Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative solutions for people living with rare diseases, today announced the recipients of its “Find For Rare” research grant program. The independently assessed, expert-led research grant initiative aims to improve patient care and management by recognising innovative research in three lysosomal diseases: Fabry disease, alpha-mannosidosis, and cystinosis.
The selected projects demonstrated significant potential to address unmet needs within these rare communities. The recipients include:
- Fabry disease: Mitra Tavakoli, University of Exeter, Exeter, UK, for her project “FAB-PAIN: Precise phenotyping of neuropathy using a range of novel biomarkers in Fabry Disease”
- Project overview: This project aims to explore a range of novel biomarkers to better understand the pain pathways and its pathophysiology in Fabry disease. The findings may lead to the development of a range of new neuropathic biomarkers, advancing the understanding of disease mechanisms and facilitating the creation of diagnostic tools and therapeutic interventions.
- Alpha-mannosidosis: Margarita Dinamarca, University of Basel, Basel, Switzerland, for her project “Investigating brain endothelial dysfunction in alpha-mannosidosis”
- Project Overview: This research is significant for its dual contributions: unraveling the mechanisms by which alpha-mannosidosis disrupts endothelial cell function and pioneering a targeted therapeutic strategy using nanocarriers
- Cystinosis: Francesco Bellomo, Ospedale Pediatrico Bambino Gesù – IRCCS, Rome, Italy, for his project “Study of molecular mechanisms underlying the effects of ketogenic diet in cystinosis”
- Project Overview: This research project investigates the potential of a ketogenic diet to treat nephropathic cystinosis, a rare genetic disorder leading to kidney disease. By studying the diet’s effects in murine models, significant reductions in symptoms such as Fanconi syndrome, inflammation, and fibrosis were observed. The project aims to develop an in vitro system to further explore the molecular mechanisms behind these benefits, potentially enabling the discovery of new therapeutic options.
Applications opened for submission on August 08, 2024, and a total of 82 applications from 23 countries were received. All submitted applications were evaluated by a steering committee of 10 independent leading experts in the field of Lysosomal Diseases, who received an honorarium for participating and reviewing the research projects. Details regarding the application criteria can be located here.
“Fabry disease, alpha-mannosidosis, and cystinosis are rare and ultra-rare lysosomal diseases causing severe, progressive, lifelong challenges, often complicated by diagnostic delays due to their complex and gradual progression,” said Enrico Piccinini, Senior Vice President, EU and International, Rare Diseases at Chiesi Group. “Further research is vital for better diagnostics, new management options, and improved patient outcomes. The selection of these three projects through ‘Find For Rare’ highlights our deep commitment to advancing Lysosomal Diseases care by fostering crucial innovation to meet the evolving needs of patients and their families.”
Chiesi hosted a ceremony on June 18, 2025, at Chiesi’s headquarters in Parma, Italy, to formally recognise the grant recipients and their innovative research.
“The quality and scientific rigor of the proposals submitted to Find For Rare this year were impressive. Each selected project stood out for its potential to address real, day-to-day challenges faced by patients and caregivers affected by lysosomal diseases,” said Prof. Christina Lampe, Chair of the Find For Rare Steering Committee. “By supporting research that is both innovative and patient-focused, this program helps accelerate the translation of science into meaningful impact for rare disease communities.”
