Abstract

The FDA approval of the first siRNA drug in 2018 marked a key milestone for RNAi therapeutics, confirming the potential of gene-silencing oligonucleotides (1). Small interfering RNAs (siRNAs) selectively silence disease-related genes, enabling targeted treatments for genetic and metabolic diseases.

Two siRNA IND Packages Delivered in 14 Months
The FDA approval of the first siRNA drug in 2018 marked a key milestone for RNAi therapeutics, confirming the potential of gene-silencing oligonucleotides (1). Small interfering RNAs (siRNAs) selectively silence disease-related genes, enabling targeted treatments for genetic and metabolic diseases.

Among current delivery strategies, conjugation with N-acetylgalactosamine (GalNAc) has shown strong clinical potential for targeted delivery to hepatocytes via subcutaneous administration. However, the synthesis of GalNAc-siRNA conjugates involves complex chemistries and stringent quality requirements, making process development and manufacturing particularly challenging.

WuXi TIDES recently supported a US-based biotech company in advancing two GalNAc-siRNA candidates to IND submission within 14 months through integrated Chemistry, Manufacturing, and Controls (CMC) services and ... ...