Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2020 Jan 14;11(3):228-231.
doi: 10.1021/acsmedchemlett.9b00582. eCollection 2020 Mar 12.

New Chemical Modalities and Strategic Thinking in Early Drug Discovery

Affiliations

New Chemical Modalities and Strategic Thinking in Early Drug Discovery

Maria-Jesus Blanco et al. ACS Med Chem Lett. .

Abstract

The classical toolbox for drug discovery is continuously expanding beyond traditional small molecules. New chemical modalities including RNA therapeutics, protein degraders, cyclopeptides, antibody drug conjugates, and gene therapy have matured, demonstrating clinical success and are now considered early in target appraisal. In this Viewpoint, we highlight recent progress in the field.

PubMed Disclaimer

Conflict of interest statement

The authors declare no competing financial interest.

Figures

Figure 1
Figure 1
Representation of three cornerstones of target validation and opportunities within the chemical modality toolbox.

References

    1. Disney M. D. Targeting RNA with small molecules to capture opportunities at the intersection of chemistry, biology, and medicine. J. Am. Chem. Soc. 2019, 141, 6776–90. 10.1021/jacs.8b13419. - DOI - PMC - PubMed
    1. Elbashir S. M.; Harborth J.; Lendeckel W.; Yalcin A.; Weber K.; Tuschl T. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 2001, 411, 494.10.1038/35078107. - DOI - PubMed
    1. Al Shaer D.; Al Musaimi O.; Albericio F.; de la Torre B. G. 2018 FDA tides harvest. Pharmaceuticals 2019, 12, 52.10.3390/ph12020052. - DOI - PMC - PubMed
    1. Stein C. A.; Castanotto D. FDA-approved oligonucleotide therapies in 2017. Mol. Ther. 2017, 25, 1069–1075. 10.1016/j.ymthe.2017.03.023. - DOI - PMC - PubMed
    1. Wan W. B.; Seth P. P. The medicinal chemistry of therapeutic oligonucleotides. J. Med. Chem. 2016, 59, 9645–9667. 10.1021/acs.jmedchem.6b00551. - DOI - PubMed