DURHAM, NC, April 11, 2023 – 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 6-month safety and efficacy data from the ongoing Phase I/II clinical trial of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1) will be presented at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting, which is being held April 23-27 in New Orleans, LA.
LCA1 is a monogenic eye disease that disrupts the function of the retina and results in early and severe vision impairment or blindness. According to previously reported interim data from the Phase I/II clinical trial, ATSN-101 has demonstrated clinically meaningful improvements in vision with no drug-related serious adverse events.
Details of the oral presentation are as follows:
Title: Six-month safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1)
Presentation Number: 1914
Session: Advances in gene therapy and gene editing for ocular diseases 1
Date / Time: Monday, April 24, 3:45 – 4:00 p.m. CT
Location: Ernest N. Morial Convention Center, R07-R08
Presenter: Christine Nichols Kay, MD, Atsena Therapeutics
In addition, Atsena is pleased to sponsor and participate in the Retinal Cell and Gene Therapy Innovation Summit 2023, jointly organized by the Foundation Fighting Blindness and the Oregon Health & Science University (OHSU) Casey Eye Institute, which is being held on Friday, April 21 in New Orleans, LA.
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 ATSN-101 for a form of LCA, one of the most common causes of blindness in children. Positive interim data presented at the American Academy of Ophthalmology 2022 Annual Meeting demonstrated that subretinal delivery of ATSN-101 was well tolerated and patients treated with the highest dose saw clinically meaningful improvements in vision. The company’s 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.