Atsena’s XLRS gene therapy shows efficacy in phase 1/2 clinical trial 

Key Takeaways

• Athena Therapeutics has reported both structural and functional vision improvements for patients receiving the low dose of the company’s XLRS gene therapy in its LIGHTHOUSE Phase 1/2 clinical trial.
• The company is currently dosing patients in the mid-dose group. 

X-linked retinoschisis (XLRS) is a genetic eye condition that results in vision impairment in boys and men. XLRS is caused by mutations in a gene on the X chromosome called RS1 which encodes a protein called retinoschisin. This protein is important for the development and maintenance of the retina (the tissue lining the back of the eye). Without normal retinoschisin protein, the layers of the retina split (“schisis”), inter-cell communication is disrupted and vision is lost.

Atsena Therapeutics, a company developing innovative gene therapies for inherited retinal diseases, has reported both structural and functional vision improvements for patients receiving the low dose of the company’s XLRS gene therapy in its LIGHTHOUSE Phase 1/2 clinical trial. Known as ATSN-201, the emerging treatment was well tolerated for all three patients in the low-dose group. The company is currently dosing patients in the mid-dose group. 

Atsena’s emerging XLRS gene therapy is injected subretinally (underneath the retina). The company says this approach gets the treatment more effectively to the area of the retina where the treatment is needed — i.e., near the damaging, schisis cavities in the central retina caused by the splitting of retinal layers in people with XLRS. ATSN-201 uses a specially designed adeno-associated viral delivery system (AAV.SPR) which laterally spreads across a larger area of the retina than other AAV systems. The AAV.SPR can reach the fragile fovea — the tiny pit in the central retina responsible for sharp central vision — with an injection outside of the foveal region.

Two of three patients in the low-dose group of LIGHTHOUSE had significant resolution of the schisis cavities. Schisis resolution was observed through 24 weeks after ATSN-201 was administered. The schisis resolution occurred inside and well outside of the injection site, affirming ATSN-201’s ability to spread broadly across the retina.

For more information on this trial, visit https://clinicaltrials.gov/study/NCT05878860 or contact Atsena Therapeutics Clinical Trials at 984-261-2001 or clinicaltrials@atsenatx.com. 

Edited by Miriam Kaplan, PhD

Source: Eye on the Cure Research News, Foundation Fighting Blindness, May 2, 2024; see source article