Vice President, Neuroscience and Head of Translational Biology
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Crossing boundaries to discover therapeutics for patients with genetic disease As a senior biologist and pharmacologist identified therapeutic opportunities and implemented drug discovery and development programs for genetic and sporadic CNS diseases. Hired and formed strong Target and Drug Discovery teams. Supervised several PhD group leaders. Led epigenetic drug target project from screening to Ph2 human proof-of-concept studies in rare monogenic disease. Implemented Ph2 primary endpoint human biomarker for orphan indication. - Developed strategy and created phenotypic screening platform for rare orphan disease generating multiple hit-to-lead (H2L) programs for novel targets - Led or supported teams that delivered five pre-clinical drug candidates tested in three clinical indications - Pioneered epigenetic therapeutic approach for haplo-insufficiency disease and validated translatable biomarker - Collaborated closely interdisciplinary in Research and with Clinical Development, external clinicians, industry partners, foundations and academic researchers to extend therapeutic efficacy from Discovery to human disease
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