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Gene therapy receives US FDA approval

Lincoln University researchers have received US FDA approval for in-human clinical trials of their gene therapy for the treatment of CLN5 Batten disease, a fatal neurodegenerative disease affecting children.

The CLN5 form of Batten disease appears early in a child’s life and causes brain degeneration manifesting in devastating symptoms including vision loss, seizures, dementia, abnormal movements and inability to communicate. Sufferers typically die in their teens.

Until now there has been no cure and no hope of treatment, but the Lincoln-developed gene therapy is a potentially transformative treatment for the CLN5 patient community.

Over the past decade, Professor David Palmer and Doctors Nadia Mitchell and Samantha Murray have been developing their gene therapy in sheep with a naturally-occurring form of the disease.

Dr Mitchell explains, “The brains of sheep with Batten disease shrink, as do the brains of humans with the condition.

“When we have replaced the missing gene in affected sheep before they display symptoms, in most cases the disease has been prevented. When we replace the gene after the sheep begin to display symptoms, the therapy has slowed the progress of the disease.”

Sponsored by Neurogene Inc (USA), a company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, the Lincoln team have received US FDA approval for their Investigational New Drug Application, clearing the way for the first in-human clinical trials of their CLN5 gene therapy.

The approved new therapy, known as NGN-101, is a one-off treatment for children with CLN5 Batten disease, whereby a functional CLN5 gene is delivered into the subject’s brain and eye via an adeno-associated virus to address the neurodegeneration and vision loss associated with the disease.

The first clinical trial is expected to initiate at the University of Rochester (New York) in the first half of 2022, under the leadership of Jonathan W Mink MD, Ph.D, the Frederick A Horner MD – Distinguished Professor in Pediatric Neurology, Chief of Child Neurology and Director of the University of Rochester Batten Center.

“CLN5 is a devastating and rapidly progressive neurodegenerative disease in children that leads to vision loss, cognitive and motor impairment, seizures and, ultimately, premature death,” said Dr Mink.

“This trial of NGN-101 will move research forward in developing a potentially disease-modifying treatment for CLN5 disease, providing hope to individuals and families where currently none exists.”

Lincoln University’s Dr Nadia Mitchell agrees: “The FDA approval is life-changing news, not just for our research team, and for the University, but especially for thousands of Batten disease patients globally.

“We’re so excited to see our science progress to the next stage, and to provide some hope for children and families impacted by CLN5 Batten disease.”

Visit for more information on the world-leading research being undertaken at Lincoln University.

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