Drug Profile

Research programme: antisense oligonucleotide therapeutics - Praxis Precision Medicines

Latest Information Update: 28 Apr 2025

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At a glance

Originator Praxis Precision Medicines
Class Antiepileptic drugs; Antisense oligonucleotides; Behavioural disorder therapies
Mechanism of Action RNA interference

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

28 Apr 2025 No recent reports of development identified for research development in Autistic-disorder in USA
28 Apr 2025 No recent reports of development identified for research development in Epilepsy in USA
31 Dec 2021 Praxis Precision Medicine and Cerebral Therapeutics signs research collaboration for antisense oligonucleotides in December 2021

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