Skip to cookie consent Skip to main content

Gene and Cell Therapy Institute

With the Gene and Cell Therapy Institute, gene and cell therapy research at Mass General Brigham is rapidly advancing, pushing the technological and clinical boundaries of this new frontier.

a strand of dna

Uniting cutting-edge research with world-class patient care

Discover the groundbreaking world of gene and cell therapy, where advanced medical technologies are revolutionizing patient care. At Mass General Brigham, we are at the forefront of this exciting frontier, pushing the boundaries of technology and clinical applications.

About the Gene and Cell Therapy Institute

The Gene and Cell Therapy Institute (GCTI) at Mass General Brigham is a hub of innovation and collaboration, uniting more than 400 researchers and clinicians dedicated to advancing gene and cell therapy. 

The GCTI is committed to finding targeted and transformative treatments that have the potential to cure diseases or halt their progression. Led by renowned cardiac gene therapy pioneer Roger Hajjar, MD, the GCTI accelerates groundbreaking research, conducts clinical trials, and facilitates the development of FDA-approved treatments.

The Institute’s multidisciplinary approach sets it apart from others in the space. The GCTI leverages expertise from across the entire ecosystem of medical innovation and fosters an environment of shared knowledge and collective impact by bringing together researchers utilizing gene and cell therapy platforms. This collaboration ensures that scientific and clinical goals align seamlessly, ultimately leading to improved patient outcomes.

The close relationships between physicians, researchers, and patients is a critical component of what makes the GCTI successful. This continuous loop, from bench to bedside and back again, enables translation to clinical applications. The GCTI is committed to expediting the pace of discovery and development by bridging the gap between research and practice.

The GCTI operates an annual Spark grant program for Mass General Brigham researchers, providing crucial funding to fuel groundbreaking projects and drive scientific progress. This investment in cutting-edge research ensures that Mass General Brigham remains at the forefront of gene and cell therapy advancements. The GCTI also offers expertise in manufacturing, regulatory, clinical trials, and core laboratory services.

Our team

The Gene and Cell Therapy Institute's leadership actively fosters and establishes both internal and external connections to drive forward the advancement of gene and cell therapy treatments and groundbreaking discoveries.

Roger J. Hajjar, MD
Head, Gene and Cell Therapy Institute 

Marcela V. Maus, MD, PhD
Associate Head & Head of Cell Therapies, Gene and Cell Therapy Institute 
Paula O’Keefe Endowed Chair, MGH Cancer Center
Director, Cellular Immunotherapy Program, Massachusetts General Hospital
Associate Professor of Medicine, Harvard Medical School
Attending Physician, Hematopoietic Cell Transplant & Cell Therapy Program, MGH

Nathan L. Yozwiak, PhD
Head of Research, Gene and Cell Therapy Institute 

Dana M. Hammill, MS, MBA
Head of CMC, Gene and Cell Therapy Institute

Angela Shen, MD, MBA
Head of Regulatory, Gene and Cell Therapy Institute  

Meini S. Shin, MBA
Head of Strategic Planning & Operations, Gene and Cell Therapy Institute 

Omar Abudayyeh, PhD
Director Gene Editing Core, Gene and Cell Therapy Institute  

Robert Alexander Wesselhoeft, PhD
Director RNA Therapeutics, Gene and Cell Therapy Institute 

Nadhitha Uma Naresh, PhD
Project Manager, Gene and Cell Therapy Institute

Isabella M. Zamora, MPH
Program Coordinator, Gene and Cell Therapy Institute

What are gene and cell therapies?

Gene and cell therapies are advanced medical treatments aimed at stopping or slowing disease progression. Gene therapy involves replacing or modifying faulty genes with corrected versions, while cell therapy replaces or repairs damaged cells. Gene editing, utilizing tools like CRISPR, enable targeted and programmable repair of damaged gene products. Mass General Brigham offers a range of FDA-approved gene and cell therapy treatments for various medical conditions, including certain types of cancer, hereditary diseases, and blood disorders.

Types of therapies

Many diseases can involve a genetic component — a missing gene, multiple copies of a gene, or a gene that is structurally defective. Gene therapy is the process of replacing or modifying genes to correct a deficiency, sometimes by altering the DNA sequence of the target gene or by modifying expression of a gene by turning it off or on. Gene therapy can have an especially profound effect in congenital diseases where the disease is associated with malfunction of a single gene. Researchers are working to identify such genes and develop therapies to replace or modify genes.

Cell therapy involves replacing or modifying cells to treat disease. Cell therapy can use cells from another person (allogenic) or the person’s own cells (autologous), which may or may not be genetically altered.

Autologous, modified cell therapy is personalized to treat an individual’s condition where scientists take the person’s own cells and genetically change them outside the body, expand the number of modified cells, and then reinfuse these modified cells to produce a therapeutic benefit. Such therapies alter the patient's cells so their immune system can now target and destroy previously stealth diseased cells.

Examples of cells that are modified to treat disease include T-cells (commercialized as chimeric antigen receptor T cells; CAR-T cells), B-cells, Natural Killer (NK) cells, Tumor Infiltrating Lymphocytes (TILs), and Dendritic Cells (DCs).

Gene editing is the modification of a cell’s DNA with great precision using tools, such as CRISPR-based biotechnologies, that enable programmable and targeted repair of damaged gene products.

Treatments

Mass General Brigham is proud to offer FDA-approved gene and cell therapy treatments for the following conditions:

  • CAR T-cell therapy for lymphoma and leukemia  
  • Multiple myeloma 
  • Beta-thalassemia
  • Retinal dystrophy
  • Cartilage defects - knee
  • Cerebral adrenoleukodystrophy
  • Spinal muscular atrophy 
  • Neurodegenerative diseases  
  • Duchenne muscular dystrophy

Meet our researchers

Get to know some of the brilliant minds driving research at Mass General Brigham.

Marcela Maus, MD, PhD

Dr. Maus and her research team design and evaluate next generation genetically modified (CAR) T cells as immunotherapy in patients with cancer. Using the immune system as treatment has the potential to induce long-term, durable remissions, and perhaps even cures for some patients. “We’re effectively hacking the immune system so patients’ own bodies can fight their disease,” says Dr. Maus.

Marcela Maus leaning on railing

Natalie Artzi, PhD

Natalie Artzi, PhD, is an Associate Professor at Harvard Medical School and researcher in the Department of Medicine at Brigham and Women’s Hospital. Dr. Artzi and her team are researching the design of smart biomaterials for disease monitoring. Additionally, they are investigating gene and cell therapy deliveries for a range of diseases and for tissue regeneration applications.

Natalie Artzi in Brigham and Women's lab

News & highlights

Mass General Brigham and its diverse teams are leading novel approaches in innovation and improved patient care.