Two UAB projects supported by the CaixaImpulse programme

The UAB projects are two of the 29 projects that have received funding from the CaixaImpulse Innovation Programme, a call for proposals from the "la Caixa" Foundation, which has selected biomedical projects from research centres, hospitals and universities in Spain and Portugal to receive a total of 3.4 million euros.

Nano-ERT, a technology that improve enzyme replacement therapies

The Nano-ERT project, led by Julia Lorenzo from the UAB Institute of Biotechnology and Biomedicine and involving the Fundación de la Comunidad Valenciana Centro de Investigación Príncipe Felipe and the Vall d'Hebron Institute of Research (VHIR), has received a grant of 150,000 euros from the programme.

Gaucher’s disease is a rare hereditary disorder characterised by the enzymatic deficiency of the lysosomal protein GBA. This deficiency causes serious problems in the “cell cleaning” mechanisms, which then gravely affects the functioning of different organs such as the liver, spleen, bones and, in some cases, even the brain.

The project’s researchers have now developed a new technology, called nanoERT, to improve the current enzyme replacement therapies used in different lysosomal disorders, such as Gaucher’s disease. This innovative technology, applied to the GBA protein, can improve current therapies being offered to patients with Gaucher’s disease, since it reduces treatment frequency and increases its effectiveness in the affected organs. That not only improves the quality of life of patients, but also contributes to reducing the cost of the treatment for the healthcare system.

This technology is also being applied to other disorders, such as Parkinson’s disease, in which GBA alterations are the main genetic risks in developing the disease. The application of the nanoERT technology means that a therapy can be developed in which, when applied nasally, is capable of transporting GBA to the brain, and thus restoring GBA activity in the neurons. This opens the door to a possible therapy for this neurodegenerative disease which currently has no cure.

Towards an effective and safe treatment for osteoporosis

CaixaImpulse has also approved a project to research an effective and safe treatment for osteoporosis, led by Joan Roig, head of the Nervous System Gene Therapy Group at the Vall d'Hebron Research Institute (VHIR), in consortium with Beatriz Almolda, researcher at the Department of Cell Biology, Physiology and Immunology at the UAB. The grant amounts to €145,850.

Osteoporosis is a degenerative disease characterised by a decrease in bone density, leading to an increased risk of fractures. Although it is often associated with ageing, there are other factors that can lead to it, such as hormone deficiency due to menopause or medical treatments associated with ovarian and prostate cancer. Bone fractures are not only a burden on families and the healthcare system, they also significantly reduce people's quality of life.

Although treatments are available to slow the progression of the disease, they have limitations and undesirable side effects. In previous studies using animal models of ageing, the researchers in this project used the Klotho protein, which is present in the membrane of various cell types and in the bloodstream, and demonstrated its beneficial effect in preventing osteoporosis. This new project aims to confirm the therapeutic efficacy and safety of this protein in order to open the door to the development of a new effective and safe therapy for osteoporosis.