Atsena Therapeutics is a biotechnology startup focusing on clinical-stage ocular gene therapy for treating inherited forms of blindness. The company's flagship programs include ATSN-201 for X-linked retinoschisis (XLRS) and ATSN-101 for GUCY2D-associated Leber congenital amaurosis (LCA1). Notably, the company's innovative spreading capsid AAV.SPR is integral to the ATSN-201 program, currently undergoing a Phase I/II clinical trial known as the LIGHTHOUSE study.
Additionally, ATSN-301, Atsena’s dual AAV vector-based gene therapy for preventing blindness caused by MYO7A-associated Usher syndrome (USH1B), stands as another promising proprietary asset. The company's Phase I/II clinical trial for patients with LCA1 has provided encouraging interim safety and efficacy data for ATSN-101, demonstrating tolerability and significant vision improvements observed 12 months post-treatment.
Atsena Therapeutics was founded in 2019 by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye from the University of Florida. Headquartered in North Carolina’s Research Triangle, an area renowned for gene therapy expertise, Atsena received a significant $24.47M Venture Round investment on 02 October 2023, affirming investor confidence in the company’s innovative approach and its potential to address unmet needs in inherited blindness treatments.
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