apceth Biopharma GmbH from Ottobrunn/Munich becomes commercial manufacturer in Europe for the gene therapy ZyntegloTM. The product from the US company bluebird bio for the treatment of transfusion-dependent ß-thalassaemia received conditional marketing authorisation yesterday from the European Commission (EC).
Transfusion-dependent ß-thalassemia is a hereditary blood disease caused by a mutation in the beta-globin gene. Patients suffer from ineffective red blood cell production and the resulting anaemia. The symptomatic treatment of people with TDT consists of lifelong blood transfusions. However, transfusion therapy and the increased uptake of iron caused by the disease lead to iron deposition in various organs, which can lead to life-threatening complications. Therefore, the administration of iron-binding medication is also part of the treatment.
ZyntegloTM (former name: LentiGlobinTM) is a gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have a β0/β0 genotype, for whom haematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available. ZyntegloTM is bluebird bio’s first gene therapy to gain regulatory approval.
“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”, commented Dr. Christine Günther, CEO of apceth Biopharma. “Being one of the very few companies worldwide to manufacture a cell-based gene therapy for commercial use marks a milestone for our company.”
apceth Biopharma is bluebird bio’s clinical and commercial manufacturing partner for Europe not only for ZyntegloTM, but also for bluebird bio’s product candidate Lenti-D for cerebral adrenoleukodystrophy (CALD). Following a successful long-term manufacturing relationship, apceth Biopharma and bluebird bio entered a commercial drug product manufacturing agreement in 2016.
As a pioneer in the field of cell therapy and regenerative medicine, apceth Biopharma is a certified partner for the development and production of cell and gene therapies for various customers.
At the beginning of the year, Hitachi Chemical Co., Ltd. took over Munich-based apceth Biopharma GmbH for 75 million euros.