Morales Saute Jonas Alex, Picanço-Castro Virginia, de Freitas Lopes Ana Carolina, da Silva Júnior João Batista, Henriques Nehm Johanna, Dos Santos Maciel Franciele, Doederlein Schwartz Ida Vanessa, Ferraz Sallum Juliana Maria, Castro Ozelo Margareth, Giugliani Roberto, Bonamino Martin, Petrs-Silva Hilda, Baldo Guilherme
Medical 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.
Lancet Reg Health Am. 2025 Jan 30;43:100995. doi: 10.1016/j.lana.2025.100995. eCollection 2025 Mar.
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.
对于巴西等中低收入国家而言，与基因治疗产品实施相关的挑战艰巨，其中包括营造适宜的技术、监管和经济环境。在本手稿中，我们概述了历史方面的情况，以及巴西目前的临床试验状况和已获批用于商业化的基因治疗产品。聚焦于基因替代疗法和嵌合抗原受体T细胞（CAR-T）疗法，我们讨论了该国在生产、审批此类产品并将其纳入公共卫生系统过程中取得的主要进展、存在的局限和困难。最后，我们强调了中低收入国家在该行业可能发挥的潜在引领作用，这不仅体现在提供自身的载体供应方面，还体现在解决与基因治疗产品的可持续性和全球长期可负担性相关的重要问题上。
Zotero 插件
