ATM-related cancer predisposition

Summary

ATM-related cancer predisposition (MONDO:0700270) is a cancer caused by ATM (GenCC Definitive), with 2 cohort genes (1 CIViC-evidence somatic driver; 288 ClinVar predisposition records).

At a glance

• Classification: Cancer
• Causal gene: ATM (GenCC Definitive)
• Cohort genes: 2
• ClinVar variants: 288

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

Field	Value
Canonical name	ATM-related cancer predisposition
Mondo ID	MONDO:0700270
GARD	0026410
Is cancer (heuristic)	yes

Data availability: 288 ClinVar variants · 136 ClinGen variant curations · 1 GenCC gene-disease record.

Disease family

Classification path: disease › human disease › disease by etiologic mechanism › disease of genetic or genomic mechanism › hereditary disease › hereditary neoplastic syndrome › ATM-related cancer predisposition

Related subtypes (116): mosaic variegated aneuploidy syndrome, tuberous sclerosis, hereditary breast ovarian cancer syndrome, hereditary multiple osteochondromas, nevoid basal cell carcinoma syndrome, leukemia, chronic lymphocytic, susceptibility to, 2, blue rubber bleb nevus, cherubism, Beckwith-Wiedemann syndrome, multiple self-healing squamous epithelioma, erythroleukemia, familial, susceptibility to, goiter, multinodular 1, with or without Sertoli-Leydig cell tumors, hyperparathyroidism 2 with jaw tumors, Kaposi sarcoma, susceptibility to, hereditary leiomyomatosis and renal cell cancer, susceptibility to uveal melanoma, melanoma and neural system tumor syndrome, nasopharyngeal carcinoma, susceptibility to, 2, WAGR syndrome, neuroblastoma, susceptibility to, 1, Rothmund-Thomson syndrome, mismatch repair cancer syndrome 1, Wiskott-Aldrich syndrome, N syndrome, hereditary thrombocytopenia and hematologic cancer predisposition syndrome, prostate cancer/brain cancer susceptibility, Brooke-Spiegler syndrome, pancreatic cancer, susceptibility to, 1, Carney-Stratakis syndrome, nasopharyngeal carcinoma, susceptibility to, 1, ovarian cancer, susceptibility to, 1, colorectal cancer, susceptibility to, 1, lung cancer susceptibility 1, leukemia, chronic lymphocytic, susceptibility to, 1, Kostmann syndrome, colorectal cancer, susceptibility to, 2, colorectal cancer, susceptibility to, 3, colorectal cancer, susceptibility to, 5, colorectal cancer, susceptibility to, 6, colorectal cancer, susceptibility to, 7, leukemia, chronic lymphocytic, susceptibility to, 3, leukemia, chronic lymphocytic, susceptibility to, 4, leukemia, chronic lymphocytic, susceptibility to, 5, lung cancer susceptibility 3, colorectal cancer, susceptibility to, 8, colorectal cancer, susceptibility to, 9, colorectal cancer, susceptibility to, 10, colorectal cancer, susceptibility to, 11, lung cancer susceptibility 4, neuroblastoma, susceptibility to, 3, neuroblastoma, susceptibility to, 4, neuroblastoma, susceptibility to, 5, neuroblastoma, susceptibility to, 6, leukemia, acute lymphocytic, susceptibility to, 1, leukemia, acute lymphocytic, susceptibility to, 2, lung cancer susceptibility 5, BAP1-related tumor predisposition syndrome, familial cutaneous telangiectasia and oropharyngeal predisposition cancer syndrome, Maffucci syndrome, basal cell carcinoma, susceptibility to, 7, colorectal cancer, susceptibility to, 12, leukemia, acute lymphoblastic, susceptibility to, 3, cholangiocarcinoma, susceptibility to, progeroid features-hepatocellular carcinoma predisposition syndrome, neuroblastoma, susceptibility to, 7, DDX41-related hematologic malignancy predisposition syndrome, nasopharyngeal carcinoma, susceptibility to, 3, familial isolated hyperparathyroidism, intestinal polyposis syndrome, dyskeratosis congenita, familial rhabdoid tumor, multiple endocrine neoplasia, hereditary pheochromocytoma-paraganglioma, PTEN hamartoma tumor syndrome, familial multiple fibrofolliculoma, hereditary retinoblastoma, familial atypical multiple mole melanoma syndrome, hereditary nonpolyposis colon cancer, Li-Fraumeni syndrome, Cobb syndrome, neurofibromatosis, susceptibility to familial cutaneous melanoma, pancreatic cancer, susceptibility to, 5, leukemia, acute myeloid, susceptibility to, diffuse gastric and lobular breast cancer syndrome with or without cleft lip and/or palate, glioma susceptibility, hemangioma, capillary infantile, susceptibility to, CDH1-related diffuse gastric and lobular breast cancer syndrome, NTHL1-deficiency tumor predisposition syndrome, SAMD9-related spectrum and myeloid neoplasm risk, neuroblastoma, susceptibility to, 2, BARD1-related cancer predisposition, BRCA1-related cancer predisposition, BRCA2-related cancer predisposition, CHEK2-related cancer predisposition, PALB2-related cancer predisposition, RAD51C-related cancer predisposition, RAD51D-related cancer predisposition, Li-fraumeni-like syndrome, breast cancer, familial, susceptibility to, 1, breast cancer, familial, susceptibility to, 2, breast cancer, familial, susceptibility to, 3, colorectal cancer, susceptibility to, 4, colorectal cancer, susceptibility to, on chromosome 15, ovarian cancer, familial, susceptibility to, 1, ovarian cancer, familial, susceptibility to, 2, ovarian cancer, familial, susceptibility to, 3, inherited hematologic cancer-predisposing syndrome, mosaic neurofibromatosis/schwannomatosis, tumor predisposition syndrome 2, prostate cancer, hereditary, X-linked 3, follicular lymphoma, susceptibility to, GPR161-related medulloblastoma predisposition, SAMD9L-related spectrum and myeloid neoplasm risk, HAVCR2-related cancer predisposition, EGLN1-related erythrocytosis and pheochromocytoma/paraganglioma predisposition

