Spinraza And Advanced Therapies: A Stakeholder Special

The approval of Spinraza (nusinersen) for the treatment of the severe rare disease Spinal muscular atrophy (SMA) is a breakthrough in the Advanced Therapies field and highlights the importance of involving all stakeholders for the successful development of therapeutics. The mechanism of action of Spinraza is based on a thorough understanding of the molecular basis of SMA and shows how genetic therapies can be rationally designed when significant knowledge about the disease has been gathered. The availability of Spinraza, a treatment with broad label, impacts a large number of people, from babies and infants through to adults. It also has implications for the possible introduction of newborn screening and showcases the difficulty in providing affordable access to advanced treatments. The Spinraza and Advanced Therapies: a stakeholder special issue of Gene Therapy covers a variety of perspectives and includes comments and review papers from SMA research funders, people affected, experts in the advanced therapies field, SMA scientists and clinicians, regulators, ethics and pricing/IP experts, and community stakeholders. The issue also covers genomics projects for personalised medicine, the importance of international collaboration in rare disease research, showcases on-going research in Asia, South America and Africa, and provides academic and corporate perspectives on the delivery of advanced therapies. It also includes a compilation of online resources of relevance. This content shows the global, inclusive and accessible way in which we want to develop the journal.