New Agreement to Expand Production Capacity for Lenti-D for CALD, Other Gene Therapies

New Agreement to Expand Production Capacity for Lenti-D for CALD, Other Gene Therapies

Hitachi Chemical Advanced Therapeutics Solutions and Apceth Biopharma GmbH, both subsidiaries of Hitachi Chemical Company, recently signed a new long-term development and manufacturing agreement with Bluebird Bio.

The agreement will expand the clinical and commercial manufacturing capacity for Lenti-D — a gene therapy that Bluebird is developing for the childhood cerebral form of adrenoleukodystrophy (CALD) — among other therapies.

“Securing long-term commercial drug product manufacturing capacity is critical to our ability to deliver for patients,” Nick Leschly, Bluebird’s CEO, said in a press release.

“Our partnership with Hitachi Chemical is a significant example of our continued progress on this front and we believe Hitachi Chemical’s recent expansion will help support our growing commercial needs. We are pleased to benefit from their expertise as well as their footprint in both the US and Europe as we work to bring transformative therapies to patients in need,” he added.

CALD is caused by mutations in the ABCD1 gene, which provide instructions for making the ALD protein. These mutations lead to a lack of functional ALD, ultimately resulting in a buildup of saturated very long chain fatty acids (VLCFAs), which cause damage to the nervous system.

Lenti-D works by using genetically modified patient-derived stem cells. These are extracted from the patient’s bone marrow, and modified with the help of a viral vector to contain a functional copy of the ABCD1 gene. The cells are then transplanted back into the patient, where they grow and differentiate into other cell types. Because they have a functional ABCD1 gene, these cells are expected to be able to remove the excess VLCFAs.

The new manufacturing agreements expand on relationships between Bluebird and the Hitachi Chemical subsidiaries that have been in place since 2011. In addition to expanded manufacturing capacity for Lenti-D, the new agreements also expand manufacturing for two other gene therapies: LentiGlobin, which is being developed for sickle cell disease, and Zynteglo, an approved treatment in Europe for transfusion-dependent beta-thalassemia.

“It is our honor to support bluebird bio in the manufacture of their potentially transformative gene therapies, to the benefit of patients in both the United States and Europe,” said Robert Preti, PhD, chief strategy officer at Hitachi Chemical Life Science Business Headquarters.

Bluebird recently announced plans to submit an application for Lenti-D as a treatment for CALD to the European Medicines Agency before the end of this year. However, it expects to delay the U.S. filing  until mid-2021 due to the COVID-19 pandemic.

“We are very happy to deepen our trustful and productive strategic partnership with bluebird bio,” said Christine Guenther, MD, CEO of Apceth. “The apceth team is proud to be part of bluebird bio’s most exciting pioneering work for the advancement of cell and gene therapies and to supply patients suffering from severe genetic illnesses with potentially life-changing treatments.”

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Patrícia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.
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