Clinical trials to gene therapy development and production in Brazil: a review
Summary: Challenges related to the implementation of gene therapy products are daunting for low-to middle-income countries, such as Brazil, and include the creation of an appropriate technical, regulatory, and economic environment. In this manuscript, we give an overview of historical aspects, as we...
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| Format: | Article |
| Language: | English |
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Elsevier
2025-03-01
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| Series: | The Lancet Regional Health. Americas |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S2667193X25000055 |
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| author | Jonas Alex Morales Saute Virginia Picanço-Castro Ana Carolina de Freitas Lopes João Batista da Silva Júnior Johanna Henriques Nehm Franciele dos Santos Maciel Ida Vanessa Doederlein Schwartz Juliana Maria Ferraz Sallum Margareth Castro Ozelo Roberto Giugliani Martin Bonamino Hilda Petrs-Silva Guilherme Baldo |
| author_facet | Jonas Alex Morales Saute Virginia Picanço-Castro Ana Carolina de Freitas Lopes João Batista da Silva Júnior Johanna Henriques Nehm Franciele dos Santos Maciel Ida Vanessa Doederlein Schwartz Juliana Maria Ferraz Sallum Margareth Castro Ozelo Roberto Giugliani Martin Bonamino Hilda Petrs-Silva Guilherme Baldo |
| author_sort | Jonas Alex Morales Saute |
| collection | DOAJ |
| description | Summary: Challenges related to the implementation of gene therapy products are daunting for low-to middle-income countries, such as Brazil, and include the creation of an appropriate technical, regulatory, and economic environment. In this manuscript, we give an overview of historical aspects, as well as the current state of clinical trials and approved gene therapy products for commercialization in Brazil. Focusing on gene replacement and CAR-T cell therapies, we discuss the main advances, limitations, and difficulties faced by the country in the production, approval, and incorporation of such products into the public health system. Finally, we highlight the potential leading role that low-to middle-income countries can have in this industry, not only by providing their own vector supply but also by addressing important issues related to the sustainability and long-term global affordability of gene therapy products. |
| format | Article |
| id | doaj-art-9c88a7baf6be44a6b3668828eb09a59e |
| institution | Kabale University |
| issn | 2667-193X |
| language | English |
| publishDate | 2025-03-01 |
| publisher | Elsevier |
| record_format | Article |
| series | The Lancet Regional Health. Americas |
| spelling | doaj-art-9c88a7baf6be44a6b3668828eb09a59e2025-01-31T05:12:39ZengElsevierThe Lancet Regional Health. Americas2667-193X2025-03-0143100995Clinical trials to gene therapy development and production in Brazil: a reviewJonas Alex Morales Saute0Virginia Picanço-Castro1Ana Carolina de Freitas Lopes2João Batista da Silva Júnior3Johanna Henriques Nehm4Franciele dos Santos Maciel5Ida Vanessa Doederlein Schwartz6Juliana Maria Ferraz Sallum7Margareth Castro Ozelo8Roberto Giugliani9Martin Bonamino10Hilda Petrs-Silva11Guilherme Baldo12Medical Genetics Service, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil; Grupo de Pesquisa Clínica em Genética Médica, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil; Graduate Program in Medicine, Medical Sciences, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil; Department of Internal Medicine, Universidade Federal do Rio Grande do Sul, Porto Alegre, Porto Alegre, Brazil; Instituto Nacional de Doenças Raras-InRaras, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil; Corresponding author. Medical Genetics Service, Hospital de Clínicas de Porto Alegre Ramiro Barcelos, 2350, Zip-code 90035-903, Porto Alegre, Brazil.Center for Cell-based Therapy, Regional Blood Center of Ribeirão Preto, University of São Paulo, Ribeirão Preto, BrazilExecutive Secretariat of the National Committee for Health Technology Incorporation (Conitec), Brazilian Ministry of Health, Brasilia, Brazil; Programa de Pós-Graduação em Saúde Coletiva, Faculdade de Medicina, Universidade de São Paulo, São Paulo, BrazilNational Health Surveillance Agency (Anvisa), Brasilia, Brazil; Department of Pharmaceutical Sciences, College of Health Sciences, University of Brasilia, Brasilia, Brazil; National Network of Experts in Advanced Therapies (Reneta), Anvisa, Brasilia, BrazilGraduate Program in Medicine, Medical Sciences, Universidade Federal do Rio Grande do Sul, Porto Alegre, BrazilGraduate Program in Medicine, Medical Sciences, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil; Department