Drug Profile

ION 356

Alternative Names: ION-356

Latest Information Update: 11 Nov 2024

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At a glance

Originator Ionis Pharmaceuticals
Class Antisense oligonucleotides
Mechanism of Action Gene silencing; PLP1 protein expression inhibitors

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Pelizaeus-Merzbacher disease
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 USA, Germany (Intrathecal) (NCT06150716)
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)

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