Atsena Therapeutics receives Orphan Drug Status from

DURHAM, NC, Sept. 17, 2024 (GLOBE NEWSWIRE) — Atsena Therapeutics, a clinical-stage gene therapy company focused on leveraging the life-changing power of genetic medicine to reverse or prevent blindness, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201, a first-in-class gene therapy product candidate, utilizes AAV.SPR, the Company's novel spreading capsid, to achieve therapeutic levels of gene expression in the photoreceptors of the central retina while avoiding the surgical risks of foveal detachment.

“We are honored that the FDA has granted orphan drug designation to ATSN-201, which was also recently designated as a rare pediatric disease. These designations mark an important turning point for the potential of this ocular gene therapy in an inherited retinal disease for which there are currently no treatment options,” said Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics. “The Atsena team is passionate and committed to our ongoing XLRS program. We look forward to providing hope to patients affected by this rare disease and are confident that these designations will accelerate our path forward.”

The FDA grants orphan drug designation to drugs and biologics intended to safely and effectively treat, diagnose, or prevent rare diseases or disorders affecting fewer than 200,000 people in the United States. Orphan drug designation provides certain incentives, such as tax credits for the cost of post-approval clinical trials and waivers of prescription drug fees. When a product receives orphan drug designation from the FDA, that product is entitled to seven years of market exclusivity for the disease for which it has orphan drug designation, regardless of intellectual property protection.

There are currently no approved treatments for XLRS, which is typically diagnosed in early childhood and affects approximately 30,000 men in the US and EU. The safety and tolerability of ATSN-201 is currently being evaluated in the LIGHTHOUSE study, an open-label, dose-escalation, dose-expansion Phase I/II clinical trial in male patients aged six years and older with a clinical diagnosis of XLRS caused by mutations in the RS1 Gen. Enrollment for this study is ongoing. For more information, visit ClinicalTrials.gov (identifier: NCT05878860).

About X-linked retinoschisis (XLRS)
XLRS is a monogenic X-linked disease caused by mutations in the RS1 Gene encoding retinoschisin, a protein secreted primarily by photoreceptors. RS1 is localized on the extracellular surface of rods, cones, and bipolar cells. XLRS is characterized by schisis, or abnormal splitting of the retinal layers, which impairs visual acuity that cannot be corrected with glasses and leads to progressive vision loss and eventually blindness. XLRS mainly affects boys and is typically diagnosed in early childhood. Approximately 30,000 males in the US and EU have XLRS, for which there are currently no approved treatments.

About AAV.SPR
AAV.SPR, one of Atsena's novel capsids, spreads laterally beyond the subretinal injection site, allowing safe and efficient transduction of the central retina (where schisis cavities predominate in retinas from XLRS patients) when injected into areas outside the macula. A preclinical study in non-human primates demonstrated that AAV.SPR promotes transgene expression well beyond the edges of the subretinal injection vesicles. This is in stark contrast to benchmark AAV vectors that remain confined to the original vesicle edges. At clinically relevant doses, AAV.SPR efficiently transduces foveal cones without the need for surgical detachment and does not cause inflammation. For more information on the preclinical study and how AAV.SPR works, visit

About Atsena Therapeutics
Atsena Therapeutics is a clinical-stage gene therapy company developing first-in-class treatments to reverse or prevent blindness due to inherited retinal diseases. The Company's lead program is evaluating ATSN-201 in an ongoing Phase I/II clinical trial for X-linked retinoschisis (XLRS), a progressive genetic disease that affects boys and men and is typically diagnosed in childhood. Another ongoing Phase I/II clinical trial is evaluating ATSN-101 for Leber congenital amaurosis type 1 (LCA1), one of the most common causes of blindness in children. Atsena's pipeline is based on a novel adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal diseases. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to the needs of patients with vision loss. For more information, visit

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