Analytical techniques currently used in the pharmaceutical industry for the quality control of RNA-based therapeutics and ongoing developments.

Liquid chromatography; Mass spectrometry; Nucleic acids; PO/PS ratio; Phosphorothioate; Quality control; RNA, Double-Stranded; Clinical Trials as Topic; Guidelines as Topic; Humans; Quality Control; RNA, Double-Stranded/chemistry; RNA, Double-Stranded/standards; RNA, Double-Stranded/therapeutic use; Chemistry, Analytic; Drug Industry; Antisense oligonucleotides; Clinical trial; Mechanism of action; Pharmaceutical industry; Phosphorothioates; Quality testing; RNA therapeutics; Small interfering RNA; Therapeutic efficacy; Analytical Chemistry; Biochemistry; Organic Chemistry; General Medicine

Abstract :

[en] The number of RNA-based therapeutics has significantly grown in number on the market over the last 20 years. This number is expected to further increase in the coming years as many RNA therapeutics are being tested in late clinical trials stages. The first part of this paper considers the mechanism of action, the synthesis and the potential impurities resulting from synthesis as well as the strategies used to increase RNA-based therapeutics efficacy. In the second part of this review, the tests that are usually performed in the pharmaceutical industry for the quality testing of antisense oligonucleotides (ASOs), small-interfering RNAs (siRNAs) and messenger RNAs (mRNAs) will be described. In the last part, the remaining challenges and the ongoing developments to meet them are discussed.

Disciplines :

Pharmacy, pharmacology & toxicology

Author, co-author :

Demelenne, Alice ; Université de Liège - ULiège > Faculté de Médecine > Doct. sc. bioméd. & pharma. (paysage)

Servais, Anne-Catherine ; Université de Liège - ULiège > Unités de recherche interfacultaires > Centre Interdisciplinaire de Recherche sur le Médicament (CIRM)

Crommen, Jacques ; Université de Liège - ULiège > Département de pharmacie

Fillet, Marianne ; Université de Liège - ULiège > Unités de recherche interfacultaires > Centre Interdisciplinaire de Recherche sur le Médicament (CIRM)

Language :

English

Title :

Analytical techniques currently used in the pharmaceutical industry for the quality control of RNA-based therapeutics and ongoing developments.

Publication date :

16 August 2021

Journal title :

Journal of Chromatography. A

ISSN :

0021-9673

eISSN :

1873-3778

Publisher :

Elsevier B.V., Netherlands

Volume :

1651

Pages :

462283

Peer reviewed :

Peer Reviewed verified by ORBi

Additional URL :

https://api.elsevier.com/content/article/PII:S0021967321004076?httpAccept=text/xml

Funders :

ULiège - Université de Liège

Available on ORBi :

since 10 March 2023

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