{"title":"The current state of Cytotherapy and the field of cell and gene therapy.","authors":"William Y K Hwang, Ezzah Mohamed Muzammil","doi":"10.1016/j.jcyt.2025.01.017","DOIUrl":null,"url":null,"abstract":"<p><p>2024 marked a transformative phase for cell and gene therapy (CGT) with significant advancements in scientific innovation, regulatory approvals and commercialization milestones. This review highlights key developments in CGT, including innovations in chimeric antigen receptor (CAR)-T therapies, mesenchymal stromal cells (MSCs), gene editing and regenerative medicine, alongside challenges in scalability, regulation and safety. Prominent breakthroughs in CAR-T technology extended its applications beyond oncology to autoimmune diseases, including lupus and systemic sclerosis. Gene therapies achieved major milestones, exemplified by regulatory approval for the treatment of hemophilia, sickle cell disease and other genetic diseases. Further advancements in delivery systems, including lipid nanoparticles and engineered viral vectors were achieved. Refinements in clustered regularly interspaced short palindromic repeats-Cas9 and base-editing tools improved precision and reduced off-target effects, enabling new approaches for genetic disorders. Global collaboration underscored the collective effort to accelerate CGT progress. MSCs remain central to CGT research, focusing on their immunomodulatory properties and clinical applications in autoimmune diseases and graft-versus-host disease. Economic and policy landscapes evolved alongside scientific advancements. Record-breaking approvals and biotech IPOs underscored CGT's economic potential, while affordability and equitable access emerged as critical challenges. Regulatory agencies advanced harmonized guidelines for manufacturing and clinical evaluation, streamlining global access to these therapies. Ethical considerations, including the affordability of therapies and the need for diverse clinical trial representation, remained prominent. Despite progress, challenges persist in scalability, safety and regulatory harmonization. Manufacturing improvements are essential to meet growing demand, while addressing safety concerns, such as off-target gene-editing effects and tumorigenicity in MSC therapies, remains paramount.</p>","PeriodicalId":50597,"journal":{"name":"Cytotherapy","volume":" ","pages":""},"PeriodicalIF":3.7000,"publicationDate":"2025-02-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Cytotherapy","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1016/j.jcyt.2025.01.017","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"BIOTECHNOLOGY & APPLIED MICROBIOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
2024 marked a transformative phase for cell and gene therapy (CGT) with significant advancements in scientific innovation, regulatory approvals and commercialization milestones. This review highlights key developments in CGT, including innovations in chimeric antigen receptor (CAR)-T therapies, mesenchymal stromal cells (MSCs), gene editing and regenerative medicine, alongside challenges in scalability, regulation and safety. Prominent breakthroughs in CAR-T technology extended its applications beyond oncology to autoimmune diseases, including lupus and systemic sclerosis. Gene therapies achieved major milestones, exemplified by regulatory approval for the treatment of hemophilia, sickle cell disease and other genetic diseases. Further advancements in delivery systems, including lipid nanoparticles and engineered viral vectors were achieved. Refinements in clustered regularly interspaced short palindromic repeats-Cas9 and base-editing tools improved precision and reduced off-target effects, enabling new approaches for genetic disorders. Global collaboration underscored the collective effort to accelerate CGT progress. MSCs remain central to CGT research, focusing on their immunomodulatory properties and clinical applications in autoimmune diseases and graft-versus-host disease. Economic and policy landscapes evolved alongside scientific advancements. Record-breaking approvals and biotech IPOs underscored CGT's economic potential, while affordability and equitable access emerged as critical challenges. Regulatory agencies advanced harmonized guidelines for manufacturing and clinical evaluation, streamlining global access to these therapies. Ethical considerations, including the affordability of therapies and the need for diverse clinical trial representation, remained prominent. Despite progress, challenges persist in scalability, safety and regulatory harmonization. Manufacturing improvements are essential to meet growing demand, while addressing safety concerns, such as off-target gene-editing effects and tumorigenicity in MSC therapies, remains paramount.
期刊介绍：
The journal brings readers the latest developments in the fast moving field of cellular therapy in man. This includes cell therapy for cancer, immune disorders, inherited diseases, tissue repair and regenerative medicine. The journal covers the science, translational development and treatment with variety of cell types including hematopoietic stem cells, immune cells (dendritic cells, NK, cells, T cells, antigen presenting cells) mesenchymal stromal cells, adipose cells, nerve, muscle, vascular and endothelial cells, and induced pluripotential stem cells. We also welcome manuscripts on subcellular derivatives such as exosomes. A specific focus is on translational research that brings cell therapy to the clinic. Cytotherapy publishes original papers, reviews, position papers editorials, commentaries and letters to the editor. We welcome "Protocols in Cytotherapy" bringing standard operating procedure for production specific cell types for clinical use within the reach of the readership.
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