Charles River Laboratories International Inc. has announced a significant milestone in the development of gene therapy for rare diseases, as part of a collaborative effort with Elly's Team, a parent-led foundation. The partnership has successfully navigated the Food and Drug Administration (FDA) approval process, receiving Investigational New Drug $(IND.AU)$ approval in March 2025. This approval enabled the first administration of IRF2BPL gene replacement therapy to a child named Elly on April 3, 2025. This achievement is a result of combined efforts and expertise from multiple organizations, including Charles River, which provided critical support in manufacturing and advisory services. The collaboration marks a pivotal moment for the IRF2BPL community, offering hope and a path to treatment for families facing similar diagnoses.
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