The Molecular Immunology Unit, Infection and Immunity Research Centre, St. George's University of London, Cranmer Terrace, London SW17 0RE, United Kingdom.
Curr Pharm Des. 2013;19(31):5478-85. doi: 10.2174/1381612811319310003.
Four major developments have taken place in the world of Molecular Pharming recently. In the USA, the DARPA initiative challenged plant biotechnology companies to develop strategies for the large-scale manufacture of influenza vaccines, resulting in a successful Phase I clinical trial; in Europe the Pharma-Planta academic consortium gained regulatory approval for a plant-derived monoclonal antibody and completed a first-in-human phase I clinical trial; the Dutch pharmaceutical company Synthon acquired the assets of Biolex Therapeutics, an established Molecular Pharming company with several clinical candidates produced in their proprietary LEX system based on aquatic plants; and finally, the Israeli biotechnology company Protalix Biotherapeutics won FDA approval for the commercial release of a recombinant form of the enzyme glucocerebrosidase produced in carrot cells, the first plant biotechnology-derived biopharmaceutical in the world approved for the market. Commercial momentum is gathering pace with additional candidates now undergoing or awaiting approval for phase III clinical trials. Filling the product pipeline is vital to establish commercial sustainability, and the selection of appropriate target products for Molecular Pharming will be a critical factor. An interesting feature of the four stories outlined above is that they span the use of very different platform technologies addressing different types of molecules which aim to satisfy distinct market demands. In each case, Molecular Pharming was an economically and technically suitable approach, but this decisionmaking process is not necessarily straightforward. Although the various technologies available to Molecular Pharming are broad ranging and flexible, competing technologies are better established, so there needs to be a compelling reason to move into plants. It is most unlikely that plant biotechnology will be the answer for the whole biologics field. In this article, we discuss the current plant biotechnology approaches that appear to hold the greatest promise and in doing so attempt to define the product areas that are most likely to benefit from different Molecular Pharming technologies.
最近，分子农业领域发生了四大进展。在美国，DARPA 计划挑战植物生物技术公司，开发大规模生产流感疫苗的策略，成功进行了一期临床试验；在欧洲，Pharma-Planta 学术联盟获得监管机构批准，可生产植物来源的单克隆抗体，并完成了首例人体一期临床试验；荷兰制药公司 Synthon 收购了 Molecular Pharming 公司 Biolex Therapeutics 的资产，该公司拥有多个临床候选药物，这些候选药物是在其基于水生植物的专有 LEX 系统中生产的；最后，以色列生物技术公司 Protalix Biotherapeutics 获得 FDA 批准，可商业发布在胡萝卜细胞中生产的重组葡萄糖脑苷脂酶，这是世界上第一个获得市场批准的植物生物技术衍生的生物制药。随着更多候选药物正在进行或等待三期临床试验的批准，商业势头正在加快。填补产品管线对于建立商业可持续性至关重要，而选择适合 Molecular Pharming 的适当目标产品将是一个关键因素。上述四个故事的一个有趣特点是，它们跨越了使用不同平台技术的范围，这些技术针对不同类型的分子，旨在满足不同的市场需求。在每种情况下，Molecular Pharming 都是一种经济和技术上合适的方法，但这种决策过程并不一定简单。尽管 Molecular Pharming 可用的各种技术范围广泛且灵活，但竞争技术更为成熟，因此需要有令人信服的理由进入植物领域。植物生物技术不太可能成为整个生物制品领域的答案。在本文中，我们讨论了目前似乎最有前途的植物生物技术方法，并试图确定最有可能受益于不同 Molecular Pharming 技术的产品领域。
