Chapter 6
Aptamers for Targeted Therapy
Ge Yang and Yuanyu Huang
Abstract This chapter introduces the aptamers for targeted therapy. Aptamers
show promising efficacy and safety in four types of clinical studies of ophthalmopathy, blood coagulation, inflammation, and cancer, and there are increasing
numbers of evidences to prove the clinical application potential of aptamers. To
date, more than a dozen aptamers have entered clinical trials as therapeutic agents
for targeted therapy. This chapter introduces their pharmacological development and
optimization processes and reviews potential treatment of diseases.
Keywords Aptamers · Target therapy · Oculopathy · Coagulation · Inflammation ·
Cancer
6.1 Introduction
The aptamer-based targeted therapeutics can be divided into the following three
approaches according to aptamers’ function mechanisms: (1) The aptamer directly
acts as the therapeutic agent to block the function of target through interaction with
it and participates in the treatment of related disease; (2) The aptamer acts as an
agonist, activates its target function, and indirectly participates in disease treatment;
(3) The aptamer serves as a targeting element to constitute a delivery vehicle for
other therapeutic agents.
In 2004, U.S. Food and Drug Administration (FDA) approved the first RNA
aptamer drug Macugen (Pipergatanil), for the treatment of wet form of agerelated macular degeneration (AMD). Macugen is the only currently commercialized
aptamer therapeutic agent, which was first developed by NeXstar and acquired by
EyeTech, and then jointly developed and sold by OSI-Eytech and Pfizer. To date, more
G. Y. wrote the manuscript, and Y. H. supervised the project.
G. Yang · Y. Huang (B)
Advanced Research Institute of Multidisciplinary Science, School of Life Science, Institute
of Engineering Medicine, Key Laboratory of Molecular Medicine and Biotherapy, Beijing
Institute of Technology, Beijing 100081, China
e-mail: yyhuang@bit.edu.cn
© Springer Nature Singapore Pte Ltd. 2021
Y. Dong (ed.), Aptamers for Medical Applications,
https://doi.org/10.1007/978-981-33-4838-7_6
129
Aptamers for Targeted Therapy
Ge Yang and Yuanyu Huang
Abstract This chapter introduces the aptamers for targeted therapy. Aptamers
show promising efficacy and safety in four types of clinical studies of ophthalmopathy, blood coagulation, inflammation, and cancer, and there are increasing
numbers of evidences to prove the clinical application potential of aptamers. To
date, more than a dozen aptamers have entered clinical trials as therapeutic agents
for targeted therapy. This chapter introduces their pharmacological development and
optimization processes and reviews potential treatment of diseases.
Keywords Aptamers · Target therapy · Oculopathy · Coagulation · Inflammation ·
Cancer
6.1 Introduction
The aptamer-based targeted therapeutics can be divided into the following three
approaches according to aptamers’ function mechanisms: (1) The aptamer directly
acts as the therapeutic agent to block the function of target through interaction with
it and participates in the treatment of related disease; (2) The aptamer acts as an
agonist, activates its target function, and indirectly participates in disease treatment;
(3) The aptamer serves as a targeting element to constitute a delivery vehicle for
other therapeutic agents.
In 2004, U.S. Food and Drug Administration (FDA) approved the first RNA
aptamer drug Macugen (Pipergatanil), for the treatment of wet form of agerelated macular degeneration (AMD). Macugen is the only currently commercialized
aptamer therapeutic agent, which was first developed by NeXstar and acquired by
EyeTech, and then jointly developed and sold by OSI-Eytech and Pfizer. To date, more
G. Y. wrote the manuscript, and Y. H. supervised the project.
G. Yang · Y. Huang (B)
Advanced Research Institute of Multidisciplinary Science, School of Life Science, Institute
of Engineering Medicine, Key Laboratory of Molecular Medicine and Biotherapy, Beijing
Institute of Technology, Beijing 100081, China
e-mail: yyhuang@bit.edu.cn
© Springer Nature Singapore Pte Ltd. 2021
Y. Dong (ed.), Aptamers for Medical Applications,
https://doi.org/10.1007/978-981-33-4838-7_6
129