Gene Therapy Program at the Bass Center

Gene therapies offer hope to transform the lives of people living with inherited diseases and cancers.

Our Gene Therapy Program has a long history of creating new gene therapies in the lab and bringing them to patients who need them. We combine deep expertise in each condition we treat with a focus on the detailed steps of the therapy to ensure that each patient receives the best care.

We currently offer a gene therapy for each of the following conditions, either through research clinical trials or as has been approved by the U.S. Food and Drug Administration (FDA).

Gene therapies for genetic diseases:

Clinical trials:

We have unique experience as the only U.S. site to treat the following conditions with gene therapy on the first clinical trials, and we hope to offer these therapies again in the near future:

Gene therapies for cancers and autoimmune diseases:

Why choose Stanford Medicine Children’s Health for gene therapy

  • If you are considering gene therapy, having an experienced team in your corner makes all the difference in bridging the gap between promise and results. At Stanford, we’ll work with you to understand if gene therapy is the right treatment option for you or your child, and we’ll be by your side through the entire process.
  • Breadth and depth of experience. Over the past 20 years, Stanford scientists have developed a number of gene therapies that are now available to our patients, making us one of the most diverse, in terms of conditions treated, and longest-running gene therapy programs in the U.S. We are one of the centers with the highest number of patients treated with a variety of FDA-approved gene therapies now available.
  • Multiple specialists working together. Every patient benefits from doctors with different specialties coming together to provide the best care. For example, to treat a blood disorder with gene therapy, our pediatric hematologists and pediatric gene therapy specialists will work side-by-side to produce the best outcomes.
  • Pioneering experts. Our team includes doctors and scientists who have worked for decades to discover breakthroughs in the science of gene therapy and successfully bring those innovations to patients. We’re continuing this legacy today, as we work to uncover, test, and apply new gene therapies for our patients—quickly and safely.
  • Dedicated stem cell collection and editing support. During gene therapy, a patient’s stem cells need to be collected using a process called apheresis. Our dedicated apheresis team has extensive experience working with kids and adults—they collect cells from hundreds of patients a year. Then, the patient’s cells need to be gene modified. Our Stanford Cellular Therapy Facility can expertly and quickly do this for patients in our clinical trials, and it works with external manufacturing sites for FDA-approved gene therapies.
  • A smooth and tailored patient experience. From our staff who help get new patients ready for care at Stanford, to our nurses and social workers, our whole team is built for the needs of gene therapy and stem cell transplant patients. We also have a dedicated space in our hospital specifically for these patients with extra infection protections in place.