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Phase 1/2 multiple ascending dose study of intrathecal ION440 in MECP2 duplication syndrome
2023-507192-22-00Phase 2Authorised
- Sponsor
- Ionis Pharmaceuticals
- Start
- Oct 2025
- 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
- 7
01
Overview
Phase 2, double-blind trial of ION440 in MECP2 duplication syndrome (M2DS) (2 arms), sponsored by Ionis Pharmaceuticals. Primary endpoint: Safety and tolerability. Planned start Oct 2025; status: authorised.
02
Drugs
ION440
03
Conditions
MECP2 duplication syndrome (M2DS)
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