Atsena Therapeutics Unveils XLRS Gene Therapy Program Leveraging Novel Spreading Capsids (Globe Newswire)

Atsena Therapeutics Unveils XLRS Gene Therapy Program Leveraging Novel Spreading Capsids

UF startup Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, unveiled its preclinical gene therapy program for X-linked retinoschisis (XLRS), a monogenic disease caused by mutations in the RS1 gene. XLRS is characterized by schisis, or abnormal splitting of the layers of the retina, which causes impaired visual acuity that is not correctable and leads to progressive vision loss. XLRS primarily affects males and is typically diagnosed in early childhood.

“We are pleased to disclose our program for XLRS, an inherited retinal disease lacking approved treatments to improve or restore vision,” said Patrick Ritschel, MBA, chief executive officer of Atsena. “Following a recent pre-IND meeting with the U.S. Food and Drug Administration, we are working expeditiously to advance our differentiated gene therapy candidate for XLRS toward the clinic.”

Atsena’s XLRS gene therapy program leverages one of the company’s novel AAV capsids, AAV.SPR, that spreads laterally beyond the subretinal injection site, enabling safe and efficient transduction of the central retina (where schisis cavities predominate in XLRS patient retinas) when injected into areas outside the macula. A preclinical study in non-human primates demonstrated that AAV.SPR promotes transgene expression well beyond subretinal injection bleb margins. This is in stark contrast to benchmark vector AAV5, which remains confined to the original bleb margins. At clinically relevant doses, AAV.SPR efficiently transduces foveal cones without the need for surgical detachment and does not cause inflammation.

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