In August 2022, the US Food and Drug Administration (FDA) approved Zynteglo®, a one-time, cell-based gene therapy for adult and pediatric patients with β-thalassaemia major, opening up new and exciting opportunities for those who are medically eligible to receive this life-changing treatment option.
To help people who are living with or caring for someone who has β-thalassaemia major get substantial information about Zynteglo®, bluebird bio, the company behind this ground-breaking therapy, has recently published two novel resources;
- a Patient Brochure, meant for people living in the US, and
- a Q&A document about getting access to gene therapy, foe people living outside the US
The Patient Brochure includes important information and details on β-thalassaemia genetics, explains how Zynteglo® addresses the disease at the genetic level, presents clinical trials studies’ results and safety information, describes the patient treatment journey with the therapy, and discusses other considerations helpful for decision-making processes with the patients’ treating physicians and other doctors.
The Q&A document is intended to answer questions from patients and caregivers residing outside of the US on both Zynteglo® and other FDA-approved bluebird bio gene therapies.
Bluebird has also created my bluebird support—a collection of resources aimed at offering guidance and support to patients and caregivers regarding Zynteglo® that ranges from helping them locate a Qualified Treatment Centre (a specialized hospital qualified to administer a bluebird bio gene therapy) to addressing non-clinical barriers to treatment access, educational materials, treatment cost and insurance-related information, and much more.