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Gene and Cell Therapy Research Symposium

The Mass General Brigham Gene and Cell Therapy Institute's annual symposium is an exploration of the dynamic research landscape within the Mass General Brigham community, uniting trailblazers who are driving advancements in gene therapies, cell therapies, and gene editing techniques to combat human diseases.

Attendees listening to a presentation at the 2023 Gene and Cell Therapy Symposium

Uniting Gene and Cell Therapy Experts

The Gene and Cell Therapy Institute (GCTI)'s annual research symposium stands as a global hub, drawing in esteemed researchers, accomplished scientists, and industry professionals from across the globe. Together, they share a common goal of disseminating knowledge, unveiling cutting-edge breakthroughs, and envisioning the transformative potential within the realm of gene and cell therapy.

What sets the symposium apart is its commitment to cultivating a unique and collaborative environment. Building on the success of the inaugural symposium in 2023 which attracted over 500 registrants, the 2024 event (November 12-13) will feature keynote speakers Crystal Mackall, MD and Erik Sontheimer, PhD and engaging discussions by leading pioneers in gene and cell therapy research, as well as plentiful opportunities for networking and collaboration among attendees.

Attendees listening to a presentation at the 2023 Gene and Cell Therapy Symposium

2024 Gene and Cell Therapy Research Symposium

The 2nd annual Gene and Cell Therapy Research Symposium will take place November 12-13, 2024, bringing together researchers, clinicians, and the greater scientific community dedicated to advancing gene and cell therapy.

Members of the Mass General Brigham research community and interested industry will have the opportunity to:

2024 Speakers

Crystal Mackall, MD

Day 1 Keynote Speaker
Crystal Mackall, MD - Stanford University

Crystal Mackall, MD, is the Ernest and Amelia Gallo Family Professor of Pediatrics and Medicine at Stanford University, the Founding Director of the Stanford Center for Cancer Cell Therapy, and Director of the Parker Institute for Cancer Immunotherapy @ Stanford. During a 27-year tenure at NCI and since 2016 at Stanford, she has led an internationally recognized translational research program focused on immune-oncology. Her work has advanced understanding of fundamental immunology and translated this understanding for the treatment of human disease with a major focus on children’s cancers. Her group advanced understanding of T cell exhaustion and developed approaches to prevent or reverse the phenomena, identified major mechanisms of resistance to CAR T cell therapies and has conducted trials with very promising results in CAR19 refractory diseases and brain tumors. She is a Member of the National Academy of Medicine, the Academy of Immuno-oncology and AACR, and is the recipient of numerous awards.

Erik Sontheimer, PhD

Day 2 Keynote Speaker
Erik Sontheimer, PhD - University of Massachusetts Chan Medical School

Erik J. Sontheimer, PhD, is the Pillar Chair in Biomedical Research and a Professor at the University of Massachusetts Chan Medical School, where he also serves as Vice Chair of the RNA Therapeutics Institute. He earned his Ph.D. from Yale in 1992, studying pre-mRNA splicing under Joan Steitz. After postdoctoral work at the University of Chicago with Joe Piccirilli, he joined Northwestern in 1999, focusing on RNA’s roles in gene expression, RNA interference mechanisms, and CRISPR systems in pathogenic bacteria. In 2008, his group was the first to demonstrate that CRISPR systems target DNA directly, pioneering RNA-guided genome engineering. He has received numerous awards, including the NSF CAREER Award, Burroughs Wellcome Fund New Investigator Award, and election to the American Academy of Microbiology. He co-founded Intellia Therapeutics in 2014 to develop clinical applications of CRISPR. At UMass Chan, he continues his research on RNA’s biomedical applications and the treatment of human disease.

