Genethon, a pioneering laboratory in gene therapy
Founded in 1990 at the initiative of a patient association, Genethon was first a pioneer in genetics, before becoming a leader in the field of gene therapy.
Genethon, pioneer in genetics
In 1987, the first Telethon provided the French muscular dystrophy association with the resources to launch large-scale research. In 1990 it created the Genethon laboratory. Its objective was to decode the human genome, establish its first maps, track the genes responsible for genetic diseases and use this knowledge to produce innovative therapies.
In less than two years, Genethon produced the first mapping of the human genome (in 1992), and continued releasing updated versions through 1996, outpacing US teams and significantly accelerated the global effort to decode the genome.
This world-first achievement solidified the laboratory’s reputation and placed France at the forefront of global genetics. Since then, Genethon has contributed to the discovery of hundreds of genes responsible for genetic diseases.
Genethon, pioneer and leader in gene therapy
In 1997, Genethon shifted its focus to gene therapy, producing its first batch of gene therapy vectors for research. In 1998, the Gene Vector Production Network (GVPN)—linking Genethon with gene therapy labs in Nantes and Marseille’s Institut Paoli-Calmettes—was created to provide innovative, standardized, high‑quality vectors for pre‑clinical trials.
Then in 1999, Genethon established a joint laboratory with Harvard Medical School. Between 1998 and 2002, the network delivered over 2,500 vector batches to 370 research teams in France and abroad.
2002: Genethon’s first therapeutic projects
In 2002, Genethon launched its first therapeutic programs targeting immune and muscle diseases. Three years later, in 2005, the laboratory acquired a “gene and cellular therapy unit” and received authorization to manufacture clinical‑grade vector batches for human trials. By 2006, Genethon had launched Europe’s first human gene therapy trial for a rare muscle disease (gamma‑sarcoglycanopathy).
Gene therapy: early successes
In 2010, Genethon initiated the first international gene therapy clinical trial targeting a severe immune deficiency: Wiskott-Aldrich syndrome (WAS). In 2015, the lab demonstrated efficacy in six treated children, whose immune systems normalized and clinical conditions improved
Since 2010, Genethon has stepped up its human trials with several early victories against diseases.
A first gene therapy medication using technologies derived from pioneering research conducted by a team from Genethon obtained marketing approval from 2019 in the United States, then in Europe and Japan, for infantile spinal muscular atrophy (type 1).Today, thousands of young children around the world have benefited from it. What once seemed impossible is now a reality.
A world-class laboratory at the heart of innovation
Today, Genethon is recognized as a leading global research and development center in the field of rare diseases. With over 240 staff members, 13 gene therapy products in clinical trials, and one treatment already on the market, the laboratory covers the entire value chain: from fundamental research to vector production and clinical trials.
As part of the Biotherapy Institute for Rare Diseases, alongside the Institute of Myology and I-Stem, Genethon embodies a collaborative and integrated approach to biomedical research.
Genethon’s efforts have paid off: gene therapy has been proving its worth. A medical revolution – involving all of us – is underway.
