ISCT细胞和基因治疗全球监管报告2024年上半年
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H1 2024 Sect on 1– Overv ew of Report & Authors Sect on 2– Report H ghl ghts Sect on 3– Global Overv ew of P pel ne Products,Recent Approvals & Regulatory Rev ews Sect on 4– H ghl ghts of Regulatory Events, Key GlobalLeg slat ve/Framework Changes, Standards & Latest Approved Products D scla mer References, Methodology, Glossary of Key Terms Report Contr butors OVERVIEW OF REPORT Welcome to the th rd b annual Cell and Gene Therapy Global Regulatory Report from ISCT and C tel ne. Th s reportprov des a global overv ew of the cell and gene therapy regulatory landscape, nclud ng p pel ne, late-stage (PhaseIII and pre-reg strat on), and approved products. It covers Cell, Genet cally-Mod f ed Cell, and Gene Therap es. Over the last few years, the regulatory agenc es have been updat ng leg slat on and frameworks to keep up w ththe rap dly advanc ng cell and gene therapy space. As a result, ISCT and C tel ne have collaborated to h ghl ghtsome of these changes and offer commentary on the updates and the r effects. In th s report, we see that year-over-year, non-genet cally mod f ed cell therap es cont nue to make up the major tyof all approved products (68%). However, the p pel ne for genet cally mod f ed cell therap es cont nues to be largerw th 1,008 therap es n development, compared to 902 non-genet cally mod f ed cell therap es and 938 genetherap es. Meanwh le, ISCT cont nues to track regulatory changes and events and prov de comments on Key GlobalLeg slat ve/Framework Changes nclud ng US FDA draft gu dance n potency assurance for CGT products and EMAreflect on paper on the use of Real-World Data n Non-Intervent onal Stud es to Generate Real-World Ev dence. ABOUT THE AUTHORS The Internat onal Soc ety for Cell & Gene Therapy (ISCT) s the global leader focused on pre-cl n cal andtranslat onal aspects of develop ng cell and gene-based therapeut cs, thereby advanc ng sc ent f c research nto nnovat ve treatments for pat ents. ISCT offers a un que collaborat ve env ronment that addresses three keyareas of translat on: Academ a, Regulatory, and Commerc al zat on. Through strong relat onsh ps w th globalregulatory agenc es, academ c nst tut ons, and ndustry partners, ISCT dr ves the advancement of research ntoa standard of care. Compr sed of over 4,000 cell and gene therapy experts across f ve geograph c reg ons and representat on fromover 60 countr es, ISCT members are part of a global commun ty of peers, thought leaders, and organ zat ons nvested n cell and gene therapy translat on. For more nformat on about the organ zat on, v s tISCT. A spec al thanks to theISCT Global Regulatory Task Forcefor the r contr but on. The ISCT GRTF works toaddress challenges and opportun t es n establ shed and evolv ng global regulatory env ronments C tel ne (formerly Pharma Intell gence) powers a full su te of complementary bus ness ntell gence offer ngs tomeet the evolv ng needs of l fe sc ence profess onals to accelerate the connect on of treatments to pat ents andpat ents to treatments. These pat ent-focused solut ons and serv ces del ver and analyze data used to dr vecl n cal, commerc al, and regulatory related-dec s ons and create real-world opportun t es for growth. Our global teams of analysts, journal sts and consultants keep the r f ngers on the pulse of the pharmaceut cal,b omed cal and medtech ndustr es, cover ng t all w th expert ns ghts: key d seases, cl n cal tr als, drug R&D andapprovals, market forecasts and more. For more nformat on on one of the world’s most trusted l fe sc encepartners, v s tC tel ne. REPORT HIGHLIGHTS THREE NEW APPROVALS HIGHLIGHT IN H1 2024 Amtagv (l f leucel)CT-053 (zevorcabtagene autoleucel)Beqvez (f danacogene elaparvovec) H1 2024 WAS A GROWTH PERIODPARTICULARLY FOR NON-GENETICALLY MODIFIED CELL THERAPIES There s a 4% ncrease n the number of non-genet cally mod f ed celltherap es n the p pel ne from H2 2023.The non-genet cally mod f ed cell category cont nues to have the mosttherap es under phase III or n pre-reg strat on phase w th a total of 46therap es n H1 2024.The non-genet cally mod f ed cell therap es cont nue to make up themajor ty of all approved products (68%) GLOBAL PIPELINE OVERVIEW – BY CATEGORY 2,848 PIPELINES As of June 25, 2024, from the global p pel nes(from precl n cal to prereg strat on phase) therap es,there are: 938 gene therap es902 non-genet cally mod f ed cell therap es1,008 genet cally-mod f ed cell therap es GLOBAL PIPELINE OVERVIEW – BY PHASE AS OF JUNE 25, 2024, THE MAJORITY OF THERAPIES ARE IN PRECLINICAL DEVELOPMENT Non-genet cally mod f ed cell category has the most therap es under regulatory rev ew w th 46therap es n Phase III or n pre-reg strat onGene therapy category has the greatest absolute number of therap es n precl n cal development(720 therap es), as well as the greatest proport on of precl n cal development (77%) OVERVIEW OF APPROVED PRODUCTS – BY CATEGORY 100 PRODUCTS As of June 25, 2024, 100 productsare approved globally:
