Mass General Brigham, the largest basic research academic medical center in the U.S., enables this fundamental science engine 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 are 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.
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.
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.
Gene and cell therapy is expected to become future treatments and potential cures for many disease areas including:
Read how Mass General Brigham and its diverse teams are leading novel approaches in innovation and improved patient care.
Breakthroughs range from restoration of sight and increasing the supply of donor organs, to treating brain cancer, hearing loss, and autoimmune diseases.
Ten biotech advancements from Mass General Brigham have been awarded Innovation Discovery Grants in the 2022 round of awards for the highly competitive grant program.
The study is the first step in testing the safety and efficacy of this new Parkinson’s disease treatment.
Physician-investigators in the Department of Neurosurgery at Brigham and Women’s Hospital are leading clinical trials of cutting-edge approaches for treating glioblastoma.
In this mouse model of retinitis pigmentosa, researchers observed that retinal pigment epithelium (RPE) cells and nearby retina cells died from oxidative stress.
Watch a video of a patient’s experience with Gene and Cell Therapy for treatment of a brain tumor.