Atsena Therapeutics has announced positive clinical results from the LIGHTHOUSE study, evaluating subretinal injection of a novel gene therapy to treat X-linked retinoschisis (XLRS).
XLRS is a monogenic X-linked disease caused by mutations in the RS1 gene. XLRS is characterised by schisis, or abnormal splitting of retinal layers, which causes impaired visual acuity. It is not correctable with glasses and ultimately leads to blindness.
Atsena’s treatment candidate, ATSN-201 leverages AAV.SPR, the company’s novel spreading capsid, to achieve therapeutic levels of gene expression in photoreceptors of the central retina while avoiding the surgical risks of foveal detachment.
“The full Part A data from the LIGHTHOUSE study reinforce the favourable safety profile and demonstrate encouraging structural and functional benefits across all three dose levels of ATSN-201,” said Atsena’s Dr Kenji Fujita.
“Interim data is available for nine adults who have been treated, and no serious adverse events related to treatment have been reported. We’re particularly pleased that the foveal schisis closure seen in seven of nine patients validates AAV.SPR’s ability to spread laterally beyond the subretinal injection blebs and enable safe gene delivery to the central retina.
It’s totally clear cut: without the contact lenses, the subject cannot see anything, but when they put them on, they can clearly see the flickering of the infrared light
“These findings represent a significant breakthrough, validating the use of our novel capsid to effectively treat inherited retinal disease and informing the safest and most effective path forward for Part B of the study, which is already underway.”
Reference
- Atsena Therapeutics, Atsena Therapeutics announces positive clinical data from Part A of Phase I/II trial evaluating ATSN-201 gene therapy to treat X-linked Retinoschisis (XLRS) (media release, 19 May 2025) available at: atsenatx.com/press-release/atsena-therapeutics-announces-positive-clinical-data-from-part-a-of-phase-i-ii-trial-evaluating-atsn-201-gene-therapy-to-treat-x-linked-retinoschis/ [accessed May 2025].
