Busulfan-Induced Infertility May Deter Some Pediatric Patients From Bluebird, Other Gene Therapies
Partial Hold On Firm's Sickle Cell Therapy Lifted
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.
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Jasper announced data on the first three patients in the trial of briquilimab in sickle cell disease and beta-thalassemia patients, not long after another player, Magenta, announced a setback in its program.
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.
The $3m gene therapy for the rare disease cerebral adrenoleukodystrophy marks bluebird’s second approval in just under a month.