CLN3

Gene Therapy for Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) or CLN3 Batten Disease

Alcyone’s adeno-associated viral vector gene therapy CLN-301 is designed to address the underlying genetic cause of CLN3 Batten disease by delivering a functional copy of the CLN3 coding sequence to patients via a one-time, lumbar intrathecal injection. This approach aims to restore CLN3 protein function, preventing cellular buildup of toxic waste products that damage neurons. By restoring these molecular mechanisms, CLN3 gene therapy addresses the disease at its root and holds the potential to ameliorate or stabilize disease progression significantly.