Drug Profile

Research programme: CNS disorders therapeutics - CAMP4 Therapeutics

Latest Information Update: 28 Jul 2025

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At a glance

Originator CAMP4 Therapeutics
Class Antisense oligonucleotides; Neuroprotectants
Mechanism of Action NAV1.1 voltage-gated sodium channel expression stimulants; Progranulin protein stimulants; 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
Available For Licensing Yes

28 Jul 2025 No recent reports of development identified for preclinical development in Dravet-syndrome in USA (Parenteral)
28 Jul 2025 No recent reports of development identified for preclinical development in Frontotemporal dementia in USA (Parenteral)
20 Jul 2022 CAMP4 Therapeutics announces intention to submit IND application for Dravet syndrome in mid-2023

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