Neurogene to Launch Gene Therapy Clinical Trial for Children with Batten Disease

Neurogene, a company developing genetic therapies for rare neurological diseases, has received clearance from the US Food & Drug Administration (FDA), to launch a Phase 1/2 clinical trial for a gene therapy for Batten disease caused by mutations in the CLN5 gene. The company expects to start the trial during the first half of 2022.

Batten disease is a group of genetic, neurological conditions that usually cause a variety of challenging symptoms, including seizures, declines in movement and cognition, and vision loss due to retinal degeneration. Batten disease is usually diagnosed in children and leads to reduced life expectancy. The condition is caused by the inability of cells to break down waste. Lysosomes, which dispose of and recycle waste in cells, don’t function properly in Batten disease, leading to the accumulation of waste products called lipofuscins. About 14 genes, when mutated, are associated with Batten disease.

Neurogene’s CLN5 gene therapy trial is for children between the ages of 3 and 8. Participants will receive a single injection of the gene therapy to the eye (intravitreal delivery) and a single injection into the ventricles of the brain (intracerebroventricular delivery). The treatment uses a human-engineered adeno-associated virus (AAV) to penetrate cells with healthy copies of CLN5 to augment the mutated copies.

There are no FDA-approved therapies for Batten disease caused by CLN5 mutations.