Flush with $8M, Durham’s Atsena acquires gene therapy for eye disease

DURHAM — Fresh off an $8 million raise, Atsena Therapeutics, a clinical-stage gene therapy company, has acquired exclusive rights to a gene therapy targeting blindness in children.

The therapy, acquired from French pharmaceutical company Sanofi, targets GUCY2D-associated Leber congenital amaurosis (LCA1), a genetic eye disease that affects the retina and is a leading cause of blindness in children.

Atsena founder and chief scientific officer Shannon Boye, Ph.D., and founder and chief technology officer Sanford Boye created the therapy at the University of Florida, from which Sanofi originally licensed it.

“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 Boye, in a statement. “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.”

LCA is the most common cause of blindness in children, impacting two to three per 100,000. 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.

Headquartered in RTP, Atsena has an ongoing Phase I/II clinical trial evaluating this gene therapy in LCA1 patients. The second cohort in the trial is expected to be dosed in the fall of 2020.

Atsena closed a Series 1 funding of $8.15 million this April, led by founding investors Hatteras Venture Partners and the Foundation Fighting Blindness’ RD Fund with participation by Osage University Partners, PBM Capital and the University of Florida.

Patrick Ritschel, co-founder and former president of gene therapy company StrideBio, serves as Atsena’s Chief Executive Officer.

“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 Ritschel. “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.”