Atsena begins study on injection for X-linked retinoschisis
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Atsena Therapeutics has dosed the first patient in its open-label, phase 1/2 LIGHTHOUSE study to evaluate the subretinal ATSN-201 injection for X-linked retinoschisis, according to a company press release.
“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 of ophthalmology at Oregon Health & Science University School of Medicine, said in the release.
X-linked retinoschisis (XLRS) is caused by mutations in the RS1 gene, found on the extracellular surface of rods, cones and bipolar cells, according to the release. The investigational ATSN-201 injection uses AAV.SPR, one of the company’s novel spreading capsids, to safely deliver RS1 to photoreceptors in the central retina while avoiding the risk for foveal detachment associated with surgery.
The dose-escalation, dose-expansion trial will examine the safety and tolerability of ATSN-201 in male patients aged 6 to 64 years who have been diagnosed with XLRS as a result of mutations in RS1.
“Dosing the first patient in the LIGHTHOUSE study marks a significant milestone for Atsena and the XLRS community,” Kenji Fujita, MD, Atsena’s chief medical officer, said in the 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.”
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