Drug Profile

CLN8 gene therapy - Amicus Therapeutics

Latest Information Update: 05 May 2026

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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 No

27 Apr 2026 Amicus Therapeutics has been acquired by BioMarin Pharmaceutical
28 Oct 2022 No recent reports of development identified for preclinical development in Neuronal ceroid lipofuscinosis in USA
20 Sep 2018 Celenex has been acquired and merged into Amicus Therapeutics

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