ATSN-201 exploits a novel diffusing capsid, AAV.SPR, to overcome the challenges associated with intravitreally delivered AAV in the treatment of XLRS. (Image courtesy of Adobe Stock)
Atsena Therapeutics today announced that the FDA has approved its Investigational New Drug (IND) application for a Phase I/II clinical trial of ATSN-201 in patients with X-linked retinoschisis (XLRS).
According to the company, ATSN-201 leverages a novel diffusing capsid, AAV.SPR, to overcome challenges associated with intravitreally-delivered AAV in the treatment of XLRS.
In a news release, Dr. Shannon Boye, founder and director of Atsena Therapeutics, noted the limitations of AAV delivered intravitreally. This is because these AAVs do not promote sufficient gene expression in photoreceptors to confer therapy and can cause vision-impairing inflammation.
“AAV.SPR is suitable for use in XLRS because it can promote therapeutic levels of photoreceptor gene expression while avoiding the surgical risk of foveal detachment. is important because the retina is fragile due to the presence of schizofoci,” Boye said. “Based on decades of research, he is excited to advance new gene therapies for patients with XLRS, who currently have no approved treatment options.”
Kenji Fujita, Chief Medical Officer of Atsena Therapeutics, noted that with the approval, the company is preparing clinics for the first program utilizing AAV.SPR for the treatment of XLRS in mid-2023.
“We look forward to evaluating ATSN-201 to address the unmet need for treatments to improve or restore vision in patients with XLRS,” said Fujita.
In the Lighthouse Study, a phase I/II, open-label, dose-escalating clinical trial, in male patients aged 6–65 years with a clinical diagnosis of XLRS caused by pathogenic or probable pathogenic mutations in RS1, Evaluate subretinal injection of ATSN-201. .
