Phase 3 Trial Shows Promising Results for Novel Encapsulated Cell Therapy in MacTel Treatment
• Phase 3 clinical trials of NT-501, an encapsulated cell therapy delivering CNTF, demonstrated significant reduction in disease progression for macular telangiectasia type 2 patients, with up to 52% reduction in ellipsoid zone loss.
• The innovative implantable device, developed by Neurotech, maintains long-term viability with CNTF production documented for up to 14.5 years, offering a potential alternative to frequent intravitreal injections.
• FDA review of the therapy is currently underway with a PDUFA date set for March 18, 2025, marking a potential breakthrough in MacTel treatment.
Gene Therapy Trial for Geographic Atrophy Shows Limited Efficacy Despite Sustained Factor I Expression
A Phase I/II clinical trial of PPY988, an AAV2-CFI gene therapy for geographic atrophy, demonstrated sustained elevation of complement Factor I levels but insufficient potency compared to existing treatments. The study provides crucial insights for future complement-targeting gene therapies while confirming complement modulation as a viable therapeutic approach for age-related macular degeneration.
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