Indication of new nanoGLA therapy. We demonstrate its composition and structure by cryomicroscopic imaging. NanoGLA has been injected into mice and has orphan drug designation. Credit: ICMAB
An international research team has developed a new nanotechnology-based therapy called nanoGLA for the treatment of Fabry disease. This new treatment has shown significant efficacy in preclinical studies. The study was published in Science Advances in December of this year.
Fabry disease is a rare genetic disorder caused by a deficiency in the enzyme GLA (alpha-galactosidase A), which results in the accumulation of fatty substrates (mainly globotriaosylceramide or Gb3) within the cells and severe disease in various organs. influence.
nanoGLA therapy is based on the use of peptide-targeted nanoliposomes to effectively deliver the defective enzyme GLA encapsulated in nanoliposomes to the organs most affected by the disease. The researchers were able to produce nanoGLA in the quality and quantity needed for preclinical testing and moving into the clinical stage.
In studies using a mouse model of Fabry disease, nanoGLA demonstrated improved efficacy compared to treatments using unencapsulated enzymes and showed efficacy in affected organs, including the brain, which is currently This is an important milestone that cannot be achieved with a cure. These results highlight the potential of nanoGLA to address both the systemic and cerebrovascular symptoms of Fabry disease.
Recognizing the importance of this innovation, the European Medicines Agency granted nanoGLA Orphan Medicinal Product Designation in 2021. This is an important step in driving its development.
Elisabeto González, a researcher at ICMAB and one of the paper’s lead authors, said: “The new nanoGLA formulation is particularly useful in addressing the neurological symptoms of Fabry disease, where current treatments have limitations that cannot be overcome. “Therefore, this represents a promising opportunity for patients with Fabry disease.” The goal is to harness the potential of nanotechnology to develop safer and more effective treatments. ”
These results provide the green light for continued drug development of nanoGLA toward clinical stages in human patients.
Further information: Judit Tomsen-Melero et al, Targeted nanoliposomes to improve enzyme replacement therapy for Fabry disease, Science Advances (2024). DOI: 10.1126/sciadv.adq4738
Source: Nanoliposomes pave the way to treatment of rare genetic diseases (December 19, 2024) from https://phys.org/news/2024-12-nanoliposomes-pave-treatment-rare-genetic.html Retrieved December 21, 2024
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