2024年第一季度基因、细胞和RNA治疗前景报告季度数据报告

Q1 2024 Quarterly Data Report About the authors TheAmerican Society of Gene & Cell Therapy(ASGCT) is the primary professionalmembership organization for scientists, physicians, patient advocates, and otherprofessionals with interest in gene and cell therapy. Our members work in a wide range of settings including universities, hospitals,government agencies, foundations, biotechnology, and pharmaceuticalcompanies. ASGCT advances knowledge, awareness, and education leading to thediscovery and clinical application of gene and cell therapies to alleviate human diseaseto benefit patients and society. Citeline, aNorstellacompany, powers a full suite of complementary businessintelligence offerings to meet the evolving needs of life science professionals toaccelerate the connection of treatments to patients and patients to treatments.These patient-focused solutions and services deliver and analyze data used to driveclinical, commercial, and regulatory-related decisions and create real-worldopportunities for growth. Our global teams of analysts, journalists, and consultants keep their fingers on thepulse of the pharmaceutical, biomedical, and medtech industries, covering it all withexpert insights: key diseases, clinical trials, drug R&D and approvals, marketforecasts, and more. For more information on one of the world’s most trusted lifescience partners, visitCiteline. Table of contents 4Introduction5Key takeaways from Q1 20246Key highlights in Q1 202416Pipeline overview18Gene therapy pipeline26Non-genetically modified cell therapypipeline31RNA therapy pipeline37Overview of dealmaking40Start-up funding44Upcoming catalysts46Appendix Introduction Following a landmark close to 2023, the cell and gene therapy field has continued its impressive momentum in the firstquarter of 2024 with several notable approvals and strong growth in all stages of clinical development. The field earned the approval of three new therapies—a CAR-T therapy for myeloma in China, an AAV gene therapy forhemophilia B in Canada, and an autologous cell therapy for melanoma in the United States. Excitingly, there are now more than 4,000 gene, cell, and RNA therapies in development. We also recorded significantincreases in the number of therapies across all three phases of development in the clinical pipeline, with notablegrowth (11 percent) in Phase I programs. While overall dealmaking rebounded with a 34 percent increase in transactions, the start-up financing landscapecontinued to face challenges. Both the number and total value of seed and Series A rounds declined in Q1.Nevertheless, the breadth of progress across approvals, clinical development, and the broader ecosystem underscoresthe progress, promise, and vitality of cell and gene therapy. David Barrett, JDCEO, ASGCT Key takeaways from Q1 2024 Three new approvals headline Q1 2024 in the advanced therapy landscape •Two new gene therapies were approved: CT-053 (zevorcabtagene autoleucel), a CAR-T therapydeveloped by CARsgen, was approved for myeloma in China; and Beqvez, an AAV gene therapydeveloped by Pfizer, was approved for hemophilia B in Canada•Amtagvi, an autologous cell therapy developed by Iovance Biotherapeutics, was approved formelanoma in the US The past quarter was one of growth at the clinical development stage (Phase I–III), in particular forgene therapies •The number of therapies at all three phases of clinical pipeline development has increased since theend of 2023•Phase I therapy numbers in particular saw their greatest growth (11%) since prior to Q4 2022 Overall dealmaking rebounded for advanced molecular therapy companies, but start-up financingcontinued to slow •The sector saw a 34% increase in total deals in Q1 2024, reaching 125 transactions, up from 93 in theprevious quarter•Driving this increase was a substantial jump in total financings; however, start-ups raising seed orSeries A funds continued the downward trend seen in previous quarters•Start-up volume declined by 33% to eight transactions in Q1 2024, and had a nearly 3x decrease invalue to $240.1m Key highlights in Q1 2024 Approved gene, cell, and RNA therapies Globally, for clinical use: •32 gene therapies have been approved(including genetically modified cell therapies)•In Q1 2024,CT-053(CARsgen) was approved formyeloma in China, andBeqvez(Pfizer) was approvedfor hemophilia B in Canada•28 RNA therapies have been approved•68 non-genetically modified cell therapies have beenapproved•In Q1 2024,Amtagviwas approved in the US for thetreatment of melanoma Pipeline overview Pipeline of gene, cell, and RNA therapies 4,002 therapies are in development,ranging from preclinical throughpre-registration •2,093 gene therapies (including geneticallymodified cell therapies such as CAR-T celltherapies) are in development, accountingfor 52% of gene, cell, and RNA therapies•885 non-genetically modified cell therapiesare in development, accounting for 22% ofgene, cell, and RNA therapies Gene therapy pipel