Генная терапия показала эффективность при лечении наследственных заболеваний сетчатки

Gene therapies show benefit in X-linked retinoschisis, Leber congenital amaurosis Key takeaways: - ATSN-201 is being investigated for X-linked retinoschisis while ATSN-101 is being investigated for Leber congenital amaurosis. - The gene therapy candidates received orphan drug designation in Europe this summer. DENVER — Two studies presented at the Association for Research in Vision and Ophthalmology meeting explored the benefits of ATSN-101 for Leber congenital amaurosis and ATSN-201 for X-linked retinoschisis. In July, Atsena Therapeutics received orphan drug designation for the two gene therapy candidates from the European Medicines Agency, according to a press release. “Receiving orphan designation from the EMA for both ATSN-101 and ATSN-201 underscores that these programs address significant unmet needs for patients who currently have no treatment options,” Atsena CEO Patrick Ritschel said in the release. “With our pivotal trial for ATSN-201 enrolling rapidly and our global pivotal trial for ATSN-101 on track to begin later this year, we are closer than ever to delivering gene therapies that can change the trajectory of these inherited retinal diseases for patients in the U.S., Europe and beyond.” ATSN-201 is a gene therapy that uses the laterally spreading capsid AAV.SPR to target photoreceptors in the central retina. In a study presented at ARVO, researchers found that ATSN-201 helped improve retinal structure as well as function. The therapy was well tolerated with no dose-limiting toxicities or treatment-emergent adverse events, according to the abstract. “We’re showing schisis closure in seven out of nine of the part A patients,” Atsena chief medical officer Kenji Fujita, MD, told Healio. “We’re showing approvable levels of microperimetry improvement in five out of nine of our patients.” In another study presented at ARVO, researchers found that ATSN-101 provided durable improvement in retinal sensitivity based on full-field stimulus testing (FST) through 3 years of treatment. There were no serious drug-related adverse events through 3 years, according to the abstract. “We’re seeing an average of a hundredfold improvement in sensitivity as measured by FST,” Fujita told Healio. “Five out of six patients in which it was studied achieved the maximum score on [multi-luminance mobility testing].” The FDA previously granted orphan drug, rare pediatric disease, fast track and regenerative medicine advanced therapy designations to ATSN-201.