Industry Insights: From a landmark Alexander disease approval to advancing CRISPR and saRNA platforms in rare disease

Abstract

Late July to mid-September 2026 saw continued regulatory, clinical, and strategic momentum across nucleic acid therapeutic modalities, with notable clustering in rare neurological and neuromuscular disorders. On the regulatory front, the US FDA approved Ionis’ antisense oligonucleotide Zanvastro™ (zilganersen) for Alexander disease, the first therapy to address the condition’s underlying cause, granted Fast Track Designation to HAYA Therapeutics’ long non-coding RNA-targeting ASO HTX-001 in nonobstructive hypertrophic cardiomyopathy, and cleared Ractigen Therapeutics’ small activating RNA candidate RAG-1 °C in proliferative vitreoretinopathy. In the clinic, Oak Hill Bio and Aligos Therapeutics initiated trials of rugonersen in Angelman syndrome and ALG-170675 in chronic hepatitis B. Strategic activity featured Aptar – Aceso and Exegenesis – Modalis collaborations in cystic fibrosis and Duchenne muscular dystrophy, alongside financings of $120 million and $31 million closed by AusperBio and Ractigen.