2025年第三季度数据报告：基因、细胞和RNA治疗前景报告

Q3 2025 Quarterly Data Report Contents Introduction The third quarter of 2025 brought notable progress across the gene, cell, and RNA therapy landscape,marked by four new global approvals — one in each major therapeutic category. In gene therapy,China’s NMPA approved Hrain Biotechnology’s Hicara CAR-T therapy for large B-cell lymphoma, while inthe US, the FDA approved Precigen’s Papzimeos for recurrent respiratory papillomatosis. The FDA alsogranted approval for Ionis Pharmaceuticals’ Dawnzera, an RNA therapy for hereditary angioedema.In Europe, ExCellThera’s Zemcelpro, a non-genetically modified cell therapy for hematologicalmalignancies, received authorization. While regulatory progress continued, clinical development activity slowed, with 125 trials initiated acrossgene, cell, and RNA therapies. The global pipeline remains robust with more than 3,200 trials currentlyunderway worldwide. Dealmaking activity increased in Q3, with 99 transactions representing a 9% risefrom the previous quarter. This growth was supported by a rebound in start-up financing, totaling $230.9million across 11 rounds. The quarter featured strategic takeovers by Kite and Biogen in areas of in vivoCAR therapies and ASO delivery, underscoring ongoing innovation and investment in next-generationmodalities. Pancreatic cancer joined the list of top five rare diseases targeted by CGTs. Additionally, the share ofgene therapy trials focused on non-oncology indications rose to 42%, signaling a notable reversal of thedownward trend observed since Q4 2024. Across these indicators, the data tell a powerful story: progress in CGT is dynamic, but not linear. Eachdata point helps us better understand the pace and direction of the field, equipping the communityto anticipate shifts and opportunities for impact. The data are critical to driving awareness, guidingresearch, and accelerating availability of cell and gene therapies for patients worldwide. David Barrett, JDJD CEO, ASGCT Key takeaways from Q3 2025 •Two gene therapy approvals: in China, the NMPA approved Hrain Biotechnology’s CAR-T therapy, Hicara, for relapsed or refractory large B-celllymphoma; while in the US, the FDA approved Precigen’s Papzimeos, a gene therapy indicated for recurrent respiratory papillomatosis (RRP)•One RNA therapy approval: the US FDA granted marketing approval for Ionis Pharmaceuticals’ Dawnzera in hereditary angioedema•One non-genetically modified cell therapy approval: ExCellThera’s Zemcelpro was granted EU approval for the treatment of adults withhematological malignancies Four new approvals acrosseach of the gene, cell, andRNA categories took placein Q3 2025 Q3 sees the lowest numberof total initiated clinicaltrials across gene, cell, andRNA therapies per quarterin over a year •A total of 125 trials were initiated in Q3 2025 across each of the gene, cell, and RNA categories, 17% less vs. the previous quarter and a 13%downturn compared to the equivalent period in 2024 (Q3)•There are a total of 3,243 trials investigating gene, cell, or RNA therapies that are currently ongoing •At 99 deals, total transaction volume for advanced molecular therapy companies represented a 9% increase over the previous quarter•Acquisitions were 4x down in Q3 from Q2, but featured takeovers by Kite and Biogen in the areas of in vivo CAR therapies and ASO delivery,respectively•Start-up financing rebounded from the low of last quarter to reach an aggregate $230.9 million from 11 rounds Dealmaking for advancedmolecular therapycompanies rebounded Key highlights in Q3 2025 Approved gene, cell, and RNA therapies Globally, for clinical use: •38 gene therapies have been approved(including genetically modified cell therapies) -Hrain Biotechnology achieved NMPA approval in China for its CAR-T therapy, Hicara,indicated for relapsed or refractory large B-cell lymphoma; the FDA approved Precigen’sPapzimeos, a gene therapy for recurrent RRP •35 RNA therapies have been approved -Ionis Pharmaceuticals’ Dawnzera was approved in the US for hereditary angioedema •71 non-genetically modified cell therapies have been approved -ExCellThera’s Zemcelpro was granted EU approval for the treatment of adults withhematological malignancies Approved gene therapies as of Q3 2025 Approved gene therapies as of Q3 2025continued Approved gene therapies as of Q3 2025continued Approved RNA therapies as of Q3 2025 Approved RNA therapies as of Q3 2025continued Approved RNA therapies as of Q3 2025continued Pipeline overview Pipeline of gene, cell, and RNA therapies 4,341 therapies are in development, ranging from preclinical through pre-registration •2,129 gene therapies (including genetically modified cell therapies such as CAR-T celltherapies) are in development, accounting for 49% of gene, cell, and RNA therapies•920 non-genetically modified cell therapies are in development, accounting for 21% of gene,cell, and RNA therapies Gene therapy pipelineGene therapy and genetically modified cell