This partnership relies on cellular models involving genomic engineering and the reprogramming of dermal cells into cardiac cells opening up new perspectives, since it is the leading cause of sudden cardiac death in young adults with no approved treatment to date.
Paris, December 10th – Sanofi and The Institute of Cardiometabolism and Nutrition (ICAN) announced today the signing of a partnership in the fight against cardiomyopathies. The partnership relies on the use of innovative cellular models with cutting-edge technologies to better understand the mechanisms underlying these diseases and identify new therapeutic targets, which may ultimately lead to new drug candidates.
Hypertrophic cardiomyopathy: the leading cause of sudden cardiac death in young adults with no approved treatment to date.
Hypertrophic cardiomyopathy affects 1 person in 500. It is associated with mutations of genes encoding for cardiac sarcomeric proteins, sarcomeres being the basic contractile units of cardiomyocytes (cardiac cells). The disease is characterised by the thickening of the left ventricular walls, which can lead to symptoms such as breathing difficulties, arrhythmias (heart rhythm disorders) and ultimately heart failure. Despite being the leading cause of non-accidental sudden death in young adults, the onset, the severity of the symptoms and the risk of cardiac death all vary greatly. Moreover, there is no specific treatment approved by healthcare authorities.
Clinicians, researchers and patients allied in a translational approach to better understand the biology of the disease.
The goal of the collaboration between the teams of Sanofi and ICAN is to identify the molecular determinants responsible for the expression of the disease (beyond the causal mutation) by exploiting cutting-edge models developed by ICAN involving inducible pluripotent stem cells (iPSC) and gene editing. The idea is to identify, starting with the patient, new therapeutic targets that could change the prognosis of hypertrophic cardiomyopathy.
A clinical study of the natural history of the disease to be launched at the end of the year in patients with genetic hypertrophic cardiomyopathy.
The ICAN clinical teams treat large cohorts of patients with cardiomyopathies, including extreme phenotypes, thanks to the Reference Centre for Hereditary Cardiac Disease (Pitié-Salpêtrière Hospital, Paris). A clinical study, called ‘iPS-CARDIOGEN’ and coordinated by Pr. Philippe Charron, is about to start late 2015 in this Centre. It will concern patients affected by hypertrophic cardiomyopathies for which the genetic origin has been fully established. Patients and their asymptomatic relatives will be asked to give a blood and skin sample for subsequent studies. The clinical and more specifically the cardiological records of the patients will be collected at the same time. This study has received full authorisation from the Health Authorities.
Study using innovative cellular models involving genomic engineering and the reprogramming of dermal cells into cardiac cells.
The technology used will enable skin cells from the patient to be reprogrammed and then differentiated into cardiac cells (cardiomyocytes) thanks to the platform coordinated by Pr. Jean-Sébastien Hulot at ICAN and Dr. Eric Villard for the genomic engineering part.
This approach opens unparalleled research opportunities for the study of human cardiomyocytes. Indeed, cardiac cells generated by this technology retain the mutations responsible for cardiomyopathy and express the biological symptomatology of the disease. These cultivated cells will be characterised and then analysed by the research teams of the Cardiovascular & Fibrosis and Translational Units of Sanofi based in Chilly-Mazarin and Vitry-sur-Seine, France in order to identify the biological targets downstream from the mutation responsible for the disease. This project, starting with the patient, is focused towards the discovery of new drugs for the treatment of genetic cardiomyopathies and, potentially by extension, certain forms of heart failure.
For Marc Bonnefoi, Vice-President of Research and Development France at Sanofi, “This partnership brings hope to patients affected by this disease. It illustrates perfectly our alliance strategy of collaborating with the best teams of clinicians and researchers in France. This collaboration relies on the complementarity of our expertise and constitutes a remarkable vector for acceleration towards the discovery of new drug candidates in a translational approach involving patients, clinicians and researchers.”
Karine Clément, Director of the ICAN University Hospital Institute adds “scientists at ICAN have considerably improved the iPSC-derived cardiomyocyte differentiation technology, the cornerstone of this partnership. The use of iPSC-derived cardiomyocytes is essential as it is impossible to have direct access to the heart of cardiomyopathy patients. This cutting-edge technology allows us to model the disease from human cells in order to understand the mechanisms involved. With this partnership, ICAN demonstrates its capacity to add value to its academic expertise and to stimulate and direct fundamental research towards applied research for the ultimate health benefit of the patients.”
This collaboration will take place over two years and will involve the contribution of more than 50 researchers and clinicians from Sanofi and ICAN.
Sanofi, a global healthcare leader, discovers, develops and distributes therapeutic solutions focused on patients’ needs. Sanofi has core strengths in diabetes solutions, human vaccines, innovative drugs, consumer healthcare, emerging markets, animal health and Genzyme. Sanofi is listed in Paris (EURONEXT: SAN) and in New York (NYSE: SNY).
ICAN is an institute created by the French government’s ‘Investments for the Future’ programme which unites the heart and metabolism centre at Pitié-Salpêtrière Hospital and 14 research teams (120 researchers), working together to better understand and better treat patients with diseases of the heart, metabolism, and nutrition, throughout their progression, and train healthcare professionals in this domain: www.ican-institute.org
About the Reference Centre for Hereditary Cardiac Disease
The Reference Centre for Hereditary Cardiac Diseases has been approved by the Minister of Health as part of the ‘French National Plan for Rare Diseases’. Coordinated by Pr. Philippe Charron at Pitié-Salpêtrière Hospital in Paris, this AP-HP pluri-site centre brings together the expertises enabling to the organization of pluridisciplinary care chains for diverse hereditary cardiac diseases, the editing of diagnostic and treatment referencials, the coordination of research activity, the medical and scientific training, all whilst developing close links with the patient associations affected by these diseases. About 6,000 patients are seen at the site every year.
Media Relations Sanofi
Tel: +33 (0) 1 57 62 00 56
Media Relations ICAN
Sylvain Gilat, PhD
Tel: +33 (0) 1 83 79 89 00
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