bluebird bio is aiming to be one of the first companies to launch a gene therapy in the UK. Often mistakenly seen as a treatment of the future, gene therapy has come of age, primed and ready to go with bluebird bio a leader in the field.
Gene therapy’s arrival on the scene is very timely as it has been swept up in Boris Johnson’s ‘new industrial revolution’ that includes a wider UK policy agenda to foster an eco-system and lattice-work of cutting-edge technological and life science innovation. The vision positions the UK as a world-leader in clinical, scientific and industrial transformation — to remain at the vanguard of pioneering science and healthcare.
bluebird bio is part of that scientific and medical revolution. Its UK office in Basingstoke sits alongside Switzerland and the US in overseeing 25 years of world-class R&D, developing gene therapies for severe genetic and rare diseases — 80 per cent of which are believed to be genetic in origin .
Gene therapy has the potential to address the underlying genetic cause of diseases through one-time treatments intended to provide lifelong benefits. bluebird bio has a vision to build a powerful gene therapy platform for eligible patients with severe genetic disorders. The name bluebird is inspired by the term ‘Bluebird Day’, first used in 1860 to describe an ideal day: a symbol of renewal and transition.
In the past 70 years, the UK has made great strides in scientific and medical innovation. The discovery of the structure of DNA, the UK’s contribution to the Human Genome project and the launch of the 100,000 Genomes project have all been game-changing breakthroughs that have helped to transform modern healthcare. More recently, inward investment into projects such as the Cell and Gene Therapy Catapult and the regional Advanced Therapy Treatment Centres have helped pave the way to build the necessary infrastructure to deliver ‘next generation’ advanced therapies such as gene therapy.
bluebird bio’s UK General Manager, Nicola Redfern, believes these initiatives are a cause for optimism but that policy barriers still exist. bluebird bio welcomes the launch this month of a review by the National Institute for Health and Care Excellence (Nice) of its methods for appraising new medicines. This review could address many of these policy barriers and accelerate access for eligible patients to these innovative treatments.
‘bluebird bio congratulates Nice for focusing attention on new technologies, such as gene therapies. We would encourage the government and NHS England to embrace and support Nice in the implementation of their recommendations so that gene therapies can be delivered quickly to the patients most likely to benefit from the technology.’
Nicola says: ‘A wave of gene therapies is coming, offering disruptive technologies that require a fundamental change in the approach to advanced therapies. We want to support the NHS in its ambition to allow sustainable access for eligible patients to these innovative treatments. We want to help fuel the “new industrial revolution” to make sure the UK leads the world in healthcare and innovation.’
1. Genomic England. Rare Disease Genomics. Available from: https://www.genomicsengland.co…
This article first appeared in a Spectator supplement: The evolution of gene therapy. Sponsored by bluebird bio.