Health: Taxpayers funds for a new gene therapy against rare diseases

Creating an innovative and affordable gene therapy to treat rare diseases and improve patients' quality of life is the goal ENEA has chosen to support with the 5x1000 funds taxpayers may allocate to the Agency in this year income tax return.

The project BIOGENERA, Sustainability and Innovation for Health: Non-Viral Gene Therapy for Rare Diseases, involves the use of an ENEA patent to develop an advanced biotechnology platform aimed in particular at the treatment of Glycogen Storage Disease Type III (GSDIII), a hereditary rare disease, and other related rare conditions.

 “The project will take 12 months to complete, and the new approach we want to adopt will also reduce the socio-economic impact of the disease” explained Doriana Triggiani at the ENEA RED Biotechnology Laboratory.

To date, there is no definitive cure for this disease, only treatments aimed at alleviating, from early childhood, symptoms that include glycogen accumulation in affected tissues, growth delay, hypoglycemia, progressive muscle weakness and liver and heart dysfunction.

The innovative method ENEA intends to develop with the 5x1000 funds includes laboratory experiments on three-dimensional (3D) cellular models, to advance the study of the disease and evaluate the therapy under conditions that better simulate the pathological microenvironment. This will make the research more effective and less expensive, also reducing the use of animal models. The use of nanoparticles capable of precisely and safely delivering therapeutic genetic material into cells, avoiding the use of viral vectors, will offer an additional advantage.

 “BIOGENERA may become a replicable model, adaptable to other rare diseases with similar characteristics, giving hope to many individuals and families who currently lack effective treatments” she concluded.

To support BIOGENERA and allocate the 2025 5x1000 to the project, simply sign the scientific research funding box on the personal income tax return forms and enter ENEA tax code: 01320740580.