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Protein / target

DNA (cytosine-5)-methyltransferase 3A

Encoded byDNMT3AQ9Y6K1Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
2
Approved medicines
Open Targets target-level
View by indication →
30
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

DNA (cytosine-5-)-methyltransferase

Strongest disease association

Leukemia, Myeloid, Acute

Via encoding gene DNMT3A · Genetic evidence · score 0.85

Therapeutic position

Established drug target

Small molecules

Derived from structured UniProt, Open Targets and literature data on this page.

Protein profile

UniProt 2026_02

Canonical identity and biological annotation from UniProt.

Function overview

Required for genome-wide de novo methylation and is essential for the establishment of DNA methylation patterns during development.

View complete UniProt function annotation

Required for genome-wide de novo methylation and is essential for the establishment of DNA methylation patterns during development (PubMed:12138111, PubMed:16357870, PubMed:30478443). DNA methylation is coordinated with methylation of histones (PubMed:12138111, PubMed:16357870, PubMed:30478443). It modifies DNA in a non-processive manner and also methylates non-CpG sites (PubMed:12138111, PubMed:16357870, PubMed:30478443). May preferentially methylate DNA linker between 2 nucleosomal cores and is inhibited by histone H1 (By similarity). Plays a role in paternal and maternal imprinting (By similarity). Required for methylation of most imprinted loci in germ cells (By similarity). Acts as a transcriptional corepressor for ZBTB18 (By similarity). Recruited to trimethylated 'Lys-36' of histone H3 (H3K36me3) sites (By similarity). Can actively repress transcription through the recruitment of HDAC activity (By similarity). Also has weak auto-methylation activity on Cys-710 in absence of DNA (By similarity)

Subcellular location

NucleusChromosomeCytoplasm
Domains and Gene Ontology detail (40)

Domains & features

PWWPADDSAM-dependent MTase C5-type

Gene Ontology

  • Cchromosome, centromeric region
  • Ccytoplasm
  • Ceuchromatin
  • Cheterochromatin
  • Cnuclear matrix
  • Cnucleoplasm
  • Cnucleus
  • CXY body
  • Fchromatin binding
  • FDNA (cytosine-5-)-methyltransferase activity
  • FDNA binding
  • FlncRNA binding

912 aa · 102 kDa · 3 isoforms

Biological roles

What this protein does, drawn together from its UniProt function, Gene Ontology terms and Reactome pathways.

Transcriptional regulationUniProt · GOMetabolic enzyme activityGO
View supporting evidence

Transcriptional regulation

  • ·Required for genome-wide de novo methylation and is essential for the establishment of D…
  • ·RNA polymerase II cis-regulatory region sequence-specific DNA binding
  • ·RNA polymerase II-specific DNA-binding transcription factor binding
  • ·transcription corepressor activity

Metabolic enzyme activity

  • ·DNA (cytosine-5-)-methyltransferase activity
  • ·protein-cysteine methyltransferase activity

Concepts derived from UniProt GO Reactome — each badge above shows which sources supported that role.

Approved medicines with mapped indications

2 medicines · 5 areas

Approved therapies targeting this protein, grouped by what they are approved to treat — the disease-first view of the medicines below. Relationships come from the canonical approved-indication graph (ChEMBL phase-4), the same source as the drug cards.

Myelodysplastic Syndromes2 medicines
Anemia1 medicine
Leukemia, Myeloid1 medicine
Leukemia, Myeloid, Acute1 medicine
Broader indication categories (1)
Neoplasms2 medicines

Broad umbrella indications (e.g. “Neoplasms”). Shown here because every medicine also appears under a more specific disease above — kept for completeness, de-emphasised for clarity.

Approved indications from ChEMBL (phase-4), via the canonical drug→disease graph.

