A look at the latest advances in gene therapy

In November 2022, Institut Imagine organized a scientific symposium dedicated to gene therapy, its challenges and advances. This event marked the end of five years of accreditation and funding as a Major Field of Interest (MFI) in the field of gene therapy by the Ile-de-France Region. Here is a review of this symposium and the major results of this DIM.

Published on 19.12.2022

Research Acceleration

A high-level symposium

Opened by Prof. Stanislas Lyonnet, Director of the Imagine Institute, Prof. Marina Cavazzana, Scientific Coordinator of the Gene Therapy DIM, and Mr. Olivier Mousson, Advisor to the Ile-de-France Region, this day brought together more than 150 key players in gene therapy.

Among the guests, Dr. Federico Mingozzi, Chief Science & Technology Officer of the biopharmaceutical company Spark Therapeutics, presented a keynote on the progress and challenges in the development of in vivo gene therapy and therapy tools, including AAV viral vectors.   

More than a dozen researchers from the network presented the research work that has benefited from the support of the Gene Therapy DIM in a number of areas: hemoglobinopathies such as sickle cell disease, metabolic and mitochondrial diseases, neuromuscular diseases, hearing disorders and hereditary kidney degenerations. Many innovative techniques and tools were also presented, such as gene editing, the CRISPR Cas9 system, and CAR T cells. 

DIM GTRU 2022

Success of the DIM-Gene Therapy 

Gene therapy is one of the areas of major interest (DIM) accredited by the Île-de-France Region. Led by the Imagine Institute and Prof. Marina Cavazzana, this DIM aimed to accelerate the development of gene therapy by regularly opening calls for projects to promote the recruitment of researchers, post-doctoral students, PhD students, engineers and technicians, the establishment or sharing of technological platforms, access to equipment, certification and the conduct of medical and economic studies. To find out more about the project.

DIM logo

This event was an opportunity for Prof. Marina Cavazzana to look back at the key results of the program and the major achievements in the field since 2017:

  • 12 million euros dedicated to gene therapy research
  • 56 projects conducted in 11 strategic therapeutic areas
  • 42 scientific publications by network members
  • 15 patents filed
  • More than €25 million in additional funding raised by the network (from industrial collaborations or public/private funding)
  • More than 10 collaborations with industrialists, big pharma, biotech and startups
  • Creation of a spin-off (Gamut Therapeutics) and a start-up in the field of metabolic diseases (creation in progress)
  • Creation of a de novo AAV platform at the Imagine Institute and strengthening of two AAV platforms in Ile-de-France (at the Vision Institute and the Myology Research Center)

The DIM has enabled the structuring of a network of excellence around a therapeutic strategy of personalized medicine, and to accelerate discoveries to bring research and care to the bedside of patients.

Gene therapy at Institut Imagine

Institut Imagine is home to research teams at the forefront of this therapeutic approach, and some of its researchers were the very first in the world to conduct clinical trials and demonstrate the success of this approach for certain diseases.

Since then, gene therapy has continued to improve and is now deployed for many other diseases. At Imagine, a reference institute in research on genetic diseases, with the reference centers and clinical services of the Hôpital Necker-Enfants malades AP-HP, this approach is being explored to treat diseases of the blood, immune system, metabolism, brain, central nervous system, eye and skin.

To learn more about the work carried out at Institut Imagine

Learn more about two studies on gene therapy in sickle cell disease, beta-thalassemia and Wiskott-Aldrich syndrome.

Learn more about a new gene therapy strategy for sickle cell disease and beta thalassemia