Genetics & variants

GWAS landscape

No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.

Variant details and genetic-evidence tiers

ClinVar germline variants

288 retrieved; paginated sample, class counts are floors:

89 pathogenic, 66 uncertain significance, 40 conflicting classifications of pathogenicity, 27 likely pathogenic, 23 benign/likely benign, 22 pathogenic/likely pathogenic, 12 benign, 9 likely benign

ClinVar	Variant (HGVS)	Gene	Classification	Review
1676606	Single allele		Pathogenic	reviewed by expert panel
127337	NM_000051.4(ATM):c.1339C>T (p.Arg447Ter)	ATM	Pathogenic	criteria provided, multiple submitters, no conflicts
127340	NM_000051.4(ATM):c.1564_1565del (p.Glu522fs)	ATM	Pathogenic	reviewed by expert panel
127374	NM_000051.4(ATM):c.3802del (p.Glu1267_Val1268insTer)	ATM	Pathogenic	reviewed by expert panel
127403	NM_000051.4(ATM):c.5228C>T (p.Thr1743Ile)	ATM	Pathogenic	reviewed by expert panel
127405	NM_000051.4(ATM):c.5290del (p.Leu1764fs)	ATM	Pathogenic	reviewed by expert panel
127463	NM_000051.4(ATM):c.8786+1G>A	ATM	Pathogenic	reviewed by expert panel
1332151	NM_000051.4(ATM):c.3662G>A (p.Trp1221Ter)	ATM	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
135780	NM_000051.4(ATM):c.8266A>T (p.Lys2756Ter)	ATM	Pathogenic	reviewed by expert panel
140818	NM_000051.4(ATM):c.6997dup	ATM	Pathogenic	reviewed by expert panel
140889	NM_000051.4(ATM):c.1402_1403del (p.Lys468fs)	ATM	Pathogenic	reviewed by expert panel
140897	NM_000051.4(ATM):c.8565_8566delinsAA (p.Ser2855_Val2856delinsArgIle)	ATM	Pathogenic	reviewed by expert panel
140907	NM_000051.4(ATM):c.8473C>T (p.Gln2825Ter)	ATM	Pathogenic	reviewed by expert panel
141037	NM_000051.4(ATM):c.378del (p.Asp126fs)	ATM	Pathogenic	criteria provided, multiple submitters, no conflicts
141325	NM_000051.4(ATM):c.2284_2285del (p.Leu762fs)	ATM	Pathogenic	reviewed by expert panel
141404	NM_000051.4(ATM):c.5908C>T (p.Gln1970Ter)	ATM	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
141474	NM_000051.4(ATM):c.1355del (p.Thr452fs)	ATM	Pathogenic	reviewed by expert panel
141647	NM_000051.4(ATM):c.7517_7520del	ATM	Pathogenic	criteria provided, multiple submitters, no conflicts
141742	NM_000051.4(ATM):c.790del (p.Tyr264fs)	ATM	Pathogenic	reviewed by expert panel
141887	NM_000051.4(ATM):c.1158del (p.Lys387fs)	ATM	Pathogenic	reviewed by expert panel
142355	NM_000051.4(ATM):c.6976-2A>C	ATM	Pathogenic	reviewed by expert panel
142700	NM_000051.4(ATM):c.8147T>C (p.Val2716Ala)	ATM	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
1713223	NM_000051.4(ATM):c.6372C>G (p.Tyr2124Ter)	ATM	Pathogenic	reviewed by expert panel
