Integrated Platform Supporting ASO Drug Discovery

Antisense oligonucleotides (ASOs) are single-stranded, synthetic chains of DNA or RNA designed to bind to specific RNA sequences through complementary base-pairing to regulate gene expression. This ability to precisely target and modulate gene expression makes ASOs a promising therapeutic modality for diseases with known genetic causes, such as genetic disorders and cancers.

WuXi Biology has established an integrated ASO drug discovery platform that encompasses sequence design, efficient oligonucleotide synthesis, and in vitro and in vivo efficacy and safety testing. To demonstrate the capabilities of our platform, WuXi AppTec scientists presented a poster at OTS 2025 highlighting a case study on the development of a potent and safe gapmer ASO targeting ATXN2 expression, as a potential treatment for amyotrophic lateral sclerosis (ALS).

Poster_OTS 2025_Platform Supporting ASO Drug Discovery

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