CCNB3

Summary

CCNB3 (cyclin B3, HGNC:18709) is a protein-coding gene on chromosome Xp11.22, encoding G2/mitotic-specific cyclin-B3 (Q8WWL7). Cyclins are positive regulatory subunits of the cyclin-dependent kinases (CDKs), and thereby play an essential role in the control of the cell cycle, notably via their destruction during cell division.

The protein encoded by this gene belongs to the highly conserved cyclin family, whose members are characterized by a dramatic periodicity in protein abundance through the cell cycle. Cyclins function as positive regulators of cyclin-dependent kinases (CDKs), and thereby play an essential role in the control of the cell cycle. Different cyclins exhibit distinct expression and degradation patterns, which contribute to the temporal coordination of each mitotic event. Studies of similar genes in chicken and drosophila suggest that this cyclin may associate with CDC2 and CDK2 kinases, and may be required for proper spindle reorganization and restoration of the interphase nucleus. Alternatively spliced transcript variants encoding different isoforms have been described for this gene.

Source: NCBI Gene 85417 — RefSeq curated summary.

At a glance

• Clinical variants (ClinVar): 218 total — 1 pathogenic
• Druggable target: yes — 17 molecules with ChEMBL bioactivity
• MANE Select transcript: NM_033031

Identifiers

Gene identifiers

Field	Value
HGNC ID	HGNC:18709
Approved symbol	CCNB3
Name	cyclin B3
Location	Xp11.22
Locus type	gene with protein product
Status	Approved
Aliases	CYCB3
Ensembl gene	ENSG00000147082
Ensembl biotype	protein_coding
OMIM	300456
Entrez	85417

Gene structure

Transcript identifiers

Ensembl transcripts: 8 — 4 protein_coding, 2 protein_coding_CDS_not_defined, 1 nonsense_mediated_decay, 1 retained_intron

ENST00000276014, ENST00000348603, ENST00000376038, ENST00000376042, ENST00000476167, ENST00000487204, ENST00000491907, ENST00000493507

RefSeq mRNA: 2 — MANE Select: NM_033031 NM_033031, NM_033670

CCDS: CCDS14331, CCDS14332

Canonical transcript exons

ENST00000376042 — 13 exons

Exon	Start	End
ENSE00000978695	50308505	50311496
ENSE00001337474	50288780	50288887
ENSE00001469251	50284542	50284616
ENSE00001469252	50204765	50204950
ENSE00002175633	50285127	50285259
ENSE00002687330	50294863	50294993
ENSE00003469079	50346652	50346807
ENSE00003521901	50313856	50313948
ENSE00003561170	50312537	50312632
ENSE00003600320	50351241	50351371
ENSE00003615123	50342202	50342339
ENSE00003624062	50347626	50347775
ENSE00003896739	50351607	50351914

Expression profiles

Bgee: expression breadth ubiquitous, 156 present calls, max score 87.89.

FANTOM5 (CAGE): breadth tissue_specific, TPM avg 0.2483 / max 16.3122, expressed in 99 samples.

FANTOM5 promoters (1 alternative TSS)

Promoter ID	TPM avg	Samples expressed
196352	0.2483	99

Top tissues by expression

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

Tissue	Anatomy ID	Expression score	Quality
primordial germ cell in gonad	CL:0000670 ∩ UBERON:0000991	87.89	gold quality
male germ line stem cell (sensu Vertebrata) in testis	CL:0000089 ∩ UBERON:0000473	87.36	gold quality
secondary oocyte	CL:0000655	87.35	gold quality
oocyte	CL:0000023	86.21	gold quality
right testis	UBERON:0004534	74.38	gold quality
left testis	UBERON:0004533	72.86	gold quality
testis	UBERON:0000473	71.83	gold quality
C1 segment of cervical spinal cord	UBERON:0006469	69.99	gold quality
pancreatic ductal cell	CL:0002079	69.37	silver quality
ileal mucosa	UBERON:0000331	67.56	silver quality
spinal cord	UBERON:0002240	67.42	gold quality
cardiac muscle of right atrium	UBERON:0003379	64.31	gold quality
left ventricle myocardium	UBERON:0006566	63.92	gold quality
ventricular zone	UBERON:0003053	62.32	gold quality
right uterine tube	UBERON:0001302	61.20	gold quality
olfactory segment of nasal mucosa	UBERON:0005386	61.10	gold quality
islet of Langerhans	UBERON:0000006	60.10	gold quality
corpus epididymis	UBERON:0004359	59.69	silver quality
granulocyte	CL:0000094	58.82	gold quality
substantia nigra	UBERON:0002038	58.29	gold quality
sperm	CL:0000019	57.11	gold quality
right adrenal gland	UBERON:0001233	57.10	gold quality
right adrenal gland cortex	UBERON:0035827	56.66	gold quality
right lobe of liver	UBERON:0001114	56.55	gold quality
gastrocnemius	UBERON:0001388	56.43	gold quality
adult organism	UBERON:0007023	56.43	gold quality
midbrain	UBERON:0001891	56.38	gold quality
pancreas	UBERON:0001264	56.27	gold quality
hindlimb stylopod muscle	UBERON:0004252	56.24	gold quality
adenohypophysis	UBERON:0002196	56.15	gold quality

Single-cell (SCXA)

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

Experiment	Marker?	Max mean expression
E-ANND-3	no	5.04

Regulation

Is transcription factor: no

miRNA regulators (miRDB)

14 targeting CCNB3, 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-6740-5P	100.00	65.64	932
HSA-MIR-450A-1-3P	100.00	69.33	1837
HSA-MIR-3646	100.00	73.56	5283
HSA-MIR-3662	99.99	73.82	5684
HSA-MIR-4775	99.98	75.00	6394
HSA-MIR-3148	99.97	75.06	6478
HSA-MIR-651-3P	99.94	73.48	5177
HSA-MIR-3143	99.93	71.96	3104
HSA-MIR-10A-5P	98.89	69.85	712
HSA-MIR-10B-5P	98.89	69.86	711
HSA-MIR-374A-3P	98.87	67.82	1531
HSA-MIR-6512-5P	98.76	69.29	1195
HSA-MIR-376C-3P	97.63	68.88	1263
HSA-MIR-217-3P	95.67	68.42	1000

