ISCT marks bluebird bio’s landmark FDA approval a celebration for the CGT sector while calling industry to attention in the pursuit of patient access

Vancouver, Canada, August 22, 2022 - The International Society for Cell & Gene Therapy (ISCT), the global society of clinicians, researchers, regulators, technologists, and industry partners dedicated to the translation of cell and gene therapy (CGT), today celebrates bluebird bio‘s announcement that ZYNTEGLO(R), a gene therapy targeting transfusion-dependent beta-thalassemia has received FDA approval. ISCT acknowledges the significance of the achievement for the CGT industry at large, while also recognising the work required to make these life-changing therapies within reach for the majority of patients. 

“The news from bluebird bio is a huge moment for the industry. Inherited blood disorders, such as thalassemia and sickle cell disease, are the most common monogenic diseases in the world and represent a growing global healthcare burden. The current standard of care for hemoglobinopathies is repeat blood transfusions or iron chelation therapy, but the approval of this therapy will begin to shift the treatment paradigm towards one-off curative treatments,” said Jacques Galipeau, President, ISCT. “We have now seen the validation of gene therapies as a curative treatment in a commercial setting. This offers patients hope that their long-term treatment plans can be replaced with one-off treatments that can vastly improve quality of life. As the field continues to mature, we will undoubtedly see more successes.” 

“Bluebird bio’s FDA approval is hugely significant and primarily for patients”, said Jaap Jan Boelens, Chair of the Stem Cell Engineering Committee, ISCT. “However, there is the important challenge for bluebird bio, payers and health systems to make sure this safe and effective product will be accessible for all patients; not only in the US, but also in the rest of world, where the majority of patients with thalassemia live”, continuesJaap Jan Boelens. “In the balance, there are a number of considerations; the need for an affordable, realistic price, alongside short-term costs, and more importantly, long term savings. Access to this FDA approved product, the first of its kind, will give transfusion dependent beta-thalassemia patients a very high chance of a normal or normalizing life”.

Since its inception, ISCT’s vision has been to support the translation of cell & gene therapies into safe and effective treatments to improve patients' lives. ZYNTEGLO(R) joins a very small group of gene therapies approved for use in the US and is the first FDA approval for a stem cell lentivirus.  Bluebird’s achievement is another demonstration that the industry is beginning to realize its potential, particularly with one-off curative therapies. The next steps for deployment of effective gene therapies are to enable global patient access.