MYT1

Summary

MYT1 (myelin transcription factor 1, HGNC:7622) is a protein-coding gene on chromosome 20q13.33, encoding Myelin transcription factor 1 (Q01538). Binds to the promoter region of genes encoding proteolipid proteins of the central nervous system.

The protein encoded by this gene is a member of a family of neural specific, zinc finger-containing DNA-binding proteins. The protein binds to the promoter regions of proteolipid proteins of the central nervous system and plays a role in the developing nervous system.

Source: NCBI Gene 4661 — RefSeq curated summary.

At a glance

• Gene–disease (curated): oculoauriculovertebral spectrum with radial defects (Strong, GenCC) — +2 more curated relationships
• GWAS associations: 23
• Clinical variants (ClinVar): 289 total — 1 pathogenic, 1 likely-pathogenic
• Druggable target: yes — 4 molecules with ChEMBL bioactivity
• MANE Select transcript: NM_004535

Identifiers

Gene identifiers

Field	Value
HGNC ID	HGNC:7622
Approved symbol	MYT1
Name	myelin transcription factor 1
Location	20q13.33
Locus type	gene with protein product
Status	Approved
Aliases	MTF1, MYTI, ZC2HC4A, NZF2, ZC2H2C1
Ensembl gene	ENSG00000196132
Ensembl biotype	protein_coding
OMIM	600379
Entrez	4661

Gene structure

Transcript identifiers

Ensembl transcripts: 9 — 8 protein_coding, 1 protein_coding_CDS_not_defined

ENST00000328439, ENST00000360149, ENST00000536311, ENST00000610671, ENST00000622439, ENST00000644172, ENST00000650655, ENST00000659024, ENST00000928401

RefSeq mRNA: 1 — MANE Select: NM_004535 NM_004535

CCDS: CCDS13558

Canonical transcript exons

ENST00000328439 — 23 exons

Exon	Start	End
ENSE00000663666	64219713	64219982
ENSE00000663667	64221893	64222047
ENSE00000856680	64205035	64205097
ENSE00000856681	64205553	64205800
ENSE00000856682	64207594	64208487
ENSE00001045080	64199892	64199922
ENSE00001045107	64236555	64236646
ENSE00001045139	64237287	64237390
ENSE00001171082	64232164	64232385
ENSE00001171090	64227888	64227971
ENSE00001171104	64223291	64223359
ENSE00001171136	64218911	64219035
ENSE00001171144	64217067	64217281
ENSE00001171153	64213534	64213647
ENSE00001171176	64211206	64211340
ENSE00001208375	64190063	64190160
ENSE00001347735	64239760	64239903
ENSE00001698818	64227415	64227477
ENSE00001735757	64223111	64223173
ENSE00001781758	64212048	64212138
ENSE00003720141	64240320	64242253
ENSE00003756160	64198862	64198916
ENSE00003841944	64164452	64164739

Expression profiles

Bgee: expression breadth ubiquitous, 101 present calls, max score 91.81.

FANTOM5 (CAGE): breadth broad, TPM avg 1.6579 / max 319.6606, expressed in 247 samples.

FANTOM5 promoters (10 alternative TSS)

Promoter ID	TPM avg	Samples expressed
185940	0.4382	89
185941	0.3590	114
185942	0.2316	87
185938	0.2166	73
185944	0.1828	64
185936	0.1097	71
185945	0.0494	19
185943	0.0274	9
185939	0.0224	11
209208	0.0208	10

Top tissues by expression

120 total, by Bgee expression score (0-100, higher = more expressed):

Tissue	Anatomy ID	Expression score	Quality
ganglionic eminence	UBERON:0004023	91.81	gold quality
cerebellum	UBERON:0002037	91.31	gold quality
cerebellar cortex	UBERON:0002129	91.28	gold quality
cerebellar hemisphere	UBERON:0002245	91.28	gold quality
right hemisphere of cerebellum	UBERON:0014890	90.63	gold quality
cortical plate	UBERON:0005343	79.20	gold quality
hypothalamus	UBERON:0001898	78.85	gold quality
amygdala	UBERON:0001876	78.14	gold quality
temporal lobe	UBERON:0001871	78.13	gold quality
brain	UBERON:0000955	76.17	gold quality
substantia nigra	UBERON:0002038	75.95	gold quality
nucleus accumbens	UBERON:0001882	75.48	gold quality
male germ line stem cell (sensu Vertebrata) in testis	CL:0000089 ∩ UBERON:0000473	75.24	gold quality
islet of Langerhans	UBERON:0000006	75.11	gold quality
corpus callosum	UBERON:0002336	74.99	gold quality
Ammon’s horn	UBERON:0001954	74.83	gold quality
pituitary gland	UBERON:0000007	74.77	gold quality
anterior cingulate cortex	UBERON:0009835	73.98	gold quality
adenohypophysis	UBERON:0002196	73.49	gold quality
putamen	UBERON:0001874	72.90	gold quality
caudate nucleus	UBERON:0001873	72.59	gold quality
ventricular zone	UBERON:0003053	71.87	gold quality
cerebral cortex	UBERON:0000956	71.80	gold quality
right frontal lobe	UBERON:0002810	71.66	gold quality
C1 segment of cervical spinal cord	UBERON:0006469	70.94	gold quality
frontal cortex	UBERON:0001870	70.73	gold quality
dorsolateral prefrontal cortex	UBERON:0009834	70.60	gold quality
prefrontal cortex	UBERON:0000451	70.22	gold quality
Brodmann (1909) area 9	UBERON:0013540	69.00	gold quality
primary visual cortex	UBERON:0002436	68.42	gold quality

Single-cell (SCXA)

Detected in 1 experiment(s), a significant marker in 0.

Experiment	Marker?	Max mean expression
E-ANND-3	no	0.41

Regulation

Is transcription factor: yes

JASPAR motifs

Motif	Name	Family
MA2506.1	MYT1	MYT

JASPAR matrix evidence (PMIDs): PMID:39671488

miRNA regulators (miRDB)

127 targeting MYT1, top 30 by miRDB confidence (max_score; target_count = how many genes the miRNA targets in total — lower means more specific):

miRNA	Max score	Avg score	miRNA target_count
HSA-MIR-5692A	100.00	74.40	6850
HSA-MIR-6833-3P	100.00	70.63	3197
HSA-MIR-3163	100.00	77.23	8605
HSA-MIR-4768-5P	100.00	69.49	2861
HSA-MIR-1277-5P	100.00	73.95	5056
HSA-MIR-5011-5P	100.00	83.46	5820
HSA-MIR-3646	100.00	73.56	5283
HSA-MIR-6873-3P	100.00	71.42	2626
HSA-MIR-200B-3P	100.00	73.31	2693
HSA-MIR-200C-3P	100.00	73.35	2685
HSA-MIR-429	100.00	73.44	2698
HSA-MIR-432-3P	100.00	67.86	705
HSA-MIR-126-5P	100.00	72.71	3180
HSA-MIR-656-3P	100.00	72.15	2788
HSA-MIR-34A-5P	99.99	71.21	1784
HSA-MIR-449A	99.99	71.05	1776
HSA-MIR-186-5P	99.99	70.83	3707
HSA-MIR-4282	99.99	75.36	6408
HSA-MIR-548C-3P	99.99	74.01	7587
HSA-MIR-27A-3P	99.98	72.13	2955
HSA-MIR-27B-3P	99.98	72.13	2955
HSA-MIR-9985	99.98	72.11	2939
HSA-MIR-4803	99.98	71.99	3117
HSA-MIR-4482-3P	99.98	72.50	3147
HSA-MIR-520D-5P	99.98	73.34	4883
HSA-MIR-524-5P	99.98	73.43	4882
HSA-MIR-302C-5P	99.97	72.56	3642
HSA-MIR-34C-5P	99.97	70.45	1577
HSA-MIR-449B-5P	99.97	70.26	1580
HSA-MIR-512-3P	99.97	67.35	1049

