Dr Lee L. Rubin

Biography

Lee Rubin has a broad experience in both academia and industry, particularly in the realms of cell-based assays and drug discovery. Prior to joining Harvard, he was Chief Scientific Officer of Curis, Inc., a Cambridge-based biotechnology company, where his group identified the first small molecule regulators of the hedgehog signalling pathway. One of these antagonists was developed by Genentech and is now (as Erivedge) approved as the first oral treatment for metastatic basal cell carcinoma. At Harvard, much of his work is focused on finding key molecular mediators of different neurodegenerative diseases and on searching for effective preclinical therapeutic candidates. His research takes advantage of his group’s ability to produce large numbers of patient-derived induced pluripotent stem cell lines and of effective means of deriving differentiated neurons from them. They have set up an array of techniques that allow them to identify early cellular and physiological changes in neurons as they become diseased. For example, they have identified new targets for the treatment of the motor neuron disorders Spinal Muscular Atrophy and Amyotrophic Lateral Sclerosis. They are also studying Psychiatric disorders, Parkinson’s disease and Alzheimer’s disease. Recently, his group discovered that a circulating protein, GDF11, has the ability to reverse some of the changes in the CNS associated with aging. They are actively exploring the therapeutic implications of these observations as well

Research Interest

neurons