1733279	NM_000051.4(ATM):c.3617T>A (p.Leu1206Ter)	ATM	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
181865	NM_000051.4(ATM):c.8264_8268del (p.Tyr2755fs)	ATM	Pathogenic	criteria provided, multiple submitters, no conflicts
181874	NM_000051.4(ATM):c.237del (p.Lys79fs)	ATM	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
181974	NM_000051.4(ATM):c.6095G>A (p.Arg2032Lys)	ATM	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
185137	NM_000051.4(ATM):c.3349C>T (p.Gln1117Ter)	ATM	Pathogenic	reviewed by expert panel
185405	NM_000051.4(ATM):c.2839-3_2839delinsGATACTA	ATM	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
186242	NM_000051.4(ATM):c.8395_8404del (p.Phe2799fs)	ATM	Pathogenic	reviewed by expert panel

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 14 · Orphanet: 8 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

Somatic driver evidence (intOGen + CIViC, cohort fanout)

Gene	intOGen role	Cancer types	CIViC
ATM	LoF	BLCA,BRCA,CCRCC,CHOL,CLLSLL,COAD,COADREAD,ESCA,HCC,LUAD,LUSC,MEL,NSCLC,PAAD,PANCREAS,PANET,PCM,PLMESO,PRAD,PROSTATE,STAD,UCEC,UTUC,WDTC	CIViC #69

GenCC gene–disease validity (cohort genes)

the Disease column is the GenCC-asserted condition — a cohort gene’s strongest validity may be for a related predisposition syndrome.

Gene	Classification	Inheritance	Disease	Records
ATM	Definitive	Autosomal dominant	ATM-related cancer predisposition	14

Orphanet rare-disease linkage (cohort genes)

Gene	Orphanet ID	Rare disease
ATM	Orphanet:100	Ataxia-telangiectasia
ATM	Orphanet:1331	Familial prostate cancer
ATM	Orphanet:145	Hereditary breast and/or ovarian cancer syndrome
ATM	Orphanet:227535	Hereditary breast cancer
ATM	Orphanet:370109	Ataxia-telangiectasia variant
ATM	Orphanet:440437	Familial colorectal cancer Type X
ATM	Orphanet:52416	Mantle cell lymphoma
ATM	Orphanet:67038	B-cell chronic lymphocytic leukemia

Cohort genes → proteins

2 cohort genes, 2 distinct canonical proteins.

Evidence partition

Subset	Genes
multi_evidence	2

Cohort genes (full)

Symbol	HGNC	Ensembl	UniProt	Name	Evidence
ATM	HGNC:795	ENSG00000149311	Q13315	Serine-protein kinase ATM	gencc,clinvar
C11orf65	HGNC:28519	ENSG00000166323	Q8NCR3	Protein MFI	clinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

Symbol	Protein name	Function (lead sentence)
ATM	Serine-protein kinase ATM	Serine/threonine protein kinase which activates checkpoint signaling upon double strand breaks (DSBs), apoptosis and genotoxic stresses such as ionizing ultraviolet A light (UVA), thereby acting as a DNA damage sensor.
C11orf65	Protein MFI	Acts as an inhibitor of mitochondrial fission.

