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Shire rare disease business unit

Shire ($SHPG) has nailed down another deal to fuel its growing rare disease unit. The pharma group has tapped the Cambridge, MA-based startup Nimbus Discovery, which will use its computational approach to find small-molecule drugs for rare genetic diseases known as lysosomal storage disorders.

The deal marks the first announcement of a corporate partnership for Nimbus, which was founded in 2009 by Atlas Venture and the chemical software company Schrödinger, and gained an early seed investment from Microsoft ($MSFT) Chairman Bill Gates. It's also a vote of confidence for the startup's use of computational screening for drug-like compounds against disease targets that have been unreachable with traditional approaches to discovery.

The companies declined to reveal any financial details of the pact, yet Nimbus's Jonathan Montagu tells FierceBiotech that the deal offers "significant" perks, including funding of all discovery research and potential milestones if Shire takes an option to acquire the program.

Shire seeks small molecules from Nimbus against an undisclosed target associated with lysosomal storage disorders (LSDs), which include 50 or so inherited diseases where missing enzymes cause buildups of certain complex carbohyrdate in the lysosomes of cells and lead to organ damage. Shire's Lexington, MA-based Human Genetic Therapies unit, Sanofi's ($SNY) Genzyme and others provide pricy enzyme-replacement therapies for LSDs, which include rare diseases such as Fabry, Gaucher and Hunter syndrome. Oral small molecules could simplify treatment for patients with the diseases, which often strike them during childhood.

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