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Protein / target

BCL11 transcription factor A

Encoded byBCL11AQ9H165Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
1
Approved medicines
Open Targets target-level
View by indication →
1
Clinical trials
Degrader-tractable
Druggability
UniProt Ubiquitination

Protein at a glance

Biological role

Transcription regulatory region nucleic acid binding

Strongest disease association

Genetic Diseases, Inborn

Via encoding gene BCL11A · Genetic evidence · score 0.84

Therapeutic position

Established drug target

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

Transcription factor.

View complete UniProt function annotation

Transcription factor (PubMed:16704730, PubMed:29606353). Associated with the BAF SWI/SNF chromatin remodeling complex (PubMed:23644491, PubMed:39607926). Binds to the 5'-TGACCA-3' sequence motif in regulatory regions of target genes, including a distal promoter of the HBG1 hemoglobin subunit gamma-1 gene (PubMed:29606353, PubMed:39423807). Involved in regulation of the developmental switch from gamma- to beta-globin, probably via direct repression of HBG1; hence indirectly repressing fetal hemoglobin (HbF) level (PubMed:26375765, PubMed:29606353, PubMed:39423807, PubMed:39607926). Involved in brain development (PubMed:27453576). May play a role in hematopoiesis (By similarity). Essential factor in lymphopoiesis required for B-cell formation in fetal liver (By similarity). May function as a modulator of the transcriptional repression activity of NR2F2 (By similarity)

Subcellular location

CytoplasmNucleusChromosomeNucleus matrix
Domains and Gene Ontology detail (37)

Gene Ontology

  • Ccytoplasm
  • Ccytosol
  • Cnuclear body
  • Cnuclear matrix
  • Cnucleoplasm
  • Cnucleus
  • Cparaspeckles
  • Cpostsynapse
  • CSWI/SNF complex
  • FDNA-binding transcription factor activity
  • FDNA-binding transcription factor binding
  • FDNA-binding transcription repressor activity, RNA polymerase II-specific

835 aa · 91 kDa · 5 isoforms

Biological roles

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

Transcriptional regulationUniProt · GO
View supporting evidence

Transcriptional regulation

  • ·Transcription factor (PubMed:16704730, PubMed:29606353). Associated with the BAF SWI/SNF…
  • ·DNA-binding transcription factor activity
  • ·DNA-binding transcription factor binding
  • ·DNA-binding transcription repressor activity, RNA polymerase II-specific

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

Approved medicines with mapped indications

1 medicine · 2 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.

Anemia, Sickle Cell1 medicine
beta-Thalassemia1 medicine

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

Drugs targeting this protein

1

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.

exagamglogene autotemcel
Narrow target profileApprovedGene editing negative modulator

B-cell lymphoma/leukemia 11A gene editing negative modulator

Indicated for Anemia, Sickle Cell, beta-Thalassemia

Direct interaction with this protein · Only this protein recorded as a target

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 BCL11A

Gene-level evidence surfaced through the gene BCL11Athat 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.

Genetic Diseases, Inborn
0.84Well supported

Genetic evidence dominant · Open Targets 0.51

Benign prostatic hyperplasia
0.81Well supported

Genetic evidence dominant · Open Targets 0.49

Intelligence
0.80Well supported

Genetic evidence dominant · Open Targets 0.49

Schizophrenia
0.77Well supported

Genetic evidence dominant · Open Targets 0.47

COVID-19
0.74Moderately supported

Genetic evidence dominant · Open Targets 0.45

View evidence synthesis (5)
Genetic Diseases, InbornWell supported
0.84
agreement 0.710.98
Genetic100%Literature0%

Open Targets aggregate 0.51 · 2 independent evidence families

Benign prostatic hyperplasiaWell supported
0.81
agreement 0.690.93
Genetic100%

Open Targets aggregate 0.49 · 1 independent evidence family

IntelligenceWell supported
0.80
agreement 0.680.92
Genetic100%

Open Targets aggregate 0.49 · 1 independent evidence family

SchizophreniaWell supported
0.77
agreement 0.630.91
Genetic98%Literature2%

Open Targets aggregate 0.47 · 2 independent evidence families

COVID-19Moderately supported
0.74
agreement 0.600.88
Genetic95%Literature5%

Open Targets aggregate 0.45 · 2 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
Anemia, Sickle Cell0.53
Genetic Diseases, Inborn0.51
Neoplasms0.51
Benign prostatic hyperplasia0.49
Intelligence0.49
Schizophrenia0.47
COVID-190.45
beta-Thalassemia0.44

Drug development

1 compounds recorded · 1 approved

Open Targets' development universe — every compound recorded against the target at any stage, not all approved medicines. Distinct from the 1 drug that targets this protein in Forefront's canonical graph (1 with a mapped approved indication, shown above): these count different sets and are not a subset relation.

View all recorded compounds (1)
EXAGAMGLOGENE AUTOTEMCELApproval

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

Protein degradersEmerging

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

View underlying tractability evidence (3)
PR · UniProt UbiquitinationPR · Database UbiquitinationPR · Half-life Data

Raw Open Targets tractability assessment buckets, by modality.

Clinical trials

1

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.

ClinicalTrials.gov via the drug-target graph.

What's happening now

1

Recent therapeutic activity around this target — regulatory actions, clinical trials and safety signals for a drug that targets 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. Regulatory approval2024-02-09

    Approval: Casgevy (EMA)

    ema · regulatory · ema · via exagamglogene autotemcel

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.