^
    Contact us  to learn more about
    our Premium Content:  News alerts, weekly reports and conference planners
    GENE:

    KAT6A (Lysine Acetyltransferase 6A)

    i
    Other names: KAT6A, Lysine Acetyltransferase 6A, MOZ, Monocytic Leukemia Zinc Finger Protein, ZC2HC6A, RUNXBP2, ZNF220, MYST3, MYST Histone Acetyltransferase (Monocytic Leukemia) 3, Runt-Related Transcription Factor Binding Protein 2, MOZ, YBF2/SAS3, SAS2 And TIP60 Protein 3, Histone Acetyltransferase KAT6A, K(Lysine) Acetyltransferase 6A, Zinc Finger Protein 220, MYST-3, Runt-Related Transcription Factor-Binding Protein 2, Histone Acetyltransferase MYST3, ARTHS, MRD32
    22h
    Revumenib in Combination With 7+3 + Midostaurin in AML (clinicaltrials.gov)
    P1, N=22, Recruiting, Richard Stone, MD | Trial completion date: Mar 2027 --> Mar 2028 | Trial primary completion date: Mar 2026 --> Mar 2027
    Trial completion date • Trial primary completion date
    |
    TP53 (Tumor protein P53) • FLT3 (Fms-related tyrosine kinase 3) • ABL1 (ABL proto-oncogene 1) • NPM1 (Nucleophosmin 1) • DNMT3A (DNA methyltransferase 1) • RUNX1 (RUNX Family Transcription Factor 1) • SF3B1 (Splicing Factor 3b Subunit 1) • ASXL1 (ASXL Transcriptional Regulator 1) • KMT2A (Lysine Methyltransferase 2A) • SRSF2 (Serine and arginine rich splicing factor 2) • CREBBP (CREB binding protein) • BCOR (BCL6 Corepressor) • U2AF1 (U2 Small Nuclear RNA Auxiliary Factor 1) • STAG2 (Stromal Antigen 2) • MECOM (MDS1 And EVI1 Complex Locus) • NUP214 (Nucleoporin 214) • GATA2 (GATA Binding Protein 2) • KAT6A (Lysine Acetyltransferase 6A) • ZRSR2 (Zinc Finger CCCH-Type, RNA Binding Motif And Serine/Arginine Rich 2)
    |
    TP53 mutation • FLT3-ITD mutation • NPM1 mutation • Chr del(5q)
    |
    midostaurin • daunorubicin • Revuforj (revumenib)
    13d
    Differentiation Therapy in Acute Myeloid Leukemia: Advances in Phenotypic Screening and CRISPR-based Functional Genomics. (PubMed, Acta Haematol)
    Notably, recent phenotypic screening studies identified Triciribine (TCN), an AKT inhibitor, as a differentiation inducer in AML cells...However, differentiation therapy outside APL has shown variable and often incomplete clinical success, frequently limited by partial maturation and context-dependent responses. Together, these approaches reveal novel therapeutic vulnerabilities in AML and support the development of differentiation-based strategies for a broader range of patients.
    Review • Journal
    |
    KAT6A (Lysine Acetyltransferase 6A) • YTHDC1 (YTH Domain Containing 1) • SLC2A1 (Solute Carrier Family 2 Member 1) • ZFP36 (ZFP36 Ring Finger Protein)
    |
    triciribine phosphate (PTX-200)
    22d
    HAP2HCT: TCRαβ-depleted Progenitor Cell Graft With Additional Memory T-cell DLI, Plus Selected Use of Blinatumomab, in Naive T-cell Depleted Haploidentical Donor Hematopoietc Cell Transplantation for Hematologic Malignancies (clinicaltrials.gov)
    P2, N=69, Active, not recruiting, St. Jude Children's Research Hospital | N=140 --> 69 | Trial completion date: Aug 2025 --> Jun 2026
    Enrollment change • Trial completion date
    |
    FLT3 (Fms-related tyrosine kinase 3) • RUNX1 (RUNX Family Transcription Factor 1) • KMT2A (Lysine Methyltransferase 2A) • ETV6 (ETS Variant Transcription Factor 6) • WT1 (WT1 Transcription Factor) • CREBBP (CREB binding protein) • NUP98 (Nucleoporin 98 And 96 Precursor 2) • NSD1 (Nuclear Receptor Binding SET Domain Protein 1) • HOXA9 (Homeobox A9) • NUP214 (Nucleoporin 214) • CBFA2T3 (CBFA2/RUNX1 Partner Transcriptional Co-Repressor 3) • GLIS2 (GLIS Family Zinc Finger 2) • KAT6A (Lysine Acetyltransferase 6A) • KDM5A (Lysine Demethylase 5A) • DEK (DEK Proto-Oncogene) • AFDN (Afadin, Adherens Junction Formation Factor) • MLLT10 (MLLT10 Histone Lysine Methyltransferase DOT1L Cofactor)
    |
    FLT3-ITD mutation
    |
    cyclophosphamide • Blincyto (blinatumomab) • melphalan • fludarabine IV • mesna • thiotepa • Neupogen (filgrastim)
    1m
    KAT6 inhibitors under investigation for solid tumors: the preclinical and early phase progress. (PubMed, Expert Opin Investig Drugs)
    Prifetrastat in combination with Fulvestrant is now being evaluated in a phase III trial for pretreated ER+ advanced breast cancer. Additionally, mitigation strategies for dysgeusia and clinically available response-predictive biomarkers should be developed. KAT6 inhibitors with different target spectra, including KAT6A-selective and KAT6/7 inhibitors, may exhibit differential efficacy and safety profiles, offering deeper insights into KAT6-targeted therapy.
