Loading

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. The RISE framework enables accelerated asset development by making academia-born technologies more attractive to industry partners, appealing to investors seeking next-generation GCT therapies, and ultimately accessible to patients in need. This session will provide an overview of the RISE framework and how GCTs can be sustainably incubated in AMCs (Dr. Nathan Yozwiak, Head of Research, GCTI). It will also feature emerging GCT platform technologies, including the first-of its-kind RNA therapeutics core for circular RNA production that is democratizing circRNA technology across many academic and industry partners (Dr. Alex Wesselhoeft, GCTI). The session will also showcase a high-throughput CAR-T platform for solid tumors, which is gaining investor interest and is on a path of new company creation with GCTI’s seed funding support (Dr. Robert Manguso, MGH Cancer Center).
Speakers
Robert Manguso, PhD
Assistant Professor of Medicine, Harvard Medical School and Krantz Family Center for Cancer Research, Massachusetts General Hospital
R Alexander Wesselhoeft, PhD
Director of RNA Therapeutics, Gene and Cell Therapy Institute (GCTI), Mass General Brigham
Nathan Yozwiak, PhD
Head of Research at the Gene and Cell Therapy Institute (GCTI)
Mass General Brigham

BIO DOUBLE HELIX SPONSORS

Our sponsors help support BIO's mission to support and advocate for the biotechnology industry.

BIO HELIX SPONSORS