Challenging the drug discovery paradigm in rare disease research

As part of MedChemNet's February focus on rare disease research, I discuss collaboration and discovery

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If there is one area where we need to challenge the traditional drug discovery paradigm, it’s rare disease research. Starting with the needs of the patient, the strong links that exist between the patient, advocacy groups and rare disease research scientists should be fostered to ensure there is a line of sight from the decisions made at the earliest stages of exploratory research through to the delivery of a transformational drug.

Scientific advances in chemical biology to understand the molecular drivers of disease will continue to drive precision medicine, and rare disease treatments have exemplified the impact such approaches can achieve. Phenotypic screening using patient-derived cells and tissues will provide new therapeutic opportunities – however, without knowing the relevant pathobiological targets and mechanisms at play, these programs will move slowly.

Therefore, we need to up our game regarding the efficiency with which phenotypic screening projects can be converted into accelerated target-based approaches. Target identification strategies will be facilitated by advances in chemoproteomics, chemogenomics and transcriptional profiling.

Medicinal chemists in particular will need to think more broadly about the ways they can deliver value to the pursuit of new medicines for rare diseases – it is imperative that we continue to drive excellence in small molecule research, but other modalities such as biotherapeutics and gene therapy should be embraced and enhanced.

Go to the profile of Lyn H. Jones

Lyn H. Jones

Head of Rare Disease Chemistry, Head of Chemical Biology, Pfizer

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