    Preclinical • Journal
    |
    ER (Estrogen receptor) • KAT6A (Lysine Acetyltransferase 6A) • KAT6B (Lysine Acetyltransferase 6B)
    |
    ER positive
    |
    fulvestrant • prifetrastat (PF-07248144)
    2ms
    Biological Activity and Structural Biology of Current KAT6A Inhibitor Chemotypes. (PubMed, J Med Chem)
    We extensively benchmark key compounds from each chemotype, augmented by new acylsulfonohydrazide analogues and a novel fused [1,2,4]thiadiazine KAT6A inhibitor subclass, which we report here for the first time, along with co-crystal structures. Additionally, we report on the in vivo activity, pharmacokinetics, and toxicology profiles of these inhibitors.
    Journal
    |
    KAT6A (Lysine Acetyltransferase 6A)
    2ms
    Dancing with KAT6A: Current advances and therapeutic potential in oncology of KAT6A inhibitors. (PubMed, Bioorg Chem)
    PF-07248144 has shown antitumor activity in estrogen receptor-positive (ER+) breast cancer models and is currently undergoing clinical evaluation...This review provides a comprehensive overview of the structural and biological functions of KAT6A, its role in tumor progression, and the therapeutic potential of its inhibition. It summarizes the advancements in KAT6A inhibitor development from 2019 to the present, emphasizing the optimization processes from lead compounds to clinical candidates.
    Review • Journal
    |
    ER (Estrogen receptor) • KAT6A (Lysine Acetyltransferase 6A)
    |
    ER positive
    |
    prifetrastat (PF-07248144)
    2ms
    Phf6 truncating mutation drives leukemogenesis via disrupted epigenetic regulation in mice. (PubMed, Leukemia)
    Treatment with CTx-648, a KAT6A/KAT6B inhibitor, restored HSC function in Phf6R274XTg mice and prolonged the survival of leukemic Phf6R274XTg mice. These findings demonstrate a gain-of-function effect for truncated PHF6aa1-273 in driving leukemogenesis and highlight KAT6B as a promising therapeutic target in PHF6 truncation-associated hematologic malignancies.
    Preclinical • Journal
    |
    PHF6 (PHD Finger Protein 6) • KAT6A (Lysine Acetyltransferase 6A) • KAT6B (Lysine Acetyltransferase 6B)
    |
    CTx-648
    2ms
    MYST acetyltransferases are a targetable therapeutic vulnerability in SETBP1-mutant leukemia. (PubMed, bioRxiv)
    To establish the efficacy of MYST inhibition in vivo , we treated mice harboring a syngeneic SETBP1 -mutant leukemia with the clinical-grade MYST inhibitor-PF-9363...In this study, we identify MYST acetyltransferases as key drivers of mutant SETBP1-driven transcription. MYST inhibitors are highly effective against SETBP1-mutant leukemia and represent a promising avenue for clinical translation.
    Journal
    |
    SETBP1 (SET Binding Protein 1) • KAT6A (Lysine Acetyltransferase 6A)
    |
    SETBP1 mutation
    |
    CTx-648
    3ms
    Histone acetyltransferase KAT6A contributes to colon cancer malignant progression by inhibiting ferroptosis. (PubMed, BMC Cancer)
    KAT6A promotes the proliferation of CC cells and suppresses ferroptosis via epigenetic regulation of GPX4. Our work presented KAT6A as a potential diagnostic and therapeutic target for treatment CC.
    Journal
    |
    GPX4 (Glutathione Peroxidase 4) • KAT6A (Lysine Acetyltransferase 6A)
    |
    erastin
    3ms
    Exploring KAT6 as a therapeutic target in breast cancer: epigenetic approaches for precision medicine. (PubMed, NPJ Breast Cancer)
    We discuss preclinical evidence supporting the therapeutic potential of KAT6 inhibition and the challenges faced in epigenetic drug development, including the need for robust biomarker-driven approaches. Finally, we explore the prospects for integrating KAT6 inhibitors with existing therapies to overcome resistance and improve patient outcomes in breast cancer.
    Review • Journal
    |
    KAT6A (Lysine Acetyltransferase 6A)
    |
    HR positive
    4ms
    Lysine Acetyltransferase 6 in Health and Disease. (PubMed, MedComm (2020))
    In this review, we summarize the currently available information regarding the physiological and pathological functions of KAT6A and KAT6B and discuss their potential as antitumor targets in drug development. We also present the discovery and development of an emerging class of KAT6 inhibitors under investigation for breast cancer, along with potential molecular mechanisms underlying the therapeutic efficacy of targeting KAT6, providing references for developing therapeutic strategies in clinical practice.
    Review • Journal
    |
    KAT6A (Lysine Acetyltransferase 6A) • KAT6B (Lysine Acetyltransferase 6B)
    4ms
    The role of histone acetyltransferases in tumorigenesis and their therapeutic potential: A review. (PubMed, Biochem Biophys Res Commun)
    It also investigates interactions between KATs and Tumor-associated signalling pathways, with a focus on emerging KAT inhibitors and their clinical progress. The aim is to provide a theoretical basis and direction for future research into epigenetic therapeutic strategies for cancer.
    Review • Journal • IO biomarker
    |
    KAT6A (Lysine Acetyltransferase 6A)