Safety first: advancing siRNA therapeutics with AI-driven design and off-target minimization

“Being able to bring molecules to patients with diseases that start outside the liver is incredibly inspirational.”Jokūbas Leikauskas, Editor, BioInsights, speaks to Marie Wikström Lindholm, Chief Scientific Officer at Silence Therapeutics, about advances in short-interfering RNA (siRNA) design, the role of artificial intelligence (AI)/machine learning (ML) in oligonucleotide development, strategies for minimizing off-target risks, and future goals for safer, broader therapeutic applications beyond the liver.