Development of new nanotechnological based Enzyme Replacement Therapy for Parkinson’s disease (NANO-ERT)

The BBVA foundation granted the NANO-ERT project in the 2019 call (Ayudas a Equipos de Investigación Científica en Biomedicina) to a consortium participated by Vall d’Hebron Research Institute (VHIR), Universitat Autónoma de Barcelona (IBB-UAB) and ICN2. 

The project ‘Development of new nanotechnological based Enzyme Replacement Therapy for Parkinson’s disease: restoration of lysosomal glucocerebrosidase activity through enzyme-polymer nanoconjugation of GBA (NANO-ERT)’ will be funded with € 124,872. The BBVA Foundation’s has selected 5 projects out of the 134 presented in the Biomedicine area.

The NANO-ERT project was focused on the development of smart nanoformulations to achieve a new therapy against Parkinson’s based on Enzimatic Replacement Therapy (ERT). The project, coordinated by the group Neurodegenerative Diseases (Vall d`Hebron-Institut de Recerca, VHIR) and led by Dr. Marta Martínez Vicente, has been stablished in collaboration with the Nanostructured Functional Materials group (NANOSFUN, ICN2) led by Dr. Daniel Ruiz-Molina and the group Protein Engineering and Proteomics led by Dr. Julia Lorenzo (Institut de Biotecnologia i Bioquímica, IBB-UAB).

The NANO-ERT project proposes the design and development of a new enzyme replacement therapy based on the administration of recombinant GBA protein, since it is known that the presence of mutations in the lysosomal enzyme GBA is the main genetic risk for developing Parkinson’s disease. The objective is to use nanotechnological approximations to improve ERT applications. The aim is to increase the metabolic stability of GBA and improve its ability to cross the blood-brain barrier and reach the brain. The technology will be validated in animal models and will serve as proof of concept in the development of protein transport systems in the central nervous system in the context of neurodegenerative diseases.