SRPK1 inhibitor

SRPK1 inhibitors are a class of small molecules that target Serine/Arginine-rich Protein Kinase 1 (SRPK1), a key regulator of mRNA splicing. SRPK1 phosphorylates SR-rich splicing factors, such as SRSF1, which in turn control the selection of splice sites in pre-mRNA. A primary therapeutic mechanism involves modulating the alternative splicing of Vascular Endothelial Growth Factor (VEGF-A) to favor anti-angiogenic isoforms (VEGF165b) over pro-angiogenic ones (VEGF165), making these inhibitors potential treatments for neovascular eye diseases like age-related macular degeneration (AMD). In oncology, SRPK1 inhibition has shown promise in treating acute myeloid leukemia (AML) by disrupting leukemogenic programs and overcoming drug resistance in metastatic colorectal cancer (mCRC). Notable compounds in this class include SCO-101 (Endovion), SPHINX31, and SRPIN340.

Other names

SRPK1 inhibitorsSRPK-1 inhibitorsSRPK 1 inhibitorsSerine/arginine-rich protein kinase 1 inhibitors