TY - JOUR
T1 - Single enzyme nanoparticle, an effective tool for enzyme replacement therapy
AU - Kim, Dong Hyun
AU - Lee, Han Sol
AU - Kwon, Tae Wan
AU - Han, Young Min
AU - Kang, Nae Won
AU - Lee, Mee Yeon
AU - Kim, Dae Duk
AU - Kim, Myeong Gyu
AU - Lee, Jae Young
N1 - Publisher Copyright:
© 2020, The Pharmaceutical Society of Korea.
PY - 2020/1/1
Y1 - 2020/1/1
N2 - 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.
AB - 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.
KW - Biologics
KW - Enzyme deficiency
KW - Enzyme replacement therapy
KW - Enzyme therapeutics
KW - Single enzyme nanoparticle
UR - http://www.scopus.com/inward/record.url?scp=85078501288&partnerID=8YFLogxK
U2 - 10.1007/s12272-020-01216-3
DO - 10.1007/s12272-020-01216-3
M3 - Review article
C2 - 31989476
AN - SCOPUS:85078501288
SN - 0253-6269
VL - 43
JO - Archives of Pharmacal Research
JF - Archives of Pharmacal Research
IS - 1
ER -