First Patient Dosed in Phase 1/2 Clinical Trial in Europe of Gene Therapy for LGMD-R9

Study to treat FKRP-related limb-girdle muscular dystrophy type 2I/R9 represents 30 years of research and is being conducted by Genethon spinout Atamyo Therapeutics.

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Meeting with Jessica Cartigny, Purification Design Engineer at Genethon

When she arrived in 2016, Jessica was drawn to Genethon for its human values. She began in the position of lab technician. Six years later, she is now a purification design engineer and is flourishing within a dynamic and compassionate team.

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The Genethon platforms obtain ISO 9001 certification

As of July 8, 2022, the Imaging-Cytometry, Preclinical Evaluation and Technological Development platforms have been officially ISO 9001 certified. An additional guarantee of the quality of the activities of these platforms.

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Anne Galy receives the WAS Foundation Callahan Award for her work on Wiskott-Aldrich syndrome

The WAS Foundation awarded the Callahan Prize for outstanding service to Anne Galy, head of the Immunology and biotherapies team. She has worked for more than 20 years on Wiskott-Aldrich syndrome, a severe immune deficiency of genetic origin.

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Sickle-cell anemia: a project involving the Gene editing team has just secured European funding

The “EDITSCD” project, that aims to improve gene editing strategies for sickle-cell anemia, this hereditary disease of the red blood cells, has just been selected by the European Union. It will involve a dozen or so research laboratories in Europe, among then the Gene editing team (Inserm UMR 951) headed by Mario Amendola at… (read more)

Meeting with Justine Klonowski, legal manager at Genethon

Recently promoted to legal manager at Genethon, Justine Klonowski plays a key role in the development of contracts between Genethon and its partners. She says she is proud to be part of the work of this atypical lab.

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Emanuèle BOURGES and Joëlle CHEUZEVILLE, USP engineers, a key role in drug production

Contrasting profiles of Emanuèle and Joëlle, both USP development engineers (cell culture processes) in Genethon’s Technology Development Department. Although they work in the same role, they each live the Genethon adventure in their own way.

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Gene Therapies Continue to Emerge as Effective Treatments for Rare Diseases, But Complex Challenges Remain

To mark International Rare Disease Day, Genethon identifies the most urgent priorities in overcoming these obstacles.

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Five post-doctoral positions open at Genethon, a leader in gene therapy for rare diseases

A pioneer and leader in gene therapy for rare disease, Genethon, a non-profit organization, recruits 5 post-doctoral fellow to develop research projects the Inserm-Genethon-UEVE Integrare unit.

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Genethon’s Lentiviral Vector-Based Gene Therapy Demonstrates Long-Term Safety and Efficacy for Wiskott-Aldrich Syndrome

Genethon announced that its lentiviral based gene therapy, developed in collaboration with French and British teams, has demonstrated long-term efficacy in eight patients with Wiskott-Aldrich syndrome, a rare and severe immune deficiency.

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From 2021, a seminal year for Genethon, to 2022, a year of challenges

After a year of significant breakthroughs in 2021, 2022 sees Genethon continuing its efforts in face of the medical and technological challenges of gene therapy for rare diseases, helping patients and their families.

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Myotubular myopathy: “Today my son Jules is able to stand”

Suffering from myotubular myopathy, a disease that’s often fatal before the age of 2, Jules received a gene therapy treatment produced by research from Genethon, as part of a clinical trial in January 2020. His progress is spectacular.

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Pierre, suffering from limb girdle muscular dystrophy: “For me, a cure would mean no more losing”

Pierre is a 14-year-old middle-schooler. Four years ago he received the diagnosis on FKRP limb girdle muscular dystrophy. This progressive disease saps his strength little by little. At Genethon, Isabelle Richard’s team has worked for over 25 years on limb girdle muscular dystrophy. A first gene therapy treatment for Pierre’s disease could… (read more)

Spinal muscular atrophy: for Victoire’s mom, treatment was a magical moment

Suffering from spinal muscular atrophy, Victoire received a gene therapy treatment derived from technologies developed at Genethon. Her mom recounts the anguish of the diagnosis, the immense relief of the treatment and the little girl’s spectacular progress in the weeks following the injection.

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Preliminary results of clinical trial for rare liver disease Crigler-Najjar syndrome presented at ESGCT congress

After a first presentation at the International Liver Congress last June, new preliminary results have just been presented at the European Society of Geneand Cell Therapy (ESGCT) congress by Dr d’Antiga, one of the investigators of the trial (Bergamo, Italy). Based on initial observations, the drug candidate is well tolerated and the first… (read more)