NanoERT, a technology that improves ERT

Julia Lorenzo

Institut de Biotecnologia i de Biomedicina, Universitat Autònoma de Barcelona, Cerdanyola del Vallès, Spain

Gaucher’s disease is a rare, hereditary condition characterised by an enzyme deficiency of the lysosomal protein GBA. This deficiency causes serious problems in the “cellular clearance” mechanisms, severely affecting the functionality of various organs such as the liver, spleen, bones and even, in some cases, the brain.

The researchers in this project have developed a new technology, called nanoERT, to improve current enzyme replacement therapies used for various lysosomal diseases, such as Gaucher’s disease. This novel technology, applied to the GBA protein, improves the current therapy offered to people with Gaucher’s disease by reducing the frequency of treatments required and increasing the effectiveness of the therapy in the affected organs. This not only improves patients’ quality of life, but also helps reduce costs for the healthcare system.

Furthermore, this technology is also being applied to other diseases, such as Parkinson’s disease, in which alterations in GBA are the main genetic risk factor for developing the condition. The application of nanoERT technology allows for the development of a therapy that, when administered via the intranasal route, is capable of delivering GBA to the brain, thereby restoring GBA activity in neurons. This opens the door to a possible therapy for this neurodegenerative disease, which currently has no cure.

NanoERT: nano-delivery platform for Enzyme Replacement Therapy in Parkinson's and Gaucher's disease

Stage 2