Shire has linked up with the USA's Nimbus Discovery to develop small molecule oral drugs for several rare genetic diseases.

The firms say that the goal of the collaboration is to use Nimbus' computational chemistry approach to discover disease-altering therapies   for lysosomal storage disorders (LSDs). Nimbus says that many LSDs remain untreated "because of challenges in creating drugs that can effectively reach the disease site", and the partners hope to develop "the first small molecule agents designed to penetrate inaccessible tissues while offering the convenience of an orally-administered pill".

The partners are keeping their cards very close to their chest, saying that 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". No financial details have been disclosed either, though Nimbus is tasked to deliver a drug candidate that is ready to enter late preclinical studies and will pocket preclinical, development and commercial stage milestones. 

__Shire is already a major player in the LSDs area, with the likes of Replagal (agalsidase alfa) for Fabry disease, Vpriv (velaglucerase alfa) for Gaucher's disease and the Hunter Syndrome therapy Elaprase (idursulfase). However, all these are enzyme replacement therapies which, like their competitors, are administered by intravenous infusion and oral treatments for LSDs are seen as a very attractive option.

Philip Vickers, Shire's R&D chief, said “Nimbus is ground-breaking in their approach to drug discovery and, in a short period of time, have already assembled an impressive track record in delivering clinical candidates for challenging disease targets". He added that this partnership is another way for the firm "to ensure that we expand into new disease areas and continue to apply cutting-edge technologies in this space".

Nimbus was set up in 2009 by chemical simulation software company Schrodinger and Atlas Venture. The latter and Shire linked up at the end of 2011 to identify early-stage investments in the rare genetic diseases arena.