Atsena Therapeutics has dosed the first patient in the Phase I/II LIGHTHOUSE study of ATSN-201 to treat X-linked retinoschisis (XLRS).
The dose-escalation and dose-expansion study intends to assess the tolerability and safety of subretinal injection of ATSN-201 for five years.
Nearly 18 male patients aged six to 64 years with a clinical diagnosis of XLRS, caused by pathogenic or likely pathogenic mutations in RS1, will be included in the study.
They will receive a one-time subretinal injection of ATSN-201 in one eye.
Safety and tolerability, as evaluated by dose-limiting toxicities and treatment-emergent adverse events, are the primary outcome measures of the study.
Atsena Therapeutics chief medical officer Kenji Fujita said: “We are excited to be utilising AAV.SPR in the clinic, as it has the potential to revolutionise the treatment of XLRS, as well as other inherited retinal disorders.
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“Spreading laterally beyond the subretinal injection site, AAV.SPR facilitates the safe delivery of RS1 to photoreceptors in the central retina/fovea.
“We look forward to advancing the LIGHTHOUSE study and the continued development of our novel gene therapies to reverse or prevent blindness.”
ATSN-201 is a part of the company’s two clinical-stage programmes that include ATSN-101 for treating GUCY2D-associated Leber congenital amaurosis.
Clinically meaningful improvements in vision were observed six months post-treatment with ATSN-101, as demonstrated in an ongoing Phase I/II study.
The company’s product portfolio also includes ATSN-301, a dual AAV vector-based gene therapy to prevent blindness from MYO7A-associated Usher syndrome.
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