Généthon initiates a new clinical trial for a severe immune deficiency (Wiskott-Aldrich syndrome)
Généthon has just obtained approval from the French and British health authorities for a Phase I/II clinical trial of gene therapy for a rare immunodeficiency, Wiskott-Aldrich syndrome (WAS). The Généthon-sponsored trial will be performed both in France (led by Professors Alain Fischer and Marina Cavazzana-Calvo at Necker Children's Hospital, Paris) and in the UK (led by Professor Adrian Thrasher at London's Great Ormond Street Hospital).
The forthcoming clinical trial is the end result of research initiated in 2002 by Anne Galy's group (UMR951) (3) in the Généthon laboratory. The lab's researchers have developed an ex vivo approach that uses an HIV-derived lentiviral vector to transfer genes into autologous CD34+ hematopoietic stem cells from the patient. Généthon has developed, produced and quality-controlled the trial's batches of gene-based medicines under GMP conditions.
The trial also demonstrates the extent of the AFM lab's know-how, ranging from preclinical research and the GMP production of gene-based drugs through to trial logistics and regulatory approval. The preparatory work took eight years and involved a €10 million investment.
"This trial is enabling the Généthon laboratory (funded almost exclusively by donations from France's annual Telethon) to demonstrates its world-class expertise in the production of gene-based medicines and the preparation and implementation of clinical trials. It confirms Généthon's ability to become a major international player in demonstrating the efficacy of gene therapy for rare diseases" emphasized AFM and Généthon President Laurence Tiennot-Herment.
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Topic world Gene therapy
Genetic diseases once considered untreatable are now at the center of innovative therapeutic approaches. Research and development of gene therapies in biotech and pharma aim to directly correct or replace defective or missing genes to combat disease at the molecular level. This revolutionary approach promises not only to treat symptoms, but to eliminate the cause of the disease itself.
Topic world Gene therapy
Genetic diseases once considered untreatable are now at the center of innovative therapeutic approaches. Research and development of gene therapies in biotech and pharma aim to directly correct or replace defective or missing genes to combat disease at the molecular level. This revolutionary approach promises not only to treat symptoms, but to eliminate the cause of the disease itself.