The great challenge with phenotypic screens is recognizing when you have something and when you don’t. How confident should you be that your library has a molecule with your desired biological function? How long will it take to advance from hit to lead? Is your lead going to look like your hit? Phenotypic screens often come with as many questions as answers. We have successfully navigated the treacherous waters of phenotypic screening to produce development candidates for the treatment of spinal muscular atrophy (SMA), gram-negative bacterial infections and cancer. I will discuss concepts that apply to hit advancement, mechanism elucidation, mechanism hopping and molecular heritage (structural characteristics carried from hit to lead).

From here to there: Recognizing paths leading to treatments for spinal muscular atrophy and other diseases

255th National Meeting (2018)

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