Silverstein Foundation Initiatives
Since our formation in March, 2017, we have made eleven research grants and established important collaborative partnerships within the pharmaceutical and biotechnology industries. Perhaps the Foundation’s most significant accomplishment to date is the launch of Prevail Therapeutics, a new gene-therapy company focused on developing novel biologic therapies for Parkinson’s Disease and other neurodegenerative diseases. Founded in July 2017, Prevail successfully raised $75 million from new and existing investors.
- A grant will be made to Dr. Craig Crews in the Crews Laboratory at Yale University. Protein aggregation has long been known to be a hallmark of Parkinson’s Disease. In particular, some forms of the alpha-synuclein protein associate into insoluble protein aggregates and correlate with PD. The Crews Lab is exploring new technologies to engage the normal cellular quality control machinery to recognize and dispose of aggregated alpha-synuclein protein. The ultimate goal is to demonstrate that pharmaceutical clearance of these disease-associated aggregates can halt PD progression.
- A grant will be made to Arvinas, a biotech company in Connecticut whose technology will focus on degrading toxic intracellular targets found in Parkinson’s Disease.
- A grant was made to resTORbio, a clinical-stage biopharmaceutical company working to target the selective inhibition of TORC1. RTB101, resTORbio’s lead drug candidate, is a selective, orally administered, TORC1 inhibitor currently being tested in a Phase 2b clinical trial as a first in-class immunotherapy for reducing the incidence of respiratory tract infections in the elderly. The Silverstein Foundation believes there is therapeutic potential for RTB101 in neurodegenerative diseases. In January 2018, the company raised $98 million in its initial public offering on the NASDAQ.
- The Foundation issued a request for proposal in the area of cell therapy. The purpose of the RFP was to potentially support research that will study and develop cell transplant therapies with disease-modifying potential and/or the potential to significantly improve symptomatic treatment beyond the standard of care for GBA-related Parkinson’s Disease.
- A grant was made to Dr. Dimitri Krainc, Chairman of Neurology at Northwestern University. Dr. Krainc is a global thought leader in GBA-Parkinson’s Disease biology and is the scientific founder of Lysosomal Therapeutics, which is one of only two companies currently running a clinical trial for GBA-PD. The Foundation is funding the development of next-generation molecules at Northwestern, and has an exclusive option to jointly start a company to bring these drugs to market. The Foundation additionally connected Dr. Krainc to world-class medicinal chemists with decades of drug-development experience, in order to expedite the research project.
- Two grants were made to Alector, a Calfornia-based biotechnology company developing novel immuno-neurology therapies. Our grants will support animal work, testing these therapies in Parkinson’s Disease models.
- A grant was made to Columbia University which was directed specifically to GBA-related research.
- A grant was made to Columbia University which was directed specifically to GBA-focused bioinformatics in search of new targets for our research.
- Prevail Therapeutics was launched by The Silverstein Foundation, REGENXBIO and OrbiMed. The company is focused on developing novel biologic therapies for Parkinson’s Disease and other neurodegenerative diseases. Prevail entered an exclusive worldwide license agreement with REGENXBIO to develop and commercialize certain gene therapy products using REGENXBIO’s proprietary gene therapy technology, NAV AAV vectors.
- A grant was made to the Michael J. Fox Foundation for Parkinson’s Research directed specifically to GBA-related research.
- A grant was made to the Parkinson’s Institute and Clinical Center in San Francisco to allow Executive Director, Dr. Carrolee Barlow and her team to complete a study of the clinical manifestations of genetic (GBA, LRRK2, etc.) as compared to sporadic Parkinsons, specifically as it relates to neurons in the GI tract. Data from this study can be used by pharmaceutical companies in collaboration with Dr. Barlow’s team and the FDA to design and quickly advance novel clinical models.