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# Abeona Therapeutics Gains Fast Track for Gene Therapy
- URL: https://www.fdaweb.com/abeona-therapeutics-gains-fast-track-for-gene-therapy/
- Published: 2019-06-18T12:00:00.000Z
- Updated: 2026-09-15T01:33:27.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5144362

FDA has granted Abeona Therapeutics a fast track designation for ABO-202, a novel one-time AAV9 gene therapy for CLN1 disease. The 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, according to the company. Abeona says it is planning to initiate a Phase 1/2 clinical trial evaluating ABO-202 in patients later this year.

Abeona says that ABO-202 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,” it says. In preclinical studies, ABO-202 reportedly normalized survival and improved neurological function in CLN1 mice. The 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, the company says.