of Internal Medicine, Universidade Federal do Rio Grande do Sul, Porto Alegre, Porto Alegre, BrazilMedical Genetics Service, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil; Instituto Nacional de Doenças Raras-InRaras, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil; Departamento de Genética, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil; Programa de Pós-Graduação em Genética e Biologia Molecular, Universidade Federal do Rio Grande do Sul, Porto Alegre, BrazilDepartment of Ophthalmology, Federal University of São Paulo, São Paulo, BrazilINCT Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, SP, BrazilMedical Genetics Service, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil; Grupo de Pesquisa Clínica em Genética Médica, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil; Graduate Program in Medicine, Medical Sciences, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil; Departamento de Genética, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil; Programa de Pós-Graduação em Genética e Biologia Molecular, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil; Instituto Nacional de Genética Médica Populacional-INAGEMP, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil; Casa dos Raros, Porto Alegre, Brazil; Dasa Genômica, São Paulo, BrazilNational Network of Experts in Advanced Therapies (Reneta), Anvisa, Brasilia, Brazil; Instituto Nacional do Câncer, Coordenação de Pesquisa, Rio de Janeiro, BrazilNational Network of Experts in Advanced Therapies (Reneta), Anvisa, Brasilia, Brazil; Laboratory of Gene Therapy and Viral Vector, Carlos Chagas Filho Institute of Biophysics, Universidade Federal do Rio de Janeiro, Rio de Janeiro, BrazilNational Network of Experts in Advanced Therapies (Reneta), Anvisa, Brasilia, Brazil; Programa de Pós-Graduação em Genética e Biologia Molecular, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil; Cell, Tissue and Gene Laboratory, Experimental Research Center, Hospital de Clínicas de Porto Alegre, Porto Alegre, RS, Brazil; Programa de Pós-Graduação em Ciências Biológicas: Fisiologia, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil; Departamento de Fisiologia, Universidade Federal do Rio Grande do Sul, Porto Alegre, BrazilSummary: Challenges related to the implementation of gene therapy products are daunting for low-to middle-income countries, such as Brazil, and include the creation of an appropriate technical, regulatory, and economic environment. In this manuscript, we give an overview of historical aspects, as well as the current state of clinical trials and approved gene therapy products for commercialization in Brazil. Focusing on gene replacement and CAR-T cell therapies, we discuss the main advances, limitations, and difficulties faced by the country in the production, approval, and incorporation of such products into the public health system. Finally, we highlight the potential leading role that low-to middle-income countries can have in this industry, not only by providing their own vector supply but also by addressing important issues related to the sustainability and long-term global affordability of gene therapy products.http://www.sciencedirect.com/science/article/pii/S2667193X25000055Gene therapy productsGene replacement therapyCAR-T cellClinical trialsLow-to middle-income countries |
| spellingShingle | Jonas Alex Morales Saute Virginia Picanço-Castro Ana Carolina de Freitas Lopes João Batista da Silva Júnior Johanna Henriques Nehm Franciele dos Santos Maciel Ida Vanessa Doederlein Schwartz Juliana Maria Ferraz Sallum Margareth Castro Ozelo Roberto Giugliani Martin Bonamino Hilda Petrs-Silva Guilherme Baldo Clinical trials to gene therapy development and production in Brazil: a review The Lancet Regional Health. Americas Gene therapy products Gene replacement therapy CAR-T cell Clinical trials Low-to middle-income countries |
| title | Clinical trials to gene therapy development and production in Brazil: a review |
| title_full | Clinical trials to gene therapy development and production in Brazil: a review |
| title_fullStr | Clinical trials to gene therapy development and production in Brazil: a review |
| title_full_unstemmed | Clinical trials to gene therapy development and production in Brazil: a review |
| title_short | Clinical trials to gene therapy development and production in Brazil: a review |
| title_sort | clinical trials to gene therapy development and production in brazil a review |
| topic | Gene therapy products Gene replacement therapy CAR-T cell Clinical trials Low-to middle-income countries |
| url | http://www.sciencedirect.com/science/article/pii/S2667193X25000055 |
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