Across January and early February 2026, activity in the nucleic acid therapeutics field spanned early clinical readouts, regulatory designations, preclinical advances, and new strategic partnerships. ProQR reported initial Phase 1 data for its editing oligonucleotide AX-0810 in cholestatic diseases, while Providence Therapeutics supported the launch of a multi-site paediatric trial evaluating personalized mRNA cancer vaccines in Australia. Regulatory momentum continued, with the US FDA granting Fast Track designation to BioNTech’s mRNA immunotherapy BNT113 for HPV16-positive head and neck cancer, and Japan awarding Orphan Drug designation to Dyne’s antisense candidate DYNE-101 for myotonic dystrophy type 1. Preclinical studies highlighted mRNA and antisense approaches in infertility and metabolic disease, alongside new collaborations to expand access to individualized antisense therapies and advance mRNA treatment for propionic acidemia.
Jokūbas Leikauskas (2026) studied this question.