PM-RARE Grant obtained

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A ZonMW PM-RARE grant of € 3.0 million involving Peter Willems (left, Dept. of Willems, PeterKoopman, WernerSmeitink, JanBiochemistry), Werner Koopman (middle, Dept. of Biochemistry and Director R&D of Khondrion BV) and Jan Smeitink (right, Dept. of Pediatrics and founding CEO of Khondrion BV), has been awarded to a national consortium consisting of the RUNMC, Khondrion BV (Nijmegen), Mercachem BV (Nijmegen) and three mitochondrial patient organizations (VSN, ESN and IMP). This grant will support an ambitious scientific and industrial collaboration to discover treatments for MELAS syndrome, a rare mitochondrial disease that affects both children and adults. During the five years' grant period, each partner of this multidisciplinary team will contribute its expertise to reach their shared goal: the development of a drug to treat MELAS. Mercachem will be responsible for the medicinal chemistry by developing and synthesizing novel molecules, Khondrion will perform the in vitro evaluation of these new chemical entities, including screening in their cell-based mitochondrial disease models, the RUNMC will be responsible for in-vivo testing. The involved patient organizations will embed the patient perspective in the project, inform patients, assist in patient recruitment and in defining clinically relevant disease outcome measures. The consortium aims at organizing a European Mitochondrial Patient conference in 2015.


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