Abeona Gets FDA Fast Track Designation for ABO-202 AAV9 Gene Therapy

Abeona Therapeutics Inc. (ABEO), a fully-integrated leader in gene and cell therapy, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its ABO-202 program. ABO-202, the Company’s novel one-time AAV9 gene therapy for CLN1 disease, 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. Abeona is preparing to initiate a Phase 1/2 clinical trial evaluating ABO-202 in patients with CLN1 disease and will provide guidance on the timing of the trial later this year.

“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.

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.

https://finance.yahoo.com/news/abeona-therapeutics-receives-fda-fast-122900142.html