bluebird bio and Généthon Announce Manufacturing Research Collaboration
The Etablissement de thérapie génique et cellulaire (ETGC – gene and cell therapy unit) at Généthon was the first organization in Europe to have produced HIV-derived lentiviral vectors in accordance with GMP (good manufacturing practice) standards. Since it was founded in 2005 and officially approved by the health authorities in 2006, the ETGC at Généthon has released GMP-compliant batches of a number of different vector types.
bluebird bio’s approach uses stem cells harvested from the patient’s bone marrow into which a healthy version of the disease causing gene is inserted. After being grown in culture, those cells are given back to the patient. This approach represents a true paradigm shift in the treatment of severe genetic diseases by eliminating the potential complications associated with donor cell transplantation and potentially presenting a one-time transformative therapy. bluebird bio has two distinct product candidates in clinical development: one for childhood cerebral adrenoleukodystrophy (CCALD), and another program (LentiGlobin®) for beta-thalassemia and sickle cell anemia. In addition to these initial disease targets, the company’s gene therapy platform is expected to be applicable to the treatment of other severe genetic diseases.
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