Atsena Therapeutics Announces Initiation of Dosing in Second Cohort of Phase I/II Clinical Trial Evaluating ATSN-201 Gene Therapy for the Treatment of X-linked Retinoschisis (XLRS)
ATSN-201, a best-in-class gene therapy product candidate, 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.
- ATSN-201, a best-in-class gene therapy product candidate, 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.
- ATSN-201 was well tolerated in all three XLRS patients in the first cohort and no serious adverse events were reported.
- The Data and Safety Monitoring Board recommended proceeding with the second cohort, in which patients will receive the next dose level of ATSN-201.
- "We are encouraged by the excellent safety observed with the low dose of ATSN-201 in the LIGHTHOUSE study, which represents the first clinical utilization of AAV.SPR.