Therapeutic oligonucleotide metabolite identification, profiling and quantitation

The effect of small strands of therapeutic oligonucleotides, or siRNAs, on gene expression has significantly impacted basic and applied pharmaceutical research. As a result, siRNA gene silencing is one of the fastest growing approaches for disease therapy. The blocking ability of siRNAs on the activity of select genetic sequences facilitates effective and controlled gene silencing. Therapeutic oligonucleotides are already being utilized for treatment of hepatitis C virus, age-related macular degeneration, cholesterol control, as well as a variety of cancer therapies. 

In this White Paper, QPS Holdings, LLC (DE, USA) summarize the siRNA interference mechanism, the barriers encountered with delivery of these therapies and the role of metabolite identification and quantitation in siRNA drug development. 

QPS_Therapeutic Oligonucleotide Metabolite_2023

In association with