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Jokūbas Leikauskas

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Open access Sep 2026

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

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 Alex...

Jokūbas Leikauskas · 0 citations
Open access Aug 2026

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

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 T...

Jokūbas Leikauskas · 0 citations
Open access Jul 2026

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

May and June 2026 saw significant clinical and regulatory momentum across nucleic acid therapeutic modalities and 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 expendit...

Jokūbas Leikauskas · 0 citations

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