Objective
Polycystic Kidney Disease (PKD) is one of the most common monogenic disorders and a major genetic cause of kidney failure. The disease is characterised by progressive formation of fluid-filled cysts originating from renal tubular epithelial cells, ultimately leading to loss of kidney function and the need for dialysis or transplantation. Both autosomal dominant and recessive forms belong to the renal ciliopathies, a group of genetic disorders caused by dysfunction of the primary cilium, a cellular organelle essential for environmental sensing and signalling.
Our laboratory has uncovered a previously unrecognised role of primary cilia in metabolic regulation. We discovered that primary cilia sense extracellular glutamine and regulate mitochondrial metabolism, revealing a new metabolic signalling axis linking ciliary function to cellular energy homeostasis. Within this pathway, we identified asparagine synthetase (ASNS) as a key mediator required for glutamine-dependent signalling and metabolic adaptation in cystic epithelial cells.
Building on these discoveries, the ERC Advanced Grant QtCilia has demonstrated that metabolic reprogramming is a central feature of PKD and represents a tractable therapeutic vulnerability. In particular, PKD cells rely on increased glutamine utilization mediated by ASNS. Inhibition of ASNS has shown strong therapeutic potential in preclinical models, supporting the development of ASNS-targeting therapies.
The QrCiliopathies Proof-of-Concept project will translate this discovery into a therapeutic innovation by developing first-in-class small-molecule inhibitors targeting ASNS. The project will optimize lead compounds identified through high-throughput screening and evaluate their therapeutic potential through a structured validation pipeline combining cellular disease models and in vivo PKD models.
Fields of science (EuroSciVoc)
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
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Programme(s)
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
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HORIZON.1.1 - European Research Council (ERC)
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Topic(s)
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Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Funding Scheme
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
HORIZON-ERC-POC - HORIZON ERC Proof of Concept Grants
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Call for proposal
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
(opens in new window) ERC-2026-POC
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Net EU financial contribution. The sum of money that the participant receives, deducted by the EU contribution to its linked third party. It considers the distribution of the EU financial contribution between direct beneficiaries of the project and other types of participants, like third-party participants.
20132 Milano
Italy
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