Abstract
The term “single enzyme nanoparticle” (SEN) refers to a chemically or biologically engineered single enzyme molecule. SENs are distinguished from conventional protein nanoparticles in that they can maintain their individual structure and enzymatic activity following modification. Furthermore, SENs exhibit enhanced properties as biopharmaceuticals, such as reduced antigenicity, and increased stability and targetability, which are attributed to the introduction of specific moieties, such as poly(ethylene glycol), carbohydrates, and antibodies. Enzyme replacement therapy (ERT) is a crucial therapeutic option for controlling enzyme-deficiency-related disorders. However, the unfavorable properties of enzymes, including immunogenicity, lack of targetability, and instability, can undermine the clinical significance of ERT. As shown in the cases of Adagen®, Revcovi®, Palynziq®, and Strensiq®, SEN can be an effective technology for overcoming these obstacles. Based on these four licensed products, we expect that additional SENs will be introduced for ERT in the near future. In this article, we review the concepts and features of SENs, as well as their preparation methods. Additionally, we summarize different types of enzyme deficiency disorders and the corresponding therapeutic enzymes. Finally, we focus on the current status of SENs in ERT by reviewing FDA-approved products.
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This work was supported by the National Research Foundation of Korea (NRF) grants funded by the Ministry of Science and ICT (NRF-2018R1C1B6005379 and NRF-2018M3A7B4071204).
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Kim, D.H., Lee, H.S., Kwon, TW. et al. Single enzyme nanoparticle, an effective tool for enzyme replacement therapy. Arch. Pharm. Res. 43, 1–21 (2020). https://doi.org/10.1007/s12272-020-01216-3
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DOI: https://doi.org/10.1007/s12272-020-01216-3