Improving healthcare for all

Généthon, the pioneer of gene therapies in France

With over 30 years of expertise, Généthon has created a complete ecosystem around gene therapies for rare diseases, from research to production. 

6 Oct 2021

Généthon was created in 1990 by the AFM (French myopathy association)-Téléthon, an association of patients and their parents. This association was founded in 1958 by Yolaine de Kepper, mother of four boys with Duchenne muscular dystrophy, with the aim of drawing attention to rare diseases, particularly neuromuscular diseases into which little research was being carried out. In addition to an innovative research laboratory, “Généthon has today become a real force to reckon with, combining research, development and gene therapy production structure, while remaining non-profit,” says Angela Columbano, Director of business development and partnerships. 

When it was created, Généthon first focused on genome mapping, published in the journal Nature in the early 90s. Once this first step had been taken, Généthon focused on finding the genes responsible for the diseases and then on developing treatments. 

Almost 150 employees work in R&D at Généthon, including 110 researchers and scientific experts, about sixty of whom are affiliated with Inserm. Zolgensma, a gene therapy product aimed at babies suffering from type-1 spinal amyotrophy, whose life expectancy is two years, is derived from technologies developed at Généthon. The company is also co-developing a treatment for Duchenne muscular dystrophy with Sarepta Therapeutics. 10 clinical trials are ongoing, and another 8 products are expected to enter trials in the next 5 years. 

Généthon has also developed a powerful ecosystem, thanks to the creation of Genosafe, a CRO specialising in the quality and safety of gene therapies, and Yposkesi, one of the leaders in the production of gene therapies in Europe.

“At Généthon, we develop gene therapies from research to the clinic. In particular, we are working to improve their safety and efficacy, and we are endeavouring to make them accessible to all those who need them,” Angela Columbano, Director of Business development and partnerships at Généthon.

Généthon was nominated for the Prix Galien Medstartup in the category “Best cooperation dedicated to vulnerable populations and developing countries”, for the partnership with Spark Therapeutics on the gene therapy programme in Pompe disease. 

“This partnership with Spark Therapeutics is key to making gene therapy available more quickly for patients with a rare liver disease that causes muscle weakness and respiratory problems that can lead to death,” says Columbano.

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