UK based Cobra Biomanufacturing Plc announced an agreement with the Italian Telethon Foundation to manufacture plasmids for two innovative gene therapy trials. The materials produced by Cobra are planned to be used in clinical trials for the treatment of the inherited disorders Metachromic Leukodystrophy and Wiskott-Aldrich Syndrome.
Cobra will produce a 5-plasmid combination under GMP for the generation of an advanced version of the lentiviral gene transfer vector. The vector is to be used to introduce therapeutic genes into haematopoietic stem cells taken from patients to treat the inherited disorders. The novel technology enables stable gene transfer with much higher efficiency and improved safety compared to currently available vectors. Researchers in Italy, France and UK have demonstrated the successful correction of both diseases in affected mice using this new gene therapy strategy.
David Thatcher, chief executive at Cobra Biomanufacturing, commented: "We are delighted to be involved in this promising project. Our position as a leading international manufacturer of DNA with a proven track record in preclinical and clinical supply puts Cobra in an ideal position to provide the Foundation with both the materials and the support and expertise needed to make this a successful and productive collaboration. The Telethon Foundation has supported many high calibre Italian research programs in genetic disease and we are proud to form this important alliance".