Genetic and Small Molecule Modifiers of Lysosomal Function

2026-07-22

LysoMod will innovate in the area of personalized medicine for disorders linked to lysosomal dysfunction. This will be achieved by implementing a collaborative staff-exchange program between highly complementary and multidisciplinary academic and non-academic partners with expertise in pharmacology, medicinal chemistry, cell biology, biochemistry, mouse and human genetics, transcriptomics, proteomics and lipidomics. Based on the critical role that lysosomes play in cells, a better understanding of lysosomal function will have a major impact on human health, fostering the development of new strategies to improve quality of life for people affected by a variety of diseases, ranging from lysosomal storage diseases (LSDs) to age-related neurodegenerative disorders. LysoMod’s specific objectives are: 1) to develop and further optimize existing therapies for LSDs; 2) to identify new targets for personalized therapies for LSDs; and 3) to investigate the cross-talk between lysosomal function, signalling pathways and gene expression regulation. The pioneer work of a participant in the consortium led to the development of a drug that is approved for clinical use. LysoMod will i) investigate the mechanisms of action of this and other drugs in lysosome-related disorder; ii) identify modifier genes involved in LSD pathology and test their potential as new targets for personalized therapeutic approaches; iii) identify candidate RNAs that can be targeted to enhance lysosomal function. The companies in the consortium will ensure a rapid transfer of new knowledge into applications for diagnostics and clinical trials. Prioritising lysosomal dysfunction as a highly relevant biomedical problem, the LysoMod consortium will implement a mentored staff-exchange program to provide young researchers with high-level training in innovative approaches for exploring biological systems, preparing the next generation of researchers for careers either in the private or public health sectors.

Portugal, United Kingdom, Israel, Italy, Romania, Germany

2026-01-15