NGN-101

NGN-101 is an investigational gene therapy developed for the treatment of CLN5 Batten disease, a rare and fatal neurodegenerative disorder primarily affecting children. The therapy utilizes an adeno-associated virus serotype 9 (AAV9) vector to deliver a codon-optimized human CLN5 transgene, aiming to restore functional CLN5 protein in patients with mutations in the endogenous gene. The loss of functional CLN5 leads to accumulation of cellular waste products, resulting in progressive neurodegeneration, vision loss, motor and cognitive decline, seizures, and premature death. NGN-101 was designed as a one-time treatment administered via intracerebroventricular (ICV) or intravitreal (IVT) injection. Preclinical data suggested potential to halt or slow disease progression across neurological and ocular symptoms[1][2][4][5][8]. As of late 2024, Neurogene discontinued further development after failing to secure FDA alignment on a streamlined registrational pathway due to the rarity of the condition[9].

Brand names

NGN-101NGN101NGN 101

Other names

AAV9-hCLN5AAV-9-hCLN5AAV 9-hCLN5CLN5 gene therapyCLN-5 gene therapyCLN 5 gene therapy