RARE Daily

Atsena Therapeutics Raises $55 Million to Advance LCA1 Gene Therapy

December 16, 2020

Rare Daily Staff

Atsena Therapeutics closed an oversubscribed $55 million series A financing that will be used to advance its clinical stage pipeline of gene therapies to treat inherited forms of blindness.

Sofinnova Investments led the financing with participation from new investors Abingworth and Lightstone Ventures, founding investors Hatteras Venture Partners and the Foundation Fighting Blindness’s RD Fund, and existing investors Osage University Partners, University of Florida, and the Manning Family Foundation.

Proceeds will be used to advance Atsena’s ongoing phase 1/2 clinical trial evaluating a gene therapy for patients with GUCY2D-associated Leber congenital amaurosis (LCA1), as well as complete manufacturing development for a phase 3 trial.

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.

The phase 1/2 LCA1 clinical trial is currently enrolling patients in the second dosing cohort. Atsena exclusively licensed the rights to the gene therapy from Sanofi, which originally licensed it from University of Florida.

The funds will also enable Atsena to expand its team to support the research and development of novel gene therapies, including the progression of two existing preclinical programs in inherited retinal diseases toward the clinic, and to advance its AAV technology platform.

“We are grateful for the support of our new and existing investors and are encouraged by their enthusiasm for the potential of our technology to overcome the unique hurdles of inherited retinal diseases to prevent or reverse blindness,” said Patrick Ritschel, CEO of Atsena. “The series A financing provides financial runway to reach the key inflection point of reading out efficacy data from our LCA1 clinical trial.”

Photo: Patrick Ritschel, CEO of Atsena

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