Imagine a tiny, protective bubble that can carry medicine safely through the body to exactly where it's needed. This project uses that bubble technology to treat Fabry disease, a severe genetic condition. By protecting the medicine, they make the treatment more effective and cheaper to produce.
Patients with Fabry disease have very few treatment options, and existing orphan medicines are often prohibitively expensive or difficult to deliver effectively.
A patent-protected nanoencapsulation platform and a specific drug candidate, nanoGLA, for treating Fabry disease.
If you are an orphan drug developer dealing with high production costs for rare disease therapies — this project developed a patent-protected nanoencapsulation platform that enables the creation of cost-effective biomolecule therapies.
If you are a drug delivery startup dealing with stability issues in biomolecule transport — this project developed nanoGLA, which is currently undergoing stability studies to ensure robustness and reproducibility.
If you are a specialized clinic dealing with limited treatment options for LSD rare diseases — this project developed a candidate called nanoGLA that has received Orphan Drug designation from the EMA.
Based on available project data, specific pricing is not mentioned, but the project aims to develop 'cost-effective therapies' using a patent-protected nanoencapsulation platform.
Yes, the project is linked to the Phoenix EU Project (#953110), which focuses on scaling up nanoGLA production to GMP conditions.
The project utilizes a patent-protected nanoencapsulation platform and a patent-protected medicinal product candidate (nanoGLA). A spin-off company is being created to commercialize these assets.
nanoGLA was designated as an Orphan Drug by the European Medicine Agency (EMA) and the European Commission in January 2021.
The project runs from 2023-11-01 to 2026-10-31, with the goal of generating safety and efficacy data to get regulatory approval to proceed to the clinical phase.
The consortium is lean, consisting of 2 partners from Spain. It features a 50% industry ratio, combining a research organization (CSIC) with one SME, which facilitates a direct path from laboratory research to a commercial spin-off.
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