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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]

YposKesi, the 1st French industrial pharmaceutical company for producing gene and cell therapy drugs for rare diseases

AFM-Téléthon and the ‘Sociétés de Projets Industriels’ SPI [Industrial Projects Companies] investment fund, managed by Bpifrance under the Programme d’Investissement d’Avenir (PIA), are creating YposKesi, the first French company to develop and produce gene and cell therapy products. This new company aims to make the first treatments available to patients and market them at a … [Read more]

I-Stem found a new therapeutic compound for progeria

A study by Xavier Nissan’s research team from I-Stem, in collaboration with Pr Nicolas Lévy’s (Aix-Marseille University – INSERM UMR_S910, Faculté de Médecine de Marseille), Dr Lino Ferreira’s (Coimbra University, Portugal) teams enabled to show the therapeutic potential of retinoic acid (Vitamin A) in the treatment of progeria. Hutchinson-Gilford progeria syndrome is a rare genetic … [Read more]

Long term correction of hyperbilirubinemia in animal models of Crigler-Najjar syndrome after AAV vector-mediated liver gene transfer

Dr. Federico MINGOZZI, Team Leader of the Immunology and Liver Gene Transfer unit at Généthon, (Inserm U951/UPMC), France, has demonstrated the long-term efficacy of an optimized AAV-UGT1A1 vector for the correction of Crigler-Najjar syndrome (CN) in two different animal models of the disease. This work, published in July 20, 2016 in Molecular Therapy Methods and … [Read more]

DMD: efficacy of gene augmentation therapy through a pre-treatment with antisense oligonucleotides

A team from the Institute of Myology research center called “RNA-repair based therapeutic strategies and skeletal muscle pathophysiology, in a study led and conducted by Stéphanie Lorain and Cécile Peccate (UPMC / Inserm / CNRS / Institute of Myology), showed that a pre-treatment made of antisense oligonucleotides improves significantly the benefit of gene therapy for … [Read more]