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实验室

10:30-11:30, Friday, Mar 29


Speaker: Matt Jacobson, Ph.D.

Topic: Challenges in drug discovery for neurodegenerative diseases

Host: Niu Huang, Ph.D.

Abstract

Neurodegenerative diseases rank among the most pressing unmet medical needs for drug discovery, but also notoriously pose many unsolved challenges.  The blood-brain barrier poses a general challenge for targeting any diseases of the CNS, and I will describe our efforts to understand and predict drug permeation through this barrier.  A second, and arguably more profound, challenge is that many idiopathic and genetic neurodegenerative diseases (in contrast to oncology, for example) largely manifest as a partial loss-of-function in critical cell processes such as autophagy.  We and our collaborators are exploring multiple approaches to restoring function at the level of specific targets and pathways, including the design of allosteric drugs.  As one example, I will discuss our recent work to discover agonists of Nurr1, a nuclear receptor controlling transcription of many genes involved in dopamine homeostasis as well as neurotropic factors, as potential therapy for Parkinson’s disease.  I will also briefly discuss my experiences as a co-founder of 4 biotech companies, focusing on Relay Therapeutics, which is dedicated to discovery of allosteric drugs.