North Carolina-based Atsena Therapeutics has secured $150 million in Series C funding to progress its two lead eye gene therapy candidates to the clinic.
The company has a candidate therapy, ATSN-201, for X-linked retinoschisis (XLRS), a rare X-linked retinal disease that usually begins in childhood and later leads to blindness, that is currently in a Phase I/II trial.
The company also has a candidate therapy, ATSN-101, for another rare inherited eye condition, Leber congenital amaurosis type 1 caused by mutations in the GUCY2D gene, which recently completed a Phase I/II trial with good results. It is currently working with a Japanese pharma partner, Nippon Shinyaku, to progress this treatment further toward the clinic.
The fundraising round was led by Bain Capital and included contributions from a new investor, Wellington Management, and existing investors: Lightstone Ventures, Sofinnova Investments, Abingworth, Foundation Fighting Blindness, Hatteras Venture Partners, Osage University Partners, and the Manning Family Foundation.
“Closing our Series C marks a pivotal moment for Atsena as we advance our transformative ocular gene therapies and fuel our next phase of growth, innovation, and clinical progress,” said Patrick Ritschel, CEO of Atsena Therapeutics.
“It follows a productive 12 months of key achievements including securing a partner to advance ATSN-101 to a global pivotal trial for Leber Congenital Amaurosis type 1 and initiating Part B of the ATSN-201 LIGHTHOUSE study for XLRS.”
Atsena uses a novel type of adeno-associated virus (AAV) vector that has a lateral spreading mechanism. This allows the treatment to spread beyond the site of injection into fragile areas of the eye that are affected by the diseases being treated by Atsena. Preclinical studies in nonhuman primates showed the therapy spread 12 times further than with conventional AAV vectors.
The company plans to use the Series C funding to progress its main lead candidate ASTN-201 further. It already has Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA, showing the potential importance of this treatment in a condition with no approved therapies.
