Drug Profile

PRAX 100

Alternative Names: PRAX-100

Latest Information Update: 31 Mar 2025

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At a glance

Originator Praxis Precision Medicines
Developer Florey Institute of Neuroscience and Mental Health; Praxis Precision Medicines
Class Antisense oligonucleotides; Behavioural disorder therapies
Mechanism of Action Gene silencing; SCN2A protein expression inhibitors

Orphan Drug Status

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

No
New Molecular Entity Yes

02 Oct 2023 Preclinical trials in Pervasive child development disorders in USA (unspecified route) before October 2023
17 Mar 2021 Praxis Precision Medicines enters into a research collaboration with The Florey Institute of Neuroscience and Mental Health to develop antisense oligonucleotides

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