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EU funding (€7,425,447): Therapies for Renal Ciliopathies Hor28 Jun 2023 EU Research and Innovation programme "Horizon"
Overview
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Therapies for Renal Ciliopathies
Ciliopathies are a large group of rare and severe genetic diseases caused by dysfunction of the primary cilium, a microtubule-based cell surface antenna that controls key signaling output required during development and tissue homeostasis. Cilium dysfunction leads to complex disorders with high genetic heterogeneity and overlapping phenotypes. Despite the broad clinical spectrum, chronic kidney disease (CKD) leading to end stage kidney disease (ESKD) is a common cause of morbidity across ciliopathies. Currently, the only available standard of care for CKD is based on dialysis and transplantation. Renal ciliopathies represent a main cause of ESKD during childhood and despite the identification of more than 40 causative genes, it remains difficult to predict the severity of the disease as well as the risk of appearance (if not present at diagnosis) and the rate of progression of renal failure. TheRaCil therefore aims: (1) to improve diagnosis and prognosis of at risk pediatric renal ciliopathy patients, and (2) to implement therapeutic approaches aimed at targeting shared pathological pathways, at modifying mRNA targets of the causative or modifier genes by antisense oligonucleotides and by the repurposing of available molecules. These goals will be achieved through the federation of our unique databases of pediatric renal ciliopathies cases available across Europe, which will allow a better stratification of patients, the identification of modifier genes and markers of disease progression. Bioinformatics approaches will be used to integrate patients’ biological and genetic data as well as multi-omics and functional analyses from patients samples and preclinical models. These analyses should lead to the identification of shared targetable pathological pathways as well as of patients eligible for the identified new therapeutic approaches which will be evaluated in robust preclinical models.
Funded Companies:
| Company name | Funding amount |
| Ciliopathy Alliance | ? |
| Assistance Publique Hopitaux de Paris | €27,955 |
| Bergmann Carsten | €91,838 |
| Imagine Institut des Maladies Genetiques Necker Enfants Malades Fondation | €1,884,483 |
| Institut National de la Sante et de la Recherche Medicale | €592,348 |
| Klinikum DER Universitaet ZU Koeln | €708,931 |
| Kobenhavns Universitet | €788,533 |
| Medetia SAS | €373,200 |
| Ospedale SAN Raffaele Srl | €338,750 |
| Ruprecht-Karls-Universitaet Heidelberg | €416,000 |
| Stichting Radboud Universitair Medisch Centrum | €809,060 |
| Thelonius Mind | €105,000 |
| Universitaet Muenster | €280,930 |
| Universitair Medisch Centrum Utrecht | €471,558 |
| Universitatsklinikum Heidelberg | €536,863 |
| Universite de Strasbourg | €0.00 |
Source: https://cordis.europa.eu/project/id/101080717
The filing refers to a past date, and does not necessarily reflect the current state. The latest known state is available on the following page: Ciliopathy Alliance, London.