bluebird bio will use Hitachi Chemical subsidiary apceth Biopharma to commercially manufacture its recently EU approved gene therapy Zynteglo.
Earlier this month, the European Medicines Agency (EMA) approved orphan drug Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene) – previously known as LentiGlobin – as a one-off treatment for beta-thalassaemia.
Zynteglo 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.
To supply the European market, Zynteglo’s marketing authorization holder bluebird bio has selected German contract development and manufacturing organization (CDMO) apceth Biopharma as its commercial manufacturer.
“We are proud to be the commercial manufacturing partner of bluebird bio and to be part of bringing this life-changing therapy to TDT patients in Europe”, Christine Guenther, CEO of apceth Biopharma, said in a statement. “Being one of the very few companies worldwide to manufacture a cell-based gene therapy for commercial use marks a milestone for our company.”
The Munich-based CDMO boasts two GMP/BSL2 cell and gene therapy production facilities, including 600 m2 of cleanroom area.
Japanese company Hitachi acquired the company earlier this year for JPY 9.4 billion ($87 million). apceth is being integrated into Hitachi’s regenerative medicine division, Hitachi Chemical Advanced Therapeutics Solutions (HCATS), which has manufacturing capabilities in the US and Japan.
apceth has also been contracted to be bluebird’s European clinical and commercial partner for the candidate Lenti-D for cerebral adrenoleukodystrophy under terms of an original supply deal in 2016.
For the production of its gene therapies, bluebird bio also uses CDMOs Brammer Bio, Novasep and MilliporeSigma. Meanwhile the firm – which is partly owned by Regeneron – recently opened its first inhouse production site; a 125,000-square-foot facility in Durham, North Carolina.