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Shire, the UK’s third largest pharmaceutical firm, has teamed up with US-based Nimbus Discovery to develop small molecule treatments for rare genetic diseases.

The partners aim to develop the first small molecule agents designed to penetrate inaccessible tissues using Nimbus’ computational approach.

No financial details of the collaboration have been disclosed.

Shire is seeking small molecules from Nimbus against an undisclosed target associated with lysosomal storage disorders, a group of around 50 rare inherited metabolic disorders that result from defects in the lysosome – a specific organelle in the body’s cells.

In a statement, the companies said the collaboration is the result of a joint assessment of a series of rare disease targets "with significant unmet medical need [and] one target was ultimately chosen to be the research focus".

Nimbus said it will extensively profile molecules against the agreed-upon disease target and deliver a drug candidate that is ready to enter preclinical studies.

Nimbus CSO Dr Rosana Kapeller said; "This collaboration validates our computationally-driven, structure-based drug discovery engine and innovative partnering model.

"Nimbus is thrilled to pursue our first alliance with Shire, a company that shares our passion for uncovering breakthroughs in highly sought-after disease targets that have proven inaccessible to traditional industry approaches."

Shire will be responsible for all clinical development and future commercialisation activities.

Shire senior vice president for R&D Dr Philip Vickers said; "As a leader in rare diseases, this partnership is another way for Shire to ensure that we expand into new disease areas and continue to apply cutting-edge technologies in this space."


Image: Lysosomal storage disorders occur when a cell’s lysosome (12) malfunctions. Photo: Courtesy of Kelvinsong.