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1、Gene,Cell,&RNATherapy Landscape ReportQ4 2025 Quarterly Data ReportJanuary 2026GENE,CELL,&RNA THERAPY LANDSCAPE REPORTContentsIntroduction2January 2026Copyright 2026 Citeline,a Norstella company.03Key takeaways from Q4 202504Key highlights in Q4 202505Pipeline overview15Gene therapy pipeline16Non-ge

2、netically modified cell therapy pipeline24RNA therapy pipeline28Overview of dealmaking34Start-up funding36Upcoming catalysts39Appendix40GENE,CELL,&RNA THERAPY LANDSCAPE REPORTIntroductionThe fourth quarter of 2025 marked continued regulatory progress across the gene,cell,and RNA therapy landscape,wi

3、th three new approvals spanning gene and RNA modalities.In gene therapy,regulatory agencies in both China and the United States advanced novel treatments for rare and serious conditions.The FDA approved GSKs Waskyra,a hematopoietic stem cellbased gene therapy for Wiskott-Aldrich syndrome,while China

4、s NMPA approved Chongqing Precision Biotechs Pulidekai,a CD19 CAR-T therapy for relapsed or refractory acute lymphoblastic leukemia.In RNA therapeutics,the FDA also granted marketing approval to Arrowhead Pharmaceuticals Redemplo for familial chylomicronemia syndrome.Despite these regulatory milesto

5、nes,the overall development pipeline continued to contract modestly in Q4.The total number of gene,cell,and RNA therapies in development declined to 2,041,representing a 4%decrease from Q1 2025 levels.This shift was largely driven by a reduction in preclinical programs,which fell from 66%to 60%of th

6、e pipeline,reflecting a broader recalibration of early-stage portfolios rather than downstream attrition.Notably,the proportion of therapies in Phase I through pre-registration remained relatively stable,underscoring continued commitment to advancing programs already in clinical development.In contr