Literature-anchored findings (GeneRIF, showing 12)

• present in brain in infancy and prenatally in infants with periventricular leukomalacia (PMID:12524179)
• These results suggesta potential role for Myt1 in the regeneration of oligodendrocyte lineage cells in response to demyelination. (PMID:17330875)
• UVA-induced caspase-3 cleavage and DNA fragmentation were suppressed by the knockdown of human Myt1 in skin cancer cells. (PMID:19204086)
• although depletion of MYT1 alone did not affect long-term cell growth, it potentiated with DNA damage to inhibit cell growth in clonogenic survival and tumor xenograft models (PMID:23146904)
• A novel missense variant affecting function, c.323C>T (p.(Ser108Leu)), was identified in MYT1, in a patient presenting with a severe form of OAVS. MYT1 overexpression downregulated all RA receptors genes in vitro. (PMID:28612832)
• wide analyses of the effects of Myt1 and Myt1l expression in a glioblastoma cell line suggest that the two proteins have largely similar effects on endogenous gene expression. Transcriptional repression is likely mediated by binding to DNA via the known consensus site, whereas this site is not associated with the transcriptional start sites of genes with higher expression in the presence of Myt1 or Myt1l. (PMID:29291346)
• Here we demonstrate that re-expression of Myt1 or Myt1l in glioblastoma (GBM) cell lines slows proliferation, that expression of both is lower in more aggressive sub-types of glioma, and that reduced expression correlates with poor prognosis in both GBM and low grade glioma, and our data suggest that Myt1 and Myt1l directly repress expression of YAP1, a protein which promotes proliferation and GBM growth. (PMID:30312684)
• PICOT knock-down in Jurkat T cells resulted in a reduced histone H3 lysine 27 trimethylation (H3K27me3) at the PRC2 target gene, myelin transcription factor 1 (MYT1), suggesting that PICOT binding to EED alters PRC2-regulated transcriptional repression, and potentially contributes to the epigenetic regulation of chromatin silencing and remodeling. (PMID:30595380)
• MYT1 attenuates neuroblastoma cell differentiation by interacting with the LSD1/CoREST complex. (PMID:32251364)
• MYT1 role in the microtia-craniofacial microsomia spectrum. (PMID:32871052)
• Novel MYT1 variants expose the complexity of oculo-auriculo-vertebral spectrum genetic mechanisms. (PMID:33880880)
• Dysregulation of Myt1 expression acts as a potential peripheral biomarker for major depressive disorder and bipolar disorder. (PMID:34011236)

Cross-species orthologs

7 orthologs

Organism	Symbol	Gene ID
danio_rerio	myt1a	ENSDARG00000074030
danio_rerio	myt1b	ENSDARG00000102879
mus_musculus	Myt1	ENSMUSG00000010505
rattus_norvegicus	Myt1	ENSRNOG00000017346
drosophila_melanogaster	Pits	FBGN0030400
caenorhabditis_elegans	ztf-11	WBGENE00009939
caenorhabditis_elegans		WBGENE00010867

Paralogs (5): IRF2BPL (ENSG00000119669), ST18 (ENSG00000147488), IRF2BP2 (ENSG00000168264), IRF2BP1 (ENSG00000170604), MYT1L (ENSG00000186487)

Protein

Protein identifiers

Myelin transcription factor 1 — Q01538 (reviewed: Q01538)

Alternative names: Myelin transcription factor I, PLPB1, Proteolipid protein-binding protein

All UniProt accessions (3): A0A2R8Y3S5, Q01538, Q6P6D5

UniProt curated annotations — full annotation on UniProt →

Function. Binds to the promoter region of genes encoding proteolipid proteins of the central nervous system. May play a role in the development of neurons and oligodendroglia in the CNS. May regulate a critical transition point in oligodendrocyte lineage development by modulating oligodendrocyte progenitor proliferation relative to terminal differentiation and up-regulation of myelin gene transcription.

Subunit / interactions. Interacts with STEAP3.

Subcellular location. Nucleus.

Tissue specificity. Mostly in developing nervous system. Expressed in neural progenitors and oligodendrocyte lineage cells. More highly expressed in oligodendrocyte progenitors than in differentiated oligodendrocytes.

Domain organisation. Contains 7 zinc fingers of the C2HC class arranged in two widely separated clusters. These two domains of DNA binding can function independently and recognize the same DNA sequence.

Similarity. Belongs to the MYT1 family.

Isoforms (2)

UniProt ID	Names	Canonical?
Q01538-1	1	yes
Q01538-2	2

RefSeq proteins (1): NP_004526* (*=MANE)

Domains & families (InterPro)

ID	Name	Type
IPR002515	Znf_C2H2C	Repeat
IPR013681	Myelin_TF	Domain
IPR036060	Znf_C2H2C_sf	Homologous_superfamily

Pfam: PF01530, PF08474

UniProt features (58 total): binding site 28, compositionally biased region 10, zinc finger region 7, sequence conflict 7, region of interest 4, chain 1, splice variant 1

Structure

Experimental structures (PDB)

1 structures.

PDB	Method	Resolution (Å)
7Q45	X-RAY DIFFRACTION	2.1

Predicted structure (AlphaFold)

Model	pLDDT	Fraction very-high
AF-Q01538-F1	58.72	0.06

Functional residue map

Curated UniProt residues grouped by drug-discovery relevance — catalytic, ligand-binding, modification, and mutation-validated positions. Source: UniProtKB sequence features.

