1、Gene,Cell,&RNA Therapy Landscape ReportQ2 2025 Quarterly Data ReportAugust 20252August 2025Copyright 2025 Citeline,a Norstella company.ContentsIntroduction 03Key takeaways from Q2 2025 04Key highlights in Q2 2025 05Pipeline overview 15Gene therapy pipeline 16Non-genetically modified cell therapy pip

2、eline 23RNA therapy pipeline 27Overview of dealmaking 32Start-up funding 34Upcoming catalysts 36Appendix 373August 2025Copyright 2025 Citeline,a Norstella company.IntroductionWelcome to 2025s second quarterly report from ASGCT,Citeline,and Evaluate!This past quarter,three new therapeutics were appro

3、ved,and the number of gene therapy programs continues to climb.In the US,FDA approved the cell-based gene therapy Zevaskyn for recessive dystrophic epidermolysis bullosa(RDEB)as well as the lower-dose mRNA vaccine mNexspike for COVID-19.In China,the countrys first gene therapy for hemophilia B was a

4、pproved.Gene therapy programs at each stage from preclinical through Phase III have continued to increase in Q2,with oncology and rare diseases remaining the top areas of development.Eight of the top 10 rare diseases in the overall pipeline are oncological,continuing a trend from the past three year

5、s.Of the 80 gene therapy trials initiated in Q2,64%are for oncology indications,the highest proportion of the past year.In the cell therapy pipeline,oncology and rare diseases also remain the top areas of non-genetically modified cell therapy development.Of the 33 cell therapy trials initiated in Q2

6、,76%were for non-oncology indications.In the RNA therapy pipeline,38 trials were initiated in Q2,up from 35 last quarter,of which 74%were for non-oncology indications.Acquisitions of CGT assets continue to increase quarter by quarter,with a 33%jump in volume,including four deals worth more than a bi