Literature-anchored findings (GeneRIF, showing 14)

• cloning of a full length cDNA; is expressed in developing male germ cells and is restricted to testis in mature tissues (PMID:12185076)
• Increased expression of cyclin B3 maybe an important marker for identifying endoderm cells differentiated from human embryonic stem cells. (PMID:21404461)
• A new fusion was observed between BCOR (encoding the BCL6 co-repressor) and CCNB3 (encoding the testis-specific cyclin B3) on the X chromosome. (PMID:22387997)
• Study provides a detailed description of the histologic spectrum, immunohistochemical features, and clinical characteristic of BCOR-CCNB3 sarcoma justifying distinction from Ewing sarcoma with its typical EWS/FUS-ETS translocations. (PMID:24805859)
• This study adds to recent reports on the clinicopathologic spectrum of BCOR-CCNB3 fusion-positive sarcomas, a newly emerging entity within the undifferentiated unclassified sarcoma category (PMID:25360585)
• CCNB3 immunostaining was useful for differentiating BCOR-CCNB3 from a group of ‘Ewing-like sarcomas’ and may contribute to the evaluation of treatment strategies for bone sarcomas. (PMID:26037154)
• Case Reports: renal sarcomas with BCOR-CCNB3 gene fusion showing histological overlap with BCOR-related clear cell sarcoma of the kidney. (PMID:28817404)
• Immunohistochemistry for either CCNB3 or BCOR is not completely sensitive and specific in undifferentiated sarcoma with BCOR-CCNB3 fusion. (PMID:28877060)
• Detection of fusion transcripts BCOR-CCNB3 in the bone marrow suggests that undifferentiated sarcoma patients with positive findings are at high risk of tumor progression. (PMID:30064235)
• report two patients with clear cell sarcoma of the kidney showing BCOR-CCNB3 (where CCNB3 is cyclin B3) fusion (PMID:31876361)
• Imaging features and clinical course of undifferentiated round cell sarcomas with CIC-DUX4 and BCOR-CCNB3 translocations. (PMID:32840647)
• Biallelic variant in cyclin B3 is associated with failure of maternal meiosis II and recurrent digynic triploidy. (PMID:32938693)
• A global collaboRAtive study of CIC-rearranged, BCOR::CCNB3-rearranged and other ultra-rare unclassified undifferentiated small round cell sarcomas (GRACefUl). (PMID:36791667)
• Mutations in CCNB3 affect its location thus causing a multiplicity of phenotypes in human oocytes maturation by aberrant CDK1 activity and APC/C activity at different stages. (PMID:37635245)

Cross-species orthologs

5 orthologs

Organism	Symbol	Gene ID
danio_rerio	ccnb3	ENSDARG00000034855
mus_musculus	Ccnb3	ENSMUSG00000051592
rattus_norvegicus	Ccnb3	ENSRNOG00000002951
drosophila_melanogaster	CycB3	FBGN0015625
caenorhabditis_elegans		WBGENE00000868

Paralogs (18): CCNE1 (ENSG00000105173), CCNP (ENSG00000105219), CCNJ (ENSG00000107443), CCND1 (ENSG00000110092), CCND3 (ENSG00000112576), CCNG1 (ENSG00000113328), CCNI (ENSG00000118816), CCND2 (ENSG00000118971), CCNA1 (ENSG00000133101), CCNB1 (ENSG00000134057), CCNJL (ENSG00000135083), CCNG2 (ENSG00000138764), CCNA2 (ENSG00000145386), CCNO (ENSG00000152669), CCNB2 (ENSG00000157456), CCNF (ENSG00000162063), CCNE2 (ENSG00000175305), CCNI2 (ENSG00000205089)

Protein

Protein identifiers

G2/mitotic-specific cyclin-B3 — Q8WWL7 (reviewed: Q8WWL7)

All UniProt accessions (1): Q8WWL7

UniProt curated annotations — full annotation on UniProt →

Function. Cyclins are positive regulatory subunits of the cyclin-dependent kinases (CDKs), and thereby play an essential role in the control of the cell cycle, notably via their destruction during cell division. Its tissue specificity suggest that it may be required during early meiotic prophase I.

Subunit / interactions. Interacts with CDK2 kinase.

Subcellular location. Nucleus.

Tissue specificity. Testis specific. In testis, it is expressed in developing germ cells, but not in Leydig cells. Weakly or not expressed in other tissues.

Post-translational modifications. Ubiquitinated. Ubiquitination leads to its degradation during anaphase entry, after degradation of CCNB1.

Domain organisation. The N-terminal destruction box (D-box) probably acts as a recognition signal for degradation via the ubiquitin-proteasome pathway.

Similarity. Belongs to the cyclin family. Cyclin AB subfamily.

Isoforms (3)

UniProt ID	Names	Canonical?
Q8WWL7-1	1	yes
Q8WWL7-2	2, Variant 1
Q8WWL7-3	3, Variant 2

RefSeq proteins (2): NP_149020, NP_391990 (=MANE)

Domains & families (InterPro)

ID	Name	Type
IPR004367	Cyclin_C-dom	Domain
IPR006671	Cyclin_N	Domain
IPR013763	Cyclin-like_dom	Domain
IPR036915	Cyclin-like_sf	Homologous_superfamily
IPR039361	Cyclin	Family

Pfam: PF00134, PF02984

UniProt features (24 total): sequence conflict 7, compositionally biased region 5, region of interest 3, mutagenesis site 3, splice variant 2, sequence variant 2, chain 1, short sequence motif 1

Structure

Experimental structures (PDB)

0 structures.

Predicted structure (AlphaFold)

Model	pLDDT	Fraction very-high
AF-Q8WWL7-F1	42.25	0.18

Functional residue map

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

Mutagenesis-validated functional residues (3):

Position	Phenotype
60	in cycb3xa; prevents its destruction after completion of anaphase; when associated with a-63 and a-68.
63	in cycb3xa; prevents its destruction after completion of anaphase; when associated with a-60 and a-68.
68	in cycb3xa; prevents its destruction after completion of anaphase; when associated with a-60 and a-63.

