| DC74241 |
Rebecsinib
|
Rebecsinib (17S-FD-895) is a selective small-molecule inhibitor of splicing-mediated ADAR1 activation, prevents leukemia stem cell generation. |
| DC70497 |
HTT-D3
|
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. |
| DC74243 |
VPC-80051
|
VPC-80051 (VPC80051) is the first small molecule inhibitor of hnRNP A1 splicing activity, targets RNA-binding domain (RBD) of hnRNP A1. |
| DC74242 |
Thailanstatin C
|
Thailanstatin C (TST-C) is a potent antiproliferative natural product, a pre-mRNA splicing inhibitor and antiproliferative agent.. |
| DC74240 |
HTT splicing modulator 27
|
HTT splicing modulator 27 is a potent, orally bioavailable, and brain-penetrant small-molecule HTT pre-mRNA splicing modulator with mHTT protein EC50 of 66 nM. |
| DC74239 |
CD33 splicing modulator 1
|
CD33 splicing modulator 1 is a small-molecule CD33 pre-mRNA splicing modulator with EC50 of 7.8 uM, enhances the exclusion of CD33 exon 2, reduces full-length V-domain containing CD33 protein. |
| DC74238 |
BPN15477
|
BPN-15477 (BPN15477) is a potent splicing modulator compound that restores correct splicing of ELP1 exon 20, increases full-length ELP1 mRNA by increasing exon 20 inclusion with EC50 of 1.9 uM in luciferase splicing assays. |
| DC70864 |
UHMCP1
|
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. |
| DC70842 |
Thailanstatin D
|
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. |
| DC70841 |
Thailanstatin B
|
Thailanstatin B is a potent antiproliferative natural product and pre-mRNA splicing inhibitor, potential antibody-drug conjugate payload. |
