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Press Releases

Atsena Therapeutics Receives Orphan Drug Designation from the FDA for Novel Gene Therapy to Treat Genetic Eye Disease LCA1

Phase I/II clinical trial is ongoing in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D

DURHAM, NC – 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 ( 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.

Atsena’s LCA1 program is based on more than 15 years of research conducted at the University of Florida. The company exclusively licensed the rights to the gene therapy from Sanofi, which originally licensed it from University of Florida.

About LCA1

Leber congenital amaurosis (LCA) is the most common cause of blindness in children. LCA1 is caused by mutations in the GUCY2D gene and results in early and severe vision impairment or blindness. GUCY2D-LCA1 is one of the most common forms of LCA, affecting roughly 20 percent of patients who live with this inherited retinal disease.

About Atsena Therapeutics

Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company’s ongoing Phase I/II clinical trial is evaluating a potential therapy for one of the most common causes of blindness in children. Its additional pipeline of leading preclinical assets is powered by an adeno-associated virus (AAV) technology platform tailored to overcome significant hurdles presented by inherited retinal disease, and its unique approach is guided by the specific needs of each patient condition to optimize treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit

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