E7107 (E-7107) is a derivative of pladienolide that targets the SF3b subunit of the spliceosome, shows significant antitumor activity in vitro and in vivo.

HTT-D3 is a potent, CNS-penetrant, orally bioavailable huntingtin (HTT) splicing modulator, affects HTT pre-mRNA splicing and reduces HTT expression with IC50 of 8 nM.Oral administration of HTT-D3 resulted in dose-dependent and approximately equivalent mHTT protein lowering in both brain and peripheral tissues in mouse models carrying human mHTT transgene, BACHD and Hu97/18.HTT-D3 (10 mg/kg) reduced mHTT mRNA and protein levels in BACHD brain in BACHD mice.

Thailanstatin B is a potent antiproliferative natural product and pre-mRNA splicing inhibitor, potential antibody-drug conjugate payload.

Thailanstatin D (TST-D) is a potent antiproliferative natural product and direct precursor of Thailanstatin A, inhibits eukaryotic RNA splicing with stiong anti-tumor activities.Thailanstatin D inhibit AR-V7 gene splicing by interfering the interaction between U2AF65 and SAP155 and Thailanstatin D preventing them from binding to polypyrimidine tract located between the branch point and the 3' splice site.Thailanstatin D exhibits a potent tumor inhibitory effect on human CRPC xenografts leading to cell apoptosis.Thailanstatins are attractive cytotoxin agent for ADC design.

UHMCP1 is a small molecule that prevents the SF3b155/U2AF65 interaction, binds to the hydrophobic pocket of the U2AF65 UHM domain (Kd=1-20 uM), impacts splicing and cell viability.