A research project that proposes the treatment of Machado-Joseph’s disease, a neurodegenerative disorder, which is rare and incurable, through the correction of the genetic defect responsible for the pathology, by using gene editing tools, has won 20 thousand euros by winning the Building Tomorrow Together competition, promoted by Roche Portugal.
The winning team, whose name if “ExoTreat”, is constituted by Sara Lopes, Luís Pereira de Almeida, Kevin Leandro, and David Ramos, researchers at the Center for Neuroscience and Cell Biology of the University of Coimbra (CNC-UC).
«The technology potential is locked with the delivery of therapeutic molecules to the affected brain regions, in a safe, efficient and highly controlled fashion”, affirms Sara Lopes, highlighting that “the success of the project may contribute to significative advances in the treatment of Machado-Joseph’s disease, as well as other neurodegenerative disorders”.
Also, Luís Pereira de Almeida, CNC-UC group leader and coordinator of this team, refers that “this prize reflects all the work that has been developed in our research group and constitutes a recognitiion to all that collaborate with it”.
The also professor at the Faculty of Pharmacy at the University of Coimbra (FFUC) denotes that “the group works in the development of gene therapies for more than 20 years with a potential therapy for this fatal disorder. All the achieved scientific advances are aimed to offer a therapy that may revert the disease and improve the patients’ life quality”.
Other than the prize, the team had access to mentoring sessions by the Roche team, in a Bootcamp of idea acceleration, which occurred before the final of the competition. According to Roche, the Building Tomorrow Together is a competition of acceleration and innovation “aimed to develop ideas that are based in science and/or technology to solve challenges in the neuroscience field”.
Credits: Cristina Pinto, David Ramos, João Cardoso, Kevin Leandro, Luís Pereira de Almeida e Sara Lopes.