Généthon I – Mapping the Human Genome
EN Généthon II – The Gene Hunters
Généthon III – Vector Study Pioneers
Généthon IV – Leading the Way in Human Trials
Gene Therapy Pioneers
Technological innovations

Généthon I – Mapping the Human Genome

J. Weissenbach, I. Chumakov, D. Cohen et J. Dausset

J. Weissenbach, I. Chumakov, D. Cohen et J. Dausset


During this initial period, Genethon made its mark by becoming the first in the world to publish a map of the human genome. This map, an unprecedented discovery, was offered to the scientific community in 1992, and advanced the human genome sequencing project by six years.

At this time, three major projects were being conducted at Genethon:

  • a physical map of the human genome, directed by Daniel Cohen;
  • a genetic map, directed by Jean Weissenbach;
  • and an inventory of gene transcripts for muscle and nerve cells, directed by Charles Auffray.

On September 18, 1992, the journal Cell published Genethon’s first physical map covering half of the human genome.

On October 1, the journal Nature published Genethon’s first map of a chromosome, chromosome 21.

On October 29, the laboratory used the same journal to publish an article about the first markers on the genetic map (814 markers).

These three world Firsts confirmed the laboratory’s outstanding reputation, and placed France on top in the world genetic research stakes.

EN Généthon II – The Gene Hunters

Genethon II took over from Genethon I in order to put the advances made in the field of human genome mapping into practice for the benefit of patients, and to speed up the work on localizing and identifying the genes involved in various genetic disorders. The samples collected by the Cell Bank, and its collection capacity for new pathologies, provided the necessary biological resources.

Either through its own teams, who were the ones creating the genome maps, or through its platform providing services to external teams, Genethon was involved in identifying the genes involved in several hundred types of genetic disorders.

In 1996, gene sequencing and genotyping activities were transferred to the French Government, so Genethon refocused its attention on therapeutic gene transfer.

Généthon III – Vector Study Pioneers

The mission of Genethon III was to develop tools for delivering therapeutic genes in a therapeutic setting.

Under the scientific management of Olivier Danos, Genethon concentrated on researching, developing and manufacturing gene therapy vectors: AAV (adeno-associated virus), and retroviral and lentiviral vectors.

Its primary goal was to provide quality vectors for preclinical animal studies for the whole of the scientific community.

Its second aim was to develop the best possible procedures for producing vectors for human use.

Genethon III eventually produced 3,000 batches of vectors that were used for preclinical studies within the GVPN (Gene Vector Production Network).

Généthon IV – Leading the Way in Human Trials

The advances made in understanding rare genetic disorders and in the development of gene therapy vectors led Genethon to set up a number of preclinical programs. While continuing with its extensive research activities, Genethon put in place the various structures that would be needed to start clinical trials in humans.

The year 2004 saw another World First for Genethon, when Olivier Danos and Luis Garcia succeeded in restoring dystrophin expression to the limb muscles in a murine model of Duchenne muscular dystrophy using exon skipping (journal Science, December 3, 2004).

In 2005, its GMP-compliant vector production site was certified by the French Healthcare Products Safety Agency (AFSSAPS), and became a Gene and Cell Therapy Facility (as per French law).

In 2006, Genethon IV was authorized to launch its first gene therapy trial for gamma-sarcoglycanopathy. At the same time, clinical grade vector preparations were underway for two other trials in Duchenne muscular dystrophy and Wiskott-Aldrich syndrome.

Gene Therapy Pioneers

Over the years, Genethon has proved capable of accompanying a candidate drug from the preclinical stages through to human clinical trials. Genethon applies its skills both to gene therapy projects that have been developed in its own laboratories and to those conducted by external teams.

Since 2005 Genethon has had its own manufacturing capacity for the production of gene therapy vectors according to the standards of Good Manufacturing Practices and In 2013, Genethon opened a new production center, Genethon BioProd, which is authorized to operate as a Pharmaceutical Establishment, to manufacture therapeutic candidates for clinical trials in France or abroad in strict compliance with regulations for pharmaceutical products.

On November 2, 2016, AFM-Téléthon and the SPI fund, managed by Bpifrance under the Programme d’Investissement d’Avenir, are creating YposKesi, the first French industrial pharmaceutical company dedicated to producing gene and cell therapy drugs for rare diseases

This new company aims to make the first treatments available to patients and market them at a fair and controlled price, consistent with the commitment made by AFM-Téléthon and its founders’ characteristic general interest strategy.

Genethon BioProd and its teams have integrated YposKesi. Ultimately, YposKesi will include three production buildings for gene and cell therapies.

Technological innovations

Today, Genethon is conducting, alone or in partnership with international excellence teams, several projects for the development of therapies for rare diseases, affecting muscles, immune system, blood, liver and vision.In 2012 Genethon received the Prix Galien France for pharmaceutical research into “Medicines destined for rare diseases – cell therapy and gene therapy” and in 2015 Genethon was the recipient of the Global Innovation Competition 2030.

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