Ionis reported first dosing in ASCEND, a Phase 1-2 study of ION337 in children with Dravet syndrome.
The trial evaluates safety and tolerability; it includes a 6-month single-ascending dose period followed by a 24-month multiple-ascending dose period with dosing every 6 months.
ION337 is an RNA-targeted antisense medicine using Ionis’ NMA chemistry, designed to boost potency and support infrequent intrathecal dosing.
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