Ligand- & substrate-binding residues (28): 30; 35; 48; 54; 442; 447; 460; 466; 486; 491; 504; 510 …

Function

Pathways and Gene Ontology

Reactome pathways

0 pathways

MSigDB gene sets: 514 (showing top): GSE18804_BRAIN_VS_COLON_TUMORAL_MACROPHAGE_UP, TAATAAT_MIR126, GOBP_RESPONSE_TO_ZINC_ION, GGTGTGT_MIR329, GGGNRMNNYCAT_UNKNOWN, TGCACTT_MIR519C_MIR519B_MIR519A, GCANCTGNY_MYOD_Q6, IVANOVA_HEMATOPOIESIS_MATURE_CELL, BROWNE_HCMV_INFECTION_12HR_UP, GGGTGGRR_PAX4_03, CAGCTG_AP4_Q5, MORF_RAD51L3, MOLENAAR_TARGETS_OF_CCND1_AND_CDK4_UP, ATGTTAA_MIR302C, GOBP_RESPONSE_TO_METAL_ION

GO Biological Process (4): regulation of DNA-templated transcription (GO:0006355), nervous system development (GO:0007399), cell differentiation (GO:0030154), regulation of transcription by RNA polymerase II (GO:0006357)

GO Molecular Function (5): DNA-binding transcription factor activity, RNA polymerase II-specific (GO:0000981), DNA binding (GO:0003677), DNA-binding transcription factor activity (GO:0003700), zinc ion binding (GO:0008270), metal ion binding (GO:0046872)

GO Cellular Component (4): chromatin (GO:0000785), nucleus (GO:0005634), nucleoplasm (GO:0005654), cytosol (GO:0005829)

GO top-level categories

Rollup of top GO terms by namespace:

Category	Terms
cellular anatomical structure	3
regulation of DNA-templated transcription	2
DNA-templated transcription	1
regulation of gene expression	1
regulation of RNA biosynthetic process	1
system development	1
cellular developmental process	1
transcription by RNA polymerase II	1
chromatin	1
RNA polymerase II transcription regulatory region sequence-specific DNA binding	1
DNA-binding transcription factor activity	1
regulation of transcription by RNA polymerase II	1
nucleic acid binding	1
transcription cis-regulatory region binding	1
transcription regulator activity	1
transition metal ion binding	1
cation binding	1
chromosome	1
intracellular membrane-bounded organelle	1
nuclear lumen	1
cytoplasm	1

Protein interactions and networks

STRING

1074 interactions, top by confidence (×1000):

Protein A	Protein B	Partner UniProt	Score
MYT1	PLP1	P04400	812
MYT1	PKMYT1	Q99640	803
MYT1	PHF20	Q9BVI0	507
MYT1	NEUROG3	Q9Y4Z2	488
MYT1	HDAC1	Q13547	478
MYT1	WEE1	P30291	453
MYT1	NPLOC4	Q8TAT6	418
MYT1	MBP	P02686	412
MYT1	VPS28	Q9UK41	405
MYT1	ASCL1	P50553	398
MYT1	SIN3B	O75182	392
MYT1	ZFHX4	Q86UP3	387
MYT1	RNF31	Q96EP0	374
MYT1	E2F7	Q96AV8	372
MYT1	ZFHX3	Q15911	372

IntAct

8 interactions, top by confidence:

A	B	Type	Score
HDAC2	KDM1A	psi-mi:“MI:0914”(association)	0.890
FHL2	CNOT1	psi-mi:“MI:0914”(association)	0.530
CDK1	MYT1	psi-mi:“MI:0217”(phosphorylation reaction)	0.440
MYT1	NPM1	psi-mi:“MI:0915”(physical association)	0.400
ATG16L1		psi-mi:“MI:0914”(association)	0.350
RCOR1	ZBTB5	psi-mi:“MI:0914”(association)	0.350
DISC1	MYT1	psi-mi:“MI:0915”(physical association)	0.000

BioGRID (12): MYT1 (Reconstituted Complex), MYT1 (Affinity Capture-RNA), MYT1 (Proximity Label-MS), MYT1 (Proximity Label-MS), MYT1 (Affinity Capture-MS), MYT1 (Affinity Capture-MS), PIN1 (Affinity Capture-Western), PIN1 (Reconstituted Complex), MYT1 (Affinity Capture-MS), MYT1 (Affinity Capture-Western), MYT1 (Affinity Capture-Western), MYT1 (Affinity Capture-MS)

ESM2 similar proteins: A0A1L8H8C0, A0A1L8HFX9, A0A2K1J5A5, A0A2R6X6S3, A0JM08, A2RUV4, B1AZP2, F1QIC4, O14490, O61366, P46012, P46582, P70047, P83510, P97836, P97839, Q01538, Q08D57, Q09314, Q09599, Q0KIC3, Q18221, Q1LY77, Q498L0, Q501J7, Q52KW0, Q5F3P8, Q5RAU1, Q5SW79, Q5TZ18, Q5XHF3, Q66J90, Q69Z38, Q6A065, Q6DFG0, Q6PEI3, Q71M21, Q80TN7, Q86TC9, Q8CFC2

Diamond homologs: A2A5N8, B1B1A0, D3YUG0, D3YXK1, D3ZWK4, E1C2V1, O02274, O60284, O95251, P39769, P59178, P70047, P70475, P78364, P97500, Q01538, Q05BQ5, Q1JQD9, Q1RNF8, Q29L50, Q32N90, Q3MIF2, Q4V7W5, Q5DTW2, Q5R737, Q5SVQ0, Q5VUG0, Q5VXD3, Q64028, Q6DIN3, Q6P5G3, Q6SPE9, Q6SPF0, Q7Z3H4, Q80TY4, Q810T5, Q8BLB7, Q8C8Y5, Q8CFC2, Q8CHP6

SIGNOR signaling

2 interactions.

A	Effect	B	Mechanism
MYT1	“down-regulates quantity by repression”	YAP1	“transcriptional regulation”
MAP2K1	“down-regulates activity”	MYT1	phosphorylation

Disease & clinical

Clinical variants and AI predictions

ClinVar

289 variants total. Per-class counts are floors (≥ shown; pagination cap):

Classification	Count (floor)
Pathogenic	1
Likely pathogenic	1
Uncertain significance	190
Likely benign	46
Benign	19

Top pathogenic / likely-pathogenic (2)

Variant ID	HGVS	Classification
58980	GRCh38/hg38 20q13.33(chr20:64206685-64261836)x1	Pathogenic
981907	NM_004535.3(MYT1):c.790G>C (p.Glu264Gln)	Likely pathogenic

SpliceAI

0 predictions. Top by Δscore:

AlphaMissense

7416 scored. Top likely-pathogenic:

Variant	Protein change	am_pathogenicity
20:64205036:T:C	C30R	1.000
20:64211238:T:A	C442S	1.000
20:64211238:T:C	C442R	1.000
20:64211239:G:A	C442Y	1.000
20:64211239:G:C	C442S	1.000
20:64211240:T:G	C442W	1.000
20:64211253:T:A	C447S	1.000
20:64211253:T:C	C447R	1.000
20:64211254:G:A	C447Y	1.000
20:64211254:G:C	C447S	1.000
20:64211254:G:T	C447F	1.000
20:64211255:T:G	C447W	1.000
20:64211260:G:A	G449D	1.000
20:64211265:G:C	G451R	1.000
20:64211266:G:A	G451D	1.000
20:64211268:C:G	H452D	1.000
20:64211269:A:G	H452R	1.000
20:64211270:C:A	H452Q	1.000
20:64211270:C:G	H452Q	1.000
20:64211272:T:A	V453D	1.000
20:64211277:G:T	G455W	1.000
20:64211278:G:A	G455E	1.000
20:64211292:C:G	H460D	1.000
20:64211294:C:A	H460Q	1.000
20:64211294:C:G	H460Q	1.000
20:64211295:C:A	R461S	1.000
20:64211298:A:C	S462R	1.000
20:64211300:C:A	S462R	1.000
20:64211300:C:G	S462R	1.000
20:64211308:G:A	G465D	1.000

