First Cohort Benefits From Atsena Therapeutics XLRS Gene Therapy

UF startup Atsena Therapeutics has announced positive preliminary data for its gene therapy candidate ATSN-201 for the treatment of X-linked retinoschisis (XLRS).

The US-based gene therapy company revealed that two of the three patients in the first cohort of the ongoing LIGHTHOUSE study showed extensive resolution of schisis, abnormal splitting of retinal layers, after dosing with ATSN-201.

The LIGHTHOUSE study (NCT05878860) is evaluating ATSN-201 in male patients aged six and older with a clinical diagnosis of XLRS caused by mutations in the RS1 gene. The technology is based on research by Shannon Boye and her team in the UF College of Medicine Department of Ophthalomy.

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