Atsena Therapeutics Receives FDA Fast Track Designation for ATSN-201 Gene Therapy in X-Linked Retinoschisis
Atsena Therapeutics has been granted Fast Track designation by the FDA for ATSN-201, its investigational gene therapy designed to treat X-linked retinoschisis (XLRS), a rare inherited retinal disease with no approved treatments.
A Milestone in XLRS Gene Therapy Development
ATSN-201 utilizes AAV.SPR, Atsena’s proprietary spreading capsid, to achieve therapeutic gene expression in central retinal photoreceptors without the need for surgical foveal detachment. This innovative approach aims to address the significant unmet need in XLRS while minimizing procedural risks.
Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics, emphasized the importance of this designation:
“We are pleased that the FDA has granted Fast Track designation to ATSN-201, reinforcing its potential to address the significant unmet need in XLRS, a rare inherited retinal disease with no approved treatments. This designation, along with the previously granted Orphan Drug and Rare Pediatric Disease designations, marks an important milestone in advancing the development of ATSN-201. The Atsena team remains dedicated to developing transformative gene therapies and improving the quality of life of individuals suffering from XLRS and other inherited retinal diseases.”
What Fast Track Designation Means for ATSN-201
Fast Track designation is granted to therapies that treat serious or life-threatening conditions with unmet medical needs. This status provides regulatory advantages, including:
• More frequent FDA interactions to facilitate drug development.
• Potential eligibility for Priority Review, expediting the approval process.
About X-Linked Retinoschisis (XLRS) and the LIGHTHOUSE Study
XLRS is a genetic retinal disorder typically diagnosed in early childhood, leading to progressive vision loss. The disease affects approximately 30,000 males in the U.S. and EU, with no FDA-approved treatments currently available.
The safety and tolerability of ATSN-201 are being evaluated in LIGHTHOUSE, a Phase 1/2 dose-escalation and dose-expansion clinical trial involving male patients aged six and older with RS1 gene mutations. Patient enrollment for the study is ongoing.
Conclusion
The FDA Fast Track designation marks a major step forward in developing ATSN-201 as a potential first-ever gene therapy for XLRS. With orphan drug and rare pediatric disease designations already in place, Atsena Therapeutics is advancing toward bringing a transformative solution to patients affected by this devastating inherited retinal disease.
