1、Gene,Cell,&RNA Therapy Landscape ReportQ3 2025 Quarterly Data ReportNovember 20252October 2025Copyright 2025 Citeline,a Norstella company.GENE,CELL,&RNA THERAPY LANDSCAPE REPORTContentsIntroduction 03Key takeaways from Q3 2025 04Key highlights in Q3 2025 05Pipeline overview 15Gene therapy pipeline 1

2、6Non-genetically modified cell therapy pipeline 24RNA therapy pipeline 28Overview of dealmaking 34Start-up funding 36Upcoming catalysts 39Appendix 403October 2025Copyright 2025 Citeline,a Norstella company.GENE,CELL,&RNA THERAPY LANDSCAPE REPORTIntroductionThe third quarter of 2025 brought notable p

3、rogress across the gene,cell,and RNA therapy landscape,marked by four new global approvals one in each major therapeutic category.In gene therapy,Chinas NMPA approved Hrain Biotechnologys Hicara CAR-T therapy for large B-cell lymphoma,while in the US,the FDA approved Precigens Papzimeos for recurren

4、t respiratory papillomatosis.The FDA also granted approval for Ionis Pharmaceuticals Dawnzera,an RNA therapy for hereditary angioedema.In Europe,ExCellTheras Zemcelpro,a non-genetically modified cell therapy for hematological malignancies,received authorization.While regulatory progress continued,cl

5、inical development activity slowed,with 125 trials initiated across gene,cell,and RNA therapies.The global pipeline remains robust with more than 3,200 trials currently underway worldwide.Dealmaking activity increased in Q3,with 99 transactions representing a 9%rise from the previous quarter.This gr

6、owth was supported by a rebound in start-up financing,totaling$230.9 million across 11 rounds.The quarter featured strategic takeovers by Kite and Biogen in areas of in vivo CAR therapies and ASO delivery,underscoring ongoing innovation and investment in next-generation modalities.Pancreatic cancer