Function

Pathways and Gene Ontology

Reactome pathways

0 pathways

MSigDB gene sets: 73 (showing top): GOBP_CELL_CYCLE_PHASE_TRANSITION, SHEPARD_BMYB_MORPHOLINO_DN, GOBP_CELL_CYCLE_G1_S_PHASE_TRANSITION, GOBP_MITOTIC_CELL_CYCLE, SHEPARD_BMYB_TARGETS, GOCC_TRANSFERASE_COMPLEX_TRANSFERRING_PHOSPHORUS_CONTAINING_GROUPS, GOBP_MEIOTIC_CELL_CYCLE, GOCC_TRANSFERASE_COMPLEX, GOCC_PROTEIN_KINASE_COMPLEX, GOBP_CELL_DIVISION, GOBP_CELL_CYCLE_PROCESS, GOCC_CYCLIN_DEPENDENT_PROTEIN_KINASE_HOLOENZYME_COMPLEX, GOMF_ENZYME_REGULATOR_ACTIVITY, HOELZEL_NF1_TARGETS_UP, SHEPARD_CRASH_AND_BURN_MUTANT_DN

GO Biological Process (4): G1/S transition of mitotic cell cycle (GO:0000082), cell division (GO:0051301), meiotic cell cycle (GO:0051321), mitotic cell cycle phase transition (GO:0044772)

GO Molecular Function (2): cyclin-dependent protein serine/threonine kinase regulator activity (GO:0016538), protein binding (GO:0005515)

GO Cellular Component (4): cyclin-dependent protein kinase holoenzyme complex (GO:0000307), nucleus (GO:0005634), nucleoplasm (GO:0005654), cytoplasm (GO:0005737)

GO top-level categories

Rollup of top GO terms by namespace:

Category	Terms
mitotic cell cycle	2
cellular anatomical structure	2
mitotic cell cycle phase transition	1
cell cycle G1/S phase transition	1
cellular process	1
cell cycle	1
sexual reproduction	1
reproductive process	1
meiotic nuclear division	1
cell cycle phase transition	1
mitotic cell cycle process	1
cyclin-dependent protein serine/threonine kinase activity	1
cyclin-dependent protein kinase regulator activity	1
binding	1
serine/threonine protein kinase complex	1
intracellular membrane-bounded organelle	1
nuclear lumen	1
intracellular anatomical structure	1

Protein interactions and networks

STRING

2547 interactions, top by confidence (×1000):

Protein A	Protein B	Partner UniProt	Score
CCNB3	CDK2	P24941	876
CCNB3	CCNY	Q8ND76	858
CCNB3	CDK1	P06493	802
CCNB3	CCNL2	Q96S94	789
CCNB3	AKAP4	Q5JQC9	774
CCNB3	DUX4L2	P0CJ85	727
CCNB3	ZC3H7B	Q9UGR2	726
CCNB3	NUTM2B	A6NNL0	647
CCNB3	EWSR1	Q01844	643
CCNB3	H1-0	P07305	605
CCNB3	CD99	P14209	542
CCNB3	PCGF1	Q9BSM1	539
CCNB3	CD99L2	Q8TCZ2	524
CCNB3	CDK3	Q00526	509
CCNB3	YWHAE	P29360	507

IntAct

13 interactions, top by confidence:

A	B	Type	Score
CDKN1A	CCNE2	psi-mi:“MI:0914”(association)	0.890
CDKN1A	CDK14	psi-mi:“MI:0914”(association)	0.770
CCNB3	Plk4	psi-mi:“MI:0217”(phosphorylation reaction)	0.440
Plk4	CCNB3	psi-mi:“MI:0217”(phosphorylation reaction)	0.440
CCNB3	KRT14	psi-mi:“MI:0915”(physical association)	0.400
CCNB3	KRT18	psi-mi:“MI:0915”(physical association)	0.400
CCNB3	CDK2	psi-mi:“MI:0915”(physical association)	0.400
CDK2	CCNB3	psi-mi:“MI:0915”(physical association)	0.400
CDKN1B	YKT6	psi-mi:“MI:0914”(association)	0.350
	EBAG9	psi-mi:“MI:0914”(association)	0.350

BioGRID (16): CCNB3 (Affinity Capture-MS), CCNB3 (Proximity Label-MS), CCNB3 (Proximity Label-MS), CCNB3 (Affinity Capture-MS), CDK2 (Reconstituted Complex), CCNB3 (Affinity Capture-MS), CCNB3 (Affinity Capture-MS), CDK1 (Affinity Capture-Western), CDK2 (Affinity Capture-Western), HIST1H2AG (Cross-Linking-MS (XL-MS)), HIST1H3A (Cross-Linking-MS (XL-MS)), CCNB3 (Cross-Linking-MS (XL-MS)), CCNB3 (Cross-Linking-MS (XL-MS)), EEA1 (Cross-Linking-MS (XL-MS)), CCNB3 (Reconstituted Complex)

ESM2 similar proteins: A0A1B0GTH6, A0A1B0GUW6, A0A1D5RMD1, A2AQH4, A4FU49, A6NJ88, C4P6S0, D3YU32, E9PAV3, F1QU13, I3L273, J3KML8, P70670, Q32L62, Q3V0E1, Q3V3Q4, Q4R729, Q5H9F3, Q5QJ38, Q5SWP3, Q5U4C1, Q5VWK0, Q5VYM1, Q68DN1, Q6AZ54, Q7TSG5, Q810T2, Q8CH19, Q8K4E0, Q8N3K9, Q8N5Q1, Q8NDH2, Q8TCU4, Q8WNU4, Q8WWL7, Q920R4, Q921B4, Q923B3, Q96JA4, Q96M34

Diamond homologs: A0MEB5, A2YH60, O48790, O77689, O93229, O95067, P04962, P07818, P10815, P13350, P13351, P13952, P14635, P14785, P15206, P18606, P20248, P20439, P24860, P24861, P24862, P24871, P25010, P25011, P25012, P29332, P30183, P30274, P30276, P30277, P30278, P30284, P34800, P34801, P37881, P37882, P37883, P39963, P42524, P43449

SIGNOR signaling

0 interactions.

