Hematology: a Health Issue and Priority for the Institut Imagine

Research must detect the nature of the disease as quickly as possible so as not to waste time… and also to spare all the children suffering from the transplants and intensive treatment that I have been through.

Sandrine M., who has a genetic disease that required intensive chemotherapy, a bone marrow transplant at 9 months old, and a lifelong treatment.

The Project

Understanding the origin and mechanisms of blood diseases, and developing innovative treatments.

Blood disease can occur due to a genetic defect in one of the cell lines of the blood. Depending on the cell concerned – white blood cells, red blood cells, plates – this disease takes a wide variety of forms: anemia, mastocytosis, hemophilia, etc. The objectives of this program are to understand the molecular origin and expression of these diseases, and to develop new therapeutic strategies (including gene and cell therapies) to treat them.

It mobilizes:

◆ 3 Imagine research laboratories
◆ 4 translational research units
◆ 2 clinical services
◆ 4 hospital laboratories
◆ 5 rare disease reference centers
◆ 1 Labex (laboratory of excellence funded through the state-funded « Investments d’Avenir » Program)
◆ DIM program (Domaine d’Intérêt Majeur) in Gene Therapy, funded by the Ile-de-France Region, with the aim of uniting all actors in the territory and accelerate the development of Gene Therapy
◆ 1 Chair Program

Context & Objectives

Genetic blood diseases affect a considerable number of people worldwide. In the case of sickle cell disease, which is just one of them, nearly 50 million patients worldwide suffer from extremely severe and painful anemia that can lead to death.

This is what motivates the Institut Imagine to make genetic research in hematology one of its 6 priority areas. These areas are real accelerators of research and treatment, entirely focused on innovative and gene-derived therapies, always with the same aim: to bring care to patients, help their families, and contribute to medical progress.

Amount to fund

1,000 K€

Overall funding over 5 years

/ For the acquisition of cutting-edge equipment

/ For the recruitments required to bring the program to life: engineers, doctoral students…

/ For the implementation of a platform: an engineering position, a room dedicated to experimentation

ABOUT THE PROJECT LEADERS

Isabelle André heads the Human Lymphohaematopoiesis laboratory at the Institut Imagine, whose research focuses on the development of the human hematopoietic immune system. Her team has identified new genes involved in severe forms of inherited immune deficiencies in children.

Elected in 2017 by the Academy of Sciences, Olivier Hermine is a Professor of Hematology at Université Pars Cité, Head of the Hematology Department at the at the Necker-Enfants malades Hospital, and Director of the “Cellular and Molecular Mechanisms of Hematological Disorders and Therapeutic Implications” laboratory at the Institut Imagine. He is also Coordinator of the Mastocytosis Reference Center (CeReMast).

Annarita Miccio is Director of the « Chromatin And Gene Regulation During Development » laboratory. In 2019, she was awarded one of the prestigious grants of the European Research Council (« ERC »), a boost for this researcher who develops the gene therapies of tomorrow.

Marina Cavazzana is Professor of Hematology at Université Paris Cité, Head of the Biological Therapy Department and the Clinical Investigation Center for Biotherapy at the Necker-Enfants malades AP-HP Hospital, and co-Director of the Human Lymphohematopoiesis laboratory at the Institut Imagine. Recognized internationally for her pioneering work in gene and cell therapies, she was elected by the American Academy of Medicine in 2019.

CONTACT

LAURENT MELLIER

Director of Development and International Philanthropy

laurent.mellier@institutimagine.org

+33 (0)1 42 75 45 79