With no approved treatments for XLRS, another promising gene therapy enters the fray.
Atsena Therapeutics (North Carolina, USA) announced that the U.S. Food and Drug Administration (FDA) has granted fast track designation to ATSN-201, an investigational gene therapy aimed at treating X-linked retinoschisis (XLRS).
FDA fast track designation is designed to expedite the development and review of treatments for serious or life-threatening conditions that lack effective medical options. XLRS retinoschisis is one of them, with no FDA-approved treatments currently available.
ATSN-201 utilizes the company’s proprietary AAV.SPR, a novel spreading capsid technology developed by Atsena Therapeutics. This innovation allows the therapy to achieve therapeutic levels of gene expression in the photoreceptors of the central retina without requiring surgical detachment of the fovea.
“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,” said Patrick Ritschel, CEO of Atsena Therapeutics.
“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.”
Atsena announces the news on LinkedIn
The designation means Atsena will have more frequent interactions with the FDA, potentially leading to a faster path to market for ATSN-201. If the therapy meets specific criteria, it may also be eligible for Priority Review, further accelerating the approval process.
Ongoing clinical trials and next steps
Currently, the safety and tolerability of ATSN-201 are being assessed in the ongoing LIGHTHOUSE study, a Phase I/II clinical trial designed to evaluate different dosing levels in male patients aged six and older with a confirmed diagnosis of XLRS. Enrollment for the study is ongoing.
With Fast Track designation in place, Atsena Therapeutics is well-positioned to advance ATSN-201 through the clinical trial process more efficiently. If successful, this therapy could represent a novel treatment for XLRS patients who currently have no approved options.For more information, see Atsena’s press release on the fast track designation.
