An international research team led by researcher Nora Ventosa from the Nanomol-Bio group of ICMAB-CSIC and CIBER-BBN has developed a new nanotechnology-based therapy called nanoGLA for the treatment of Fabry disease. The new therapeutic solution has demonstrated remarkable efficacy in preclinical studies. The study, which was carried out in collaboration with the Vall d’Hebron Research Institute (VHIR), was published this December in the open access journal Science Advances.

Fabry disease is a rare genetic disease caused by a deficiency of the enzyme GLA (alpha-galactosidase A) which causes the accumulation of fatty substrates (mainly globotriaosylceramide or Gb3) in cells and has serious repercussions on different organs. NanoGLA therapy, based on the use of peptide-targeted nanoliposomes, effectively delivers the deficient GLA enzyme, encapsulated in nanoliposomes, to the organs most affected by this disease. Researchers have managed to manufacture nanoGLA in the quality and quantity necessary for preclinical testing, and to be able to enter the clinical phases.

In studies with mouse models of Fabry disease, nanoGLA has demonstrated improved efficacy compared to therapies that use the unencapsulated enzyme, showing effectiveness in the affected organs, and even in the brain, a key milestone that current therapies fail to achieve. These results highlight the potential of nanoGLA to address both systemic and cerebrovascular manifestations of Fabry disease.

In recognition of the importance of this innovation, the European Medicines Agency granted nanoGLA Orphan Medicinal Product Designation in 2021, a decisive step to boost its development.

This breakthrough has been possible thanks to the collaboration between researchers from several international institutions, including the Vall d'Hebron Research Institute (VHIR), the Barcelona Institute of Materials Science (ICMAB-CSIC), CIBER-BBN; companies such as Nanomol Technologies SL and Leanbio SL, the Institute of Advanced Chemistry of Catalonia (IQAC-CSIC), the Institute of Biotechnology and Biomedicine (IBB-UAB) and international collaborators such as the Joanneum Research-Institute for Biomedical Research and Technologies (HEALTH) (Austria), the Technion-Israel Institute of Technology (Israel), the Guangdong-Technion Israel Institute of Technology (China), Aarhus University (Denmark) and Labcorp Drug Development (UK).

Elisabet González, researcher at ICMAB and one of the main authors of the article explains “the new nanoGLA formulation represents a promising opportunity for patients with Fabry disease, especially in addressing the neurological manifestations of the disease, a limitation that current therapies cannot overcome. Our goal is to develop safer and more effective treatments by taking advantage of the potential of nanotechnology”.

These results, obtained within the framework of the European Smart4Fabry project, funded by the European Union’s Horizon 2020 research and innovation program, have given the green light to continue the pharmaceutical development of nanoGLA towards the clinical phases with human patients. The European Commission, through the EU Phoenix and Nano4Rare projects, has granted the necessary funding to complete the preclinical phase and obtain approval to start the clinical phase.