ION 356
Alternative Names: ION-356Latest Information Update: 25 Apr 2024
At a glance
- Originator Ionis Pharmaceuticals
- Class Antisense oligonucleotides
- Mechanism of Action Gene silencing; PLP1 protein expression inhibitors
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- Phase I Pelizaeus-Merzbacher disease
Most Recent Events
- 10 Apr 2024 Phase-I clinical trials in Pelizaeus-Merzbacher disease (In children, In adolescents) in France (Intrathecal) (NCT06150716)
- 10 Apr 2024 Phase-I clinical trials in Pelizaeus-Merzbacher disease (In children, In adolescents) in Netherlands (Intrathecal) (NCT06150716)
- 04 Oct 2023 Ionis Pharmaceuticals plans a first-in-patient trial for Pelizaeus-Merzbacher disease in 2024