Disease & clinical

Clinical variants and AI predictions

ClinVar

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

Classification	Count (floor)
Pathogenic	1
Likely pathogenic	0
Uncertain significance	112
Likely benign	25
Benign	5

Top pathogenic / likely-pathogenic (1)

Variant ID	HGVS	Classification
619178	NM_033031.3(CCNB3):c.3752T>A (p.Val1251Asp)	Pathogenic

SpliceAI

2250 predictions. Top by Δscore:

Variant	Effect	Δscore
X:50288774:TTTTA:T	acceptor_loss	1.0000
X:50288775:TTTA:T	acceptor_loss	1.0000
X:50288776:TTA:T	acceptor_loss	1.0000
X:50288777:TAGA:T	acceptor_loss	1.0000
X:50288778:A:AG	acceptor_gain	1.0000
X:50288778:AGAC:A	acceptor_gain	1.0000
X:50288778:AGACG:A	acceptor_gain	1.0000
X:50288779:G:GA	acceptor_gain	1.0000
X:50288779:GAC:G	acceptor_gain	1.0000
X:50288779:GACG:G	acceptor_gain	1.0000
X:50288779:GACGG:G	acceptor_gain	1.0000
X:50288888:G:GG	donor_gain	1.0000
X:50294990:AATG:A	donor_gain	1.0000
X:50294990:AATGG:A	donor_loss	1.0000
X:50294991:ATG:A	donor_gain	1.0000
X:50294991:ATGG:A	donor_loss	1.0000
X:50294992:TG:T	donor_gain	1.0000
X:50294992:TGGT:T	donor_loss	1.0000
X:50294993:GG:G	donor_gain	1.0000
X:50294993:GGTG:G	donor_loss	1.0000
X:50294994:G:GG	donor_gain	1.0000
X:50294994:GT:G	donor_loss	1.0000
X:50311494:G:T	donor_gain	1.0000
X:50312999:TC:T	donor_gain	1.0000
X:50342286:GA:G	donor_gain	1.0000
X:50346806:GG:G	donor_gain	1.0000
X:50346807:GG:G	donor_gain	1.0000
X:50347624:A:AG	acceptor_gain	1.0000
X:50347624:AGT:A	acceptor_gain	1.0000
X:50347625:G:GA	acceptor_gain	1.0000

AlphaMissense

9255 scored. Top likely-pathogenic:

Variant	Protein change	am_pathogenicity
X:50347663:G:C	R1283P	0.997
X:50351305:T:C	L1342P	0.996
X:50313931:T:A	W1167R	0.994
X:50313931:T:C	W1167R	0.994
X:50313915:G:C	R1161S	0.993
X:50313915:G:T	R1161S	0.993
X:50342248:T:C	L1188P	0.993
X:50342254:A:T	D1190V	0.993
X:50346787:T:C	F1264L	0.992
X:50346789:T:A	F1264L	0.992
X:50346789:T:G	F1264L	0.992
X:50342254:A:C	D1190A	0.991
X:50347744:T:C	L1310P	0.991
X:50351284:T:C	L1335P	0.991
X:50342253:G:C	D1190H	0.990
X:50342263:T:C	L1193P	0.990
X:50347732:C:A	A1306D	0.990
X:50313923:T:C	L1164P	0.989
X:50346791:T:C	L1265P	0.988
X:50347741:T:C	L1309P	0.988
X:50351296:T:A	V1339D	0.988
X:50313914:G:C	R1161T	0.987
X:50342238:G:C	A1185P	0.987
X:50347750:T:C	L1312P	0.987
X:50313862:T:C	F1144L	0.986
X:50313864:T:A	F1144L	0.986
X:50313864:T:G	F1144L	0.986
X:50342313:G:C	A1210P	0.986
X:50342325:G:C	A1214P	0.986
X:50342328:G:C	A1215P	0.986

dbSNP variants (sampled 300 via entrez): RS1000057984 (X:50224162 T>C), RS1000270569 (X:50314979 C>T), RS1000333513 (X:50215193 G>A), RS1000403267 (X:50215437 G>A), RS1000469728 (X:50284131 T>G), RS1000817769 (X:50327824 A>T), RS1000885410 (X:50325781 G>T), RS1000890695 (X:50293763 T>A), RS1000900613 (X:50352244 A>G), RS1000915852 (X:50283734 G>A,T), RS1000963716 (X:50294407 A>G), RS1001015526 (X:50305995 C>T), RS1001107394 (X:50293377 C>T), RS1001142205 (X:50217746 C>T), RS1001492219 (X:50226035 G>C,T)

Disease associations

OMIM: gene MIM:300456 | disease phenotypes: MIM:300434, MIM:300299, MIM:300894, MIM:300896, MIM:301000, MIM:313900, MIM:614389

GenCC curated gene-disease

Mondo (7): X-linked intellectual disability, Stocco dos Santos type (MONDO:0010325), X-linked severe congenital neutropenia (MONDO:0010294), neurodegeneration with brain iron accumulation 5 (MONDO:0010476), SLC35A2-congenital disorder of glycosylation (MONDO:0010478), Wiskott-Aldrich syndrome (MONDO:0010518), thrombocytopenia 1 (MONDO:0010743), pregnancy loss, recurrent, susceptibility to, 1 (MONDO:0013727)

Orphanet (7): X-linked intellectual disability, Stocco Dos Santos type (Orphanet:85288), Hereditary thrombocytopenia with normal platelets (Orphanet:268322), Beta-propeller protein-associated neurodegeneration (Orphanet:329284), SLC35A2-CDG (Orphanet:356961), X-linked thrombocytopenia with normal platelets (Orphanet:852), X-linked severe congenital neutropenia (Orphanet:86788), Wiskott-Aldrich syndrome (Orphanet:906)

HPO phenotypes

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

GWAS associations

0 associations (top):

MeSH disease descriptors (4)

Descriptor	Name	Tree numbers
D014923	Wiskott-Aldrich Syndrome	C15.378.100.100.970; C15.378.243.750.605.900; C15.378.463.960; C15.378.553.546.605.900; C16.320.099.970; C16.320.322.937; C16.320.798.875; C20.673.627.900; C20.673.795.875
C564539	Neutropenia, Severe Congenital, X-Linked (supp.)
C537495	Stocco dos Santos syndrome (supp.)
C564052	Thrombocytopenia 1 (supp.)

