Chiesi launches research grant initiative for Fabry, other LSDs
'Find For Rare' seeking proposals from scientists outside the Americas
Chiesi Global Rare Diseases is launching a research grant initiative to support innovative studies into Fabry disease, alpha-mannosidosis, and cystinosis — all three of which are lysosomal storage disorders (LSDs).
“Whilst the disorders can be rare individually, their prevalence is significant at a global level with an estimated 1 in 8,000 people diagnosed,” the company stated in a press release announcing the new grant program.
The initiative, called Find For Rare, is seeking proposals from researchers outside the Americas, who can apply for grants up to €50,000 (about $55,000) with projects lasting up to two years. Applications are open until Oct. 31, and grants totaling €150,000 (approximately $166,000) will be announced early next year.
“Find For Rare is calling for all principal investigators and scientists with an interest in lysosomal storage disorders to apply for this fantastic research opportunity,” said Christoph Wanner, MD, chair of the Find For Rare steering committee and a professor at the University Hospital of Würzburg in Germany.
Independent scientific board will review research grant applications
Lysosomal storage disorders, or LSDs for short, are a group of conditions in which certain enzyme deficiencies impair the function of lysosomes — organelles that work as the cells’ recycling centers — resulting in an abnormal accumulation of specific molecules inside cells.
In Fabry disease, patients have mutations that result in a deficiency in the alpha-galactosidase A enzyme, which is needed to break down fatty molecules such as globotriaosylceramide (Gb3). As a consequence, Gb3 toxically accumulates inside lysosomes, which leads to progressive organ damage, particularly to the heart, kidneys, and nervous system.
Alpha-mannosidosis, also a rare LSD, is a genetic condition characterized by a deficiency of the enzyme alpha-D-mannosidase, which is needed to break down certain sugar molecules. Cystinosis is marked by a deficiency in the cystinosin enzyme that transports the amino acid cysteine out of the lysosomes.
“Whilst we have expanded our knowledge into LSDs in the last decade, we still need to generate greater understanding into the factors affecting the disorders from pathogenesis [disease development] to progression,” Wanner said.
The initiative will fund projects meant to better understand the factors affecting these diseases, including the mechanisms associated with their onset and progression. Chiesi also intends to fund studies designed to personalize disease management. Importantly, preclinical or clinical studies assessing the safety and efficacy of drugs will not be considered.
Whilst we have expanded our knowledge into LSDs in the last decade, we still need to generate greater understanding into the factors affecting the disorders from pathogenesis [disease development] to progression.
After submission, proposals will be evaluated by a steering committee — an independent scientific board of 10 experts in the field of lysosomal storage disorders. The committee will assess the projects’ strength, relevance, and innovation, as well as their potential for success. Chiesi noted that the company will not be involved in or influence the grant selection process.
“Find For Rare represents an opportunity to discover new thinking and ambition to meet our ultimate goal of improved patient care,” Wanner said. “The steering committee is eagerly awaiting the opportunity to review new ideas from across the globe and hopefully identify excellent candidates to receive the grants.”
Chiesi collaborated with Protalix to develop Elfabrio (pegunigalsidase alfa), one of the two enzyme replacement therapies approved in the U.S. for people with Fabry. It’s now solely responsible for selling the therapy in the U.S.
“Similar to all of our activities, the inspiration behind this initiative is the rare disease community,” said Enrico Piccinini, head of Europe and International Chiesi Global Rare Diseases. “We pride ourselves on listening closely to their needs and delivering what will truly benefit patients – our real hope is this year’s Find For Rare initiative will provide future solutions to today’s challenging rare conditions.”
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