Atsena Initiates Dosing in Phase I/II XLRS Gene Therapy Trial's Second Cohort

In Durham, N.C., Atsena Therapeutics, a gene therapy company focused on reversing or preventing blindness, announced that the second group in their LIGHTHOUSE study has started receiving doses of ATSN-201. This Phase I/II trial is testing subretinal injections of ATSN-201 to treat X-linked retinoschisis (XLRS), using the novel spreading capsid AAV.SPR.

The first cohort of the study showed that ATSN-201 was well received by all patients without any serious adverse events, leading the Data and Safety Monitoring Board to approve moving forward with the next dosage level. Dr. Kenji Fujita, the Chief Medical Officer, mentioned that the initial low dose demonstrated excellent safety and potential structural and functional improvements in the retina, indicating the effective spread of the vector.

The LIGHTHOUSE study is designed to evaluate the safety and tolerability of ATSN-201 in males aged 6 and older, who have a clinical diagnosis of XLRS caused by mutations in the RS1 gene. The trial continues to enroll patients.

XLRS is caused by mutations in the RS1 gene, impacting visual acuity and leading to progressive vision loss and potential blindness. This disease primarily affects males and is commonly diagnosed in early childhood, with approximately 30,000 cases in the U.S. and EU.

AAV.SPR is a capsid that spreads beyond the subretinal injection site, allowing safe and effective gene transduction in the retina without surgical detachment. Preclinical studies in primates have shown that AAV.SPR enables extensive transgene expression beyond the injection area, which is a significant improvement over traditional AAV vectors.

Atsena Therapeutics also has ongoing trials evaluating ATSN-101 for Leber congenital amaurosis type 1 (LCA1). The company's pipeline is built on adeno-associated virus technology tailored for inherited retinal diseases, with a team dedicated to developing gene therapies for vision loss.

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