Protein-family classification

Druggable: 1 · Difficult: 0 · Unknown: 1 · Druggable fraction: 0.5

Family distribution

Cohort families vs a genome-wide background (hypergeometric, BH-FDR; fold = observed/expected). Counts kept; sorted by enrichment, so the catch-all Other/Unknown bucket no longer leads.

Family	Genes	Fold	FDR
Kinase	1	13.9×	0.142
Other/Unknown	1	0.9×	0.805

Per-gene assignment

Symbol	Family	Druggable?	EC	InterPro (top 3)
ATM	Kinase	yes	2.7.11.1	PI3/4_kinase_cat_dom, PIK-rel_kinase_FAT, FATC_dom
C11orf65	Other/Unknown	no

Expression context

Cohort genes with no expression data: 0.

2 cohort genes are a single-cell marker in ≥1 SCXA experiment.

Breadth distribution (Bgee present_calls)

Bucket	Genes
narrow (1-5 tissues)	0
moderate (6-20)	0
broad (>20)	2
unknown	0

Top tissues across cohort

Tissue	Cohort genes
calcaneal tendon	1
colonic epithelium	1
corpus callosum	1
left testis	1
right testis	1
sperm	1

Per-gene tissue summary (top 30)

Symbol	Bgee breadth	FANTOM5 breadth	SCXA	Top tissues
ATM	286	ubiquitous	marker	calcaneal tendon, colonic epithelium, corpus callosum
C11orf65	163	ubiquitous	marker	sperm, left testis, right testis

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

Symbol	Interactor count
ATM	7,383
C11orf65	312

Structural data

PDB: 1 · AlphaFold-only: 1 · No structure: 0

Cohort genes with PDB structures (top 30)

Symbol	UniProt	PDB entries
ATM	Q13315	14

AlphaFold-only cohort genes (top 30 by pLDDT)

Symbol	UniProt	pLDDT
C11orf65	Q8NCR3	74.69

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 61. Enrichment computed across 2 evidence-associated genes (1 with Reactome annotation).

Pathways by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 1 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

Pathway	Cohort genes	Fold	FDR	Sample cohort genes
Sensing of DNA Double Strand Breaks	1	1903.3×	0.007	ATM
TP53 Regulates Transcription of Caspase Activators and Caspases	1	951.7×	0.007	ATM
Pexophagy	1	951.7×	0.007	ATM
Defective homologous recombination repair (HRR) due to PALB2 loss of function	1	951.7×	0.007	ATM
Diseases of DNA Double-Strand Break Repair	1	815.7×	0.007	ATM
Defective homologous recombination repair (HRR) due to BRCA2 loss of function	1	815.7×	0.007	ATM
Stabilization of p53	1	761.3×	0.007	ATM
p53-Dependent G1 DNA Damage Response	1	713.8×	0.007	ATM
p53-Dependent G1/S DNA damage checkpoint	1	713.8×	0.007	ATM
G1/S DNA Damage Checkpoints	1	671.8×	0.007	ATM
Resolution of D-Loop Structures	1	634.4×	0.007	ATM
Diseases of DNA repair	1	571.0×	0.007	ATM
TP53 Regulates Transcription of Cell Death Genes	1	543.8×	0.007	ATM
TP53 Regulates Transcription of Genes Involved in Cytochrome C Release	1	543.8×	0.007	ATM
Regulation of TP53 Activity through Methylation	1	543.8×	0.007	ATM
Regulation of TP53 Expression and Degradation	1	519.1×	0.007	ATM
DNA Double Strand Break Response	1	475.8×	0.007	ATM
Impaired BRCA2 binding to PALB2	1	456.8×	0.007	ATM
Defective homologous recombination repair (HRR) due to BRCA1 loss of function	1	423.0×	0.007	ATM
Defective HDR through Homologous Recombination Repair (HRR) due to PALB2 loss of BRCA1 binding function	1	423.0×	0.007	ATM
Defective HDR through Homologous Recombination Repair (HRR) due to PALB2 loss of BRCA2/RAD51/RAD51C binding function	1	423.0×	0.007	ATM
Cellular response to heat stress	1	393.8×	0.007	ATM
Resolution of D-loop Structures through Synthesis-Dependent Strand Annealing (SDSA)	1	393.8×	0.007	ATM
Homologous DNA Pairing and Strand Exchange	1	380.7×	0.007	ATM
Homology Directed Repair	1	308.6×	0.007	ATM
HDR through Homologous Recombination (HRR) or Single Strand Annealing (SSA)	1	308.6×	0.007	ATM
Impaired BRCA2 binding to RAD51	1	308.6×	0.007	ATM
Resolution of D-loop Structures through Holliday Junction Intermediates	1	300.5×	0.007	ATM
HDR through Single Strand Annealing (SSA)	1	292.8×	0.007	ATM
Regulation of TP53 Degradation	1	292.8×	0.007	ATM

