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A novel mechanism to regulate gene expression in the brain (DD-PD)
Date du début: 1 nov. 2012, Date de fin: 31 oct. 2013 PROJET  TERMINÉ 

"Development of advanced therapeutics based on non-small molecule drugs for brain diseases is an area of intense research. Experimental work using viral vector-mediated in vivo gene therapy showed great potential as a competitive therapeutic strategy for clinical use in neurodegenerative diseases. AAV vectors are now in early clinical trials stage for treatment of Parkinson’s disease. Although it is deemed very important to have controlled expression in the brain, none of the currently tested strategies have implemented an approach that can be exogenously controlled. Regulation of the therapeutic intervention is important not only for safety reasons but has important implications for tuning the delivery of the therapeutic molecule to the individual needs of the patients, i.e., personalizing their treatment. The core of this proof-of-concept application is to develop a novel gene regulation system as a product for clinical use in patients with Parkinson’s disease. The applicant has assembled a strong team of experts that can take this exciting idea that has spun out of his ERC-funded research to an early business strategy and bridge to commercialization. The work will include filing of new IP, a proactive use of novelty search, formation of a dedicated start-up company for handling the new IPR as well as in-licensing of related IP, and sketching a path towards commercialization via obtaining regulatory authorization, investigation of strategies for large-scale production of the product."



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