Single enzyme nanoparticle, an effective tool for enzyme replacement therapy

Dong Hyun Kim, Han Sol Lee, Tae Wan Kwon, Young Min Han, Nae Won Kang, Mee Yeon Lee, Dae Duk Kim, Myeong Gyu Kim, Jae Young Lee

Research output: Contribution to journalReview articlepeer-review

6 Scopus citations

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.

Original languageEnglish
JournalArchives of Pharmacal Research
Volume43
Issue number1
DOIs
StatePublished - 1 Jan 2020

Keywords

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

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