Press releases

Media contact: Stéphanie Bardon – communication@genethon.fr – 01.69.47.12.78

Généthon, Winner of the World Innovation Competition 2030

Tuesday April 28, during a ceremony at the Elysee Palace, Généthon, the laboratory of the AFM-Telethon, was named recipient of the Global Innovation Competition 2030 in the category “Risk lift.” A prize that rewards the expert laboratory and world leader in the field of gene therapy for the development of an industrial… (plus)

New gene therapy success in a rare disease of the immune system: Wiskott-Aldrich syndrome

French teams from CIC Biothérapie (AP-HP/Inserm), from pediatric hematology department of Necker Hospital for Children (AP-HP), led by Marina Cavazzana, Salima Hacein Bey Albina and Alain Fischer and from Genethon led by Anne Galy (Genethon/Inserm UMR-S951), and English teams from UCL Institute of Child Health and Great Ormond… (plus)

Effectiveness of innovative gene therapy treatment demonstrated in canine model of Duchenne muscular dystrophy

A collaboration involving three laboratories supported by the AFM-Telethon, Atlantic Gene Therapies (AFM-Telethon, Inserm UMR 1089, Université de Nantes, Nantes University Hospital), Généthon (Evry) and the Institute of Myology (Paris), demonstrated the effectiveness of an innovative gene therapy treatment in the canine model… (plus)

GENETHON and ESTEVE announce agreement to manufacture the gene therapy for the treatment of Sanfilippo Syndrome

France – March 11, 2014 –ESTEVE, a Spanish pharmaceutical company devoted to the research, development, manufacturing and commercialization of novel medicines and Genethon, a non‐profit organization dedicated to the research and development of gene therapies for orphan genetic diseases, announce that they have entered into an agreement to… (plus)

Audentes Therapeutics and Genethon Announce Agreement to Develop Treatment for Severe Genetic Disease X‐Linked Myotubular Myopathy

SAN FRANCISCO, CA – February 5, 2014 –Audentes Therapeutics, Inc., a biotechnology company dedicated to the development of innovative treatments for rare muscle diseases, and Genethon, a non‐profit organization dedicated to the research and development of biotherapies for orphan genetic diseases, announce that they have entered… (plus)

Efficacy of gene therapy demonstrated in canine and murine models of myotubular myopathy

Evry (France), 22 January, 2014. A team of French researchers, led by Dr. Ana Buj-Bello (Genethon/Inserm) and teams at the University of Washington and Harvard Medical School in the United States, have demonstrated the efficacy of gene therapy in models of myotubular myopathy, an extremely severe neuromuscular disease in children. Transfer of the… (plus)

Généthon, the French AFM-Telethon laboratory, becomes the first not-for-profit to obtain authorization from ANSM to be a pharmaceutical manufacturer

Evry, June 27, 2013. Généthon, the AFM-Telethon laboratory, has received the authorization delivered by
the National Agency for Drug Safety (ANSM) to become a pharmaceutical manufacturer. Its production
center, Généthon BioProd, is now authorized to produce drugs for innovative treatments. This is a first
for a laboratory created by an… (plus)

The European charity Myotubular Trust is supporting Généthon in developing gene therapy for myotubular myopathy

Evry (France), June 24, 2013. The European charity Myotubular Trust announces its
support to the development of a pre-clinical gene therapy trial for myotubular
myopathy, a project co-ordinated by Dr. Anna Buj-Bello at Généthon (Evry, France). (plus)

Genethon receives approval from the UK medicine regulatory agency to start a new clinical trial for an inherited immune deficiency: chronic granulomatous disease

On January 10th, the British Medicine and Healthcare Regulatory Agency (MHRA) approved Genethon’s Phase I / II gene therapy clinical trial application in X-linked chronic granulomatous disease (XCGD) to start at Great Ormond Street Hospital in London. Genethon is also seeking approval for this multicenter trial in Germany, Switzerland and France…. (plus)

Decree “advanced therapy medicinal products”: a new milestone for the AFM-Telethon and laboratory Genethon!

The decree relating to “advanced therapy medicinal products” was published in the Journal Officiel on 8 November. A decree awaited since the adoption of the Law of 22 March 2011 allowing organizations to become non-profit pharmaceutical establishments. In the coming days, Genethon submit its application for accreditation by the National… (plus)

Genethon receives the 2012 Prix Galien France

Genethon receives the 2012 Prix Galien France for its innovative treatments of rare diseases using gene therapy and awarded it the prize in the category of “Medicines destined for rare diseases – cell therapy and gene therapy.” (plus)

Genethon, member of the Biotherapies Institute for Rare Diseases

The Biotherapies Institute for Rare Diseases, a unique task force to accelerate therapeutic developments, includes 4 laboratories founded and supported by the AFM Telethon : the Institute of Myology, Genethon, I-Stem and Atlantic Gene Therapies (Nantes’ gene therapy research centre). (plus)

Alain Schwenck, an Expert on Pharmaceutical Prodcution, becomes Director of Genethon Bioprod

As of 2 April this year, Alain Schwenck, 51, has joined Genethon as Director of Genethon Bioprod, the new site for the production of clinical grade-gene therapy medicines for the treatment of rare diseases, created by AFM-Telethon. Mr. Schwenck is a seasoned expert in manufacturing of pharmaceutical products. He began his career at Rhône Poulenc,… (plus)

Fulvio Mavilio, the internationally acknowledged expert in gene therapy for rare diseases, is appointed as Genethon’s new Scientific Director

Fulvio Mavilio, PhD (aged 58), an internationalacknowledged expert and pioneer in gene therapy for rare diseases for more than twenty years, was appointed as the new Scientific Director of Généthon (the not-for-profit biotherapy lab funded by the French Muscular Dystrophy Association (AFM) with donations from France’s annual Telethon) on January… (plus)

Gene therapy : Encouraging results in a Phase I clinical trial in limb-girdle muscular dystrophy type 2C

Evry (France), January 12, 2012 – The results of a Phase I clinical trial of gene therapy for limb-girdle muscular dystrophy type 2C (a rare neuromuscular disease) have just been published in the journal Brain on January 11, 2012. The… (plus)