Drugs & pharmacology

Drug and pharmacology data

Is drug target: yes

ChEMBL targets (1): CHEMBL2094127 (PROTEIN COMPLEX GROUP)

Molecules with ChEMBL bioactivity

17 molecules (phase ≥1), by development phase (incl. off-target/promiscuous compounds). Patent mentions across the top 20 by phase: 198,081 (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
CHEMBL189963	PALBOCICLIB	4	13,102
CHEMBL2103840	DINACICLIB	3	2,257
CHEMBL428690	ALVOCIDIB	3	27,781
CHEMBL50	QUERCETIN	3	74,559
CHEMBL1230165	SILMITASERTIB	2	593
CHEMBL1276127	INDIRUBIN	2	181
CHEMBL14762	SELICICLIB	2	3,787
CHEMBL151	LUTEOLIN	2	23,523
CHEMBL2347597	ASNUCICLIB	2	100
CHEMBL31574	FISETIN	2	7,745
CHEMBL3545283	RIVICICLIB	2	968
CHEMBL445813	AT-7519	2	2,614
CHEMBL150	KAEMPFEROL	1	25,940
CHEMBL258805	SU-9516	1	76
CHEMBL269538	HARMINE	1	4,346
CHEMBL296468	BMS-387032	1	2,075
CHEMBL412142	LADUVIGLUSIB	1	8,434

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

ChEMBL bioactivities

500 potent at pChembl≥5 of 612 total, top 50 by pChembl (potency: 10 = 0.1 nM, 6 = 1 µM).

pChembl	Type	Value	Unit	Molecule
9.22	IC50	0.6	nM	CHEMBL261720
9.22	IC50	0.6	nM	CHEMBL384350
9.22	IC50	0.6	nM	CHEMBL426509
9.00	IC50	1	nM	CHEMBL212552
8.72	IC50	1.9	nM	CHEMBL377449
8.70	IC50	2	nM	CHEMBL212299
8.68	IC50	2.1	nM	CHEMBL361900
8.62	IC50	2.4	nM	STAUROSPORINE
8.60	IC50	2.52	nM	STAUROSPORINE
8.52	IC50	3	nM	CHEMBL363607
8.52	IC50	3	nM	CHEMBL212491
8.52	Ki	3	nM	DINACICLIB
8.49	IC50	3.2	nM	CHEMBL425720
8.49	IC50	3.2	nM	CHEMBL187395
8.49	IC50	3.2	nM	CHEMBL364927
8.49	IC50	3.2	nM	STAUROSPORINE
8.49	IC50	3.2	nM	CHEMBL1684800
8.43	IC50	3.7	nM	CHEMBL370698
8.40	Ki	4	nM	ASNUCICLIB
8.40	IC50	4	nM	CHEMBL430653
8.40	IC50	4	nM	PURVALANOLA
8.35	IC50	4.5	nM	CHEMBL190643
8.32	IC50	4.8	nM	CHEMBL363130
8.30	Ki	5	nM	CHEMBL5286978
8.30	Ki	5	nM	CHEMBL5277074
8.30	Ki	5	nM	CHEMBL5286090
8.22	IC50	6	nM	PURVALANOL B
8.22	IC50	6	nM	JNJ-7706621
8.22	IC50	6	nM	CHEMBL310840
8.22	Ki	6	nM	CHEMBL5286271
8.22	Ki	6	nM	CHEMBL5281179
8.22	IC50	6	nM	STAUROSPORINE
8.22	IC50	6	nM	CHEMBL325023
8.19	IC50	6.4	nM	JNJ-7706621
8.15	IC50	7	nM	CHEMBL310567
8.15	IC50	7	nM	CHEMBL5287128
8.15	IC50	7	nM	CHEMBL328406
8.10	IC50	8	nM	CHEMBL363607
8.05	IC50	9	nM	CHEMBL212833
8.05	IC50	9	nM	CHEMBL434217
8.02	IC50	9.5	nM	CHEMBL319467
8.00	IC50	10	nM	CHEMBL126997
8.00	IC50	10	nM	CHEMBL420463
8.00	IC50	10	nM	CHEMBL100012
7.92	Ki	12	nM	CHEMBL5277565
7.92	IC50	12	nM	CHEMBL318564
7.92	IC50	12	nM	STAUROSPORINE
7.89	IC50	13	nM	CHEMBL81211
7.89	IC50	13	nM	CHEMBL380301
7.85	IC50	14	nM	CHEMBL385478

