Seattle Children’s Research Institute, the research division of Seattle Children’s Hospital and a leader in pediatric research, and Genezen, a gene and cell therapy contract development and manufacturing organization, have unveiled a strategic manufacturing partnership for Seattle Children’s Research Institute’s X-linked agammaglobulinemia (XLA) program.
This collaborative effort is focused on leveraging Genezen’s viral vector process development and cGMP manufacturing expertise to advance Seattle Children’s XLA gene therapy program. XLA is a rare primary immunodeficiency condition characterized by abnormally low levels of immunoglobulins, also referred to as antibodies. XLA is an X-chromosome-linked inherited disorder that affects males and is caused by defects in the BTK gene required for B cell generation and function. The inability to produce B cells or the immunoglobulins (antibodies) reduces the body’s ability to fight infection. Infants with XLA may initially appear healthy as they are protected by maternal antibodies during their early months. However, as these maternal antibodies wane, these infants often begin to grapple with severe, recurring bacterial infections affecting various parts of their body, including the ears, lungs, sinuses, and skin, which can pose life-threatening risks.
No cure presently exists for XLA. The primary objective of XLA gene therapy is to genetically modify stem cells from the bone marrow to permit the generation of new B cells and antibody-producing B cells to restore antibody production and bolster the immune system.
Steve Favaloro, Chief Executive Officer of Genezen, commented on the partnership: “I am extremely excited for our team to support the world-class researchers at Seattle Children’s Research Institute as they progress their XLA gene therapy into the clinic. With this program, Seattle Children’s is one of our first customers to leverage our Cytegrity™ producer cell line offering from CSL. The Genezen team is dedicated to a science-first, service-focused approach for our innovator clients – I know our team will add tremendous value to Seattle Children’s Hospital and XLA patients.”
Furthermore, David J. Rawlings, MD, the Director of the Center for Immunity and Immunotherapies and Division Chief of Immunology at Seattle Children’s, as well as a professor of pediatrics and immunology at the University of Washington School of Medicine, added his perspective: “Our team has developed a novel lentiviral platform for XLA gene therapy that is safe and effective in extensive animal and human cell pre-clinical studies. As we move forward to treat XLA patients, we are excited to work with Genezen to generate the high-quality clinical vector required for this important program.”
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