Citeline & ASGCT：2026年第一季度基因、细胞和 RNA 疗法概况报告（英文版）（46页）.pdf

1、Gene,Cell,RNA Therapy Landscape ReportGene,Cell,&RNATherapy Landscape ReportQ1 2026 Quarterly Data ReportApril 2026GENE,CELL,&RNA THERAPY LANDSCAPE REPORTContentsContents2April 2026Copyright 2026 Citeline,a Norstella company.Introduction03Keytakeawaysfrom Q1 202604KeyhighlightsinQ1 202605Pipelineove

2、rview16Genetherapypipeline17Non-geneticallymodifiedcelltherapy pipeline25RNAtherapypipeline29Overviewofdealmaking35Start-up funding37Upcomingcatalysts40Appendix42GENE,CELL,&RNA THERAPY LANDSCAPE REPORTIntroductionIntroductionThe first quarter of 2026 marked a slower period for regulatory approvals a

3、cross gene,cell,and RNA therapies,with two new approvals globally both non-genetically modified cell therapies.Japan granted marketing authorization to Sumitomos raguneprocel for Parkinsons disease and Cuorips ReHeart for heart failure,while no additional gene or RNA therapies were approved.Global t

4、otals now stand at 40 approved gene therapies(including genetically modified cell therapies),37 RNA therapies,and 75 non-genetically modified cell therapies.Despite one fewer approval than in Q4 2025,regulatory momentum remained strong.Accelerated pathways expanded notably,with 13 additional designa

5、tions granted quarter over quarter.Broad use of Fast Track,RMAT,Breakthrough,and Orphan designations continues to reflect active regulatory engagement and steady pipeline advancement.Industry activity also remained resilient.Dealmaking rose 3%to 103 transactions,driven by increased financings and al

6、liances and stable acquisition volume.Two major CAR-T acquisitions Gileads$7.8 billion purchase of Arcellx and Eli Lillys acquisition of Orna Therapeutics for up to$2.4 billion underscore continued confidence in the space.Early-stage activity held steady at 13 seed or Series A rounds,though total ca