Atsena Therapeutics Announces First Patient Dosed in Phase 1/2 Clinical Trial of ATSN-201 for the Treatment of X-linked Retinoschisis
Atsena Therapeutics announced the first patient has been dosed in the LIGHTHOUSE study, a phase 1/2 clinical trial evaluating subretinal injection of ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201 uses AAV.SPR, the company’s novel spreading capsid, to achieve therapeutic levels of gene expression in photoreceptors of the central retina while avoiding the surgical risks of foveal detachment.
“Dosing the first patient in the LIGHTHOUSE study marks a significant milestone for Atsena and the XLRS community,” Kenji Fujita, MD, Chief Medical Officer of Atsena Therapeutics, said in a company news release. “We are excited to be utilizing AAV.SPR in the clinic, as it has the potential to revolutionize the treatment of XLRS, as well as other inherited retinal disorders. Spreading laterally beyond the subretinal injection site, AAV.SPR facilitates the safe delivery of RS1 to photoreceptors in the central retina/fovea. We look forward to advancing the LIGHTHOUSE study and the continued development of our novel gene therapies to reverse or prevent blindness.”
The LIGHTHOUSE study is a phase 1/2, open-label, dose-escalation and dose-expansion clinical trial evaluating the safety and tolerability of ATSN-201 in male patients ages 6-64 with a clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1. For more information, visit ClinicalTrials.gov (Identifier: NCT05878860).
“Considering the lack of available therapies for X-linked retinoschisis, this is very exciting news for the inherited retinal disease community,” Mark Pennesi, MD, PhD, Professor in Ophthalmology and Chief of the Paul H. Casey Ophthalmic Genetics Division Molecular and Medical Genetics, School of Medicine at Oregon Health & Science University (OHSU), said in a news release. “While attempts to deliver gene therapy through intravitreal routes faced challenges, subretinal treatment utilizing spreading AAV vectors has the potential to be the breakthrough we need to achieve efficacy.”
XLRS is a monogenic X-linked disease caused by mutations in the RS1 gene which encodes retinoschisin, a protein secreted primarily by photoreceptors. RS1 is localized to the extracellular surface of rods, cones, and bipolar cells. XLRS is characterized by schisis, or abnormal splitting of the layers of the retina, which causes impaired visual acuity that is not correctable with glasses and leads to progressive vision loss. XLRS primarily affects males and is typically diagnosed in early childhood. Approximately 30,000 males in the U.S. and EU have XLRS, for which there are currently no approved treatments.