In order to develop gene therapy treatments and take them from basic research to clinical trials, Genethon relies on a team of experts, including five research teams and three advanced technology platforms, working closely with the clinical development teams as well as with the technological and bioprocess development teams.
Almost 150 people work on R&D at Genethon, including 110 researchers and scientific experts.
Five research teams, working in a mixed unit managed in partnership with Inserm and University Paris Saclay, work on Genethon’s areas of priority. Headed by Anne Galy, the “Integrated genetic approaches in therapeutic discovery for rare diseases” unit (Integrare), conducts high-level translational research on gene therapy for rare diseases, to provide a basic approach to areas of therapeutic and technological interest for Genethon.
Three high-performing technological platforms are there to support Genethon’s scientific teams.
Immunology and biotherapies – Anne Galy – UMR 951
The immune and hematopoietic system team has two areas of expertise: one is developing new gene therapy treatments using genetically modified immune or blood cells; the other is studying unwanted immune responses in gene therapy in order to improve vectors.
Progressive dystrophies – Isabelle Richard
Isabelle Richard’s team works on muscular dystrophy. These progressive neuromuscular diseases are disabling, and sometimes fatal, without treatment to slow or halt progression of the disease.
Neuromuscular diseases and gene therapy – Ana Buj-Bello
The team is focused on the development and evaluation of therapeutic strategies to correct genetic defects that may affect the neuromuscular system. It is working on congenital muscle disorders such as myotubular myopathy.
Gene editing – Mario Amendola
The team is looking at the development of effective and safe gene editing techniques, as well as traditional gene therapy approaches, to treat rare genetic diseases in vivo and ex vivo.
Immunology and liver diseases – Giuseppe Ronzitti
The Immunology and liver diseases team lends its technical and scientific expertise to work in vectorology, cellular biology and immunology to provide innovative therapeutic solutions to patients suffering from rare hereditary metabolic diseases. Its work ranges from proof of concept to clinical trials, and is focused in three particular areas.
Imaging-cytometry platform (ImCy)- Daniel Stockholm
The mission of the ImCy facility is to provide imaging and flow cytometry technologies to support Genethon research. The facility, named Genopole, is also open to external users.
DNA bank – Safaa Saker
Since Genethon was founded in 1990, its DNA and Cell bank has provided the scientific community with the high-quality services of a cell and human products bank.
Europe’s leading bank for genetic diseases, it operates as a service to the entire medical and scientific community. It has been certified according to the AFNOR NF-900 standard, since June 2009.
Preclinical assessment platform – Nathalie Danièle
The preclinical assessment facility enables the evaluation of gene therapy treatments in animal models in compliance with regulatory and ethical procedures for research studies and preclinical studies.
The department is split into four teams.
- Bio-experimentation team that administers treatments and monitors animals under protocol.
- Functional exploration team that measures the effectiveness of treatments on muscle, respiratory and cardiac function.
- Histology-immunolabeling team that measures the effectiveness and tolerance of treatments on all aspects of the tissues.
- Molecular evaluation team that measures the molecular improvements related to treatments on all of the body’s tissues and fluids.