Industry Insights: Advancing oligonucleotide therapeutics through regulatory approvals, clinical data, and AI-enabled discovery

Across April and May 2026, the nucleic acid therapeutics field saw broad regulatory momentum, continued clinical advancement, and significant manufacturing investment. Regulatory highlights included MHRA authorisation of the ligand-conjugated ASO donidalorsen for hereditary angioedema prophylaxis and US FDA acceptance of GSK's NDA for the ASO bepirovirsen in chronic hepatitis B, the latter also receiving Breakthrough Therapy Designation. Clinical milestones featured the 50th patient treated under n-Lorem Foundation's personalized ASO programme and first results from HF-REVERT, the inaugural randomized trial of microRNA inhibition in heart failure. Manufacturing expansion featured prominently, with both Agilent Technologies and WuXi AppTec announcing major capacity increases for oligonucleotide production. AI-driven drug discovery also advanced, with RareLabs launching a robotics- and AI-enabled platform for rare disease ASO and siRNA development, and Ribo Life Science partnering with Insilico Medicine to integrate AI across its siRNA pipeline.