Project: Advanced chemical technologies and predictive experimental models leading to new treatments against rare diseases.

Advanced technologies which proved their effectiveness in drug discovery have been seldom applied in the field of rare diseases. This is true both for repositioning known products and for hit and lead selection using "classical" drug discovery approaches. In order to exploit the possibilities offered by recent advancements in various research field, three different companies with complementary expertise joined their forces, trying to cover relevant medical needs in rare diseases of neurometabolic origin, of the respiratory system and of the blood elements. This project will apply cutting edge medicinal chemistry techniques and novel cellular models predictive of efficacy of seven rare diseases, to explore efficiently the whole chemical space of known drugs, and to identify potential candidates to be used for the treatment of such diseases. On the basis of the data obtained in this phase of the project, it will be possible to reposition a known compound toward treatment of a rare disease, and to start a focused discovery program including chemistry and biology profiling of analogs of the selected known products, whose later clinical development will be more likely to succeed.

Acronym	Re.Mark.RareDis
Network	ETB-PRO
Call	7th call for proposals (Call 2012)

Project partner

Number	Name	Role	Country
	Italy		Italy
	Minoryx Therapeutics S.L.	Coordinator	Spain