Phase III data on bluebird bio's Zynteglo, as well as Vertex and CRISPR Therapeutics' CTX001, showed patients achieving transfusion independence via two gene therapy approaches.
An analyst wrote that it is unlikely bluebird would provide full data from its registration-directed study of the CAR-T bb2121 by year end, which is more likely to come early next year.
By reducing administrative burden and redesigning workflows around human needs, it creates space for what matters most: connection between clinicians and patients.
Two beta-thalassemia advocacy organizations - one British and one Italian - issued a press release Monday that claimed the EMA had approved the gene therapy under the brand name Zynteglo.
A company analysis deemed the patient's myelodysplastic syndrome was unrelated to LentiGlobin, but a news report indicated it may have stemmed from the conditioning regimen used in the trial.