We have assembled a Scientific Advisory Board filled with world-renowned researchers and drug developers, in addition to thought leaders and GBA-mutation experts. This high level of expertise will help us rapidly comb through all GBA-related research and advance our goals expeditiously.


Roy Alcalay, M.D., is the Alfred and Minnie Bressler Associate Professor of Neurology at Columbia University Irving Medical Center. He obtained his medical degree from Tel Aviv University, Israel, his neurology training from the Harvard University residency program at Massachusetts General Hospital and Brigham and Women’s Hospital and his movement disorders training at Columbia University.

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He is a graduate of the Patient Oriented Research and earned Master’s in biostatistics from Columbia University. His research focuses on biomarkers and genetics in Parkinson’s disease and cognitive functioning. He is a Brookdale Leadership in Aging fellow, and his research is supported by the NIH, the DOD, the Parkinson’s Foundation and the Michael J Fox Foundation.


Carolee Barlow, MD, Ph.D, was appointed to the Board of Directors in June 2018. Dr. Barlow is a renowned expert in neuroscience, neurodegeneration, and rare diseases, and is currently the Chief Medical Officer of ESCAPE Bio.

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Dr. Barlow’s previous work spanned clinical care, laboratory and clinical research, academia, and industry. She is the former CEO of the Parkinson’s Institute and Clinical Center, an independent nonprofit organization providing research, clinical trials, and clinical care, as well as formed partnerships with biotech and pharmaceutical companies. Before joining the Parkinson’s Institute, Dr. Barlow was acting Chief Medical Officer at Amicus Therapeutics and also was a co-founder, Chief Scientific Officer, and Chief Medical Officer of BrainCells, Inc. Prior to BrainCells, she served as the Director of Molecular Neuroscience and Worldwide Therapeutic Area Head for Stroke and Neurodegeneration at Merck Research Laboratories, responsible for neuroscience biology, global exploratory, licensing and development efforts. She serves as an advisory board member and is on the Board of Directors of several biotechnology companies and disease foundations advancing therapies for rare diseases and disorders of the central nervous system. Dr. Barlow has held a faculty position at the Laboratory of Genetics at the Salk Institute for Biological Studies in La Jolla, California.

Dr. Barlow received her Bachelor of Arts in English from the University of Utah and Doctor of Medicine from the University of Utah School of Medicine. In addition, she received her Doctor of Philosophy in molecular and development biology from the Karolinska Medical Nobel Institute in Stockholm, Sweden. She is an author of approximately 100 peer-reviewed research papers, and book chapters, and is an inventor on numerous patents.


Dr. Grabowski is Professor Emeritus in the Department of Pediatrics, and the Department of Molecular Genetics, Biochemistry and Microbiology in the College of Medicine of the University of Cincinnati as well as a member of the Division of Human Genetics at the Cincinnati Children’s Hospital Research Foundation.


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From 2015 to 2019 he was the Chief Scientific Officer of Kiniksa Pharmaceuticals. Prior to that he was Chief Scientific Officer of Synageva BioPharma, before its acquisition by Alexion in June 2015.

From 1992 to 2014 Dr. Grabowski was the A. Graeme Mitchell Chair of Human Genetics and Director of Human Genetics at the Cincinnati Children’s Hospital Research Foundation. During that period, he was also a Professor in the Department of Molecular Genetics, Biochemistry, and Microbiology, and the Department of Pediatrics at the University of Cincinnati College of Medicine.

From 1979 to 1992, Dr. Grabowski rose to be Professor of Pediatrics at the Mount Sinai School of Medicine in New York City, where he developed the world’s largest clinic for patients afflicted with Gaucher disease and other lysosomal diseases. He established basic research and diagnostic laboratories for genetic diseases as well as the first treatment center for enzyme therapy in Gaucher disease.

Dr. Grabowski received his M.D. from the University of Minnesota where he also completed his residency in Pediatrics and fellowship training in Human Genetics. During his research career, he has published over 350 scholarly works on the basic and clinical sciences of lysosomal storage and genetic diseases, while maintaining active clinical and treatment programs for genetic diseases.


Richard Hargreaves holds a BSc and Ph.D. from Chelsea/Kings College, London University UK and is an Honorary Fellow of the British Pharmacological Society. He is currently Senior VP Head of Neuroscience Research and Early Development at Bristol Myers Squibb where he leads a portfolio built through external partnerships and collaborations connected to a focused internal effort and core company expertise.

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Previously Corporate VP, Head of Neuroscience and Imaging Research at Celgene, VP, Head of the New Indications Research Unit and Research & Early Development Centers of Excellence at Biogen and VP Discovery Head for Neuroscience and VP Imaging in Merck Research Laboratories. Richard has led teams that have advanced numerous novel CNS PET imaging agents and neuroscience drug candidates to the clinic contributing to the successful registration of MAXALT® for acute migraine, EMEND® and EMEND-IV® for chemotherapy-induced nausea and vomiting, ZIOPTAN® for glaucoma, BELSOMRA® for insomnia, UBRELVY® for acute migraine, AJOVY® and QULIPTA® for prevention of migraine and Zeposia® for multiple sclerosis and ulcerative colitis. Richard has published more than 220 journal articles and been recognized by awards from the ASPCT for his work on CNS discovery imaging and the BPS with the Sir James Black Award for Drug Discovery.


Dr. Franz Hefti is Chief Executive Officer, at Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly and Company. He has more than 15 years of experience in the biotech industry as executive, board member, co-founder and scientific advisor of many early-stage neurology companies.

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Prior to Prevail, he was Chief Executive Officer of Acumen Pharmaceuticals, a biotech company developing novel therapies for Alzheimer’s disease (ABOS), Chief Scientific Officer at Avid Radiopharmaceuticals (acquired by Eli Lilly and Company), a company that developed Amyvid® for Alzheimer’s disease brain imaging and was Executive Vice President of Drug Development at Rinat Neuroscience (acquired by Pfizer), which was responsible for early development of tanezumab and fremanezumab (Ajovy®) for pain and migraines. Earlier in his career, Dr. Hefti held senior management positions in neuroscience at Merck and Genentech. He also held positions in academia as a Professor at the University of Southern California and Associate Professor at the University of Miami, where he carried out discovery research on therapeutic approaches to neurodegenerative diseases. He has published more than 250 papers on neurotrophic factors and topics in neuropharmacology. He holds a Ph.D. from the University of Zurich and completed his postdoctoral research at the Massachusetts Institute of Technology.


Karl Kieburtz M.D. M.P.H is a Professor of Neurology at the University of Rochester, and President of Clintrex Research Corporation. He was the founding Director of the Center for Health & Technology, the initial Robert J. Joynt Professor in Neurology, and served as the Senior Associate Dean for Clinical Research and Director of the Clinical & Translational Science Institute in Rochester.

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Dr Kieburtz’s primary research interest is novel therapeutics for neurodegenerative diseases. He led the NINDS sponsored trials of neuroprotective agents for PD (NET-PD), and served as the Chair of the Executive Committee of the Parkinson Study Group. He previously served on and chaired the FDA Advisory Committee on Peripheral and Central Nervous System Disorders. In 2009, he was one of the co-founders of Clintrex Research Corporation which has collaborated with over 150 organizations developing products for brain and neurological disorders.

He received his BA in Neuroscience from Amherst College. He completed his MD and MPH degrees at the University of Rochester, as well as his Neurology training and Fellowship in Experimental Therapeutics.