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Enabling Gene and Cell Therapy translation from bench to clinic at Mass General Brigham’s Gene and Cell Therapy Institute (MGB-GCTI)

June 19, 2025
Breakout Session
Cell and Gene Therapy and Genome Editing
With more than 30 approved gene and cell therapy (GCT) products and the rapid emergence of new GCT technologies, academic medical centers (AMCs) play a crucial role in advancing genetic medicines. However, translating these therapies from bench to clinic requires overcoming numerous challenges, including high costs, complex regulatory pathways, and navigating the translational "valley of death."To address these challenges, Mass General Brigham’s Gene and Cell Therapy Institute (MGB-GCTI) has implemented the RISE model—an institutional framework emphasizing Resource sharing, Interdisciplinary collaboration, Sustainable funding, and Education. In this session speakers will describe the pipeline of latest GCT advancements emerging from academia, including ways AMCs can sustainably incubate GCT development (Dr. Nathan Yozwiak, Head of Research at GCTI). The session will also highlight emerging GCT platform technologies, including the first-of-its-kind RNA therapeutics core to produce circular RNA for research and therapy development (Dr. Alex Wesselhoeft, Director of the RNA therapeutics, GCTI) and a high-throughput platform for generating CAR-T therapies for solid tumors (Dr. Robert Manguso, MGH Cancer Center) among others. These projects demonstrate how MGB-GCTI's structured support accelerates novel GCTs toward clinical applications.

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