Open AccessRisdiplam, the First Approved Small Molecule Splicing Modifier Drug as a Blueprint for Future Transformative Medicines
Author(s) -
Hasane Ratni,
Renata S. Scalco,
Alexander Stephan
Publication year - 2021
Publication title -
acs medicinal chemistry letters
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.065
H-Index - 66
ISSN - 1948-5875
DOI - 10.1021/acsmedchemlett.0c00659
Subject(s) - blueprint , spinal muscular atrophy , small molecule , rna splicing , transformative learning , drug discovery , sma* , drug development , computational biology , medicine , nanotechnology , drug , disease , computer science , data science , engineering ethics , pharmacology , bioinformatics , chemistry , psychology , biology , rna , engineering , materials science , biochemistry , gene , mechanical engineering , pedagogy , algorithm
Not too long ago, the concept of selectively targeting mRNA with small molecules was perceived as a formidable scientific challenge. The discovery of small molecule splicing modifiers and the development of risdiplam for the treatment of spinal muscular atrophy (SMA) have firmly established proof of concept for this exciting new platform and transformed a scientific curiosity into a viable technology to target disease. Today, several approaches to target mRNA with small molecules, supported by biophysical and screening methods, are in place to deliver new drugs with high therapeutic relevance.