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Moins de deux ans après sa création, EspeRare se place dans le top 8 des start-ups dans lesquelles investir. Le magazine Blian présente, dans son édition de février, la sélection 2015 des 50 start-ups suisses dans lesquelles investir, tous secteurs d'activités confondus. Dans ce contexte, la fondation a reçu les recommandations spéciales de deux membres du jury.

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EspeRare explore le potentiel thérapeutique de Cilengitide dans la glomérulosclérose segmentaire focale avec l'Université de Bristol, Royaume-Uni.

Cilengitide is an investigational compound developed by Merck Serono, the biopharmaceutical division of Merck.Focal Segmental Glomerulosclerosis (FSGS) is a rare disease that causes impair renal function due to scaring of glomeruli (structures within the kidneys composed of capillary blood vessels actively involved in filtering harmful substances out of the blood). The underlying disease mechanism involves the alteration of cells called podocytes within those glomeruli. Published scientific data suggests that the inhibition of αvβ3 integrin could potentially modulate the pathologic processes characterized by inappropriate motility of podocytes in this rare renal disease.

EspeRare has initiated a collaboration with Professor Moin Saleem at University of Bristol to test the ability of cilengitide, a αvβ3 integrin inhibitor, to activate the motility of podocytes in several FSGS experimental models. If demonstrated, such effect could potentially translate into a reduced or halted decline of renal function and progression to end stage kidney disease in humans. In that context, cilengitide represents an attractive investigational candidate for a potential future therapy for FSGS patients.

This first assessment of the therapeutic potential of cilengitide in non clinical models of Focal Segmental Glomerulosclerosis is set to be completed by the end of 2014, and is co-funded by Merck Serono.

About Cilengitide
Cilengitide is an investigational compound developed by Merck Serono. The small molecule is a potential first in a class of αvβ3 integrin inhibitor that was in clinical development in oncology until Phase III. The development of this drug candidate was halted in 2013.
Merck Serono has also previously provided support to EspeRare in the development of another of its investigational products, Rimeporide, in the deadly pediatric disease Duchenne Muscular Dystrophy, through initial fund donation, and transfer of compound rights to EspeRare. This joint project is still ongoing.

About Focal Segmental Glomerulosclerosis (FSGS)
FSGS is a rare form of a nephrotic syndrome that affects both children and adults with peaks at 6-8 and 20-30 years of age, respectively. The life expectancy of a 10 year-old child on hemodialysis due to end stage kidney disease is dramatically reduced.
The cause of FSGS is unknown. The disease effects a specific part of the kidney called glomeruli. The role of the glomeruli is to filter soluble waste such as by-products of cellular metabolism from the blood, the first step to form urine. When barriers in this filter are damaged, proteins begin to leak into the urine and over time cause kidney failure. The diagnosis of FSGS is based on the presence of proteinuria, possibly signs and symptoms secondary to proteinuria (oedema), and histologic examination of kidney biopsy.The disease burden is tremendous to patients with this lifelong chronic disease; particularly it requires management of dialysis and in some cases transplantation. Currently, there is unfortunately no cure for this debilitating disease.

About Professor Moin Saleem, University of Bristol
Professor Moin Saleem, Professor of Paediatric Renal Medicine, at University of Bristol, School of Clinical Sciences, is a world renowned expert in glomerular diseases and particularly the nephrotic syndromes. He is a pioneer in the field of podocyte biology and originated the gold standard of a conditionally immortalised human podocyte cell line, now in use worldwide in academia and industry. In 2007 he was awarded a Medical Research Council grant to set up the UK renal rare disease registry (www.renalradar.org), which has grown into a national strategy adopted by the UK Renal Association.

La présidente et la directrice d’EspeRare sont les auteurs d’un chapitre décrivant le rôle des associations de patients dans le développement de médicaments orphelins dans une monographie intitulée « Orphan Drugs and Rare Diseases » publiée par la « Royal Society of Chemistry » du Royaume-Uni. Pour voir un extrait du chapitre cliquer ici

The book is an up-to-date monograph written by a combination of academic and industry experts working in the field of orphan diseases drug development, this text brings together expert authors in the regulatory, drug development, genetics, biochemistry, patient advocacy group, medicinal chemistry and commercial domains to create a unique and timely reference for all biomedical researchers interested in finding out more about orphan drugs and the rare diseases they treat.

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EspeRare et Merck Serono sont l'un des trois lauréats du concours «co-création sociale & commerciale: l'impact de la collaboration" 

The prize was awarded for its project “Advancing treatment for Duchenne” at the 5th Zermatt Summit “The Courage to Dare” on June 27th. The jury was constituted by Ashoka, the Zermatt Summit Foundation, Fondation Guilé, DPD and Boehringer Ingelheim, they reviewed 338 projects, selected 15 finalists and named 3 winning Social & Business Co-Creation projects.

