bluebird bio gets CHMP nod for gene therapy Zynteglo

PharmaPhorum | April 01, 2019

A European Medicines Agency advisory panel has recommended a conditional approval for Zynteglo, bluebird bio’s lead gene therapy for thalassemia, a rare blood disorder. The Committee for Medicinal Products for Human Use (CHMP) confirmed on Friday that it had issued a positive opinion for the therapy – previously known as LentiGlobin – as a treatment for patients aged 12 years and older with transfusion-dependent beta-thalassemia (TDT) who do not have a specific β0/β0 genotype. The announcement came after a couple of medical charities jumped the gun and prematurely issued notices of the panel’s endorsement last week, hailing it as a major advance for patients with TDT who typically need frequent blood transfusions to manage the disease. The positive opinion for Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene) puts bluebird bio on course for its first gene therapy approval in the coming weeks and could give thalassaemia patients a new one-shot treatment option that tackles the underlying genetic cause of the disease.

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Is solid form characterization critical in your line of work, but you lack expertise in X-ray diffraction (XRD)? Do you need an efficient tool to support you with lead optimization? Do you need an easy way to validate the stability of your solid form following processing?

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