ATSN-101 has demonstrated clinically meaningful improvements in vision with no drug-related serious adverse events
DURHAM, NC, February 8, 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 positive interim data from the ongoing Phase I/II clinical trial of ATSN-101 will be presented at the 46th Annual Macula Society Meeting, which is being held February 15-18 in Miami, FL. This is an encore presentation of data that were presented as a late-breaker at the American Academy of Ophthalmology 2022 Annual Meeting.
ATSN-101, Atsena’s lead investigational gene therapy product, 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.
“We are pleased to have the opportunity to share the positive interim safety and efficacy data from our ongoing Phase I/II trial of ATSN-101 with the retinal disease community at the upcoming Macula Society Annual Meeting,” said Kenji Fujita, MD, Chief Medical Officer of Atsena Therapeutics. “Subretinal delivery of ATSN-101 was well tolerated and demonstrated clinically meaningful improvements in vision in patients treated with the highest dose. With no approved treatments available for patients with LCA1, we are honored to be at the forefront of research for this inherited retinal disease and look forward to reporting additional data from our Phase I/II trial at medical meetings later this year.”
Details of the presentation are as follows:
Title: Safety and Efficacy of ATSN-101 in Patients with Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D (LCA1)
Section: Session III – Inherited Retinal Disorders
Date / Time: Wednesday, February 15, 6:16 p.m. EST
Location: Fontainebleau Miami Beach
Presenter: Christine Nichols Kay, MD, Atsena Therapeutics
About GUCY2D-associated Leber congenital amaurosis (LCA1)
LCA1 is a monogenic eye disease that disrupts the function of the retina. It 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 group of inherited retinal diseases. There are currently no approved treatments for LCA1.
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.
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