Partnership for a gene therapy clinical trial

Généthon, the not-for-profit biotherapy laboratory funded by the French Muscular Dystrophy Association (AFM) thanks to the donations collected during the French Telethon, and Children's Hospital Boston announce that they have initiated a partnership to conduct a gene therapy clinical trial for Wiskott-Aldrich Syndrome (WAS). This rare genetic condition is a life-threatening immunodeficiency disease.

Genethon is sponsoring parallel trials at Great Ormond Street Hospital in London and Hopital Necker-Enfants Malades in Paris and will be supplying the vector for the trial at the US site. The vector is a latest-generation lentiviral vector that incorporates several safety features designed to avoid the complications caused by early-generation vectors. The vector is manufactured by Genethon in Evry. Altogether, the WAS gene therapy trials in London, Paris and Boston will constitute a unique multicentre collaboration to accelerate the testing of new advanced therapies for rare conditions.

The Wiskott Aldrich Syndrome (WAS) is a rare primary immune deficiency disease causing significant bleeding due to low platelets and increased numbers of serious infections. Most patients have mild to severe eczema and are also at a higher risk of developing autoimmune disorders and malignancies such as lymphoma.

Généthon to use Polyplus-transfection’s jetPEI transfection reagent for producing viral vectors

Polyplus-transfection SA, which specializes in the development of solutions for cellular and molecular biology research, announced today that Généthon, a not-for-profit biotherapy laboratory run by the French Muscular Dystrophy Association (AFM – Association Française contre les Myopathies) and which is financed by donations raised through the annual Téléthon, has selected Polyplus-transfection’s jetPEI transfection reagent for the production of its viral vectors. The financial terms of the deal were not disclosed.