Industry Insights: Antisense oligonucleotide milestones in rare genetic disease and new approaches to delivery and manufacturing

Mid-September to mid-October 2026 saw clinical, regulatory, and strategic progress across antisense, RNA-editing, and small interfering RNA modalities, with a focus on rare genetic disease. In the clinic, SpliSense and Korro Bio initiated trials of inhaled antisense oligonucleotide SPL84 in cystic fibrosis and RNA-editing oligonucleotide KRRO-121 in urea cycle disorders, while Otsuka reported positive Phase 3 data for ulefnersen in FUS-associated amyotrophic lateral sclerosis. On the regulatory front, Stoke Therapeutics completed a US FDA meeting on its planned New Drug Application for zorevunersen in Dravet syndrome, and Nitto Avecia received a No Action Indicated inspection classification. Market activity included a National Institutes of Health grant to AlphaRose Therapeutics and a $135 million series B for Soufflé Therapeutics, alongside collaborations spanning enzymatic oligonucleotide manufacturing (Codexis), small interfering RNA candidate selection (Wave Life Sciences – GSK), and AI-guided antisense design (TychoBio – PacBio). In this issue: Clinical Trials and Research Regulatory Changes and Updates Market Trends Collaborations, Partnerships, and Acquisitions

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