Atsena Therapeutics Receives Orphan Drug Designation from the FDA for Novel Gene Therapy to Treat Genetic Eye Disease LCA1Phase I/II clinical trial is ongoing in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D

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 that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for its investigational gene therapy product for the treatment of GUCY2D-associated Leber congenital amaurosis (LCA1), a genetic eye disease that affects the retina. The safety and efficacy of the gene therapy are being evaluated in a Phase I/II clinical trial, which is currently enrolling patients (ClinicalTrials.gov Identifier: NCT03920007).

“Receiving orphan drug designation from the FDA is an important milestone for our LCA1 gene therapy clinical program,” said Kenji Fujita, MD, Chief Medical Officer of Atsena. “We look forward to the continued progression of our Phase I/II clinical trial as we seek to develop a new treatment for children and adults who have severe visual impairment or blindness due to GUCY2D-associated LCA1.”

The FDA may grant orphan drug designation to drugs and biologics intended to treat diseases or conditions that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain benefits, such as tax credits for qualified clinical testing, exemptions from certain FDA application fees, and seven years of market exclusivity, if approved.

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