Exploring multifunctional design-for-delivery approaches for nucleic acid therapeutics

“[...] there have been three key categories of strategies that have had a transformative impact on the field – chemical design, conjugation, and formulation-based approaches.”Jokūbas Leikauskas, Editor, BioInsights, speaks with Sasha Ebrahimi, Scientific Leader, Emerging Drug Delivery Platforms team at GSK, about advanced strategies to improve the developability and targeted delivery of nucleic acid therapeutics. In particular, they discuss emerging design-for-delivery approaches, such as ligand- and formulation-based multifunctional approaches for extrahepatic delivery. They also highlight key translational challenges such as immune activation, safety considerations under repeat dosing, and the growing need for scalable chemical and enzymatic synthesis methods to support increasingly complex oligonucleotide constructs.