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 2 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO term	Cohort genes	Fold	FDR	Sample cohort genes
establishment of RNA localization to telomere	1	4213.0×	0.005	ATM
establishment of protein-containing complex localization to telomere	1	4213.0×	0.005	ATM
positive regulation of telomerase catalytic core complex assembly	1	4213.0×	0.005	ATM
pre-B cell allelic exclusion	1	2808.7×	0.005	ATM
cellular response to nitrosative stress	1	2808.7×	0.005	ATM
peptidyl-serine autophosphorylation	1	1685.2×	0.005	ATM
negative regulation of mitochondrial fission	1	1685.2×	0.005	C11orf65
negative regulation of telomere capping	1	1685.2×	0.005	ATM
regulation of telomere maintenance via telomerase	1	1404.3×	0.005	ATM
obsolete negative regulation of protein targeting to mitochondrion	1	1404.3×	0.005	C11orf65
positive regulation of telomere maintenance via telomere lengthening	1	1404.3×	0.005	ATM
lipoprotein catabolic process	1	1203.7×	0.005	ATM
V(D)J recombination	1	1053.2×	0.005	ATM
meiotic telomere clustering	1	936.2×	0.005	ATM
female meiotic nuclear division	1	842.6×	0.005	ATM
histone mRNA catabolic process	1	842.6×	0.005	ATM
cellular response to X-ray	1	842.6×	0.005	ATM
DNA double-strand break processing	1	766.0×	0.005	ATM
regulation of autophagosome assembly	1	561.7×	0.006	ATM
pexophagy	1	526.6×	0.006	ATM
regulation of cellular response to heat	1	526.6×	0.006	ATM
positive regulation of DNA damage response, signal transduction by p53 class mediator	1	495.6×	0.006	ATM
replicative senescence	1	495.6×	0.006	ATM
negative regulation of B cell proliferation	1	468.1×	0.006	ATM
oocyte development	1	468.1×	0.006	ATM
cellular response to stress	1	421.3×	0.006	ATM
signal transduction in response to DNA damage	1	401.2×	0.006	ATM
positive regulation of telomere maintenance via telomerase	1	366.4×	0.007	ATM
cellular response to gamma radiation	1	300.9×	0.008	ATM
mitotic spindle assembly checkpoint signaling	1	280.9×	0.008	ATM

Therapeutics

Drug target analysis

Approved (phase 4): 1 · Phase ≥3: 1 · Phased (≥1): 1 · Undrugged: 1

Druggability breadth: 1 of 2 evidence-associated genes (50%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).

Genes with an approved drug

The molecule shown is one approved compound that hits the gene — not necessarily a drug of choice or one indicated for this disease.

