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A major research project in gene therapy is financed thanks to the RHU call of projects

Leaded by the Pr. Marina Cavazzana, the IRIS project was selected among the 15 laureates of the 4th Hospital Research University (RHU) call of projects. With a financial support of more than € 9.9 million, this project is dedicated to the development of gene therapies in the field of severe hereditary immunodeficiencies.

The IRIS project aims to reinforce the role of gene therapy in the therapeutic arsenal of severe hereditary immunodeficiencies and to offer a treatment for patients who cannot benefit from the reference treatment (bone marrow transplant), due to a lack of a compatible donor. Monogenic hereditary diseases of the immune system result from a defective gene that has undergone one or more mutations within blood cells, leading to the dysfunction of the immune system. Bone marrow transplant (source of hematopoietic stem cells) from a compatible donor allows the patient to correct the dysfunction of the immune system. However, in the absence of a compatible donor, the success of transplants remains limited.

The approach proposed in the IRIS project is to develop gene therapy products based on genetically modified stem cell autograft. Autografting involves extracting blood cells from the patient carrying the mutation, modifying them in the laboratory to introduce a functional copy of the defective gene, and then introducing them into the patient. The pathologies targeted by this approach are severe immunodeficiencies like severe combined immunodeficiency-X linked (SCID-X1), chronic granulomatous disease (AR-CGD), FHL3 (Munc 13.4 deficiency), and immune dysregulation disease (IPEX). This approach should overcome the lack of compatible donor and meet patients expectations. The accessibility of these new therapies is often challenged, in particular because of the high price of treatments that can reach more than € 1.5 million on the market. One of the challenges of the IRIS project is also to reduce the costs of the proposed therapy which could be more easily managed by the health system. Medical and economic studies will be designed to evaluate a cost / benefit / effectiveness ratio of the therapy compared to the total societal and economic costs of the treatments provided in the absence of a compatible donor. The IRIS project mobilizes, for a period of 5 years, a strong public-private partnership of international recognized experts. Coordinated by the Assistance Publique-Hôpitaux de Paris (APHP) and leaded by Prof. Marina Cavazzana at the Imagine Institute for Genetic Diseases, the project has also been labeled by the Medicen Paris Region competitiveness cluster. The IRIS project illustrates the scientific and clinical excellence and innovation capacity of Ile-de-France actors in the field of innovative therapies and in particular gene therapy, and the leverage effect conferred by the DIM Gene Therapy labeled by the Paris Region.

Aurélie Laubier, PhD, Project manager DIM Thérapie Génique





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