Three personal drug discovery case studies will be presented from the medicinal chemistry perspective:
- the design and discovery of the small molecule Netupitant, an FDA approved anti-emetic medicine since 2014,
- the design and discovery of the peptide therapeutic Dasiglucagon for the treatment of severe hypoglycemia in diabetic patients, currently in late stage clinical phase 3 trials, and
- the design and preclinical characterization of a therapeutic GalNAc-siRNA conjugate for the treatment of rare blood disorders, for which the CTA has been recently approved.