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Atsena Gets FDA Clearance to Start Phase I/II Trial of X-linked Retinoschisis Gene Therapy

NEW YORK – The US Food and Drug Administration has given Atsena Therapeutics permission to study its gene therapy ATSN-201 in patients with X-linked retinoschisis (XLRS) in a Phase I/II trial, the company said Monday.

After getting FDA clearance for its investigational new drug application for ATSN-201, Durham, North Carolina-based Atsena is gearing up to launch the Lighthouse Study by mid-year. In the Phase I/II open-label trial, the firm will enroll male XLRS patients between 6 and 65 years old and evaluate the activity of the gene therapy at various doses.

XLRS is a rare ocular disorder caused by mutations in RS1, a gene that encodes a protein secreted by photoreceptors called retinoschisin. As a monogenic X-linked disease, XLRS tends to occur largely in males, who as a result suffer from progressive vision loss. There are currently no treatments for XLRS.

According to Atsena, ATSN-201 will use AAV.SPR, its novel adeno-associated virus capsid that spreads laterally beyond the subretinal injection site to deliver a normal copy of RS1 to photoreceptors in the central retina. The Phase I/II trial of ATSN-201 will be the first evaluation of the AAV.SPR capsid, which Atsena has designed to overcome the inability of intravitreally delivered AAVs to drive therapeutically sufficient gene expression in photoreceptors. 

"AAV.SPR is well-suited for use in XLRS as it can drive therapeutic levels of gene expression in photoreceptors while avoiding the surgical risks of foveal detachment, which is important because XLRS patients have fragile retinas due to the presence of schisis lesions," Atsena Founder and Director Shannon Boye said in a statement. "Building on decades of research, we're excited to progress our novel gene therapy for patients with XLRS who currently lack an approved treatment option."