dbSNP variants (sampled 300 via entrez): RS1000014079 (20:64176848 T>C), RS1000023345 (20:64211461 T>C), RS1000031556 (20:64182445 C>T), RS1000061962 (20:64230381 C>T), RS1000123445 (20:64218733 T>A,C), RS1000162763 (20:64197909 C>T), RS1000167467 (20:64236608 G>A,T), RS1000276873 (20:64235002 C>CT), RS1000293577 (20:64202477 C>T), RS1000319773 (20:64242333 C>T), RS1000324264 (20:64202303 A>C), RS1000360278 (20:64185067 G>A), RS1000472674 (20:64180833 G>A,C,T), RS1000489433 (20:64219009 A>C), RS1000500310 (20:64190020 C>G)

Disease associations

OMIM: gene MIM:600379 | disease phenotypes: MIM:303350

GenCC curated gene-disease

Disease	Classification	Inheritance
oculoauriculovertebral spectrum with radial defects	Strong	Autosomal dominant
intellectual disability	Limited	Autosomal dominant
craniofacial microsomia	Limited	Autosomal dominant

Mondo (5): hereditary spastic paraplegia (MONDO:0019064), autism spectrum disorder (MONDO:0005258), intellectual disability (MONDO:0001071), oculoauriculovertebral spectrum with radial defects (MONDO:0007712), craniofacial microsomia (MONDO:0015397)

Orphanet (3): Hereditary spastic paraplegia (Orphanet:685), NON RARE IN EUROPE: Autism (Orphanet:106), NON RARE IN EUROPE: Unexplained intellectual disability (Orphanet:319658)

HPO phenotypes

0 total (0 of 0 shown, HPO-id order):

GWAS associations

23 associations (top):

Study	Trait	p-value
GCST001474_9	Hypothyroidism	9.000000e-06
GCST002318_80	Rheumatoid arthritis	3.000000e-12
GCST002318_81	Rheumatoid arthritis	6.000000e-09
GCST003476_2	Eyebrow thickness	7.000000e-06
GCST003988_3	Hypothyroidism	7.000000e-06
GCST005042_20	Restless legs syndrome	2.000000e-34
GCST006959_154	Rheumatoid arthritis	6.000000e-12
GCST006959_22	Rheumatoid arthritis	5.000000e-09
GCST007001_16	Cerebrospinal AB1-42 levels in normal cognition	3.000000e-07
GCST007096_29	Pulse pressure	2.000000e-11
GCST007097_117	Pulse pressure	1.000000e-07
GCST007097_118	Pulse pressure	7.000000e-10
GCST008529_16	Tea consumption	4.000000e-08
GCST010173_151	Triglyceride levels	9.000000e-09
GCST011096_14	Systemic lupus erythematosus	2.000000e-10
GCST011097_7	Systemic lupus erythematosus	6.000000e-06
GCST011995_19	Restless legs syndrome	2.000000e-18
GCST90002401_14	Platelet distribution width	1.000000e-10
GCST90011866_1	Systemic lupus erythematosus	6.000000e-06
GCST90020024_810	A body shape index	9.000000e-09
GCST90020025_1768	Waist-to-hip ratio adjusted for BMI	5.000000e-12
GCST90020027_387	Waist-hip index	2.000000e-12
GCST90020029_158	Waist circumference adjusted for body mass index	4.000000e-09

EFO canonical traits (7, from GWAS)

EFO ID	Trait name
EFO:0004670	beta-amyloid 1-42 measurement
EFO:0005763	pulse pressure measurement
EFO:0010091	tea consumption measurement
EFO:0004530	triglyceride measurement
EFO:0007984	platelet component distribution width
EFO:0007789	BMI-adjusted waist circumference
EFO:0007788	BMI-adjusted waist-hip ratio

MeSH disease descriptors (3)

Descriptor	Name	Tree numbers
D006053	Goldenhar Syndrome	C05.116.099.370.231.576.410; C05.660.207.231.576.410; C16.131.621.207.231.576.410
D008607	Intellectual Disability	C10.597.606.360; C23.888.592.604.646; F01.700.687; F03.625.539
D015419	Spastic Paraplegia, Hereditary	C10.500.300.820; C10.574.500.495.820; C10.668.829.800.300.820; C16.131.666.300.820; C16.320.400.375.820

Drugs & pharmacology

Drug and pharmacology data

Is drug target: yes

ChEMBL targets (1): CHEMBL2331044 (SINGLE PROTEIN)

Molecules with ChEMBL bioactivity

4 molecules (phase ≥1), by development phase (incl. off-target/promiscuous compounds). Patent mentions across the top 20 by phase: 56,147 (via chembl_molecule»patent_compound — counts attach to the compound, not the gene–compound relationship, so off-target/promiscuous molecules can dominate).

Molecule	Name	Phase	Patents
CHEMBL1421	DASATINIB ANHYDROUS	4	55,003
CHEMBL5416410	DASATINIB	4	655
CHEMBL5199076	LUNRESERTIB	2	34
CHEMBL49120	PD-0166285	1	455

PharmGKB: 1 entry (VIP=true, CPIC=false)

Binding affinities (BindingDB)

591 measured of 920 human assays (920 total across all organisms); most potent 50 below. Values come from heterogeneous assays and are not directly comparable.