Symbol	Example approved molecule
ATM	AMIODARONE HYDROCHLORIDE

Top cohort targets by molecule count

Symbol	Molecules	Max phase
ATM	35	4
C11orf65	0	0

Drugs targeting cohort genes (top 30)

Molecule	Max phase	Targets in cohort
AMIODARONE HYDROCHLORIDE	4	ATM
FURAZOLIDONE	4	ATM
ESTRADIOL ACETATE	4	ATM
NAFTIFINE HYDROCHLORIDE	4	ATM
METHYSERGIDE MALEATE	4	ATM
AMITRIPTYLINE HYDROCHLORIDE	4	ATM
XYLOMETAZOLINE HYDROCHLORIDE	4	ATM
FLUVOXAMINE MALEATE	4	ATM
ESTRADIOL VALERATE	4	ATM
PERMETHRIN	4	ATM
MITOTANE	4	ATM
TICLOPIDINE HYDROCHLORIDE	4	ATM
ENOXIMONE	4	ATM
METHYLENE BLUE ANHYDROUS	4	ATM
DITHIAZANINE IODIDE	4	ATM
ETHACRYNIC ACID	4	ATM
SECNIDAZOLE	4	ATM
MENADIONE	4	ATM
FENOFIBRATE	4	ATM
DIPYRIDAMOLE	4	ATM
DACTOLISIB	3	ATM
STREPTONIGRIN	2	ATM
CALCIMYCIN	2	ATM
ENPIROLINE	2	ATM
OXACEPROL	2	ATM
TOLONIUM CHLORIDE	2	ATM
ESTRADIOL BENZOATE	2	ATM
BERZOSERTIB	2	ATM
LARTESERTIB	2	ATM
ALTHIAZIDE	2	ATM

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 1.

Cohort genes with ChEMBL bioactivity (full, sorted by assay count)

Symbol	Assays	Type breakdown
ATM	240	Binding:233, Functional:5, ADMET:2

Cohort enzymes (BRENDA EC)

Symbol	EC numbers	Names
ATM	2.7.11.1	non-specific serine/threonine protein kinase

Cohort genes with high screening signal

≥100 ChEMBL assays — a studied-ness signal; see Therapeutics for approved-drug status.

Symbol	ChEMBL assays
ATM	240

Pharmacogenomics

Cohort genes with a PharmGKB record: 2; with CPIC/DPWG dosing guidelines: 0.

No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).

Drug repurposing candidates

30 approved/phased drugs hit cohort targets but don’t yet appear in disease-level clinical trials. Target-inhibition rationale is strongest for cancer driver genes; a bioactivity hit is a screening signal, not a treatment claim.

Compound	Max phase	Cohort target (bioactivity)
AMIODARONE HYDROCHLORIDE	4	ATM
FURAZOLIDONE	4	ATM
ESTRADIOL ACETATE	4	ATM
NAFTIFINE HYDROCHLORIDE	4	ATM
METHYSERGIDE MALEATE	4	ATM
AMITRIPTYLINE HYDROCHLORIDE	4	ATM
XYLOMETAZOLINE HYDROCHLORIDE	4	ATM
FLUVOXAMINE MALEATE	4	ATM
ESTRADIOL VALERATE	4	ATM
PERMETHRIN	4	ATM
MITOTANE	4	ATM
TICLOPIDINE HYDROCHLORIDE	4	ATM
ENOXIMONE	4	ATM
METHYLENE BLUE ANHYDROUS	4	ATM
DITHIAZANINE IODIDE	4	ATM
ETHACRYNIC ACID	4	ATM
SECNIDAZOLE	4	ATM
MENADIONE	4	ATM
FENOFIBRATE	4	ATM
DIPYRIDAMOLE	4	ATM
DACTOLISIB	3	ATM
STREPTONIGRIN	2	ATM
CALCIMYCIN	2	ATM
ENPIROLINE	2	ATM
OXACEPROL	2	ATM
TOLONIUM CHLORIDE	2	ATM
ESTRADIOL BENZOATE	2	ATM
BERZOSERTIB	2	ATM
LARTESERTIB	2	ATM
ALTHIAZIDE	2	ATM

Druggability pyramid

Cohort genes binned by druggability tier (high → low):

Tier	Definition	Genes	Symbols
A	Approved (phase 4 drug)	1	ATM
B	Phased (≥1) drug, not yet approved	0
C	Druggable family + PDB, no drug	0
D	Druggable family + AlphaFold only, no drug	0
E	Difficult family or no structure, no drug	1	C11orf65

Undrugged target profiles

1 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).

Symbol	ChEMBL assays	Drugged partners (top 3)
C11orf65	0	—

Clinical trials & evidence

Clinical trials

Clinical trials: 0.