CLN-301 in Juvenile Batten Disease (CLN3)

CLN-301 is Alcyone’s innovative adeno-associated viral vector gene therapy program for CLN3 Batten disease, also known as Juvenile Neuronal Lipofuscinosis (JNCL), an inherited neurodegenerative disorder affecting approximately 2 to 4 out of every 100,000 children worldwide. CLN3 Batten disease is marked by progressive loss of motor skills, vision, cognitive abilities, and seizure occurrence, ultimately resulting in premature death. Symptoms often begin in early childhood and progressively impair quality of life as they advance.

CLN-301 targets the root cause of this disorder—a mutation in the CLN3 gene—that leads to lysosomal dysfunction and cellular toxicity in affected cells, especially in the nervous system. This one-time gene therapy is designed to deliver a functional CLN3 coding sequence to the cerebrospinal fluid (CSF), allowing it to reach the central nervous system more directly. This replacement CLN3 gene allows the functional protein to be made, restoring the deficit in lysosomal function, preventing toxic buildup within neurons and other cells, supporting neuronal health, and stabilizing neurodevelopment.