Atsena Therapeutics announced that the FDA has approved its investigational new drug (IND) application for ATSN-201 to treat X-linked retinoschisis (XLRS).
Refresh me on Atsena.
The clinical-stage gene therapy company targets the development of novel treatments for inherited forms of blindness, including GUCY2D-associated Leber congenital amaurosis (LCA1), X-linked retinoschisis (XLRS), and MYO7A-associated Usher syndrome (USH1B).
Atsena’s next-generation, adeno-associated virus (AAV) technologies feature laterally spreading capsids, dual vectors, and intravitreal capsids.
Tell me about ATSN-201.
ATSN-201 is an investigational gene therapy candidate utilizing AAV.SPR, one of Atsena’s novel capsids, that spreads laterally beyond the subretinal injection site to ensure the safe delivery of retinoschisin (RS1)—a protein secreted primarily by photoreceptors that, when mutated, causes XLRS— to photoreceptors within the central retina/fovea.
How about this novel capsid?
The AAV.SPR vector was developed to spread laterally beyond the subretinal injection bleb margins in order to reach therapeutic levels of gene expression in photoreceptors within the central retina—all without the surgical risks associated with foveal detachment to potentially restore retinal structure and function
The capsid is currently being investigated in Atsena’s XLRS and USH1B programs.
What makes it unique?
According to Shannon Boye, PhD, Atsena’s founder and director, AAVs delivered intravitreally have limitations that could lead to vision-compromising inflammation.
“AAV.SPR is well-suited for use in XLRS [because] it can drive therapeutic levels of gene expression in photoreceptors while avoiding the surgical risks of foveal detachment,” Boye stated, “which is important because XLRS patients have fragile retinas due to the presence of schisis lesions.”
So what’s next?
Atsena is now moving forward with the LIGHTHOUSE study, a phase 1/2 , open-label, dose-escalation clinical trial that will assess ATSN-201 as a subretinal injection for male patients (ages 6 to 65) diagnosed with XLRS caused by pathogenic or likely pathogenic mutations in RS1.
When is it slated to begin?
According to the company, the trial is expected to commence in mid-2023. Stay tuned!