Atsena Therapeutics has received fast track designation from the US Food and Drug Administration (FDA) for its gene therapy product candidate, ATSN-201, to treat X-linked retinoschisis (XLRS).
Utilising the company’s AAV.SPR spreading capsid, ATSN-201 will attain therapeutic gene expression levels in photoreceptors of the central retina.
This approach ensures the elimination of the surgical risks typically associated with foveal detachment.
AAV.SPR is tailored to transduce the central retina, which is particularly affected by schisis cavities in XLRS sufferers.
The capsid’s ability to spread laterally from the subretinal injection area allows treatment beyond the macula.
Atsena Therapeutics CEO Patrick Ritschel stated: “We are pleased that the FDA has granted fast track designation to ATSN-201, re-inforcing its potential to address the significant unmet need in XLRS, a rare inherited retinal disease with no approved treatments.
“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.”
XLRS, a condition diagnosed typically in early childhood, is estimated to affect 30,000 males across the US and the European Union. It is marked by schisis, or splitting of retinal layers, which causes impaired visual acuity and may lead to blindness.
The Phase I/II LIGHTHOUSE clinical trial will assess the safety and tolerability of the gene therapy in male subjects aged six and above who are clinically diagnosed with XLRS caused by retinoschisin 1 gene mutations.
FDA fast track designation is crucial for treatments that target serious conditions with unmet medical needs. It allows for more frequent communication with the agency and the possibility of priority review.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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