SK-2407

SK-2407 (also known as SN-001) is an antisense oligonucleotide (ASO) being co-developed by Sanwa Kagaku Kenkyusho and Nissan Chemical Corporation for the treatment of dentatorubral-pallidoluysian atrophy (DRPLA). DRPLA is a rare, autosomal dominant neurodegenerative disorder caused by the expansion of a CAG repeat in the ATN1 gene, which leads to the accumulation of toxic polyglutamine-expanded atrophin-1 protein. SK-2407 is designed to target and reduce the levels of this abnormal protein in the central nervous system. As of early 2025, the drug is in the preclinical stage of development, with the companies conducting Good Laboratory Practice (GLP) studies to support the initiation of clinical trials in Japan.