2025年第二季度基因、细胞与RNA疗法概览报告

Q2 2025 Quarterly Data Report Introduction03Key takeaways from Q2 202504Key highlights in Q2 202505Pipeline overview15Gene therapy pipeline16Non-genetically modified cell therapy pipeline23RNA therapy pipeline27Overview of dealmaking32Start-up funding34Upcoming catalysts36Appendix37 Introduction Welcome to 2025’s second quarterly report from ASGCT, Citeline, and Evaluate! This past quarter,three new therapeutics were approved, and the number of gene therapy programs continues toclimb. In the US, FDA approved the cell-based gene therapy Zevaskyn for recessive dystrophicepidermolysis bullosa (RDEB) as well as the lower-dose mRNA vaccine mNexspike for COVID-19. InChina, the country’s first gene therapy for hemophilia B was approved. Gene therapy programs at each stage from preclinical through Phase III have continued to increasein Q2, with oncology and rare diseases remaining the top areas of development. Eight of the top 10rare diseases in the overall pipeline are oncological, continuing a trend from the past three years. Ofthe 80 gene therapy trials initiated in Q2, 64% are for oncology indications, the highest proportionof the past year. In the cell therapy pipeline, oncology and rare diseases also remain the top areasof non-genetically modified cell therapy development. Of the 33 cell therapy trials initiated in Q2,76% were for non-oncology indications. In the RNA therapy pipeline, 38 trials were initiated in Q2, upfrom 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 billion dollars each. There were three start-up financings,which raised $197 million in Q2, continuing the decline of the past three quarters in both volumeand value. Thank you,David Barrett, JDCEO, ASGCT Gene, Cell, & R NA Therapy L andscape R eport Key takeaways from Q2 2025 •The US FDA approved Abeona’s cell-based gene therapy,Zevaskyn, for recessive dystrophic epidermolysis bullosa(RDEB), as well as Moderna’s next-generation, lower-dosemRNA vaccine, mNexspike, for COVID-19 Three new approvalsacross the gene, cell,and RNA landscapein Q2 2025 •In China, Belief BioMed achieved NMPA approval forBBM-H901 — China’s first approved hemophilia Bgene therapy •679 asset-indications are at the clinical trial stage comparedto 645 in Q1 2025 — a 5.3% rise quarter on quarter anda two-percent-point increase in relation to respectivepreclinical asset-indications Over the past quarter,more RNA therapiesthat are beingpursued in oncologyindications arereaching the clinic •26% of RNA therapy trials initiated in Q2 2025 weretargeting oncology indications, as opposed to 17% in Q12025, marking the highest proportion of RNA oncology trialsinitiated in a quarter, for over two years •Advanced molecular therapy companies signed 91 deals inQ2 2025, nearly equivalent to last quarter’s volume•Acquisition activity picked up, with 12 such deals in Q2compared with nine in the previous quarter, highlighted bythree billion-dollar takeovers by big pharma•Financing activity overall was down, especially amongstart-ups which saw a total $197 million raised from onlythree transactions Overall dealmakingwas flat, and start-upfinancing continueddownward trend Gene, Cell, & R NA Therapy L andscape R eport Key highlights in Q2 2025 APPROVED GENE, CELL, AND RNA THERAPIES Globally, for clinical use: •36 gene therapies have been approved(including genetically modified cell therapies) -Belief BioMed achieved NMPA approval inChina for its hemophilia B gene therapy,BBM-H901; the FDA approved Abeona’s cell-based gene therapy, Zevaskyn, for recessivedystrophic epidermolysis bullosa (RDEB). •36 RNA therapies have been approved -Moderna’s next-generation, lower-dosemRNA vaccine, mNexspike, was approvedin the US for COVID-19. •71 non-genetically modified cell therapieshave been approved Source: Pharmaprojects | Citeline, July 2025 Gene, Cell, & R NA Therapy L andscape R eport Gene, Cell, & R NA Therapy L andscape R eport Gene, Cell, & R NA Therapy L andscape R eport Gene, Cell, & R NA Therapy L andscape R eport Gene, Cell, & R NA Therapy L andscape R eport Gene, Cell, & R NA Therapy L andscape R eport Gene, Cell, & R NA Therapy L andscape R eport Gene, Cell, & R NA Therapy L andscape R eport Gene, Cell, & R NA Therapy L andscape R eport Pipeline overview PIPELINE OF GENE, CELL, AND RNA THERAPIES 4,469 therapies are in development,ranging from preclinical throughpre-registration •2,210 gene therapies (includinggenetically modified cell therapiessuch as CAR-T cell therapies) are indevelopment, accounting for 49%of gene, cell, and RNA therapies •962 non-genetically modified celltherapies are in development,accounting for 22% of gene, cell,and RNA therapies Gene, Cell, & R NA Therapy L andscape R eport Gene therapy pipelineGene therapy and genetically modified cell therapies GENE THERAPY PIPELINE: QUARTERLY C