2025年第三季度基因、细胞与RNA疗法领域报告（英）

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 16Non-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 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 in the US, the FDA approved Precigen’s Papzimeos for recurrent respiratory papillomatosis. The FDA also granted approval for Ionis Pharmaceuticals’ Dawnzera, an RNA therapy for hereditary angioedema. In Europe, ExCellThera’s Zemcelpro, a non-genetically modified cell therapy for hematological malignancies, received authorization. While regulatory progress continued, clinical 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 growth 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 joined the list of top five rare diseases targeted by CGTs. Additionally, the share of gene therapy trials focused on non-oncology indications rose to 42%, signaling a notable reversal of the downward trend observed since Q4 2024. Across these indicators, the data tell a powerful story: progress in CGT is dynamic, but not linear. Each data point helps us better understand the pace and direction of the field, equipping the community to anticipate shifts and opportunities for impact. The data are critical to driving awareness, guiding research, and accelerating availability of cell and gene therapies for patients worldwide.David Barrett, JDJD CEO, ASGCT4October 2025Copyright ©️ 2025 Citeline, a Norstella company.GENE, CELL, & RNA THERAPY LANDSCAPE REPORT• Two gene therapy approvals: in China, the NMPA approved Hrain Biotechnology’s CAR-T therapy, Hicara, for relapsed or refractory large B-cell lymphoma; 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