FDA accepts Abeona IND for CLN1 disease gene therapy


The FDA has accepted Abeona Therapeutics (NASDAQ:ABEO) IND application for AB-202 for the treatment of CLN1 disease.

CLN1 disease, also known as infantile Batten disease, is caused by a mutation in the PPT1 gene. In CLN1, lipopigments accumulate in cells of the central nervous system, causing progressive neurological decline and death.

ABO-202 is a one-time gene therapy that delivers a functional copy of the PPT1 gene to central nervous system cells and peripheral organs, restoring proper lysosomal function.

“ABO-202 is a promising gene therapy that extended survival and improved neurological function in the animal model of CLN1 disease,” Dr. Steven Gray, associate pediatrics professor, at UT Southwestern Medical Center, said in a statement.

“Importantly, the combined intravenous and intrathecal administration approach showed additional benefits compared to a single route of delivery, providing a new treatment paradigm for patients with devastating neurological diseases,” he added.

ABO-202 has been granted FDA orphan drug and rare pediatric disease designation and orphan medicinal product designation in the EU. Abeona will provide guidance on the timing of the clinical trial later in 2019.

Michelle Carr