Frontotemporal dementia (FTD) and Parkinson’s disease (PD) are heterogeneous neurodegenerative disorders in terms of clinical expression and disease pathology. It is therefore unlikely that there will be a one-therapy-fix all solution. Rather, tailored therapies for specific disease subtypes are more likely to succeed.
Recent scientific progress have demonstrated a pivotal pathogenic role for lysosomal dysfunction in FTD and PD and created novel opportunities for therapeutic interventions. We are focusing on subtypes of FTD and PD associated with mutations in the genes encoding the lysosomal factors progranulin (PGRN, encoded by the GRN gene) and glucocerebrosidase (GCase, encoded by the GBA1 gene), respectively. PGRN and GCase belong to the same lysosomal pathway and their activities are reduced in FTD/GRN and PD/GBA1. By targeting the same pathway for different indications we benefit from major synergies in product development. One major challenge is to successfully restore PGRN and GCase signaling in the brain. We provide novel solutions for both manufacturing and drug delivery specifically tailored for the treatment of FTD/GRN and PD/GBA1.