BioCentury
ARTICLE | Clinical News

Amicus’ Batten disease gene therapy shows preserved speech, motor function

August 2, 2019 12:48 AM UTC

Interim data for Amicus’ most advanced Batten disease gene therapy suggest that the biotech’s recent addition of the modality to its pipeline could be paying off.

On Thursday, Amicus reported interim Phase I/II data showing its AAV-CLN6 gene therapy stabilized motor and speech functions in children with neuronal ceroid lipofuscinosis type 6 (CLN6), a form of Batten disease...