Lysosomal storage disorders (LSD) diseases are a group of rare diseases that currently lack a definitive cure. In the case of Fabry LSD Disease, the deficiency in α-Galactosidase A (GLA) enzyme activity results in the cellular accumulation of neutral glycosphingolipids, leading to widespread vasculopathy with particular detriment to the kidneys, heart and nervous system. The current treatment for FD is the Enzyme Replacement Therapy (ERT), in which free GLA recombinant protein is administered intravenously to patients. ERT exhibits several drawbacks mainly related to the instability, high immunogenicity and low efficacy of the exogenously administered GLA to cross biological barriers, such as cell membranes and blood brain barrier. Hence, the aim of Smart-4-Fabry project is to achieve excellent quality control over the assembly of the different molecular components of a new liposomal nanoformulation of GLA, nano-GLA, for the treatment of Fabry disease (FD). Nanoformulated GLA has already shown to have better PK/PD profile than free GLA and higher efficacy in vivo. Smart-4-Fabry project will advance nano-GLA from an experimental PoC (TRL3) to preclinical regulatory phase (TRL5-6). The final GLA nanoformulation will have tailored transport of GLA through cell membranes and BBB. Fulfilment of Smart-4-Fabry will impact on a major health problem, the existence of new therapies for rare diseases, which constitutes a priority societal challenge as shown in the H2020 Work Programmes. Another important impact is related to its contribution to support the European Strategy for KETs, which aims to reverse the decline in manufacturing as this will stimulate growth and jobs. Smart-4-Fabry is strongly focusing on three KETs: nanotechnology, industrial biotechnology and advanced materials.
Project duration: January 2017 – December 2020 (Kick-off-Meeting: 16.01. - 18.01.2017)
Total number of partners: 10 beneficiaries
Coordinator: CENTRO DE INVESTIGACION BIOMEDICA EN RED (CIBER), Spain
Project volume: 5 845 258,63 €
Role of BioNanoNet: Conducting nano-safety assessment and safe-by-design approaches along the manufacturing and encapsulation processes of GLA-nanoliposomes (contributing towards the framework of EU nanosafety and regulatory strategies).