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.


Dr. Carrolee Barlow, M.D., Ph.D., B.A., has been Chief Executive Officer of The Parkinson’s Institute since March 5, 2013. Dr. Barlow most recently served as the Chief Scientific and Medical Officer of BrainCells, Inc. a company she joined in 2004.

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She also served as the Director of Molecular Neuroscience and the Therapeutic Area Head for Stroke and Neurodegeneration at Merck Research Laboratories, a company she joined in 2002.
Prior to joining Merck, she held a faculty position in the Laboratory of Genetics at the Salk Institute for Biological Studies in La Jolla, California, where she maintained an adjunct appointment. She served as a Scientific Advisor of Bionomics Ltd. until September 22, 2016. At the Salk Institute, her research laboratory focused on developing animal models of human neurological disease.

Dr. Barlow received her M.D. from the University of Utah followed by a residency at The New York Hospital, Cornell Medical Center in Internal Medicine. She obtained a Ph.D. in molecular and developmental biology at the Karolinska Medical Nobel Institute in Stockholm, Sweden. She completed medical subspecialty training in the field of endocrinology at the NIH and a post-doctoral fellowship in neurogenetics at the National Human Genome Research Institute.


Dr. Grabowski is Professor Emeritus of Pediatrics and Genetics at the Cincinnati Children’s Hospital Research Foundation, and Chief Scientific Officer of Kiniksa Pharmaceuticals. He most recently served as Chief Scientific Officer of Synageva BioPharma, before its acquisition by Alexion in June 2015.


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Prior to that, Dr. Grabowski spent over 20 years at Cincinnati Children’s Hospital Medical Center, where he was the A. Graeme Mitchell Chair of Human Genetics and Director of Human Genetics at the Children’s Hospital Research Foundation.
He was also a Professor in the Departments of Molecular Genetics and Biochemistry, and Pediatrics, at the University of Cincinnati College of Medicine. Previously, Dr. Grabowski spent 13.5 years at the Mount Sinai School of Medicine, 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 330 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. He is currently Senior VP Head of Neuroscience Research and Early Development at Bristol Myers Squibb where he leads a research 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 (NIRU) and Research & Early Development Centers of Excellence (RED-CoEs) 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, BELSOMRA ® for insomnia, Ubrelvy® for migraine, Ajovy® for prevention of migraine and Zeposia® for multiple sclerosis and. Richard has published >200 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 Development Officer, at Prevail Therapeutics. 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 Operations Officer at Proclara Biosciences, a biotech company developing novel therapies for neurodegenerative diseases caused by protein misfolding. Previously, he was 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 currently serves as Board member at Cadent Therapeutics, Embera Neuro, Proclara Biosciences, and Spinogenix. 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. He was the founding Director of the Center for Human Experimental Therapeutics, which conducts learning phase clinical trials in a wide spectrum of disorders.

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He was also 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, where he continues to have a senior advisory role. Dr Kieburtz’s primary clinical and research interests are neurodegenerative diseases affecting the basal ganglia. He was the principal investigator (PI) for the NINDS sponsored trials of neuroprotective agents for PD (NET-PD), served as the Chair of the Parkinson Study Group, and directs the Clinical Core for the Fox Foundation sponsored Parkinson Progression Marker Initiative. He has served as the PI for many multicenter clinical trials in Huntington disease (HD), including the first NIH-funded multicenter trial in HD (CARE HD), and the initial dosage ranging trials of Pridopidine. 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 LLC, and continues to serve as President of the organization.