A small molecule is a small, chemically manufactured compound with a low molecule weight. Small molecule drugs help regulate a biological target such as an enzyme, channel or receptor to modify a disease process.

The large majority of drugs on the market today are small molecules and most are delivered orally. Small molecule drugs are part of what Syncona calls, the “First Wave” of technologies in healthcare and are dominated by large pharmaceutical companies.

Key facts*


value of portfolio


Syncona Board seats

Our Small Molecule portfolio companies

The small molecule pipeline

Drug discovery Pre-clinical Clinical

Our domain areas

Syncona has deep domain expertise across four key therapeutic areas.

*Unless stated all financials at 30 June 2021