The aim of the Smart4Fabry project is to obtain a final nanoformulation of GLA enzyme, ready to enter future clinical testing, with a solid pre-clinical proof of concept (PoC) to demonstrate that nano-GLA has a higher targeting of the enzyme to affected organs and a lower clearance rate than free GLA. Our objective is that the new nanoformulation of GLA will allow a significant increase in the efficacy (30-80% higher) compared to the current ERT with non-nanoformulated GLA
Achieve quality control over the self-assembly of nano-GLA molecular components and its physicochemical parameters.
Develop a nano-GLA capable of crossing the BBB.
Construction of a GMP plant for the production of the quantities of the nanoformulation needed for regulatory pre-clinical and future clinical trials.
Development of the GLA nanoformulation following the requirements of the pharmaceutical industry and regulatory authorities.
Create a realistic exploitation plan and business model, based on experience from the companies in the rare diseases market, in order to ensure the translation of the project results to patients suffering from Fabry disease.