Atsena Therapeutics’ ATSN-201 Gene Therapy Receives FDA RMAT Designation for X-Linked Retinoschisis
DURHAM, NC, April 15, 2025 – Atsena Therapeutics, a clinical-stage gene therapy company focused on reversing blindness, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201 utilizes Atsena’s novel AAV.SPR capsid, enabling targeted gene expression in central retina photoreceptors without foveal detachment risks.
“This RMAT designation underscores ATSN-201’s potential to address the urgent need for XLRS treatments,” said Patrick Ritschel, CEO of Atsena Therapeutics. “Combined with our recent $150 million Series C financing, this milestone accelerates our mission to deliver transformative gene therapies for inherited retinal diseases.”
RMAT designation, established under the 21st Century Cures Act, expedites development for promising regenerative therapies. It provides intensive FDA guidance, including discussions on accelerated approval pathways and priority review.
XLRS, an inherited retinal disease affecting approximately 30,000 males in the U.S. and EU, currently lacks approved treatments. ATSN-201’s safety and tolerability are being evaluated in the ongoing LIGHTHOUSE Phase I/II trial (NCT05878860).
About X-linked Retinoschisis (XLRS):
XLRS is caused by RS1 gene mutations, leading to retinal splitting and progressive vision loss. It primarily affects males and is diagnosed in early childhood.
About AAV.SPR:
AAV.SPR, Atsena’s novel capsid, enables lateral spread beyond the injection site, achieving efficient central retina transduction without foveal detachment. Preclinical studies demonstrate its superior spread and safety profile compared to standard AAV vectors.
Atsena Therapeutics is committed to developing life-changing gene therapies to prevent and reverse blindness.
Source:
https://atsenatx.com/press-release/atsena-therapeutics-granted-us-fda-regenerative-medicine-advanced-therapy-designation-for-atsn-201-gene-therapy-to-treat-x-linked-retinoschisis/
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