Industry Insights: Clinical and regulatory momentum across nucleic acid modalities, with advances in ALS and obesity

Abstract

June and July 2026 saw continued clinical, regulatory, and strategic momentum across nucleic acid therapeutic modalities, with notable clustering in antisense approaches to ALS and RNA-based approaches to obesity and cardiometabolic disease. On the regulatory front, Praxis Precision Medicines secured FDA Breakthrough Therapy Designation for the ASO elsunersen in SCN2A developmental and epileptic encephalopathy, and Atrium Therapeutics received IND clearance for siRNA ATR-1072 in PRKAG2 syndrome. In the clinic, Wave Life Sciences and Resalis Therapeutics advanced siRNA and ASO candidates in obesity, while Leal Therapeutics and Trace Neuroscience initiated ASO trials in ALS. Preclinical highlights included CDW Holding’s spleen-targeted LNP-LANFA delivery platform and Confluence Genetics’ Cas-CLEAR CRISPR oncology platform. Strategic activity featured a Ribo–Madrigal siRNA milestone in MASH and a Vect-Horus–Servier agreement to develop brain-targeted oligonucleotides for rare CNS disorders.