Ligand	Measure	Value	Patent
3-amino-6-(2-amino-4-pyridinyl)-4-(3-hydroxy-2,6-dimethylphenyl)pyridine-2-carboxamide	IC50	2 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-4-(3-hydroxy-2,6-dimethylphenyl)-6-(5-methoxy-2-pyridinyl)pyridine-2-carboxamide	IC50	2 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-4-(3-hydroxy-2,6-dimethylphenyl)-6-(6-methoxy-2-pyridinyl)pyridine-2-carboxamide	IC50	2 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-4-(3-hydroxy-2,6-dimethylphenyl)-6-[4-(trifluoromethyl)-2-pyridinyl]pyridine-2-carboxamide	IC50	2 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-4-(3-hydroxy-2,6-dimethylphenyl)-6-[6-(oxan-4-yloxy)-2-pyridinyl]pyridine-2-carboxamide	IC50	2 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-4-(5-methyl-1H-indazol-4-yl)-6-[6-[(3R)-oxolan-3-yl]oxy-2-pyridinyl]pyridine-2-carboxamide	IC50	2 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-6-[6-[(2R)-2-amino-2-phenylethoxy]-2-pyridinyl]-4-(5-methyl-1H-indazol-4-yl)pyridine-2-carboxamide	IC50	2 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-6-[2-[[(1R)-2-hydroxy-1-phenylethyl]amino]pyrimidin-4-yl]-4-(5-methyl-1H-indazol-4-yl)pyridine-2-carboxamide	IC50	2 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-6-[2-[[(2R)-1-hydroxy-3-phenylpropan-2-yl]amino]pyrimidin-4-yl]-4-(5-methyl-1H-indazol-4-yl)pyridine-2-carboxamide	IC50	2 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
5-amino-2-[2-(2,2-difluoroethylamino)phenyl]-6-(5-methyl-1H-indazol-4-yl)pyrimidine-4-carboxamide	IC50	2 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
5-amino-6-(5-chloro-1H-indazol-4-yl)-2-[2-(2,2-dimethylpropanoylamino)phenyl]pyrimidine-4-carboxamide	IC50	2 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
7-amino-8-(5-methyl-1H-indazol-4-yl)-[1]benzofuro[3,2-b]pyridine-6-carboxamide	IC50	2 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
5-amino-2-[2-(cyclopropylsulfonylamino)-5-fluorophenyl]-6-(5-methyl-1H-indazol-4-yl)pyrimidine-4-carboxamide	IC50	2 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
5-amino-2-[3-(2,2-dimethylpropanoylamino)-4-pyridinyl]-6-(5-methyl-1H-indazol-4-yl)pyrimidine-4-carboxamide	IC50	2 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
5-amino-6-(5-methyl-1H-indazol-4-yl)-2-[2-[(3-methyloxan-4-yl)amino]-3-pyridinyl]pyrimidine-4-carboxamide	IC50	2 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
5-amino-2-[2-[[(1R)-2,2-difluoro-1-pyridin-3-ylethyl]amino]-3-pyridinyl]-6-(5-methyl-1H-indazol-4-yl)pyrimidine-4-carboxamide	IC50	2 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
5-amino-2-[2-[[(1S)-2,2-difluoro-1-pyridin-3-ylethyl]amino]-3-pyridinyl]-6-(5-methyl-1H-indazol-4-yl)pyrimidine-4-carboxamide	IC50	2 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-4-(3-hydroxy-2,6-dimethylphenyl)-6-pyrazin-2-ylpyridine-2-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-4-(3-hydroxy-2,6-dimethylphenyl)-6-(1,2-thiazol-5-yl)pyridine-2-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-4-(3-hydroxy-2,6-dimethylphenyl)-6-pyrimidin-4-ylpyridine-2-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-4-(3-hydroxy-2,6-dimethylphenyl)-6-(1,3-thiazol-5-yl)pyridine-2-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-4-(3-hydroxy-2,6-dimethylphenyl)-6-(6-methyl-2-pyridinyl)pyridine-2-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-6-[2-(2-methoxyethoxy)pyrimidin-4-yl]-4-(5-methyl-1H-indazol-4-yl)pyridine-2-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-4-(5-methyl-1H-indazol-4-yl)-6-[2-(pyrimidin-2-ylmethoxy)pyrimidin-4-yl]pyridine-2-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-4-(5-methyl-1H-indazol-4-yl)-6-[2-(pyridin-3-ylmethoxy)pyrimidin-4-yl]pyridine-2-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-6-[6-[[(1S)-2-hydroxy-1-phenylethyl]amino]-2-pyridinyl]-4-(5-methyl-1H-indazol-4-yl)pyridine-2-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-6-[2-[(2R)-2-methoxybutoxy]pyrimidin-4-yl]-4-(5-methyl-1H-indazol-4-yl)pyridine-2-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-6-[2-[(2R)-2-amino-2-phenylethoxy]pyrimidin-4-yl]-4-(5-methyl-1H-indazol-4-yl)pyridine-2-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
5-amino-2-[2-(2,2-dimethylpropanoylamino)phenyl]-6-(5-methyl-1H-indazol-4-yl)pyrimidine-4-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-6-[6-[(1-aminocyclopropyl)methoxy]-2-pyridinyl]-4-(5-methyl-1H-indazol-4-yl)pyridine-2-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-2-(3-hydroxy-2,6-dimethylphenyl)-6-[2-[(1-methylcyclopropanecarbonyl)amino]phenyl]pyridine-4-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
5-amino-2-[2-[(2-fluoro-2-methylpropanoyl)amino]phenyl]-6-(5-methyl-1H-indazol-4-yl)pyrimidine-4-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
5-amino-2-[2-[(2,2-difluoro-1-methylcyclopropanecarbonyl)amino]phenyl]-6-(5-methyl-1H-indazol-4-yl)pyrimidine-4-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
5-amino-2-[5-fluoro-2-[(1-methylcyclopropanecarbonyl)amino]phenyl]-6-(5-methyl-1H-indazol-4-yl)pyrimidine-4-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
5-amino-2-[2-[(1-fluorocyclopropanecarbonyl)amino]phenyl]-6-(5-methyl-1H-indazol-4-yl)pyrimidine-4-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
5-amino-2-[2-(2-methoxyethylamino)phenyl]-6-(5-methyl-1H-indazol-4-yl)pyrimidine-4-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
5-amino-2-[2-(cyclopropylamino)phenyl]-6-(5-methyl-1H-indazol-4-yl)pyrimidine-4-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
5-amino-2-[2-[(1-cyanocyclopropanecarbonyl)amino]-4,5-difluorophenyl]-6-(5-methyl-1H-indazol-4-yl)pyrimidine-4-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
5-amino-6-(5-methyl-1H-indazol-4-yl)-2-[2-[[(2R)-oxolan-2-yl]methylamino]-3-pyridinyl]pyrimidine-4-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
5-amino-2-[2-[(5-cyano-2-methylpyrimidin-4-yl)amino]-4-fluorophenyl]-6-(5-methyl-1H-indazol-4-yl)pyrimidine-4-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
5-amino-2-[2-[(2-cyano-4-pyridinyl)amino]-4-fluorophenyl]-6-(5-methyl-1H-indazol-4-yl)pyrimidine-4-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
5-amino-2-[2-[(3-fluoro-2-pyridinyl)amino]-3-pyridinyl]-6-(5-methyl-1H-indazol-4-yl)pyrimidine-4-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
5-amino-2-[4-cyano-2-[(2-methylpyrimidin-4-yl)amino]phenyl]-6-(5-methyl-1H-indazol-4-yl)pyrimidine-4-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
5-amino-2-[2-(2,2-dimethylpropanoylamino)-3-pyridinyl]-6-(5-methyl-1H-indazol-4-yl)pyrimidine-4-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
5-amino-2-[2-fluoro-5-(oxan-4-ylamino)-4-pyridinyl]-6-(5-methyl-1H-indazol-4-yl)pyrimidine-4-carboxamide	IC50	3 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-6-(2-amino-4-pyridinyl)-4-(3-hydroxy-2,6-dimethylphenyl)pyridine-2-carboxamide	IC50	4 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-4-(3-hydroxy-2,6-dimethylphenyl)-6-pyridin-2-ylpyridine-2-carboxamide	IC50	4 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-4-(3-hydroxy-2,6-dimethylphenyl)-6-(5-methyl-2-pyridinyl)pyridine-2-carboxamide	IC50	4 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-4-(3-hydroxy-2,6-dimethylphenyl)-6-(5-methoxy-2-pyridinyl)pyridine-2-carboxamide	IC50	4 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME
3-amino-4-(5-methyl-1H-indazol-4-yl)-6-[2-(3-methyloxetan-3-yl)oxypyrimidin-4-yl]pyridine-2-carboxamide	IC50	4 nM	US-20250304537: HETEROARENES, PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME, AND METHODS OF USING THE SAME

