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.
Initial TNF-α Levels Predict Infliximab Response in Pediatric Crohn's Disease
• A recent study reveals that initial levels of TNF-α can predict the treatment response to Infliximab (IFX) in pediatric Crohn's Disease (CD).
• Higher initial TNF-α concentrations correlate with lower IFX trough concentrations, potentially leading to suboptimal therapeutic effects.
• A TNF-α cutoff value of 27.6 pg/mL was identified, above which higher IFX doses may be needed to maintain therapeutic concentrations.
• Measuring initial cytokine levels could help personalize IFX dosing strategies, improving treatment outcomes for pediatric CD patients.
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