RARE Daily

Genezen Enters Partnership with Seattle Children’s for XLA Cell Therapy Program

March 6, 2024

Rare Daily Staff

Seattle Children’s Research Institute and Genezen, a gene and cell therapy contract development and manufacturing organization, have entered into a strategic manufacturing partnership to advance Seattle Children’s X-linked agammaglobulinemia program.

This collaborative effort is focused on leveraging Genezen’s viral vector process development and cGMP manufacturing expertise to advance Seattle Children’s X-linked agammaglobulinemia (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.

Seattle Children’s Research Institute, the research division of Seattle Children’s Hospital, has developed a novel lentiviral platform for XLA gene therapy that has been shown to be safe and effective in extensive animal and human cell pre-clinical studies.

The Institute will be one of Genezen’s first customers to leverage its cutting-edge Cytegrity technology from biotech leader CSL for stable and scalable high-titer lentivirus production.

“With this program, Seattle Children’s is one of our first customers to leverage our Cytegrity producer cell line offering from CSL,” said Steve Favaloro, CEO of Genezen.

Photo: Steve Favaloro, CEO of Genezen

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