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Durham biotech uses new funding to acquire gene therapy for eye disease

A Hatteras-backed, early-stage life science company has used its new funding to acquire the exclusive rights to gene therapy platform targeting eye disease.

On Wednesday, Durham-based Atsena Therapeutics announced that it has acquired the exclusive rights to a gene therapy from French pharma giant Sanofi. The therapy targets GUCY2D-associated Leber congenital amaurosis (LCA1) – a genetic eye disease that affects the retina and is a leading cause of blindness in children.

The acquisition follows a $8.1 million funding round Atsena closed earlier this year. The round was led by Durham-based Hatteras Venture Partners. In addition to Hatteras, the company’s investors include the Foundation Fighting Blindness’ RD Fund, Osage University Partners, PBM Capital and the University of Florida.

Sanofi originally licensed the technology from the University of Florida. It was first created in the laboratory of the Atsena’s founders Dr. Shannon Boye and Sanford Boye.

“We are thrilled that our gene therapy for LCA1 is coming home to Atsena and that we will have the opportunity to further its development,” said Sanford Boye, who serves as the company’s chief technology officer. “Atsena was founded to advance treatments for inherited retinal diseases and believes in centering patients’ perspectives and needs in all we do. We are honored to continue to work with LCA1 patients and their families as we strive to treat this debilitating disease.”

According to the company, LCA is the most common cause of blindness in children, impacting about 2 to 3 children per 100,000. The condition is caused by a generic mutation that results in “early and severe vision impairment or blindness.” The form targeted by the company affects roughly 20 percent of all patients with the retinal disease.

The company has an ongoing Phase 1/2 clinical trial evaluating the therapy in patients. it expects the second cohort of patients in the trial to be dosed in the fall.

“Atsena is pleased to have the support of an enthusiastic investor base that shares our dedication to bringing the life-changing power of genetic medicine to patients living with LCA1 and other forms of blindness,” said Patrick Ritschel, the company’s CEO and the former co-founder of StrideBio. “We look forward to working closely with our investors and patients as we continue to grow, and expect to announce additional milestones later this year.”

Original Article Source: Triangle Business Journal