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Genezen and Seattle Children's Research Institute Forge Strategic Alliance for X-linked Agammaglobulinemia (XLA) Cell Therapy Program Development and Manufacturing

Thursday, March 07, 2024

Seattle Children's Research Institute, the pediatric research division of Seattle Children's Hospital, has announced a strategic partnership with Genezen, a prominent contract development and manufacturing organization specializing in gene and cell therapy.

This collaboration aims to utilize Genezen's expertise in viral vector process development and cGMP manufacturing to advance Seattle Children's Research Institute's X-linked agammaglobulinemia (XLA) program. XLA is a rare genetic disorder characterized by low levels of immunoglobulins, or antibodies, leading to compromised immune function, primarily affecting males due to mutations in the BTK gene, essential for B cell development and function.

Infants with XLA initially benefit from maternal antibodies but become susceptible to severe bacterial infections as these antibodies decline. Currently, treatment options for XLA are limited, prompting the exploration of gene therapy to modify bone marrow stem cells, enabling the production of functional B cells and antibodies to bolster the immune system.

Steve Favaloro, CEO of Genezen, expressed enthusiasm for the partnership, emphasizing Genezen's commitment to supporting Seattle Children's Research Institute's innovative work in advancing XLA gene therapy. He highlighted the importance of Genezen's Cytegrity™ producer cell line tailored for this program.

Dr. David J. Rawlings, Director of the Center for Immunity and Immunotherapies at Seattle Children's, shared optimism about the collaboration. He highlighted the safety and efficacy of their lentiviral platform for XLA gene therapy, validated through rigorous pre-clinical studies. Dr. Rawlings looks forward to leveraging Genezen's capabilities to ensure the production of high-quality clinical vectors necessary for advancing this critical program.



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