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 it has closed an oversubscribed $55 million Series A financing led by Sofinnova Investments with participation from additional new investors Abingworth and Lightstone Ventures. Founding investors Hatteras Venture Partners and the Foundation Fighting Blindness’ RD Fund, along with existing investors Osage University Partners, University of Florida, and Manning Family Foundation, also participated in the round. Sarah Bhagat, PhD, Partner at Sofinnova, Jackie Grant, PhD, Principal at Abingworth, and Jason Lettmann, General Partner at Lightstone, will join Atsena’s board of directors.

Proceeds will be used to advance Atsena’s ongoing Phase I/II clinical trial evaluating a gene therapy for patients with GUCY2D-associated Leber congenital amaurosis (LCA1), one of the most common causes of blindness in children, as well as complete manufacturing development for Phase 3. In addition, the funds will 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 advancement of the company’s innovative adeno-associated virus (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, MBA, Chief Executive Officer 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. While we continue expeditiously advancing this trial and our preclinical programs, we are excited to accelerate our growth as a leading ophthalmic gene therapy company.”

The Phase I/II 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. 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.

“We believe Atsena’s foundation in ocular gene therapy and potentially game-changing novel AAV vectors position the company to become a partner of choice,” said Dr. Bhagat. “Sofinnova is delighted to support Atsena and we look forward to helping the team further its mission to develop life-changing gene therapies for patients with inherited retinal diseases.”

Source: Atsena Therapeutics Raises $55 Million Series A Financing to Advance LCA1 Gene Therapy Clinical Program, Two Preclinical Assets, and Novel Capsid Development for Ocular Diseases