The EMA has recommended approving Zynteglo, bluebird bio’s one-off treatment for beta-thalassaemia. Meanwhile the cell and gene therapy firm has cut the ribbon on a 125,000-square-foot facility in Durham, North Carolina.

The EMA’s Committee for Advanced Therapies (CAT) recommended Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene) – also know as LentiGlobin – today, paving the way for bluebird bio to bring its gene therapy product to market.

The product uses a lentiviral viral vector to add functional copies of a modified β-globin gene into a patient’s own stem cells, addressing the underlying genetic cause of patients for whom a related donor for stem cell transplantation is not available.

Image: iStock/metamorworks

The European Commission (EC) will now review the product before granting bluebird marketing approval or not. The Committee for Medicinal Products for Human Use (CHMP) meeting was the first to be held in Amsterdam following the EMA’s arrival in the Netherlands due to the current Brexit debacle.

NC manufacturing plant

In November 2017, bluebird bio acquired a facility in Durham, North Carolina and after investing $80 million in the site, the firm has now cut the ribbon on the lentiviral vector facility.

The plant will support bluebird’s cell and gene therapy candidates including bb2121 and bb21217 for the treatment of multiple myeloma and LentiGlobin for the treatment of transfusion-dependent β-thalassemia (TDT) and sickle cell disease.

The 125,000 square-foot facility is the first inhouse production site for bluebird, which has relied up to now solely on contract development and manufacturing organizations (CDMOs) and services firms including Brammer Bio, Novasep and MilliporeSigma. The new plant will not affect these multi-year contracts, the firm has said.

The facility is “a crucial step toward our mission of bringing a new generation of treatments to people living with severe genetic diseases and cancer,” said bluebird CEO Nick Leschly.

The company did not respond to this publication when contacted for more details.

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