Drug Profile

AX 2404

Alternative Names: AX-2404

Latest Information Update: 30 May 2025

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At a glance

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

18 Mar 2025 Early research in Rett syndrome in Netherlands (Parenteral) (ProQR Therapeutics pipeline, March 2025)
13 Mar 2025 ProQR Therapeutics has patent pending for Axiomer® program in multiple countries
13 Mar 2025 ProQR Therapeutics has patent protection for Axiomer® program in Australia, Brazil, Europe,India, Japan, New Zealand, China, Canada, Israel, New Zealand, Japan, Russia, South Africa, South Korea, USA and Europe

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