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Mol Ther Nucleic Acids. 2017 Sep 15;8:132-143. doi: 10.1016/j.omtn.2017.06.005. Epub 2017 Jun 12.

Therapeutic miRNA and siRNA: Moving from Bench to Clinic as Next Generation Medicine.

Author information

1
Department of Bioinformatics and Biochemistry, Galgotias University, Greater Noida 201306, Uttar Pradesh, India; Institute for Skeletal Aging & Orthopedic Surgery, Hallym University-Chuncheon Sacred Heart Hospital, Chuncheon 24252, Republic of Korea. Electronic address: drchiranjib@yahoo.com.
2
Institute for Skeletal Aging & Orthopedic Surgery, Hallym University-Chuncheon Sacred Heart Hospital, Chuncheon 24252, Republic of Korea.
3
Department of Integrative Biology, VIT University, Vellore 632014, Tamil Nadu, India.
4
Institute for Skeletal Aging & Orthopedic Surgery, Hallym University-Chuncheon Sacred Heart Hospital, Chuncheon 24252, Republic of Korea. Electronic address: 123sslee@gmail.com.

Abstract

In the past few years, therapeutic microRNA (miRNA) and small interfering RNA (siRNA) are some of the most important biopharmaceuticals that are in commercial space as future medicines. This review summarizes the patents of miRNA- and siRNA-based new drugs, and also provides a snapshot about significant biopharmaceutical companies that are investing for the therapeutic development of miRNA and siRNA molecules. An insightful view about individual siRNA and miRNA drugs has been depicted with their present status, which is gaining attention in the therapeutic landscape. The efforts of the biopharmaceuticals are discussed with the status of their preclinical and/or clinical trials. Here, some of the setbacks have been highlighted during the biopharmaceutical development of miRNA and siRNA as individual therapeutics. Finally, a snapshot is illustrated about pharmacokinetics, pharmacodynamics with absorption, distribution, metabolism, and excretion (ADME), which is the fundamental development process of these therapeutics, as well as the delivery system for miRNA- and siRNA-based drugs.

KEYWORDS:

drug development; miRNA; siRNA

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