Drugs targeting this protein

2

How approved and investigational drugs engage this protein — mechanism and action type, direct vs complex targeting, and how broadly each acts across other targets. A drug-first view (the section above groups the approved ones disease-first); direct binders with few recorded targets are listed first.

azacitidine
Narrow target profileApprovedInhibitor

DNA (cytosine-5)-methyltransferase 3A inhibitor

Indicated for Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Neoplasms

Direct interaction with this protein · 1 of 2 recorded protein targets — narrow recorded profile

decitabine
Narrow target profileApprovedInhibitor

DNA (cytosine-5)-methyltransferase 3A inhibitor

Indicated for Anemia, Leukemia, Myeloid, Myelodysplastic Syndromes, Neoplasms

Direct interaction with this protein · 1 of 2 recorded protein targets — narrow recorded profile

ChEMBL mechanism, action type, target identity and approved indications. Open Targets clinical status.

Translational evidence

Open Targets 26

Why this target matters therapeutically, strongest evidence first. Disease associations are gene-level (via the gene that encodes this protein) and open into the full confidence synthesis; the development universe, tractability and safety annotations are target-level, from Open Targets.

Strongest disease associations · via encoding gene DNMT3A

Gene-level evidence surfaced through the gene DNMT3Athat encodes this protein — not a direct protein–disease relationship. Ranked by Forefront's causal-directness weighting, so genetically- and clinically-evidenced diseases lead over ones that merely share the literature.

Leukemia, Myeloid, Acute
0.99Well supported

Genetic evidence dominant · Open Targets 0.86

Leukemia, Myeloid
0.92Well supported

Genetic evidence dominant · Open Targets 0.68

Myelodysplastic syndrome
0.86Well supported

Clinical evidence dominant · Open Targets 0.74

Neoplasms
0.84Well supported

Clinical evidence dominant · Open Targets 0.57

Chronic myelomonocytic leukemia
0.82Well supported

Clinical evidence dominant · Open Targets 0.67

View evidence synthesis (5)
Leukemia, Myeloid, AcuteWell supported
0.99
agreement 0.901.00
Genetic34%Clinical30%Somatic mutation20%Pathway10%Literature6%Genetic literaturedup

Open Targets aggregate 0.86 · 5 independent evidence families · 1 not counted as duplicate

Leukemia, MyeloidWell supported
0.92
agreement 0.811.00
Genetic56%Clinical42%Literature2%

Open Targets aggregate 0.68 · 3 independent evidence families

Myelodysplastic syndromeWell supported
0.86
agreement 0.740.98
Clinical58%Somatic mutation31%Literature11%

Open Targets aggregate 0.74 · 3 independent evidence families

NeoplasmsWell supported
0.84
agreement 0.750.93
Clinical43%Genetic33%Somatic mutation14%Literature10%

Open Targets aggregate 0.57 · 4 independent evidence families

Chronic myelomonocytic leukemiaWell supported
0.82
agreement 0.700.94
Clinical69%Somatic mutation28%Literature4%

Open Targets aggregate 0.67 · 3 independent evidence families

The evidence agreement range shows how closely the independent evidence families agree — it is not a statistical confidence interval, and nothing here is fitted to outcome data. Derived from Open Targets evidence types under Forefront weighting; the per-type scores above show the calculation.

Show all associations
Leukemia, Myeloid, Acute0.86
Myelodysplastic syndrome0.74
Leukemia, Myeloid0.68
Chronic myelomonocytic leukemia0.67
Neoplasms0.57
Myeloproliferative disorder0.56

Drug development

2 compounds recorded · 2 approved

Open Targets' development universe — every compound recorded against the target at any stage, not all approved medicines. Distinct from the 2 drugs that target this protein in Forefront's canonical graph (2 with a mapped approved indication, shown above): these count different sets and are not a subset relation.

View all recorded compounds (2)
AZACITIDINEApproval
DECITABINEApproval

Open Targets known-drugs universe. Drug name and highest clinical stage only — the disease relationship is NOT read from this slice (it carries trial-context noise); approved indications come from the canonical graph above.

