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Single enzyme nanoparticle, an effective tool for enzyme replacement therapy

Archives of Pharmacal Research 2020년 43권 1호 p.1 ~ 21
 ( Kim Dong-Hyun ) - Chungnam National University College of Pharmacy

이한솔 ( Lee Han-Sol ) - Chungnam National University College of Pharmacy
 ( Kwon Tae-Wan ) - Chungnam National University College of Pharmacy
 ( Han Young-Min ) - Chungnam National University College of Pharmacy
 ( Kang Nae-Won ) - Seoul National University College of Pharmacy
 ( Lee Mee-Yeon ) - Seoul National University Seoul Metropolitan Government Boramae Medical Center Department of Pharmacy
김대덕 ( Kim Dae-Duk ) - Seoul National University College of Pharmacy
 ( Kim Myeong-Gyu ) - CHA University Graduate School of Clinical Pharmacy
이재영 ( Lee Jae-Young ) - Chungnam National University College of Pharmacy

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.

키워드

Single enzyme nanoparticle; Biologics; Enzyme therapeutics; Enzyme deficiency; Enzyme replacement therapy
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