An international research team led by ICMAB with the participation of the UAB has developed a new nanotechnology-based therapy called nanoGLA for the treatment of Fabry disease. The new therapeutic solution has shown remarkable efficacy in preclinical studies. The study was published this December in Science Advances in open access.
Fabry disease is a rare genetic disorder caused by a deficiency of the enzyme GLA (alpha-galactosidase A), which leads to the accumulation of fatty substrates (mainly globotriaosylceramide or Gb3) in cells, with severe effects on various organs. The nanoGLA therapy, based on the use of peptide-targeted nanoliposomes, effectively delivers the deficient enzyme GLA, encapsulated in the nanoliposomes, to the organs most affected by this disease. The researchers have managed to produce nanoGLA with the necessary quality and quantity for preclinical testing, as well as for advancing into clinical phases.
In studies with mouse models of Fabry disease, nanoGLA demonstrated improved efficacy compared to therapies using the unencapsulated enzyme, showing effectiveness in affected organs, including the brain, a key milestone that current therapies do not achieve. These results highlight the potential of nanoGLA to address both the systemic and cerebrovascular manifestations of Fabry disease.
In recognition of the importance of this innovation, the European Medicines Agency granted nanoGLA orphan drug designation (Orphan Medicinal Product Designation) in 2021, a crucial step in driving its development.
This breakthrough has been made possible through the collaboration among researchers from several international institutions, including the Institute of Materials Science of Barcelona (ICMAB-CSIC), at the UAB campus; the CIBER-BBN; the Institute of Biotechnology and Biomedicine of the UAB (IBB-UAB); the Vall d'Hebron Research Institute (VHIR), a UAB-partner institute; companies such as Nanomol Technologies SL (at the UAB campus) and Leanbio SL, the Advanced Chemistry Institute of Catalonia (IQAC-CSIC), and international collaborators such as the Joanneum Research-Institute for Biomedical Research and Technologies (HEALTH) (Austria), Technion-Israel Institute of Technology (Israel), Guangdong-Technion Israel Institute of Technology (China), Aarhus University (Denmark), and Labcorp Drug Development (UK).