Many diseases still lack effective or curative treatments, leaving patients with limited options that focus mainly on managing symptoms rather than addressing the root cause. Conventional therapies such as enzyme replacement or small molecules offer only partial relief, while viral vectors face issues of immunogenicity, scalability, and safety.
In this context, researchers from the University of the Basque Country (EHU) have developed a non-viral gene therapy platform that consists of solid lipid nanotechnologies that enables the targeted administration of different types of nucleic acids (DNA, RNA, siRNA) allowing both gene silencing and the enhancement of specific enzyme expression. This platform has shown potential in ocular pathologies and rare lysosomal diseases, such as Fabry disease, where current therapies remain insufficient. The technology incorporates condensing peptides and proteins, which optimise the stability of the nanoparticles and significantly increase the transfection capacity in target cells.
In different cell and ex vivo models, the system bearing pDNA or mRNA has shown the ability to transfect and to induce the expression of the encoded proteins, without affecting cell viability. The ability of the system bearing shRNA or siRNA to silence proteins has also been demonstrated. In relevant mouse models, after intravenous administration different organ and tissues were transfected. In addition, the system was able to transfect the cornea after topical instillation, and different layers of the retina after intravitreal injection, producing a therapeutic protein, which lead to structural improvement of the retinas, without signs of toxicity.
- The technology uses non-viral vectors with favourable safety profile and simpler and scalable production.
- Highly versatile, as it allows for both gene silencing and protein expression in several different applications.
- It has shown promising results in diseases with unmet medical needs, such as rare eye diseases and Fabry syndrome.
- Condensing peptides and proteins or gold nanoparticles improve the effective delivery of different types of genetic material (DNA, RNA, siRNA).
The represented institution is looking for a collaboration that leads to commercial exploitation of the presented invention.
Institution: PharmaNanoGene Group from the Universidad del País Vasco (EHU)
TRL: 4-5
Protection Status: The technology is protected by three currently active patents:
- PCT/EP2020/087608A1, filed on December 2020.
- PCT/ES2011/070883, filed on December 2010.
- PCT/ES2010/070519, filed on July 2010.
Contacto: Elisa Sáenz Gómez | e.saenz@viromii.com