PubChem BioAssay actives

496 with measured affinity, of 879 total; 50 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
N-[2-methyl-4-(2-pyrrolidin-1-ylethyl)phenyl]-4-(1H-pyrrolo[2,3-b]pyridin-3-yl)pyrimidin-2-amine	270821: Inhibition of CDK1/cyclinB by Flashplate assay	ic50	0.0006	uM
2-[3-methyl-4-[[4-(1H-pyrrolo[2,3-b]pyridin-3-yl)pyrimidin-2-yl]amino]phenyl]ethanol	270821: Inhibition of CDK1/cyclinB by Flashplate assay	ic50	0.0006	uM
5-amino-N-(2,6-difluorophenyl)-3-(4-sulfamoylanilino)-1,2,4-triazole-1-carbothioamide	241014: Inhibition of Cyclin B-cyclin-dependent kinase 1	ic50	0.0006	uM
N-[2-methyl-4-(2-piperidin-1-ylethyl)phenyl]-4-(1H-pyrrolo[2,3-b]pyridin-3-yl)pyrimidin-2-amine	270821: Inhibition of CDK1/cyclinB by Flashplate assay	ic50	0.0010	uM
N-[2-methyl-4-(2-morpholin-4-ylethyl)phenyl]-4-(1H-pyrrolo[2,3-b]pyridin-3-yl)pyrimidin-2-amine	270821: Inhibition of CDK1/cyclinB by Flashplate assay	ic50	0.0019	uM
4-N-[4-(1H-pyrrolo[2,3-b]pyridin-3-yl)pyrimidin-2-yl]cyclohexane-1,4-diamine	270821: Inhibition of CDK1/cyclinB by Flashplate assay	ic50	0.0020	uM
4-[[5-amino-1-(3-fluorothiophene-2-carbonyl)-1,2,4-triazol-3-yl]amino]benzenesulfonamide	241014: Inhibition of Cyclin B-cyclin-dependent kinase 1	ic50	0.0021	uM
(2S,3R,4R,6R)-3-methoxy-2-methyl-4-(methylamino)-29-oxa-1,7,17-triazaoctacyclo[12.12.2.12,6.07,28.08,13.015,19.020,27.021,26]nonacosa-8,10,12,14,19,21,23,25,27-nonaen-16-one	1960664: Inhibition of C-terminal His6 tagged human full length CDK1/N-terminal GST-tagged human full length Cyclin B expressed in baculovirus infected Sf21 cells using 5-FAM-QSPKKG-CONH2 as substrate incubated for 60 mins in presence of ATP	ic50	0.0024	uM
4-[[1-(2,6-difluoro-3-methylbenzoyl)-5-methyl-1,2,4-triazol-3-yl]amino]benzenesulfonamide	241014: Inhibition of Cyclin B-cyclin-dependent kinase 1	ic50	0.0030	uM
N-(2-methylphenyl)-4-(1H-pyrrolo[2,3-b]pyridin-3-yl)pyrimidin-2-amine	270821: Inhibition of CDK1/cyclinB by Flashplate assay	ic50	0.0030	uM
2-[(2S)-1-[3-ethyl-7-[(1-oxidopyridin-1-ium-3-yl)methylamino]pyrazolo[1,5-a]pyrimidin-5-yl]piperidin-2-yl]ethanol	1940563: Inhibition of CDK1/Cyclin B (unknown origin) assessed as inhibition constant	ki	0.0030	uM
4-[[5-methyl-1-(2,3,6-trifluorobenzoyl)-1,2,4-triazol-3-yl]amino]benzenesulfonamide	241014: Inhibition of Cyclin B-cyclin-dependent kinase 1	ic50	0.0032	uM
4-[[5-amino-2-(2,6-difluorobenzenecarbothioyl)-1,2,4-triazol-3-yl]amino]benzenesulfonamide	241014: Inhibition of Cyclin B-cyclin-dependent kinase 1	ic50	0.0032	uM
N-[5-amino-1-[(4-methoxyphenyl)methyl]pyrazol-4-yl]-5-[[4-chloro-3-(trifluoromethyl)phenyl]carbamoylamino]-2-methylbenzamide	578724: Inhibition of CDK1/cyclin B	ic50	0.0032	uM
4-[[5-amino-1-(3,5-dimethylthiophene-2-carbonyl)-1,2,4-triazol-3-yl]amino]benzenesulfonamide	241014: Inhibition of Cyclin B-cyclin-dependent kinase 1	ic50	0.0032	uM
5-amino-N-(2,6-difluorophenyl)-3-(4-sulfamoylanilino)-1,2,4-triazole-1-carboxamide	241014: Inhibition of Cyclin B-cyclin-dependent kinase 1	ic50	0.0037	uM
(2R)-2-[[6-(3-chloroanilino)-9-propan-2-ylpurin-2-yl]amino]-3-methylbutan-1-ol	53206: In vitro inhibitory activity against Cyclin-dependent kinase 1-cyclin B complex from starfish oocytes	ic50	0.0040	uM
5-[(5-tert-butyl-1,3-oxazol-2-yl)methylsulfanyl]-N-pyridin-2-yl-1,3-thiazol-2-amine	53348: Inhibition of cyclin-dependent kinase 1-cyclin B	ic50	0.0040	uM
3-[[5-fluoro-4-[4-methyl-2-(methylamino)-1,3-thiazol-5-yl]pyrimidin-2-yl]amino]benzenesulfonamide	1940563: Inhibition of CDK1/Cyclin B (unknown origin) assessed as inhibition constant	ki	0.0040	uM
4-[[5-amino-1-(thiophene-2-carbonyl)-1,2,4-triazol-3-yl]amino]benzenesulfonamide	241014: Inhibition of Cyclin B-cyclin-dependent kinase 1	ic50	0.0045	uM
4-[[5-amino-1-(3-methylthiophene-2-carbonyl)-1,2,4-triazol-3-yl]amino]benzenesulfonamide	240969: Inhibition of Cyclin B-cyclin-dependent kinase 1	ic50	0.0048	uM
4-[[5-chloro-4-(1-methylpyrazol-4-yl)pyrimidin-2-yl]amino]benzenesulfonamide	1940568: Inhibition of CDK1/Cyclin B (unknown origin) assessed as inhibition constant incubated for 30 to 60 mins presence of dithiothreitol by Cheng-Prusoff equation analysis	ki	0.0050	uM
3-[[5-fluoro-4-(1-methylpyrazol-4-yl)pyrimidin-2-yl]amino]benzenesulfonamide	1940568: Inhibition of CDK1/Cyclin B (unknown origin) assessed as inhibition constant incubated for 30 to 60 mins presence of dithiothreitol by Cheng-Prusoff equation analysis	ki	0.0050	uM
3-[[5-chloro-4-(1-methylpyrazol-4-yl)pyrimidin-2-yl]amino]benzenesulfonamide	1940568: Inhibition of CDK1/Cyclin B (unknown origin) assessed as inhibition constant incubated for 30 to 60 mins presence of dithiothreitol by Cheng-Prusoff equation analysis	ki	0.0050	uM
