The Brigham Gene and Cell Therapy Symposium program at the Brigham on June 6 attracted more than 100 attendees for the opening panel discussion, moderated by Kathryn Rexrode, MD, MPH, Director, Brigham Research Institute. The event highlighted extensive gene and cell therapy expertise at Mass General Brigham and identified opportunities to ensure the system remains a leader in the field.

The panelists were Paul Anderson, MD, PhD, Acting Chief Academic Officer, Mass General Brigham
Lindsey Baden, MD, Vice President of Clinical Research, Brigham and Women’s Hospital
Chris Coburn, Chief Innovation Officer, Mass General Brigham
Roger Hajjar, MD, Director, Gene and Cell Therapy Institute, Mass General Brigham

Anderson discussed the success of the Institute to date in setting up the infrastructure necessary to accommodate more than the 400 PIs current working in GCT research in the system. Baden offered that an “action arm” of the Institute, an intensified response effort, will move projects along quickly and efficiently. Hajjar emphasized that the Institute will be a leader in manufacturing and regulatory resources needed for innovation and announced that “core” areas of the Institute include in vivo testing, human tissue testing and novel editing.

When asked what makes the Institute different from other similar ones, Hajjar said it is patient-focused, contrasting it to other centers elsewhere, where technology is key. “Our clinician-scientists are empowered by GCT being patient-focused.” Within the next five years, Hajjar predicted “we’ll be number one in the nation for GCT therapies and the main center for clinical projects.”

Coburn acknowledged that it’s an exciting time for the system because of its “uniform infrastructure,” which entails the vast expertise within the Mass General Brigham hospitals, who are working collaboratively. He also assured “this is a great time to invest,” noting the system’s productive 55 portfolio companies to date.

A robust, user-friendly GCT website for PIs, as well as the public, is in the works. The first Spark Grants, from the 60 submitted proposals, will be awarded in August. Other symposia are planned to keep PIs informed about the Institute and its developments.

A Q&A followed the panel discussion before nine PIs made curated presentations of their work, from four categories.  Nathan Yozwiak, PhD, Head of Research, GCTI, moderated.

• Viral Gene Therapy
• Non-viral Platforms
• Cell Therapy (Preclinical/Clinical)
• Translation

Presenters and their projects:

Viral Gene Therapy
AAV-based gene delivery platform for the central nervous system
Fengfeng Bei, PhD, Assistant Professor of Neurosurgery

Strategies to Cure Hypertrophic Cardiomyopathy
Christine Seidman, MD, Professor of Medicine

Nociceptor-specific gene therapy for chronic pain
William Renthal, MD, PhD, Assistant Professor of Neurology

Non-viral Platforms
Focused ultrasound for delivery of gene therapy vectors across the blood-brain barrier
Nicholas Todd, PhD, Assistant Professor of Radiology

Beyond Conventional Approaches: Engineering Nanodiscs and Nanoshells for Next-Generation Non-viral Gene Delivery
Mahmoud Nasr, PhD, Assistant Professor of Medicine

Biomaterials for gene therapy and immunomodulation
Natalie Artzi, PhD, Associate Professor of Medicine

Cell Therapy (Preclinical/Clinical)
Engineering skeletal muscle and brown adipose tissue for cell therapy of muscle and metabolic diseases
Olivier Pourquie, PhD, Professor of Pathology

Breaking barriers in cancer research: Leveraging a patient-derived translational research platform to elucidate cell therapy activity
Aaron Goldman, PhD, Instructor in Medicine

Translation
The Concept of Innovation and Orchestration: Translating Cell and Gene Therapies from Bench to Bedside
Khalid Shah, PhD, Professor of Neurosurgery

A reception followed the event.

-Margie Coloian