Gene therapy for X-linked retinoschisis receives rare pediatric disease designation
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Key takeaways:
- ATSN-201 is being investigated for the treatment of X-linked retinoschisis.
- The designation means a priority review voucher would be issued upon approval.
The FDA granted rare pediatric disease designation to ATSN-201, a gene therapy candidate for the treatment of X-linked retinoschisis, according to a press release from Atsena Therapeutics.
“There are currently no approved treatments for X-linked retinoschisis (XLRS),” Patrick Ritschel, CEO of Atsena, told Healio. “If approved, ATSN-201 would be the first time that ophthalmologists could offer a therapy to their patients with XLRS to address the underlying genetic cause of the disease.”
ATSN-201 leverages AAV.SPR, a novel spreading capsid from Atsena, to spur therapeutic levels of gene expression in the central retina’s photoreceptors while mitigating the risk for foveal detachment. The use of AAV.SPR may “potentially open the doors to developing treatments for a broad range of inherited retinal diseases that have otherwise been untreatable with subretinal injection,” Ritschel said. “With its ability to spread beyond the margins of the subretinal injection bleb, AAV.SPR could make it possible to deliver gene therapies to a much larger area of the retina than traditional [adeno-associated viruses].”
The designation would allow ATSN-201 to receive a priority review voucher upon approval that could be used to advance an additional program for Atsena or be sold.
“Atsena is currently enrolling adult participants in a phase 1/2 clinical trial and collecting safety and efficacy data,” Ritschel said. “We are hopeful that we will see schisis resolution (ie, improvements in the structure of the retina) with ATSN-201 and also improvements in visual function for these patients. After collecting safety and efficacy data in adults, we will include pediatric patients ages 6 to 17 in the trial to evaluate the potential for ATSN-201 to treat children with XLRS.”
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