Tractability

Small moleculesStrong

Approved Drug and Structure with Ligand support this modality.

Protein degradersEmerging

Feasibility evidence (uniprot ubiquitination and database ubiquitination) — no clinical-stage drug of this modality recorded.

View underlying tractability evidence (8)
SM · Approved DrugSM · Structure with LigandSM · High-Quality LigandSM · Druggable FamilyPR · UniProt UbiquitinationPR · Database UbiquitinationPR · Half-life DataPR · Small Molecule Binder

Raw Open Targets tractability assessment buckets, by modality.

Clinical trials

30

Trials of drugs that target this protein — reached indirectly through those drugs, so a trial listed here studies the drug, not the protein. Ranked by active status, then clinical phase and recency, and spread across the targeting drugs.

View all trials (26)

ClinicalTrials.gov via the drug-target graph.

What's happening now

12

Recent therapeutic activity around this target — regulatory actions, clinical trials and safety signals for 2 drugs that target this protein, plus publications where such a drug is a genuine subject. Every item is reached indirectly through the drug, not the protein itself; incidental mentions (a paper that merely measures a drug) and press items are excluded.

  1. Trial status changed2026-08-25

    Phase I/II Study of Neoadjuvant Inhaled Azacytidine With Platinum-Based Chemotherapy and Durvalumab (MEDI4736) - a Combined Epigenetic-Immunotherapy (AZA-AEGEAN) Regimen for Operable Early-Stage Non-Small Cell Lung Cancer (NSCLC)

    Status changed to Suspended · ClinicalTrials.gov · via azacitidine

  2. Trial status changed2026-08-20

    A Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combined With Filgrastim for Children and Young Adults With AML, MDS and Related Myeloid Malignancies

    Status changed to Active, not recruiting · ClinicalTrials.gov · via decitabine

  3. Trial status changed2026-07-31

    Study of Cladribine+Venetoclax After Failure of Venetoclax+Hypomethylating Agent in Monocytic AML

    Status changed to Active, not recruiting · ClinicalTrials.gov · via azacitidine

  4. Trial status changed2026-07-14

    Phase 1b Study of Pembrolizumab, Decitabine +/- Venetoclax Combination Therapy in Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome

    Status changed to Suspended · ClinicalTrials.gov · via decitabine

  5. Label change2026-06-09

    Label change: AZACITIDINE (ANDA204949)

    fda · regulatory · fda · via azacitidine

  6. Label change2026-06-09

    Label change: AZACITIDINE (ANDA204949)

    fda · regulatory · fda · via azacitidine

  7. Label change2026-06-09

    Label change: AZACITIDINE (ANDA204949)

    fda · regulatory · fda · via azacitidine

  8. Regulatory approval2024-01-05

    Approval: Azacitidine Kabi (EMA)

    ema · regulatory · ema · via azacitidine

  9. New publication2021-08-23
    Results of a randomized phase 3 study of oral sapacitabine in elderly patients with newly diagnosed acute myeloid leukemia (SEAMLESS).

    Cancer · 2021 · 6 citations · Europe PMC · via decitabine

  10. New publication2021-03-01
    Low-dose decitabine for refractory prolonged isolated thrombocytopenia after HCT: a randomized multicenter trial.

    Blood advances · 2021 · 18 citations · Europe PMC · via decitabine

  11. New publication2009-12-21
    Azacitidine prolongs overall survival compared with conventional care regimens in elderly patients with low bone marrow blast count acute myeloid leukemia.

    Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2010 · 701 citations · Europe PMC · via azacitidine

  12. New publication2009-02-21
    Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study.

    The Lancet. Oncology · 2009 · 1,943 citations · Europe PMC · via azacitidine

Objective event titles are shown unmodified; the event kind and significance line are derived from structured fields. Forefront AttentionEvent stream aggregating Europe PMC Regulatory filings ClinicalTrials.gov.