(4-butoxy-2H-pyrazolo[3,4-b]pyridin-5-yl)-(2,6-difluoro-4-methylphenyl)methanone	53347: Inhibition of Cyclin-dependent kinase 1-cyclin B	ic50	0.0060	uM
4-[[5-fluoro-4-(1-methylpyrazol-4-yl)pyrimidin-2-yl]amino]benzenesulfonamide	1940568: Inhibition of CDK1/Cyclin B (unknown origin) assessed as inhibition constant incubated for 30 to 60 mins presence of dithiothreitol by Cheng-Prusoff equation analysis	ki	0.0060	uM
5-chloro-4-(1-methylpyrazol-4-yl)-N-(4-methylsulfonylphenyl)pyrimidin-2-amine	1940568: Inhibition of CDK1/Cyclin B (unknown origin) assessed as inhibition constant incubated for 30 to 60 mins presence of dithiothreitol by Cheng-Prusoff equation analysis	ki	0.0060	uM
2-chloro-4-[[2-[[(2R)-1-hydroxy-3-methylbutan-2-yl]amino]-9-propan-2-ylpurin-6-yl]amino]benzoic acid	53206: In vitro inhibitory activity against Cyclin-dependent kinase 1-cyclin B complex from starfish oocytes	ic50	0.0060	uM
1-[3-(2,4-dimethyl-1,3-thiazol-5-yl)-4-oxo-2H-indeno[1,2-c]pyrazol-5-yl]-3-(4-methylpiperazin-1-yl)urea	53342: Inhibition of cyclin-dependent kinase 1-cyclin B	ic50	0.0060	uM
4-[[5-amino-1-(2,6-difluorobenzoyl)-1,2,4-triazol-3-yl]amino]benzenesulfonamide	241014: Inhibition of Cyclin B-cyclin-dependent kinase 1	ic50	0.0060	uM
(4-butoxy-2H-pyrazolo[3,4-b]pyridin-5-yl)-(2,6-difluoro-4-methoxyphenyl)methanone	53347: Inhibition of Cyclin-dependent kinase 1-cyclin B	ic50	0.0070	uM
(2R,3R)-3-[[2-[4-(cyclopropylsulfonimidoyl)anilino]-5-(trifluoromethyl)pyrimidin-4-yl]amino]butan-2-ol	1921461: Inhibition of CDK1/Cyclin B (unknown origin)	ic50	0.0070	uM
5-[(5-tert-butyl-1,3-oxazol-2-yl)methylsulfanyl]-N-pyridin-4-yl-1,3-thiazol-2-amine	53348: Inhibition of cyclin-dependent kinase 1-cyclin B	ic50	0.0070	uM
N-(2-chlorophenyl)-4-(1H-pyrrolo[2,3-b]pyridin-3-yl)pyrimidin-2-amine	270821: Inhibition of CDK1/cyclinB by Flashplate assay	ic50	0.0090	uM
4-[[6-(cyclohexylmethoxy)-7H-purin-2-yl]amino]-N-methylbenzenesulfonamide	53195: Inhibitory activity against cyclin-dependent kinase 1-cyclin B.	ic50	0.0090	uM
4-[[6-(cyclohexylmethoxy)-7H-purin-2-yl]amino]benzenesulfonamide	53195: Inhibitory activity against cyclin-dependent kinase 1-cyclin B.	ic50	0.0095	uM
1-[5-[(5-tert-butyl-1,3-oxazol-2-yl)methylsulfanyl]-1,3-thiazol-2-yl]-3-(2,6-difluorophenyl)urea	55520: Inhibitory activity against baculovirus expressed Cyclin dependent kinase 1-cyclinB	ic50	0.0100	uM
5-[(5-tert-butyl-1,3-oxazol-2-yl)methylsulfanyl]-N-pyridin-3-yl-1,3-thiazol-2-amine	53348: Inhibition of cyclin-dependent kinase 1-cyclin B	ic50	0.0100	uM
4-[[5-[(5-tert-butyl-1,3-oxazol-2-yl)methylsulfanyl]-1,3-thiazol-2-yl]amino]-N-(2-hydroxyethyl)benzenesulfonamide	53348: Inhibition of cyclin-dependent kinase 1-cyclin B	ic50	0.0100	uM
5-chloro-4-(1,3-dimethylpyrazol-4-yl)-N-(1-methylsulfonylpiperidin-4-yl)pyrimidin-2-amine	1940568: Inhibition of CDK1/Cyclin B (unknown origin) assessed as inhibition constant incubated for 30 to 60 mins presence of dithiothreitol by Cheng-Prusoff equation analysis	ki	0.0120	uM
5-[(5-tert-butyl-1,3-oxazol-2-yl)methylsulfanyl]-N-phenyl-1,3-thiazol-2-amine	53348: Inhibition of cyclin-dependent kinase 1-cyclin B	ic50	0.0120	uM
(4-butoxy-2H-pyrazolo[3,4-b]pyridin-5-yl)-(4-chloro-2,6-difluorophenyl)methanone	53347: Inhibition of Cyclin-dependent kinase 1-cyclin B	ic50	0.0130	uM
N-(2-bromophenyl)-4-(1H-pyrrolo[2,3-b]pyridin-3-yl)pyrimidin-2-amine	270821: Inhibition of CDK1/cyclinB by Flashplate assay	ic50	0.0130	uM
N-cyclohexyl-4-(1H-pyrrolo[2,3-b]pyridin-3-yl)pyrimidin-2-amine	270821: Inhibition of CDK1/cyclinB by Flashplate assay	ic50	0.0140	uM
(4-bromo-2,6-difluorophenyl)-(4-butoxy-2H-pyrazolo[3,4-b]pyridin-5-yl)methanone	53347: Inhibition of Cyclin-dependent kinase 1-cyclin B	ic50	0.0170	uM
3-(3-nitroso-1H-indol-2-yl)-1H-indol-2-ol	512487: Inhibition of CDK1/cyclinB	ic50	0.0180	uM
propan-2-yl N-[5-[(5-tert-butyl-1,3-oxazol-2-yl)methylsulfanyl]-1,3-thiazol-2-yl]carbamate	55520: Inhibitory activity against baculovirus expressed Cyclin dependent kinase 1-cyclinB	ic50	0.0180	uM
3-[[6-[[5-[(5-tert-butyl-1,3-oxazol-2-yl)methylsulfanyl]-1,3-thiazol-2-yl]amino]-3-pyridinyl]methylamino]-2,2-dimethylpropan-1-ol	53348: Inhibition of cyclin-dependent kinase 1-cyclin B	ic50	0.0180	uM
1-[5-[(5-tert-butyl-1,3-oxazol-2-yl)methylsulfanyl]-1,3-thiazol-2-yl]-3-(4-fluorophenyl)urea	55520: Inhibitory activity against baculovirus expressed Cyclin dependent kinase 1-cyclinB	ic50	0.0190	uM
2-[[[2-[[(2R)-1-hydroxybutan-2-yl]amino]-9-propan-2-ylpurin-6-yl]amino]methyl]phenol	53346: Inhibition of recombinant Cyclin-dependent kinase 1-cyclin B	ic50	0.0200	uM

