The US Food and Drug Administration (FDA) has cleared Abeona Therapeutics to begin a Phase 1/2 clinical trial evaluating its novel, one-time gene therapy ABO-202 for the treatment of CLN1 disease. ABO-202 is designed to deliver a functional copy of the PPT1 gene to cells of the central nervous system and peripheral organs using a combined intravenous and intrathecal delivery via the AAV9 vector. 

In a company press release, Timothy J. Miller, PhD, Co-Founder, President & Chief Scientific Officer of Abeona said, “This significant step brings hope to people impacted by this devastating disease and was achieved in partnership with Dr. Steven Gray and Taylor’s Tale. We are very encouraged that ABO-202 was well-tolerated and demonstrated significant efficacy in preclinical studies. These results are consistent with findings in our other pre-clinical studies for AAV9-based programs for lysosomal storages diseases, MPS IIIA and MPS IIIB.”

In preclinical studies, ABO-202 effectively delivered a functional copy of the PPT1 gene to the central nervous system and peripheral organs. IND-enabling studies in a CLN1 animal model demonstrated that ABO-202 normalized survival and led to improvement of neurological function in affected mice. These studies also showed that combination intravenous and intrathecal dosing provided incremental efficacy over either delivery route alone and thus may enhance the therapeutic potential of this gene therapy.

ABO-202 is a one-time AAV gene therapy designed to enable cells to produce the normal PPT1 enzyme, which is critical for proper lysosomal function. Lack of this enzyme in patients with CLN1 disease results in neuroinflammation and neurodegeneration. ABO-202 has been granted Orphan Drug and Rare Pediatric Disease designations by the FDA and has received Orphan Medicinal Product designation in the EU. The global trial will be conducted at centers of excellence in CLN1 research, including the University of Rochester Medical Center in the U.S. and the University of Hamburg-Eppendorf in Germany.

“ABO-202 is a promising AAV9 gene therapy that extended survival and improved neurological function in the animal model of CLN1 disease. 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,” said Steven J. Gray, Ph.D., Batten disease researcher and Associate Professor, Pediatrics, UT Southwestern Medical Center.