CLN7 gene therapy

CLN7 gene therapy is an investigational **AAV9-based gene therapy** for **neuronal ceroid lipofuscinosis type 7** caused by pathogenic variants in **MFSD8**. The therapy uses an adeno-associated virus serotype 9 vector to deliver a functional human **MFSD8** transgene, reportedly under a **JeT promoter**, with the goal of restoring MFSD8 protein expression in the central nervous system after **intrathecal** administration. It originated from work at **UT Southwestern** and was clinically evaluated in a first-in-human Phase 1 study in pediatric patients with CLN7 disease; development rights to a next-generation version were later optioned by **Taysha Gene Therapies** and subsequently became associated with **Neurogene** following acquisition-related pipeline changes.

Other names

AAV9 MFSD8 gene therapyAAV-9 MFSD8 gene therapyAAV 9 MFSD8 gene therapyintrathecal AAV9 MFSD8 gene therapy