ChEMBL bioactivities

33 potent at pChembl≥5 of 33 total, top 33 by pChembl (potency: 10 = 0.1 nM, 6 = 1 µM).

pChembl	Type	Value	Unit	Molecule
8.82	IC50	1.5	nM	LUNRESERTIB
8.72	IC50	1.9	nM	CHEMBL5562687
8.59	IC50	2.6	nM	CHEMBL5566629
8.54	IC50	2.9	nM	CHEMBL5200005
8.44	IC50	3.6	nM	CHEMBL5573176
8.38	IC50	4.2	nM	CHEMBL5572884
8.37	IC50	4.3	nM	CHEMBL5569464
8.34	IC50	4.6	nM	CHEMBL5573390
8.32	IC50	4.8	nM	CHEMBL5575890
8.32	IC50	4.8	nM	CHEMBL5567277
8.31	IC50	4.9	nM	CHEMBL5573971
8.26	IC50	5.5	nM	CHEMBL5569840
8.24	IC50	5.8	nM	CHEMBL5571889
8.18	IC50	6.6	nM	CHEMBL5573662
8.15	IC50	7.1	nM	CHEMBL5563675
8.14	IC50	7.2	nM	PD-0166285
7.99	IC50	10.3	nM	CHEMBL5570485
7.98	IC50	10.4	nM	CHEMBL5566455
7.94	IC50	11.4	nM	CHEMBL5565137
7.79	IC50	16.2	nM	CHEMBL5564808
7.76	IC50	17.3	nM	CHEMBL5566287
7.63	IC50	23.2	nM	CHEMBL5574963
7.62	IC50	24	nM	PD-0166285
7.60	IC50	25.3	nM	CHEMBL5571632
7.31	IC50	48.9	nM	CHEMBL5572439
7.20	IC50	63	nM	DASATINIB
7.16	IC50	69	nM	CHEMBL5573978
6.65	IC50	226	nM	DASATINIB ANHYDROUS
6.65	IC50	222.9	nM	CHEMBL5574840
6.45	IC50	359.1	nM	CHEMBL5569793
6.15	IC50	713.8	nM	CHEMBL5570533
5.92	IC50	1213	nM	CHEMBL5572096
5.83	IC50	1495	nM	CHEMBL5593294

PubChem BioAssay actives

33 with measured affinity, of 94 total; 32 most potent distinct compounds. Largely complementary to BindingDB; screening values are coarse (µM, 4 dp), so sub-nM hits tie at the floor.

