因编辑、细胞治疗与RNA技术前沿报告行业现状（英）

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 pipeline 23RNA therapy pipeline 27Overview of dealmaking 32Start-up funding 34Upcoming catalysts 36Appendix 373August 2025Copyright ©️ 2025 Citeline, a Norstella company.IntroductionWelcome 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 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 country’s first gene therapy for hemophilia B was approved.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 years. 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, 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 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 volume and value.Thank you,David Barrett, JD CEO, ASGCT4August 2025Copyright ©️ 2025 Citeline, a Norstella company.Gene, Cell, & RNA Therapy Landscape ReportKey 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-dose mRNA vaccine, mNexspike, for COVID-19• In China, Belief BioMed achieved NMPA approval for BBM-H901 — China’s first approved hemophilia B gene therapy• 679 asset-indications are at the clinical trial stage compared to 645 in Q1 2025 — a 5.3% rise quarter on quarter and a two-percent-point increase in relation to respective preclinical asset-indications• 26% of RNA therapy trials initiated in Q2 2025 were targeting oncology indications, as opposed to 17% in Q1 2025, marking the highest proportion of RNA oncology trials initiated in a quarter, for over two years• Advanced molecular therapy companies signed 91 deals in Q2 2025, nearly equivale