Industry Insights: Clinical and regulatory momentum for antisense and mRNA therapeutics

Across February and March 2026, activity in the nucleic acid therapeutics field highlighted continued clinical progression of antisense oligonucleotides (ASOs) alongside expanding regulatory momentum and strategic industry developments. Dyne Therapeutics initiated a Phase 3 trial of its ASO candidate DYNE–101 in myotonic dystrophy type 1, while Vico Therapeutics and Stoke Therapeutics advanced early–stage clinical studies targeting polyglutamine disorders and autosomal dominant optic atrophy, respectively. Regulatory updates included the US FDA IND clearance for Innorna’s mRNA therapy in refractory gout and Fast Track designations for RNA–based therapies from Argo Biopharma and ARTHEx Biotech. Industry activity also featured Novartis’ acquisition of Avidity Biosciences, a collaboration to advance personalized RNA medicines, new company formation in mRNA, and funding to support mRNA therapeutics and delivery platforms.

Dyne Therapeutics initiated a Phase 3 trial of DYNE-101 for myotonic dystrophy type 1; Clinical Trials and Research. Credit: stock.adobe.com.

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