The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to a novel one-time AAV9 gene therapy for CLN1 disease. The gene therapy is designed to deliver a functional copy of the PPT1 gene to the central nervous system and peripheral organs using a combination of intravenous and intrathecal administrations.
CLN1 disease, also known as Infantile Neuronal Ceroid Lipofuscinosis or infantile Batten disease, is a rapidly-progressing rare lysosomal storage disease with no approved treatment. It primarily affects the central nervous system and typically manifests during the first year of life with vision impairment that progresses to blindness, motor and cognitive decline, seizures and ultimately early death. The underlying cause of the disorder is mutations in the PPT1 gene that encodes the enzyme of the same name, resulting in lysosome dysfunction that leads to cellular dysfunction, neuroinflammation and neurodegeneration.
“Receiving Fast Track designation acknowledges the urgency for developing a therapy for children suffering from this rapidly-progressing and fatal disease and highlights the significant potential of ABO-202 to address this unmet need,” said João Siffert, M.D., Chief Executive Officer of Abeona Therapeutics, the biopharmaceutical company developing the ABO-202 program.
ABO-202 is administered as a one-time adeno-associated virus 9 (AAV9) gene therapy that delivers a functional copy of the PPT1 gene to cells of the central nervous system and peripheral organs. This enables cells to produce a functioning PPT1 enzyme, which is critical for proper metabolism in lysosomes. The absence of this enzyme in patients with CLN1 disease results in malfunctioning cells, including brain cells, neuroinflammation, and neurodegeneration. In preclinical studies, ABO-202 normalized survival and improved neurological function in CLN1 mice. These studies also showed that a combination of intravenous and intrathecal administrations of ABO-202 improved efficacy over either delivery route alone, and that early treatment significantly improved outcomes.
Fast Track designation, granted by the FDA, is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier through more frequent interactions with FDA, potential eligibility for accelerated approval, priority regulatory review, and rolling BLA review. ABO-202 has also received Orphan Drug designations in the U.S. and EU, and Rare Pediatric Disease designation from the FDA.