CTD chemical–gene interactions

17 total (human), top 17 by PubMed support.

Chemical	Actions (top 5)	PubMed papers
Valproic Acid	affects expression, increases expression	2
FR900359	increases phosphorylation	1
bisphenol A	affects expression	1
sodium arsenite	decreases expression	1
S-(1,2-dichlorovinyl)cysteine	affects response to substance, increases expression	1
polyhexamethyleneguanidine	decreases expression	1
CGP 52608	affects binding, increases reaction	1
abrine	decreases expression	1
Sunitinib	decreases expression	1
Arsenicals	increases expression	1
Benzo(a)pyrene	affects methylation, decreases methylation	1
Lipopolysaccharides	affects response to substance, increases expression	1
N-Nitrosopyrrolidine	decreases expression	1
Niclosamide	decreases expression	1
Rotenone	increases expression	1
Okadaic Acid	increases expression	1
Particulate Matter	increases expression	1

ChEMBL screening assays

148 unique, capped per target: 147 binding, 1 functional

Representative assays (with source publication via chembl_document):

Assay ID	Type	Description	Source paper
CHEMBL1037380	Binding	Inhibition of human CDK1/cyclin B at 20 uM by FRET assay	Synthesis and biological evaluation of p38alpha kinase-targeting dialkynylimidazoles. — Bioorg Med Chem Lett
CHEMBL700201	Functional	In vitro antiproliferative activity against myeloid leukemia K562 cell line	Pyrazolo[4,3-d]pyrimidines as new generation of cyclin-dependent kinase inhibitors. — Bioorg Med Chem Lett

Clinical trials (associated diseases)

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

Trial	Phase	Status	Title
NCT01289847	PHASE4	COMPLETED	A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency
NCT00220766	PHASE3	COMPLETED	Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency Patients
NCT00278954	PHASE3	COMPLETED	Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases.
NCT03837483	PHASE3	ACTIVE_NOT_RECRUITING	A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome
NCT04340700	PHASE3	WITHDRAWN	Characterization of the Pharmacodynamic Response to Vaped THC
NCT00909363	PHASE2	TERMINATED	Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients
NCT01529827	PHASE2	COMPLETED	Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
NCT01821781	PHASE2	ACTIVE_NOT_RECRUITING	Immune Disorder HSCT Protocol
NCT02512679	PHASE2	TERMINATED	Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells
NCT03019809	PHASE2	UNKNOWN	A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients
NCT03333486	PHASE2	TERMINATED	Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
NCT04371939	PHASE2	UNKNOWN	Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome
NCT04360044	PHASE2	COMPLETED	Efficacy of Inhaled Cannabis for Acute Migraine Treatment
NCT05427630	PHASE2	SUSPENDED	Dose-Ranging Trial of Inhaled Cannabis for Acute Migraine Treatment
NCT05514899	PHASE2	RECRUITING	Effects of Cannabidiol and Tetrahydrocannabinol on Microbiome and Neuroinflammation in HIV
NCT05641766	PHASE2	NOT_YET_RECRUITING	Multimodal Magnetoencephalography and Electroencephalography Exploration of the Acute Effects of THC Exposure on Neural Noise and Information Transmission Within Working Memory Networks
NCT05999383	PHASE2	RECRUITING	Understanding the Clinical Pharmacology of Marijuana-Tobacco Co-administration
NCT06647524	PHASE2	RECRUITING	Pilot fMRI Studies of Aging-Related Effects of THC
NCT00160355	PHASE1	COMPLETED	Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome
NCT00774358	PHASE1	COMPLETED	Interleukin-2 Treatment for Wiskott-Aldrich Syndrome
NCT01917708	PHASE1	COMPLETED	Bone Marrow Transplant With Abatacept for Non-Malignant Diseases
NCT03098940	PHASE1	UNKNOWN	A Bioavailability Study on Dronabinol
NCT04130633	PHASE1	COMPLETED	Behavioral Pharmacology of THC and Alpha-pinene
NCT05121506	PHASE1	COMPLETED	A Study to Investigate the Bioavailability and Skin Absorption of CBD and THC From GT4 Technology in Healthy Adults
NCT06378957	PHASE1	RECRUITING	Behavioral Pharmacology of Orally Administered THC and D-limonene
NCT02699190	Not specified	COMPLETED	LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
NCT03047369	Not specified	RECRUITING	The Myelin Disorders Biorepository Project
NCT03572114	Not specified	UNKNOWN	Imaging Neuromelanin and Iron in Dystonia/Parkinsonism
NCT05522374	Not specified	RECRUITING	TIRCON International NBIA Registry
NCT06938542	Not specified	ENROLLING_BY_INVITATION	Palliative Care Needs of Children With Rare Diseases and Their Families
NCT00730314	PHASE1/PHASE2	COMPLETED	Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
NCT00885833	PHASE1/PHASE2	COMPLETED	Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS)
NCT01347242	PHASE1/PHASE2	COMPLETED	Gene Therapy for Wiskott-Aldrich Syndrome (WAS)
NCT01347346	PHASE1/PHASE2	COMPLETED	Gene Therapy for WAS
NCT01410825	PHASE1/PHASE2	COMPLETED	Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome
NCT01515462	PHASE1/PHASE2	COMPLETED	Gene Therapy for Wiskott-Aldrich Syndrome
NCT01852370	PHASE1/PHASE2	ENROLLING_BY_INVITATION	Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases
NCT02333760	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome
NCT03513328	PHASE1/PHASE2	COMPLETED	Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
NCT00004341	Not specified	UNKNOWN	Study of Genetic and Molecular Defects in Primary Immunodeficiency Disorders