FDA Clears IND for Neurogene’s Gene Therapy for Batten Disease

FDA recently cleared Neurogene’s Investigational New Drug (IND) application for its CLN5 Batten disease gene therapy, NGN-101. 

NGN-101 is an adeno-associated virus (AAV) gene therapy and Neurogene’s lead product candidate in its Batten disease franchise. 

“We believe our preclinical data strongly support the potential of NGN-101 to stop the progression of CLN5 Batten disease, including the associated vision, motor, cognitive, and behavioral declines,” Rachel McMinn, PhD, founder and CEO of Neurogene, said in the announcement.  

“FDA clearance of our first IND is an incredibly significant milestone for Neurogene, and moves us one step closer to delivering on our mission to bring transformative treatments to patients living with rare neurologic diseases,” McMinn continued. 

CLN5 disease is one of a group of disorders known as neuronal ceroid lipofuscinoses (NCLs), collectively referred to as Batten disease. Batten disease is a rare and fatal neurodegenerative disorder with no approved disease-modifying therapies available.

With the IND clearance, Neurogene can initiate a Phase 1/2 trial to assess the safety, tolerability, and efficacy of NGN-101 in patients with Batten disease. 

The open-label trial will evaluate a single dose of NGN-101 administered through intracerebroventricular and intravitreal routes. 

The University of Rochester will be the lead trial site in the US. Leading the study will be Jonathan W. Mink, MD, PhD, the Frederick A. Horner MD professor in pediatric neurology, chief of child neurology, and director of the University of Rochester Batten Center.

Mink stated that the trial will move research forward in developing a potentially disease-modifying treatment for Batten disease, which will provide hope to individuals and families “where currently none exists.” 
In April 2017, FDA approved Brineura to treat a specific form of Batten disease. Brineura was the first FDA-approved treatment to slow loss of walking ability in symptomatic patients three years of age and older with late infantile neuronal ceroid lipofuscinosis type 2.