Compound	Assay	Type	Value	Unit
2-amino-1-(3-hydroxy-2,6-dimethylphenyl)-5,6-dimethylpyrrolo[2,3-b]pyridine-3-carboxamide	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0015	uM
5-[6-amino-5-(3-hydroxy-2,6-dimethylphenyl)-2,3-dimethylpyrrolo[2,3-b]pyrazine-7-carbonyl]-6,7-dihydro-4H-pyrazolo[1,5-a]pyrazine-3-carbonitrile	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0019	uM
[6-amino-2-cyclopropyl-5-(3-hydroxy-2,6-dimethylphenyl)-3-methylpyrrolo[2,3-b]pyrazin-7-yl]-(6,7-dihydro-4H-pyrazolo[1,5-a]pyrazin-5-yl)methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0026	uM
6-amino-5-(3-hydroxy-2,6-dimethylphenyl)-2,3-dimethylpyrrolo[2,3-b]pyrazine-7-carboxamide	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0029	uM
[6-amino-5-(3-hydroxy-2,6-dimethylphenyl)-2,3-dimethylpyrrolo[2,3-b]pyrazin-7-yl]-(3-chloro-6,7-dihydro-4H-pyrazolo[1,5-a]pyrazin-5-yl)methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0036	uM
[6-amino-5-(3-hydroxy-2,6-dimethylphenyl)-2,3-dimethylpyrrolo[2,3-b]pyrazin-7-yl]-(6,7-dihydro-4H-pyrazolo[1,5-a]pyrazin-5-yl)methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0042	uM
[6-amino-5-(3-hydroxy-2,6-dimethylphenyl)-2,3-dimethylpyrrolo[2,3-b]pyrazin-7-yl]-(6,8-dihydro-5H-[1,2,4]triazolo[4,3-a]pyrazin-7-yl)methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0043	uM
[6-amino-5-(3-hydroxy-2,6-dimethylphenyl)-2,3-dimethylpyrrolo[2,3-b]pyrazin-7-yl]-[2-(trifluoromethyl)-6,7-dihydro-4H-pyrazolo[1,5-a]pyrazin-5-yl]methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0046	uM
[6-amino-5-(3-hydroxy-2,6-dimethylphenyl)-2,3-dimethylpyrrolo[2,3-b]pyrazin-7-yl]-[3-(trifluoromethyl)-6,7-dihydro-4H-pyrazolo[1,5-a]pyrazin-5-yl]methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0048	uM
[6-amino-5-(3-hydroxy-2,6-dimethylphenyl)-2,3-dimethylpyrrolo[2,3-b]pyrazin-7-yl]-(3-methyl-6,7-dihydro-4H-[1,2]oxazolo[4,3-c]pyridin-5-yl)methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0048	uM
[6-amino-2-cyclopropyl-5-(3-hydroxy-2,6-dimethylphenyl)pyrrolo[2,3-b]pyrazin-7-yl]-(6,7-dihydro-4H-pyrazolo[1,5-a]pyrazin-5-yl)methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0049	uM
[6-amino-5-(3-hydroxy-2,6-dimethylphenyl)-2,3-dimethylpyrrolo[2,3-b]pyrazin-7-yl]-(6,8-dihydro-5H-imidazo[1,5-a]pyrazin-7-yl)methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0055	uM
[6-amino-5-(3-hydroxy-2,6-dimethylphenyl)-2,3-dimethylpyrrolo[2,3-b]pyrazin-7-yl]-(6,8-dihydro-5H-imidazo[1,2-a]pyrazin-7-yl)methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0058	uM
[6-amino-5-(3-hydroxy-2,6-dimethylphenyl)-2,3-dimethylpyrrolo[2,3-b]pyrazin-7-yl]-(6,8-dihydro-5H-[1,2,4]triazolo[1,5-a]pyrazin-7-yl)methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0066	uM
[6-amino-5-(3-hydroxy-2,6-dimethylphenyl)-2,3-dimethylpyrrolo[2,3-b]pyrazin-7-yl]-(2-methyl-6,7-dihydro-4H-pyrazolo[1,5-a]pyrazin-5-yl)methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0071	uM
6-(2,6-dichlorophenyl)-2-[4-[2-(diethylamino)ethoxy]anilino]-8-methylpyrido[2,3-d]pyrimidin-7-one	729550: Binding affinity to human full-length His-tagged Myt1 kinase expressed in HEK293 cells by TR-FRET based binding assay	ic50	0.0072	uM
[6-amino-5-(3-hydroxy-2,6-dimethylphenyl)-2-methyl-3-(1-methylpiperidin-4-yl)pyrrolo[2,3-b]pyrazin-7-yl]-(6,7-dihydro-4H-pyrazolo[1,5-a]pyrazin-5-yl)methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0103	uM
[6-amino-5-(3-hydroxy-2,6-dimethylphenyl)-3-[(3R)-3-hydroxypiperidin-1-yl]-2-methylpyrrolo[2,3-b]pyrazin-7-yl]-(6,7-dihydro-4H-pyrazolo[1,5-a]pyrazin-5-yl)methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0104	uM
2-[6-amino-5-(3-hydroxy-2,6-dimethylphenyl)-2,3-dimethylpyrrolo[2,3-b]pyrazine-7-carbonyl]-3,4-dihydro-1H-pyrido[1,2-a]pyrazin-6-one	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0114	uM
[6-amino-5-(3-hydroxy-2,6-dimethylphenyl)-2,3-dimethylpyrrolo[2,3-b]pyrazin-7-yl]-[3-(2-hydroxypropan-2-yl)-6,8-dihydro-5H-imidazo[1,5-a]pyrazin-7-yl]methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0162	uM
[6-amino-5-(3-hydroxy-2,6-dimethylphenyl)-2,3-dimethylpyrrolo[2,3-b]pyrazin-7-yl]-[2-(trifluoromethyl)-6,8-dihydro-5H-[1,2,4]triazolo[1,5-a]pyrazin-7-yl]methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0173	uM
[6-amino-5-(3-hydroxy-2,6-dimethylphenyl)-2,3-dimethylpyrrolo[2,3-b]pyrazin-7-yl]-(7,8-dihydro-5H-pyrido[3,4-b]pyrazin-6-yl)methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0232	uM
[6-amino-5-(3-hydroxy-2,6-dimethylphenyl)-3-methyl-2-prop-1-ynylpyrrolo[2,3-b]pyrazin-7-yl]-(6,8-dihydro-5H-[1,2,4]triazolo[1,5-a]pyrazin-7-yl)methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0253	uM
[2-amino-5-chloro-1-(3-hydroxy-2,6-dimethylphenyl)pyrrolo[2,3-b]pyridin-3-yl]-(6,7-dihydro-4H-pyrazolo[1,5-a]pyrazin-5-yl)methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0489	uM
N-(2-chloro-6-methylphenyl)-2-[[6-[4-(2-hydroxyethyl)piperazin-1-yl]-2-methylpyrimidin-4-yl]amino]-1,3-thiazole-5-carboxamide;hydrate	729550: Binding affinity to human full-length His-tagged Myt1 kinase expressed in HEK293 cells by TR-FRET based binding assay	ic50	0.0630	uM
[6-amino-2-(3,6-dihydro-2H-pyran-4-yl)-5-(3-hydroxy-2,6-dimethylphenyl)-3-methylpyrrolo[2,3-b]pyrazin-7-yl]-(6,7-dihydro-4H-pyrazolo[1,5-a]pyrazin-5-yl)methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.0690	uM
[6-amino-7-(3-hydroxy-2,6-dimethylphenyl)-2-methylpyrrolo[2,3-d]pyrimidin-5-yl]-(6,8-dihydro-5H-[1,2,4]triazolo[1,5-a]pyrazin-7-yl)methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.2229	uM
Dasatinib	2093196: Inhibition of MYT1 (unknown origin)	ic50	0.2260	uM
[6-amino-5-(3-hydroxy-2,6-dimethylphenyl)-3-(trifluoromethyl)pyrrolo[2,3-b]pyrazin-7-yl]-(6,7-dihydro-4H-pyrazolo[1,5-a]pyrazin-5-yl)methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.3591	uM
[6-amino-5-(3-hydroxy-2,6-dimethylphenyl)-2,3-dimethylpyrrolo[2,3-b]pyrazin-7-yl]-(1,1-dioxo-1,4-thiazinan-4-yl)methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	0.7138	uM
1-[4-[6-amino-5-(3-hydroxy-2,6-dimethylphenyl)-2,3-dimethylpyrrolo[2,3-b]pyrazine-7-carbonyl]piperazin-1-yl]ethanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	1.2130	uM
[2-amino-1-(3-hydroxy-2,6-dimethylphenyl)-5-methylpyrrolo[3,2-b]pyridin-3-yl]-[2-(trifluoromethyl)-6,8-dihydro-5H-[1,2,4]triazolo[1,5-a]pyrazin-7-yl]methanone	2093197: Inhibition of human recombinant MYT1 incubated for 60 mins in presence of tracer178 by HTRF analysis	ic50	1.4950	uM

CTD chemical–gene interactions

34 total (human), top 30 by PubMed support.

Chemical	Actions (top 5)	PubMed papers
Benzo(a)pyrene	affects methylation, decreases expression	3
propionaldehyde	increases expression, increases methylation	2
sodium arsenite	increases methylation, decreases expression	2
butyraldehyde	increases expression, increases methylation	2
pentanal	increases expression, increases methylation	2
Lead	affects expression, decreases expression	2
Phenylmercuric Acetate	decreases expression, affects cotreatment	2
Valproic Acid	affects expression, increases methylation	2
Aflatoxin B1	decreases methylation, increases methylation	2
aristolochic acid I	decreases expression	1
LCS1269	affects expression	1
bisphenol A	increases expression	1
nonanal	increases methylation	1
n-hexanal	increases methylation	1
arsenite	increases methylation	1
cryptolepine	decreases expression	1
benzo(e)pyrene	increases methylation	1
aflatoxin B2	increases methylation	1
caprylic aldehyde	increases methylation	1
beta-methylcholine	affects expression	1
heptanal	increases methylation	1
16-hydroxycleroda-3,13(14)-dien-15,16-olide	increases expression	1
CGP 52608	affects binding, increases reaction	1
4-(5-benzo(1,3)dioxol-5-yl-4-pyridin-2-yl-1H-imidazol-2-yl)benzamide	affects cotreatment, decreases expression	1
dorsomorphin	decreases expression, affects cotreatment	1
(+)-JQ1 compound	decreases expression	1
Vorinostat	decreases expression	1
Colchicine	increases phosphorylation	1
Estradiol	increases expression	1
Etoposide	decreases expression	1

ChEMBL screening assays

9 unique, capped per target: 9 binding

Representative assays (with source publication via chembl_document):

Assay ID	Type	Description	Source paper
CHEMBL2341271	Binding	Binding affinity to human full-length His-tagged Myt1 kinase expressed in HEK293 cells at 3 ug/ml by TR-FRET based binding assay	Evaluation of potential Myt1 kinase inhibitors by TR-FRET based binding assay. — Eur J Med Chem

Cellosaurus cell lines

6 cell lines: 3 embryonic stem cell, 3 cancer cell line

First 10 cell lines (id-ordered, not curated):

Cellosaurus	Name	Category	Sex
CVCL_A4J1	SEES3-1V human MYT1, clone1	Embryonic stem cell	Male
CVCL_A4J2	SEES3-1V human MYT1, clone2	Embryonic stem cell	Male
CVCL_A4J3	SEES3-1V human MYT1, clone3	Embryonic stem cell	Male
CVCL_SZ69	HAP1 MYT1 (-) 1	Cancer cell line	Male
CVCL_SZ70	HAP1 MYT1 (-) 2	Cancer cell line	Male
CVCL_SZ71	HAP1 MYT1 (-) 3	Cancer cell line	Male

Clinical trials (associated diseases)

507 trials via MONDO — disease-level, not drug-specific.

