FOLD - Amicus Therapeutics receives PRIME designation for CLN6 batten disease gene therapy

Amicus Therapeutics (FOLD) announced that the European Medicines Agency has granted priority medicines designation to AT-GTX-501, the Company’s investigational gene therapy for children living with variant late infantile neuronal ceroid lipofuscinosis 6 (vLINCL6) disease, also known as CLN6 Batten disease. The PRIME designation is based on data from the ongoing Phase 1/2 clinical trial evaluating a single dose of AT-GTX-501 for the treatment of children with CLN6 Batten disease.Additional data from the ongoing Phase 1/2 clinical study will be presented at the Child Neurology Society Annual Meeting in October.

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Amicus Therapeutics receives PRIME designation for CLN6 batten disease gene therapy