- Data from Phase I/II trial of ATSN-101 in patients with GUCY2D-associated Leber congenital amaurosis (LCA1) to be presented
DURHAM, NC – 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 that data from the Phase I/II clinical trial of ATSN-101 will be presented in a Late Breaking Developments session during the Retina Subspecialty Day at the American Academy of Ophthalmology Annual Meeting (AAO 2022), which is being held September 30 – October 3, 2022 in Chicago.
ATSN-101, Atsena’s lead investigational gene therapy product formerly known as SAR439483, is being evaluated in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). LCA1 is a monogenic eye disease that disrupts the function of the retina and results in early and severe vision impairment or blindness.
Details of the presentation are as follows:
Title: Safety and Efficacy of SAR439483 in Patients with Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D (LCA1)
Abstract Number: 30071742
Section: Section X: Late Breaking Developments, Part II
Date / Time: Saturday, October 1, 9:15 a.m. CST
Location: McCormick Place – Arie Crown
Presenter: Christine Nichols Kay, MD, Atsena Therapeutics
About Atsena Therapeutics
Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company’s ongoing Phase I/II clinical trial is evaluating a potential therapy for LCA1, one of the most common causes of blindness in children. Its additional pipeline of leading preclinical assets is powered by an adeno-associated virus (AAV) technology platform tailored to overcome significant hurdles presented by inherited retinal disease, and its unique approach is guided by the specific needs of each patient condition to optimize treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com.
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