RNA splicing modulator-4

RNA splicing modulator-4 (Compound A) is an orally active, brain-penetrant and selective RNA splicing modulator. RNA splicing modulator-4 regulates the inclusion or exclusion of specific exons in precursor mRNA (pre-mRNA) by modulating alternative splicing events, thereby altering the coding sequence and function of mature mRNAs. RNA splicing modulator-4 is promising for research of neurodegenerative diseases (e.g., Huntington’s disease) and cancers[1].

Product Specifications

CAS Number

[3067915-73-2]

UNSPSC

12352005

Target

DNA/RNA Synthesis

Related Pathways

Cell Cycle/DNA Damage

Applications

Cancer-programmed cell death

Field of Research

Cancer; Neurological Disease

Smiles

OC1=C(C2=CC=C(N=N2)N([C@@H]3[C@@H]([C@]4([H])N[C@](CCC4)([H])C3)F)C)C=C5C(N=C(O5)C)=C1

Molecular Formula

C21H24FN5O2

Molecular Weight

397.45

References & Citations

[1]Wester DE POEL, et al. Crystalline forms of 6- (6- (((1r,2r,3s,5s) -2-fluoro-9-azabicyclo[3.3.1]nonan-3-yl) (methyl) amino) pyridazin-3-yl) -2-methylbenzo[d]oxazol-5-ol, a splicing modulator for the treatment of huntington's disease. WO2025006887A1. 2025-01-02.