Bluebird’s Zynteglo Shows Long-Term Efficacy And Safety Amid US Commercialization Push
Data presented at ASH show patients maintaining transfusion independence for years after infusion, months after bluebird won FDA approval for the beta-thalassemia gene therapy.
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Ovum preservation and sperm banking are options for post-puberty patients, but busulfan – used for myeloablative conditioning before certain gene therapies – may cause hesitation to use some treatments in children, experts at ASH told Scrip.
Bluebird bio will not see revenue for Zynteglo until it is infused into beta-thalassemia patients, which will take months due to the ex vivo gene therapy’s complex manufacturing and quality control process.
Bluebird bio’s Tom Klima told Scrip launch preparations are well under way, including negotiations with payers on outcomes-based reimbursement agreements.