Trial	Phase	Status	Title
NCT05657860	PHASE4	COMPLETED	Guanfacine Extended Release for the Reduction of Aggression and Self-injurious Behavior Associated With Prader-Willi Syndrome
NCT05744479	PHASE4	RECRUITING	Metformin for Antipsychotic-induced Weight Gain in Adults With Intellectual Disability
NCT06107829	PHASE4	WITHDRAWN	Valbenazine Treatment of Tardive Dyskinesia in Adults With Intellectual/Developmental Disabilities
NCT06997198	PHASE4	NOT_YET_RECRUITING	Deutetrabenazine Treatment for Tardive Dyskinesia in Intellectual/Developmental Disabilities
NCT07542548	PHASE4	COMPLETED	D-Cycloserine for Serine Palmitoyltransferase Inhibition
NCT00391261	PHASE4	COMPLETED	An Open-label Trial of Metformin for Weight Control of Pediatric Patients on Antipsychotic Medications.
NCT01028820	PHASE4	COMPLETED	FMRI Brain Activation of Aripiprazole Treatment in Autism Spectrum Disorders
NCT01333865	PHASE4	COMPLETED	A Study of Memantine Hydrochloride (Namenda®) for Cognitive and Behavioral Impairment in Adults With Autism Spectrum Disorders
NCT01337700	PHASE4	COMPLETED	Milnacipran in Autism and the Functional Locus Coeruleus and Noradrenergic Model of Autism
NCT01695200	PHASE4	COMPLETED	Omega-3 Fatty Acids in Autism Spectrum Disorders
NCT02096952	PHASE4	COMPLETED	Methylphenidate ER Liquid Formulation in Adults With ASD and ADHD
NCT02235467	PHASE4	COMPLETED	Multisite Study: Parental Training Using Video Modelling to Develop Social Skills in Children With Autism
NCT02940574	PHASE4	COMPLETED	Neural and Behavioral Effects of Oxytocin in Autism Spectrum Disorders
NCT03333629	PHASE4	COMPLETED	Promoting Positive Outcomes for Individuals With ASD: Linking Early Detection, Treatment, and Long-term Outcomes
NCT03337646	PHASE4	COMPLETED	Evaluation of the Effect and Safety of Lisdexamfetamine in Children Aged 6-12 With ADHD and Autism
NCT03538431	PHASE4	COMPLETED	Improving Driving in Young People With Autism Spectrum Disorders
NCT03757585	PHASE4	COMPLETED	Natural Treatments for the Management of Emotional Dysregulation in Youth With Non-verbal Learning Disability (NVLD) and/or Autism Spectrum Disorders (ASD)
NCT04903353	PHASE4	COMPLETED	Pragmatic Trial Comparing Weight Gain in Children With Autism Taking Risperidone Versus Aripiprazole
NCT05063656	PHASE4	COMPLETED	Biomarker-Driven Pharmacological Treatment of Adolescents With Autism Spectrum Disorder With Gabapentin
NCT05146245	PHASE4	UNKNOWN	Safety and Pharmacokinetics of Antipsychotics in Children 2: Studying TDM in an RCT
NCT05916339	PHASE4	RECRUITING	AWARE: Management of ADHD in Autism Spectrum Disorder
NCT05954052	PHASE4	TERMINATED	A Study of Glutathione in Children With Autism Spectrum Disorder
NCT06853665	PHASE4	RECRUITING	The TEAM Study - Treatment Efficacy for Autism/Attention Using Mixed Amphetamine
NCT07054697	PHASE4	COMPLETED	Pilot-RCT With Individualized Homeopathic Treatment in the Children With Autism Spectrum Disorder
NCT07161804	PHASE4	COMPLETED	Pilot RCT Using Homeopathic Medicines in ASD
NCT07439042	PHASE4	NOT_YET_RECRUITING	Buspirone for Anxiety in Autistic Youth
NCT02270736	PHASE3	COMPLETED	Clinical Study to Investigate the Efficacy and Safety of NT 201 Compared to Placebo in the Treatment of Chronic Troublesome Drooling Associated With Neurological Disorders and/or Intellectual Disability
NCT01302964	PHASE3	COMPLETED	Mirtazapine Treatment of Anxiety in Children and Adolescents With Pervasive Developmental Disorders
NCT01706523	PHASE3	TERMINATED	Open Label Extension Study of STX209 (Arbaclofen) in Autism Spectrum Disorders
NCT01825798	PHASE3	COMPLETED	Treatment of Overweight Induced by Antipsychotic Medication in Young People With Autism Spectrum Disorders (ASD)
NCT01972074	PHASE3	COMPLETED	Behavioral and Neural Response to Memantine in Adolescents With Autism Spectrum Disorder
NCT02985749	PHASE3	COMPLETED	A Study of Oxytocin for the Treatment of Social Impairment in Individuals With High Functioning Autism Spectrum Disorder
NCT03197922	PHASE3	COMPLETED	Treatment of Encopresis in Children With Autism Spectrum Disorders
NCT03504917	PHASE3	TERMINATED	A Study of Balovaptan in Adults With Autism Spectrum Disorder With a 2-Year Open-Label Extension
NCT03553875	PHASE3	TERMINATED	Memantine for the Treatment of Social Deficits in Youth With Disorders of Impaired Social Interactions
NCT03640156	PHASE3	COMPLETED	Modulating Socially Adaptive Mirror System Functioning in Autism by Oxytocin
NCT03715153	PHASE3	TERMINATED	Efficacy and Safety of Bumetanide Oral Liquid Formulation in Children Aged From 2 to Less Than 7 Years Old With Autism Spectrum Disorder.
NCT03715166	PHASE3	TERMINATED	Efficacy and Safety of Bumetanide Oral Liquid Formulation in Children and Adolescents Aged From 7 to Less Than 18 Years Old With Autism Spectrum Disorder
NCT04233502	PHASE3	WITHDRAWN	Efficacy and Safety of Slenyto for Insomnia in Children With ASD
NCT04578756	PHASE3	COMPLETED	Open-Label, Flexible-dose Study to Evaluate the Long-Term Safety and Tolerability of Cariprazine in the Treatment of Pediatric Participants With Schizophrenia, Bipolar I Disorder, or Autism Spectrum Disorder