International Consortium for Gene and Cell Therapy
… we have a societal obligation to the rare-disease community to collaborate and build the infrastructure to meet it. With an international consortium in place, it is more likely that therapies will be established that help patients not only in the developed world but also in less developed parts of our planet. Importantly, current scientific developments make this a timely challenge, and, in the long term, gene therapies for these diseases should become cost-effective.