OncoSignal
Live from Open Targets Platform, fetched 2026-11-11 15:37 UTC. Every row links to its source record.

ASCL1 achaete-scute family bHLH transcription factor 1

ENSG00000139352 · protein coding · Open Targets profile

Transcription factor that plays a key role in neuronal differentiation: acts as a pioneer transcription factor, accessing closed chromatin to allow other factors to bind and activate neural pathways. Directly binds the E box motif (5'-CANNTG-3') on promoters and promotes transcription of neuronal genes. The combination of three transcription factors, ASCL1, POU3F2/BRN2 and MYT1L, is sufficient to reprogram fibroblasts and other somatic cells into induced neuronal (iN) cells in vitro. Plays a role at early stages of development of specific neural lineages in most regions of the CNS, and of several lineages in the PNS. Essential for the generation of olfactory and autonomic neurons. Acts synergistically with FOXN4 to specify the identity of V2b neurons rather than V2a from bipotential p2 progenitors during spinal cord neurogenesis, probably through DLL4-NOTCH signaling activation. Involved in the regulation of neuroendocrine cell development in the glandular stomach (By similarity). .

In 5 of 15 cancers, the evidence for ASCL1 comes mainly from inherited variants

Each row is a cancer, strongest overall evidence first. The two dots show how strong the inherited (germline) and tumour-mutation (somatic) evidence is; the line between them is the gap.

Inherited (germline) variantsMutations in tumours (somatic)
00.250.50.751gastric cancergastric cancergastric cancer: germline 0.43small cell lung carcinomasmall cell lung carcinomauterine corpus leiomyomauterine corpus leiomyomauterine corpus leiomyoma: germline 0.26cervical carcinomacervical carcinomacervical carcinoma: germline 0.22neuroblastomaneuroblastomaglioblastomaglioblastomaovarian neoplasmovarian neoplasmovarian neoplasm: germline 0.18hepatocellular carcinomahepatocellular carcinomagliomagliomacentral nervous system cancercentral nervous system cancercolorectal carcinomacolorectal carcinomabreast cancerbreast cancerbreast cancer: germline 0.04neuroendocrine carcinomaneuroendocrine carcinomacarcinoid tumorcarcinoid tumornasopharyngeal carcinomanasopharyngeal carcinoma

Scores run from 0 (no evidence) to 1 (strongest). A missing dot at 0 means Open Targets holds no evidence of that kind. Source: Open Targets Platform, fetched live.

Tractability

No positive tractability buckets.

gnomAD loss of function constraint: o/e 0.1244800016283989 (upper bound 0.3918600082397461). Low values mean loss of function is poorly tolerated, a safety flag for inhibition.

Who runs trials of these drugs

No named investigators on registry trials of ASCL1's approved or late-stage drugs in the last three years.

No terminated, withdrawn or suspended trials of ASCL1's drugs are registered

All 0 drugs and clinical candidates as a table

Each drug, stage and indication links to its Open Targets record, where the trials and approvals behind it are listed. Salt forms (for example a mesylate) are listed as Open Targets records them, as separate entries.

DrugTypeHighest stageIndicationsSource records
No drugs or clinical candidates recorded.