Lee L. Rubin, Ph.D.

Lee L. Rubin, Ph.D.

Harvard Department of Stem Cell and Regenerative Biology
Principal Faculty, Harvard Stem Cell Institute
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Dr. Lee Rubin, co-leader of the HSCI Nervous System Diseases Program, has a broad experience in both academia and industry, particularly in the realms of cell-based assays and drug discovery. He received his PhD in neuroscience from the Rockefeller University and had postdoctoral training, also in Neuroscience, at Harvard Medical School and Stanford University School of Medicine.Prior to coming to Harvard, Rubin was Chief Scientific Officer of Curis, Inc., a Cambridge-based biotechnology company, where his group identified the first small molecule regulators of the hedgehog signaling 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 Rubin's work is focused on finding key molecular mediators of different neurodegenerative diseases and on searching for effective preclinical therapeutic candidates. His research leverages 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. 

The Rubin group has 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, they discovered that a circulating protein, GDF11, has the ability to reverse some of the changes in the CNS associated with aging. The Rubin group is actively exploring the therapeutic implications of these observations.

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