May 2, 2023

Atsena Receives FDA Authorization to Launch a Gene Therapy Clinical Trial for XLRS

Eye On the Cure Research News

Known as The Lighthouse Study, the Phase ½ trial is expected to begin in mid-2023

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Atsena Therapeutics, a company developing gene therapies for inherited retinal diseases (IRDs), has received authorization from the US Food and Drug Administration (FDA) to launch a Phase 1/2 clinical trial for its X-linked retinoschisis (XLRS) gene therapy, ATSN-201. Known as The Lighthouse Study, the clinical trial will evaluate ATSN-201 in male patients ages 6-65 with a clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in the gene RS1. The company plans to initiate the trial in mid-2023.

Atsena’s emerging XLRS gene therapy is injected subretinally (underneath the retina). The company says this approach gets the treatment more effectively to the area of the retina where the treatment is needed – i.e., the cavities in the central retina caused by the splitting of retinal layers. The gene therapy uses a specially designed adeno-associated viral delivery system (AAV.SPR) that is able to reach the fragile fovea, the tiny pit in the central retina responsible for visual acuity, without an injection in the foveal region.

"The FDA’s clearance of the IND application for ATSN-201 marks a significant milestone for Atsena," said Kenji Fujita, MD, chief medical officer of Atsena Therapeutics. "We're excited to advance this investigational gene therapy that leverages our novel spreading capsid known as AAV.SPR into the clinic for people living with XLRS. XLRS is the leading cause of macular degeneration in young males and there are currently no approved treatments, so we have a compelling opportunity to address a significant unmet need."

The RD Fund, the Foundation’s venture philanthropy fund for advancing emerging treatments into and through early stage clinical trials, is a founding investor in Atsena.

“We are delighted to see Atsena launch its second clinical trial for an IRD,” said Rusty Kelley, PhD, managing director of the Foundation’s RD Fund and an Atsena Board observer. “XLRS is a challenging retinal condition and a critical unmet need.  We believe Atsena’s innovative, spreading-vector technology, developed by co-founder Shannon Boye’s group, provides an excellent opportunity to overcome delivery hurdles faced by other XLRS gene therapy developers that did not advance past early stage clinical trials.”

XLRS is caused by mutations in the gene RS1 which expresses a protein called retinoschisin — a protein that plays a critical role in the maintenance of the retinal structure and cell-to-cell adhesion. As an X-linked condition, XLRS usually affects males with females as unaffected carriers. XLRS is usually diagnosed in boys before the age of 10. Approximately 30,000 people in the US and EU are affected by XLRS.”

Atsena also recently reported vision improvements for patients in a Phase 1/2 gene therapy clinical trial for Leber congenital amaurosis 1 (LCA1) which is caused by GUCY2D mutations. The company also has a dual-vector gene therapy in preclinical development for Usher syndrome 1B, which is caused by MYO7A mutations.