Please follow this link to see all winners

About Merck Serono
Merck Serono is the biopharmaceutical division of Merck. With headquarters in Darmstadt, Germany, Merck Serono offers leading brands in 150 countries to help patients with cancer, multiple sclerosis, infertility, endocrine and metabolic disorders as well as cardiovascular diseases. In the United States and Canada, EMD Serono operates as a separately incorporated subsidiary of Merck Serono.
Merck Serono discovers, develops, manufactures and markets prescription medicines of both chemical and biological origin in specialist indications. We have an enduring commitment to deliver novel therapies in our core focus areas of neurology, oncology, immuno-oncology and immunology.

About the “Social & Business Co-Creation: collaboration for impact” competition:
The competition Social & Business Co-Creation: collaboration for impact, has been developed by Ashoka, the Zermatt Summit Foundation, Fondation Guilé, DPD and Boehringer Ingelheim to recognize innovative Co-Creation projects in Europe led by social-mission organisations, traditional businesses and public institutions.

The 3 winners receives coaching and a first prize of € 20,000, two runner-up prizes of € 10,000. Two early entry prizes of €1,000, a last mile and a special jury prize are also awarded.

About the Zermatt Summit: 
Created in 2010 as a primary international platform, the Zermatt Summit promotes a constructive, action oriented dialogue between key stakeholders from different universes who share the same purpose. Its role is to inspire, connect and empower high profile entrepreneurs, economists, political and spiritual leaders and NGO representatives who strive to be catalysts of a more inclusive and human globalization. Through a uniquely designed program, the Zermatt Summit team takes participants on an intense three-day journey to share a vision for a better society, develop models for a more human and sustainable world and change their hearts and minds in the process. 

La fondation a été présentée dans le 25ème volume de la revue de la Société Suisse de Pédiatrie.

Cliquez ici pour consulter l'article en page 22-23 de l'édition de Pediatrica en cours.

La Loterie Romande attribue un don à la fondation pour financer l’infrastructure informatique de sa plateforme de recherche translationnelle.

Cette plateforme, actuellement en cours de développement, facilitera:
- L’identification systématisée de nouvelles opportunités de développement thérapeutique dans les maladies rares
- La gestion standardisée des données biomédicales générées lors du développement de nouveaux traitements
- Le partage des informations avec les collaborateurs externes (associations de patients, Universités et partenaires pharmaceutiques)

Ce projet s’étend sur deux ans. Une première version de la plateforme sera opérationnelle d’ici la fin du premier semestre 2014. Jusqu’en décembre 2015, les performances de la plateforme seront optimisées et d’autres sources de données telles que les librairies de composés pharmaceutiques du NIH (National Institute of Health) y seront intégrées. Cette plateforme favorisera le développement du portefeuille de projets dans les maladies rares d’EspeRare et permettra d’appliquer une gestion des données répondant aux critères de qualités de l’industrie et des autorités règlementaires.

Par son soutien, la Loterie Romande contribue au développement des activités d’EspeRare dans le domaine de la santé, en aidant la fondation à étendre ses actions de développement thérapeutique dans les maladies rares.

A propos de la Loterie Romande

EspeRare représente la Suisse au sein d’une Action pour la Coopération Européenne dans le domaine de la recherche scientifique et technique (COST) sur le thème de la Santé des Citoyens.

Bruxelles, le 27 Novembre 2013. En tant que membres du comité, Sharon Terry et Caroline Kant ont participé au lancement de l’Action COST IS1303 sur le thème de la Santé des Citoyens à travers l’initiative publique-privée « Santé publique, Marché et Perspectives Ethiques ». Vingt-deux pays sont représentés au sein de cette Action, dont l’objectif est de développer des directives légales et scientifiques dans le domaine de la Santé des citoyens, en particulier les tests génétiques et le dépistage, les biobanques, l’éthique et la justice dans le domaine de la santé et la démocratisation de la recherche.

*COST (Coopération européenne dans le domaine de la recherche scientifique et technique) est un programme financé par la Commission Européenne permettant de coordonner et d’aider la circulation du savoir à l’échelle pan-européenne.

EspeRare démarre deux collaborations de recherche visant à démontrer l'efficacité préclinique de Rimeporide dans Duchenne.

Geneva, Switzerland, August 19, 2013 - The foundation announces that it has started two collaborations with the University of Geneva (Switzerland) and the Children National Medical Center in Washington DC (USA) to develop Rimeporide, its lead programme in Duchenne Muscular Dystrophy (DMD) patients. The objective is to assess the therapeutic utility of this compound, a safe and specific Na+/H+ exchanger type 1 (NHE 1) inhibitor previously developed for cardiac failure, for the treatment of DMD.

Rimeporide preclinical efficacy will be tested in DMD mice models. The assessment will be conducted under the leadership of Dr. Kanneboyina Nagaraju specialized in DMD at Prof. Eric Hoffman's research center for genetic medicine, Children National Medical Center, Washington DC.
In parallel, Dr Olivier Dorchies, a recognised DMD preclinical expert at the Pharmaceutical biochemistry lab of Prof. Leonardo Scappozza, University of Geneva, will also investigate the dose / efficacy relationship of Rimeporide in a mice model  and evaluate the efficacy in various cell lines.

Preliminary data from both collaborations are expected by second quarter 2014.

This preclinical work together with the already available data will support further development of this compound in DMD patients. This program has ultimately the potential to address muscle degeneration and cardiomyopathy in a broad segment of DMD children.