November 12, Day 1:

Keynote Speaker
Crystal Mackall, MD - Stanford University

Gene and Cell Therapy in Neurology
Jeff Schweitzer, PhD, MD
Brian Wainger, MD, PhD

Platform Technology
Jeff Karp, PhD,
Mahmoud Nasr, PhD, RPh

Fireside Chat: Patient Advocacy
Pat Furlong
Nick Vita
Sonia Vallabh, PhD

Flash Talks (Day 1)
Marcela Maus, MD, PhD
Natalie Artzi, PhD
Patricia Musolino, MD, PhD

Gene and Cell Therapy in Oncology
Max Jan, MD, PhD
Edwin Choy, MD, PhD

Reception and Poster Session

November 13, Day 2:

Keynote Speaker
Erik Sontheimer, PhD - University of Massachusetts Chan Medical School

RNA Medicine I
Clotilde Lagier-Tourenne, MD, PhD
Suma Babu, MBBS, MPH

RNA Medicine II
Robert A. Wesselhoeft, PhD
Omar Abudayyeh, PhD
Jonathan Gootenberg, PhD

Flash Talks (Day 2)
E. Antonio Chiocca, MD, PhD
Allan Goldstein, MD
Sharl Azar, MD
Florian Eichler, MD

Ophthalmic and Auditory Gene Therapies
Luk Vandenberghe, PhD
Zheng-Yi Chen, DPhil

Regulatory Landscape and Commercialization of Gene and Cell Therapies
Dana Hammill, MS, MBA
Angela Shen, MD, MBA

2024 Spark Program Awards and Presentations

2023 Symposium Highlights

The Gene and Cell Therapy Institute held its inaugural Gene and Cell Therapy Research Symposium in November 2023 in collaboration with the Mass General Research Institute and the Brigham Research Institute. The symposium welcomed approximately 500 registrants, primarily from the Mass General Brigham community. Over the course of the day-long event, Mass General Brigham investigators discussed their research across four areas: viral gene therapy, platform technology, translation and cell therapy.

2023 Keynote Speakers

2023 Speakers, topics and more

Cell Therapy
Allan Goldstein, MD, Massachusetts General Hospital
Marcela Maus, MD, PhD, Massachusetts General Hospital, Gene and Cell Therapy Insitute
Angela Shen, MD, MBA, Mass General Brigham Innovation, Gene and Cell Therapy Institute

Platform Technology
Omar Abudayyeh, PhD, Brigham and Women's Hospital
Natalie Artzi, PhD, Brigham and Women's Hospital
Jonathan Gootenberg, PhD, Brigham and Women's Hospital
Dana Hammill, MS, MBA, Gene and Cell Therapy Institute
Robert Alexander Wesselhoeft, PhD, Gene and Cell Therapy Institute

Translation
Sharl Azar, MD, Massachusetts General Hospital
Florian Eichler, MD, Massachusetts General Hospital

Viral Gene Therapy
Xandra Breakfield, PhD, Massachusetts General Hospital
E. Antonio Chiocca, MD, PhD, Brigham and Women's Hospital
Patricia Musolino, MD, PhD, Massachusetts General Hospital

The 2023 symposium included an overview of the GCTI’s Spark Grant program, which funds gene and cell therapy projects by Mass General Brigham investigators that demonstrate tangible advancements toward clinical applications and commercialization outcomes including licensing, partnerships and new company creation. In its inaugural year the program received 56 proposals from Mass General Brigham investigators, and six grant recipients were each awarded $250,000 to continue their innovative work in disease areas such as Huntington's disease, cancer, chronic pain and deafness.

Learn more about the Spark Grant program

At the 2023 symposium researchers from the Mass General Brigham community had the opportunity to submit abstracts for consideration to present at the poster session. This platform provided a way for participants to showcase the work they have been doing in the gene and cell therapy field, allowing them to share their research and insights.

What did you find most valuable about the symposium?

The range of the presentations - from historical overviews to current and emerging technologies - provided a valuable teaching-and-learning framework.

The ability to hear about what others are doing, the journey of CAR-T at Mass General Brigham, and the networking with keynote speakers and others.

Attendee asking a question during a session at the 2023 Gene and Cell Therapy Research Symposium