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NoStop: Personalised Small Molecule Gene Therapy

CORDIS · observation · Publication date unknown

apy offers this potential; however, its clinical impact has so far been limited by disappointing trial outcomes. Using a combination of in-house–developed high-throughput human readthrough assays and machine learning, we recently demonstrated that a key factor underlying this limited success is the strong sequence specificity of readthrough drugs, which varies markedly among compounds. With the NoStop platform, our goal is to build on this validated technology to transform the readthrough therapy field by systematically generating comprehensive PTC-specificity maps for all known readthrough compounds, while simultaneously establishing a sequence-tailored high-throughput drug discovery pipeline to identify novel proprietary readthrough drugs. If successful, NoStop will generate invaluable data to enable the transition of readthrough therapy from a one-size-fits-all approach to a personalized medicine paradigm.

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recordType

award

status

SIGNED

region

EU

value

150000

unit

EUR