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First-in-human dose-escalation study of intrathecal ION582 in Angelman syndrome
MOH_2022-06-21_011863Phase 2Recruiting
- Sponsor
- Ionis Pharmaceuticals
- Start
- Jun 2022
- 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
- 1
01
Overview
Phase 2, open-label trial of obudanersen in Angelman syndrome (AS) (5 cohorts), sponsored by Ionis Pharmaceuticals. Primary endpoint: Safety and tolerability. Started Jun 2022; status: recruiting.
02
Drugs
obudanersen
03
Conditions
Angelman syndrome (AS)
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