Gene Therapy and Repair

Program in Cell and Gene Therapy

David RawlingsLed by Dr. David Rawlings, the Program in Cell and Gene Therapy is working toward a day when children with immune disorders won’t need lifelong treatments. Instead, patients will receive cell therapy – an infusion of cells that addresses a disease’s cause and could potentially cure it forever.

To create cell therapies, researchers remove a small number of cells – either stem cells or immune cells – from a patient. Then they change or “reprogram” the genetic instructions in those cells and infuse them back into the patient.

Developing Gene Therapy Treatments for Children

Gene therapy is a type of cell therapy. Gene therapies fix the genes that cause disease by disabling them, repairing them or replacing them with new, synthetic genes.

Rawlings and his colleagues in the Center for Immunity and Immunotherapies have developed tools that make it easier to repair or replace faulty genes. Our researchers are building a pipeline of gene therapies that could eventually be used to treat hundreds of genetic immune disorders.

We’re conducting a clinical trial of a gene therapy for severe combined immunodeficiency (SCID). We plan to open similar trials for Wiskott-Aldrich syndrome by 2019 and X-linked agammaglobulinemia and Hyper-IGM syndrome (CD40L deficiency) soon thereafter.

These trials will help us test gene therapies and make them more effective, so we can apply them to many other diseases and potentially revolutionize treatment for children worldwide.

Cell Therapies for Autoimmune Diseases

We’re also partnering with leading biotech companies like Casebia to develop cell therapies for autoimmune diseases, which strike when immune cells turn against the body and attack it. Our researchers are using gene editing to generate a T cell that functions as a peacekeeper, telling attacker cells to calm down.

We have used this approach to stop autoimmune disease in preclinical models. We plan to start clinical trials in children by 2020, beginning with rare diseases for which there are no effective treatments.

The long-term goal is to use cell therapies to replace today’s treatments for diseases such as lupus and diabetes and to promote tolerance following bone marrow or solid organ transplantation.