Gene & Cell Therapy
Mass General Brigham is the largest basic research academic medical center in the U.S. and this fundamental science engine enables us to generate novel approaches to treating disease, develop therapies to address them, and evaluate these discoveries in proof-of-concept first-in-human clinical studies. Most gene and cell therapy development is conducted for specific disorders where a treatment emerges based on the underlying molecular mechanism of that disease.
We bring together 400 leading gene and cell therapy researchers from five nationally ranked academic medical centers — Massachusetts General Hospital, Brigham & Women’s Hospital, Mass Eye and Ear, McLean Hospital, and Spaulding Rehabilitation — with multidisciplinary expertise to accelerate innovation and ultimately improve patient care for a variety of congenital and non-congenital diseases. Clinical gene and cell therapy research at Mass General Brigham is rapidly advancing, pushing the technological and clinical boundaries of this new frontier.
What Are Gene and Cell Therapies?
Researchers at Mass General Brigham are leaders in gene and cell therapies, translating to treatment for patients through clinical trials. This work benefits patients from all over the world with rare and high prevalence conditions. Gene and cell therapies have the potential to be a one-time curative treatment in the future for conditions like ALS, multiple sclerosis, hemophilia, cancer, and more.
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). These cells may or may not be genetically altered. Cell therapy is personalized to treat an individual’s condition, like CAR-T cell therapy, where scientists take the person’s own cells and genetically modify them outside the body, expand the number of modified cells and then re-infuse 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 cancer cells.
Gene editing technology directly edits entire genes or parts of genes within the cell either within (in vivo) or outside (ex vivo) the body and can be used both for gene and cell therapies.
Conditions Potentially Treated by Gene and Cell Therapies
Gene and cell therapies are expected to become future treatments and potential cures for many disease areas including:
- Amyotrophic lateral sclerosis (ALS)
- Cystic fibrosis
- Heart disease
- HIV and AIDS
- Parkinson’s disease
- Multiple myeloma, leukemia, and other blood cancers
- Multiple sclerosis (MS)
At the Frontier of Gene & Cell Therapy
From groundbreaking research to successfully conducting clinical trials, Mass General Brigham researchers, scientists, and clinicians are continuously working to help patients globally with rare and complex disorders. Through groundbreaking gene and cell therapy research and treatments, we are always looking for potential cures. Our priorities are to imagine and create what is scientifically possible in conjunction with the highest clinical need.
With more than $2.3 billion in total research funding, Mass General Brigham has more than 400 researchers who are conducting gene and cell research in two categories:
- Platforms (viral vector delivery, gene editing etc.), which apply across cohorts
We will continue to partner with industry to advance gene and cell therapy.
Natalie Artzi, Ph.D., is leading the way in gene therapy
Natalie Artzi, Ph.D., is an Assistant 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. In this Q&A, she discusses the work of her lab to advance gene therapies to provide improved care for patients living with a variety of diseases.
Eric Pierce, MD, PhD, brings therapies for inherited retinal degenerations into focus
As the director of the Ocular Genomics Institute, at Mass Eye & Ear and Harvard Medical School, Eric Pierce, MD, PhD, is leading the way to understand the molecular mechanisms of inherited retinal degenerations and, importantly, to develop gene therapy interventions for these blinding conditions.
World Medical Innovation Forum
The World Medical Innovation Forum brings together Mass General Brigham and presenting sponsor Bank of America to advance patient benefiting technologies and including review of The Disruptive Dozen — the twelve most disruptive technologies most likely to have significant impact on gene and cell therapy in the near future.