Advancing the Research and Development of Enzyme Replacement Therapies for Lysosomal Storage Diseases.
Advancing the Research and Development of Enzyme Replacement Therapies for Lysosomal Storage Diseases.
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DOI：
10.1089/genbio.2021.0013
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发表时间：
2022-04
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--
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随着人们对开发罕见病基因疗法的兴趣越来越大，人们很容易忽视许多罕见的溶酶体贮积病（LSD）的治疗方法已被美国和欧洲的监管机构批准。这些主要包括酶替代疗法（ERT），这是重组人蛋白，通过不同的途径在患者的生命中提供，与基因疗法相比，可能具有明显的安全性和分布优势。ERT的研究和开发是一个漫长而昂贵的过程，通常在转移到制药公司之前在学术实验室进行，因此是一个成熟的中断过程。ERT可能仍有相当大的科学和投资潜力，但我们需要开发类似于在一些开放科学努力中创造的蛋白质管道，并应用技术降低制造成本。在这个角度来看，我们说明了机会，以填补罕见的LSD治疗空白与Erts，而基因疗法正在开发这些缩短生命的疾病。
With the increasing interest in developing gene therapies for rare diseases, it is easy to overlook that there are numerous rare lysosomal storage diseases (LSD) with treatments that have been approved by regulatory agencies in the United States and Europe. These primarily consist of enzyme replacement therapies (ERT), which are recombinant human proteins that are delivered for the life of the patient via different routes and may have distinct safety and distribution advantages over gene therapies. The research and development of ERT is a lengthy and expensive process, which is usually performed in academic laboratories before transfer to pharmaceutical companies and is hence a process ripe for disruption. There may still be considerable scientific and investment potential for ERT, however we need to develop a pipeline of proteins analogous to what has been created in some open science efforts as well as apply technologies to decrease manufacturing costs. In this Perspective, we illustrate the opportunity to fill the rare LSD treatment gap with ERTs while gene therapies are in development for these life-shortening diseases.
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