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ION337 ascending-dose study in pediatric Dravet syndrome
2025-523835-20-00Phase 2Authorised
- Sponsor
- Ionis Pharmaceuticals
- Start
- Jun 2026
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 10
01
Overview
Phase 2, open-label trial of ION337 in Dravet syndrome (2 arms), sponsored by Ionis Pharmaceuticals. Primary endpoint: Safety and tolerability. Planned start Jun 2026; status: authorised.
02
Drugs
ION337
03
Conditions
Dravet syndrome
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