Risdiplam, the First Approved Small Molecule Splicing Modifier Drug as a Blueprint for Future Transformative Medicines
Hasane Ratni, R. Scalco, Alexander Stephan
Abstract
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.
Topics & Concepts
BlueprintSpinal muscular atrophySmall moleculeRNA splicingTransformative learningDrug discoverySMA*Drug developmentComputational biologyMedicineNanotechnologyDrugDiseaseComputer scienceData scienceEngineering ethicsPharmacologyBioinformaticsChemistryPsychologyBiologyRNAEngineeringMaterials scienceInternal medicineBiochemistryGeneMechanical engineeringPedagogyAlgorithmNeurogenetic and Muscular Disorders ResearchRNA Research and SplicingViral Infections and Immunology Research