Agreements

Date: 2016-12-15

Type of information: Production agreement

Compound: Lenti-D™ and LentiGlobin™ drug products

Company: apceth (Germany) Bluebird bio (USA - MA)

Therapeutic area: Technology - Services - Rare diseases - Genetic diseases - Hematological diseases

Type agreement: manufacturing - bioproduction

Action mechanism:

gene therapy/stem cell therapy. Lenti-D™ drug produt is autologous CD34+ cell-enriched population that contains cells transduced with Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein, suspended in a cryopreservative solution. Lenti-D Drug Product is administered by IV infusion following myeloablative conditioning with busulfan and cyclophosphamide.
LentiGlobin BB305 Drug Product consists of autologous CD34+ hematopoietic stem cells transduced with lentiviral vector LentiGlobin BB305 encoding the human Beta A-T87Q-globin gene and suspended in cryopreservative solution.

Disease: childhood cerebral adrenoleukodystrophy (CCALD - X-linked adrenoleukodystrophy cerebral childhood) transfusion-dependent ?-thalassemia.

Details:

• On December 15, 2016, bluebird bio and apceth Biopharma announced that they have entered into a strategic manufacturing agreement providing for the future European commercial production of bluebird bio’s Lenti-D™ product candidate for cerebral adrenoleukodystropy and its LentiGlobin™ product candidate for transfusion-dependent ?-thalassemia.
This agreement follows a successful multi-year manufacturing relationship and provides bluebird bio with European commercial manufacturing capabilities, including dedicated production suites within apceth Biopharma’s state-of-the-art GMP facility.
Under this multi-year agreement, apceth Biopharma will perform clinical manufacturing, process validation activities and commercial manufacturing for LentiGlobin and Lenti-D drug product to support the treatment of European patients with transfusion-dependent beta thalassemia and cerebral adrenoleukodystrophy, respectively.

Financial terms:

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