Atsena Therapeutics Receives FDA Designation for Treatment of Severe Congenital Eye Disease Using Gene Therapy

Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced the U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy designation to ATSN-101, the company’s lead investigational gene therapy for patients with Leber congenital amaurosis.

The company was created by ocular gene therapy pioneers Dr. Shannon Boye and Associate Scientist Sanford Boye, who also run the Shannon E. Boye Laboratory in the UF College of Medicine’s Department of Pediatrics. The lab creates technology aimed at restoring retinal function and visually-guided behavior, as well as preserving retinal structure during early-onset retinal dystrophy. The goal is to take this technology from the lab and apply it in a patient setting, restoring the missing gene and combatting vision loss. After successful tests on mice, this approval from the FDA will help the project move closer to human trials.

Outside the lab, Dr. Shannon Boye was recently named “Innovator of the Year” at UF Innovate’s annual Standing InnOvation event for her groundbreaking work in the fields of ophthalmology and gene therapy.

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