TY - JOUR
T1 - A Guide to Chemical Considerations for the Pre-Clinical Development of Oligonucleotides
AU - on behalf of the N = 1 Collaborative
AU - O’Reilly, Daniel
AU - van Roon-Mom, Willeke
AU - Aartsma-Rus, Annemieke
N1 - Publisher Copyright:
Copyright 2024, Mary Ann Liebert, Inc., publishers.
PY - 2024/12/1
Y1 - 2024/12/1
N2 - Oligonucleotide therapeutics, a pioneering category of modern medicinal drugs, are at the forefront of utilizing innate mechanisms to modulate gene expression. With 18 oligonucleotide-based FDA-approved medicines currently available for treating various clinical conditions, this field showcases an innovative potential yet to be fully explored. Factors such as purity, formulation, and endotoxin levels profoundly influence the efficacy and safety of these therapeutics. Therefore, a thorough understanding of the chemical factors essential for producing high-quality oligonucleotides for preclinical studies is crucial in their development for further clinical application. This paper serves as a concise guide to these chemical considerations, aiming to inspire and equip researchers with the necessary knowledge to advance in this exciting and innovative field.
AB - Oligonucleotide therapeutics, a pioneering category of modern medicinal drugs, are at the forefront of utilizing innate mechanisms to modulate gene expression. With 18 oligonucleotide-based FDA-approved medicines currently available for treating various clinical conditions, this field showcases an innovative potential yet to be fully explored. Factors such as purity, formulation, and endotoxin levels profoundly influence the efficacy and safety of these therapeutics. Therefore, a thorough understanding of the chemical factors essential for producing high-quality oligonucleotides for preclinical studies is crucial in their development for further clinical application. This paper serves as a concise guide to these chemical considerations, aiming to inspire and equip researchers with the necessary knowledge to advance in this exciting and innovative field.
KW - chemical engineering
KW - oligonucleotide synthesis
KW - pre-clinical guidelines
UR - http://www.scopus.com/inward/record.url?scp=85201075907&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85201075907&partnerID=8YFLogxK
U2 - 10.1089/nat.2024.0031
DO - 10.1089/nat.2024.0031
M3 - Article
C2 - 39110607
AN - SCOPUS:85201075907
SN - 2159-3337
VL - 34
SP - 295
EP - 298
JO - Nucleic Acid Therapeutics
JF - Nucleic Acid Therapeutics
IS - 6
ER -