Industry insights: Clinical advances and regulatory momentum across nucleic acid therapeutic modalities

Abstract

May and June 2026 saw significant clinical and regulatory momentum across nucleic acid therapeutic modalities. Regulatory highlights included FDA Breakthrough Therapy Designation for Biogen’s intrathecally administered ASO salanersen in spinal muscular atrophy and MHRA authorisation of Moderna and the University of Oxford’s INTERCEPT-Lynch Phase 1/2 trial of mRNA-4194, the first investigational mRNA cancer prevention vaccine. In clinical development, Novartis reported positive Phase 1/2 biomarker data for the AOC del-brax in facioscapulohumeral muscular dystrophy, GSK published Phase 3 data demonstrating a 19% functional cure rate for the ASO bepirovirsen in chronic hepatitis B, and SpliSense secured $13 million from the Cystic Fibrosis Foundation to advance inhaled ASO SPL84 into Phase 2b. Strategic activity included a multi-target ASO collaboration between Servier and n-Lorem Foundation in rare neurodevelopmental disorders, and WuXi AppTec’s announcement of a 17% increase in capital expenditure to expand oligonucleotide and peptide manufacturing capacity globally.