The Staying Power of Small Molecules

We asked the 2026 Power List whether newer modalities have eclipsed small molecules – and where the traditional mainstay still wins

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Small molecules retain advantages in oral administration, which can reduce treatment burden and support convenient long-term use.
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Their ability to cross the blood-brain barrier and reach intracellular targets makes small molecules useful for central nervous system and intracellular therapies.
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Advances in medicinal chemistry and structure-based design have improved small-molecule potency and selectivity, while computational methods support optimization.
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Protein degraders are extending small-molecule approaches to targets previously considered undruggable; Ascentage Pharma is advancing a BTK degrader into global phase 1 trials.
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Small molecules generally offer scalable manufacturing and broad accessibility, and their use is expanding beyond traditional rule-of-five property limits.

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