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Cell & gene therapy insights最新文献

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Developing non-viral delivery & cell engineering technologies for ex vivo T cell immunotherapies 开发用于体外T细胞免疫治疗的非病毒递送和细胞工程技术
Pub Date : 2023-07-19 DOI: 10.18609/cgti.2023.091
Julie Shi, N. Bhattacharjee
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引用次数: 0
Investing long-term in the cell & gene therapies of the future 对未来的细胞和基因疗法进行长期投资
Pub Date : 2023-07-19 DOI: 10.18609/cgti.2023.047
Geeta Vemuri
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引用次数: 0
Driving the expansion of mRNA into the therapeutic sphere 推动信使核糖核酸向治疗领域的扩展
Pub Date : 2023-07-19 DOI: 10.18609/cgti.2023.098
Alejandro J. Becerra, Andreas Kuhn, Metin Kurtoglu
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引用次数: 0
An optimized & streamlined approach for upstream & downstream lentiviral production 优化和流线型的方法为上游和下游慢病毒生产
Pub Date : 2023-07-19 DOI: 10.18609/cgti.2023.092
Gregory Piscitello, Paul Turiano
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引用次数: 0
Gene therapy CMC and analytics—Foreword 基因治疗CMC与分析——前言
Pub Date : 2023-07-19 DOI: 10.18609/cgti.2023.113
Lauren Drouin
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引用次数: 0
Facing potential chemistry, manufacturing, & control (CMC) development challenges with recombinant adeno-associated viral vectors: available regulatory guidance & recommendations 面对重组腺相关病毒载体潜在的化学、制造和控制(CMC)开发挑战:可用的监管指导和建议
Pub Date : 2023-07-19 DOI: 10.18609/cgti.2023.101
Stuart Beattie
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引用次数: 0
Can novel bioreactors improve the cost of goods of viral vectors? 新型生物反应器能提高病毒载体的商品成本吗?
Pub Date : 2023-07-19 DOI: 10.18609/cgti.2023.087
C. Stamatis, A. Chatel, S. Farid
Lentiviral and adeno-associated viral vectors make up the vast majority of gene therapy candidates for in-vivo and in-vitro applications. While effective for treating a range of debilitating diseases, they are also currently very expensive to produce, which hampers patient accessibility. While other biologics have been studied and optimized for several decades, viral vectors still suffer from relatively low titers, difficulty in scaling up and poor downstream recovery. A review of available technologies focusing on upstream solutions highlights that despite the development of randomly packed bed bioreactors for adherent cells and the move to suspension cell cultures in stirred tank bioreactors, technology design flaws hamper efforts to cost-effectively bring new therapies to the market. In this paper, the scale-X™ and NevoLine™ technologies are shown to provide conditions that support two to ten-fold increase in cell specific productivity for AAV and LVV relative to alternative technologies, which results in drug substance cost of goods reduction between −18% and −61%. Furthermore, increased titers, smaller footprint and reduced complexity could improve the efficacy of facility utilization.
慢病毒和腺相关病毒载体构成了体内和体外应用的绝大多数基因治疗候选物。虽然它们对治疗一系列使人衰弱的疾病有效,但目前它们的生产成本也非常昂贵,这阻碍了患者的获取。虽然其他生物制剂已经研究和优化了几十年,但病毒载体仍然存在滴度相对较低、扩大规模困难和下游回收能力差的问题。对上游解决方案的现有技术的回顾强调,尽管针对贴壁细胞的随机填充床生物反应器的发展以及在搅拌槽生物反应器中悬浮细胞培养的发展,但技术设计缺陷阻碍了将新疗法经济有效地推向市场的努力。在本文中,scale-X™和NevoLine™技术提供的条件可以使AAV和LVV的细胞特异性生产力比替代技术提高2到10倍,从而使原料药成本降低- 18%到- 61%。此外,提高滴度、减少占地面积和降低复杂性可以提高设施利用效率。
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引用次数: 0
Leveraging derisked AAV vector development & delivery approaches to enhance speed to market for rare disease gene therapies 利用无风险的AAV载体开发和递送方法,加快罕见病基因疗法的上市速度
Pub Date : 2023-07-07 DOI: 10.18609/cgti.2023.063
A. Lemoine
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引用次数: 0
Navigating the gene & cell therapy regulatory landscape: when, why and how to deploy GMP gene editing materials 导航基因和细胞治疗监管景观:何时,为什么以及如何部署GMP基因编辑材料
Pub Date : 2023-07-07 DOI: 10.18609/cgti.2023.076
Michael Lau
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引用次数: 0
Cell culture in immune cell therapies: do more with less 免疫细胞疗法中的细胞培养:事半功倍
Pub Date : 2023-07-07 DOI: 10.18609/cgti.2023.070
Anastasiya Smith, Josh Ludwig, D. Hermanson
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引用次数: 0
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Cell & gene therapy insights
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