Press Releases
RSS feedKlaudia Kuranda joins Genethon and the Institute of Myology to create a center of expertise dedicated to immunology
An internationally recognized expert in the field of immunology and the development of gene therapies, Klaudia Kuranda is joining the AFM-Téléthon laboratories to establish and lead a new Immunology Center that will bring together the complementary expertise of Genethon, a leading gene therapy non-profit biotech organization, and the Institute of Myology, a center of expertise … [Read more]
Duchenne Muscular Dystrophy: Start of the pivotal phase of Genethon’s gene therapy trial
A new milestone has been reached in the clinical trial of gene therapy conducted by the Généthon laboratory for Duchenne muscular dystrophy. Building on the positive results of the dose escalation phase, which demonstrated the safety and efficacy of the second dose tested, Genethon has just obtained authorization from the European Medicines Agency (EMA) (with … [Read more]
AFM-Telethon and Muscular Dystrophy Association Announce Research Grant Awards Advancing Treatments for Mitochondrial Myopathies and ALS
AFM-Téléthon and The Muscular Dystrophy Association (MDA) are proud to announce the awarding of two cutting-edge research grants to accelerate the development of treatments for mitochondrial myopathies and amyotrophic lateral sclerosis (ALS). “This partnership with MDA is an essential step to build a strong collaboration between our two associations, through the support of these two … [Read more]
CRISPR-Cas9 : First in vivo proof-of-concept in Steinert’s myotonic dystrophy, a neuromuscular disease
Ana Buj Bello’s team, a researcher in an Inserm unit at Genethon, the AFM-Telethon laboratory, has made the proof-of-concept of a CRISPR-Cas9 approach in a mouse model of Steinert’s myotonic dystrophy, the most common neuromuscular disease in adults. Indeed, thanks to this genome editing approach, the expanded CTG triplet repeat in the DMPK gene, which … [Read more]
The first FDA-approved gene therapy for a neuromuscular disease in the United States is a wonderful hope for patients, also a key challenge for France
On May 24, the Food and Drug Administration approved Zolgensma®, a gene therapy drug for the treatment of spinal muscular atrophy developed by AveXis (Novartis). AFM-Telethon welcomes this historic step for patients and families affected by this neuromuscular disease, whose most severe form kills toddlers before the age of two. AFM-Telethon urges the European Agency … [Read more]
Gene therapy : Towards a clinical trial for gamma-sarcoglycanopathy, limb-girdle muscular dystrophy
Isabelle Richard’s team, a CNRS researcher in an Inserm unit at Genethon, has demonstrated the efficacy of gene therapy and determined the effective dose for treating a rare muscle disease, gamma-sarcoglycanopathy, in mouse models of the disease. Based on these encouraging results, published in Molecular Therapy – Methods and Clinical Development, the researchers are preparing … [Read more]
Myotubular Myopathy : Genethon welcomes interim data of a phase I / II clinical trial with a gene therapy product developed in its laboratories
Audentes Therapeutics, a biotechnology company, launched the first gene therapy clinical trial in children with myotubular myopathy disease using a gene therapy product designed at Genethon. At the 12-week timepoint, the first 3 children treated show early signs of efficacy. With the support of the AFM-Téléthon and the Myotubular Trust, Dr Ana Buj-Bello led the … [Read more]
Généthon and Orchard Therapeutics announce a gene therapy alliance in X-linked chronic granulomatous disease
Orchard Therapeutics (“Orchard”), a clinical-stage biotechnology company dedicated to transforming the lives of patients with rare disorders through innovative gene therapies announces today a strategic alliance with Généthon, to develop gene therapy for X-linked chronic granulomatous disease (“X-CGD”). Under the terms of the agreement, Orchard has obtained an exclusive option to license Généthon’s rights and … [Read more]
YposKesi welcomes Alain Lamproye, an expert in industrial bioproduction, as its Chief Executive Officer
YposKesi, the first French pharmaceutical company to develop and manufacture gene and cell therapy products for rare diseases, created by the nonprofit organization AFM-Téléthon and the SPI fund managed by Bpifrance, have announced the appointment of Alain Lamproye as its Chief Executive Officer. Alain Lamproye brings more than 25 years of experience in Europe and … [Read more]
Metformin, a therapeutic compound highlighting for a rare aging disease : Progeria
A recent study, conducted by Dr Xavier Nissan (I-Stem, INSERM U861) in collaboration with Pr Nicolas Lévy (Aix Marseille University, INSERM UMR_S910, Faculty of Medicine of Marseille) shows that the anti-diabetic drug metformin is able to rejuvenate cells from patient suffering of a rare accelerated aging disease. This work supported by AFM-Telethon, was published in … [Read more]
