We are currently facing enormous challenges in preventing and successfully treating neurodegenerative diseases. Traditional pharmacological approaches and approaches using stem cells have made some progress, but their impact continues to be limited.
As clinical results in Canavan's and Parkinson's disease suggest, gene transfer has considerable potential. However, this therapeutic intervention strategy also presents some unique obstacles: particularly the need to deal with viability, efficacy and safety. BrainCAV is based on the potential of type-2 canine adenovirus vectors (CAV-2), which perfectly transduce neurons and reach their target located at a considerable distance in a very efficient way by means of axonal transport. Furthermore, long-term episomal expression allows for safe and efficient neurospecific administration to the cell.
We have proposed a structured translational approach that includes basic research thanks to the viability, efficacy and safety of the preclinical model. To provide proof of principle of the efficacy of CAV-2, we use mucopolysaccharidosis type VII, a rare global disease that generally affects children, and Parkinson's disease, a focal degeneration of the dopaminergic neurons that usually affects the elderly.
In order to carry out this project, BrainCAV has brought together a multidisciplinary group of associates with unique knowledge, which will bring CAV-2 vectors to the threshold of clinical trials.
Centre National de la Recherche Scientifique; Cancer Research UK;University of Rome La Sapienza; Instituto de Biologia Experimental e Tecnologica; Universidad Autónoma de Barcelona; University of Glasgow; University of Leipzig; Institut National de la Santé et Recherche Médicale; GenIBET Biopharmaceuticals