechanistic 21.0 nM (IC50)
CHEMBL3780756 inhibits Mechanistic 600.0 nM (IC50)
CHEMBL3781757 inhibits Mechanistic 5000.0 nM (IC50)
CHEMBL3781384 inhibits Mechanistic 3300.0 nM (IC50)
CHEMBL3780785 inhibits Mechanistic 3500.0 nM (IC50)
CHEMBL3781548 inhibits Mechanistic 1100.0 nM (IC50)
CHEMBL3780235 inhibits Mechanistic 1200.0 nM (IC50)
CHEMBL3781347 inhibits Mechanistic 1000.0 nM (IC50)
CHEMBL3780772 inhibits Mechanistic 900.0 nM (IC50)
CHEMBL3780043 inhibits Mechanistic 10000.0 nM (IC50)
CHEMBL3781625 inhibits Mechanistic 2600.0 nM (IC50)
CHEMBL1551510 inhibits Mechanistic 10000.0 nM (IC50)
CHEMBL3780470 inhibits Mechanistic 880.0 nM (IC50)
CHEMBL3780729 inhibits Mechanistic 2100.0 nM (IC50)
CHEMBL3780349 inhibits Mechanistic 8000.0 nM (IC50)
CHEMBL3781797 inhibits Mechanistic 6000.0 nM (IC50)
CHEMBL3780845 inhibits Mechanistic 4400.0 nM (IC50)
CHEMBL3781535 inhibits Mechanistic 7000.0 nM (IC50)
CHEMBL3780527 inhibits Mechanistic 2000.0 nM (IC50)
QUERCETIN inhibits Mechanistic 12000.0 nM (IC50)
MYRICETIN inhibits Mechanistic 12000.0 nM (IC50)
CHEMBL4277734 inhibits Mechanistic 10000.0 nM (IC50)

Showing top 40 of 115 agents ranked by evidence tier.


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 “Type 1 Diabetes” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 9-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) — 59 not found by the gene-target search above.

Insulin LisproNOT in gene-target list

Phase 3 · 3 trials · source: ClinicalTrials.gov, Open Targets
NCT07282054 NCT03465878 NCT03433677

LY900014NOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT03433677 NCT03465878

Alpha 1-Antitrypsin (AAT, Aralast NP)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01319331

BHT-3021NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00453375

BHT-PlaceboNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00453375

Usual algorithmNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02521675

Diabrasport algorithmNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02521675

RestNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02521675

Holter Glycemic Ipro2, MedtronicNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02521675

Ertugliflozin 2 mg single doseNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01054300

Ertugliflozin 2 mg split into twice dailyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01054300

Ertugliflozin 4 mg single doseNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01054300

Ertugliflozin 4 mg split into twice dailyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01054300

LiraglutideNOT in gene-target list

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT02516657

Pancreas transplantation aloneNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03869281

PembrolizumabNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04305054

VibostolimabNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04305054

Pembrolizumab/QuavonlimabNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04305054

LenvatinibNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04305054

Favezelimab/PembrolizumabNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04305054

ATRANOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04305054

Allogeneic CD7-Targeted CAR-T Cell InjectionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07528105

PuraPly AMNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06618612

PuraPly XTNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06618612

THDB0206NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07282054

Insulin GlargineNOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT07282054

Acetylated and Butyrylated High Amylose Maize StarchNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04114357

Automated insulin delivery system algorithm with a dual-hormone configuration (insulin and glucagon) for glucose controlNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06082973

Automated insulin delivery system with a single-hormone configuration (insulin) with rescue carbohydrate recommendations for glucose controlNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06082973

Sensor Based Glucose Monitoring SystemNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02232698

Standard Blood Glucose MonitoringNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02232698

IDN-6556NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01653899

Maridebart cafraglutideNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07160257

AcetazolamideNOT in gene-target list

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT06981234

DİABOT (mobile application robot )NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05333393

Guided meditationNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06151821

Medtronic Extended Infusion SetNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06074965

GSK962040 (5 mg tablet)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01262898

GSK962040 (25 mg tablet)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01262898

GSK962040 (125 mg tablet)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01262898

Plan, Reflect, and Engage with Providers for Diabetes CareNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03734107

Coin2DoseNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05280184

DM-StandardNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00675311

Disease Management PlusNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00675311

SitagliptinNOT in gene-target list

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01718093

Metformin

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01718093

Sitagliptin + MetforminNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01718093

DiamydNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03345004

Vitamin DNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03345004

Placebo for DiamydNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03345004

Placebo for Vitamin DNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03345004

Glucagon Sensitivity test consisting of an amino acid tolerance test and a bolus infusion of glucagonNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04907721

MyDiaMateNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06308549

Information-Motivation-Behavioral Skills model-based interventionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04486547

Heart healthy interventionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03531021

Immunosuppression and autologous stem cell transplantationNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00315133

Blood drawNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03945747

Urine sample collectionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03945747

DXA scanNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03945747

Minimed 780GNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06978387

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