Gene Therapy at City of Hope

Gene therapy is a term used to describe several innovative new treatments that treat inherited disorders and diseases caused by changes in a patient’s genes. Commonly, these changes result in too much or not enough of a protein in the patient’s cells, which leads to the disorder or disease. Gene therapy delivers genetic material, which contains instructions to change how a cell makes a protein, to the affected patient’s cells.

The gene therapy program at City of Hope^® Cancer Center Duarte offers Food and Drug Administration (FDA)-approved treatment options for adults and children with conditions including beta thalassemia and sickle cell disease. Currently, these therapies require collecting precursor blood-forming stem cells from a patient’s body and transfer of the genetic material that is designed to correct these genetic changes. The stem cells containing this new genetic material is stored frozen and shipped back to the hospital. They are then thawed on site, and infused back into the patient’s body, where it can start to address the genetic changes that cause the patient’s disease or disorder. Our multidisciplinary team of physicians and researchers, which includes nationally renowned experts in gene therapy, guides each patient and their loved ones through the benefits, risks and steps involved in undergoing gene therapy.

As an internationally recognized research center, City of Hope has a longstanding history of developing breakthrough treatments for a wide range of life-threatening conditions, including cancer, diabetes and HIV/AIDS. The process of undergoing gene therapy currently is similar to a stem cell or bone marrow transplant. As one of the nation’s largest bone marrow transplantation centers, City of Hope is uniquely placed to deliver these innovative new gene therapies. Our Center for Gene Therapy is a global leader in cellular therapy research and is home to a team of physicians, researchers and academics committed to unlocking the potential of stem cell therapies and bringing new treatments from the laboratory to patients’ bedsides. City of Hope’s gene therapy program offers patients access to:

• The latest FDA-approved gene therapy options, including Casgevy®, Lyfgenia® and Zynteglo®
• Gene therapy treatment options for both children and adults diagnosed with blood disorders such as thalassemia and sickle cell disease
• A multidisciplinary care team of physicians, nursing staff and gene therapy experts
• Supportive care for patient and loved ones, including fertility preservation experts, social workers and behavioral health counselors
• One of the nation’s most successful centers for delivering gene therapy
• A world-class team of gene therapy researchers, who are continually working to develop new gene therapy treatments
• Clinical trials investigating new gene therapy treatments
• Follow-ups are, including support managing the long-term side effects of thalassemia and sickle cell disease and the gene therapy process, as well as tips and strategies for managing recovery at home
   

Different gene therapies may work slightly differently. However, during most gene therapy treatments:

• A sample of cells is collected from the patient, in much the same way that stem cells or bone marrow are collected prior to a hematopoietic stem cell transplant procedure
• This sample is introduced to a vector. The vector “packages” the sample cells with newly added or altered genetic material that is designed to treat a patient’s disease. This added or edited genetic material is intended to provide the patient’s body with a “fix” for the genetic change that has caused their disorder or disease
• The patient undergoes chemotherapy to make room for the new treated sample of cells
• The sample of cells is reintroduced into the patient’s body as an infusion into a vein, where it begins to be taken up by the body’s cells

Read about patients' experience with the newest treatment

• Sickle Cell Disease Pediatric Gene Therapy Program Offers Children Hope for a Cure | The Downey Patriot, June 17, 2025
• The World's First Crispr Drug Gets a Slow Start | Wired, December 23, 2024

City of Hope has been conducting translational research in the field of gene therapy for several decades. Our team of expert physicians and researchers also lead numerous clinical trials designed to bring breakthrough gene therapies to more patients who need them, with recent clinical trials targeting conditions such as Rett syndrome.

Gene Therapy for CD19-Positive Hematologic Malignancies (SENTRY-CD19)

NCT06533579: This is a Phase 1/2, first-in-human, open-label, dose-escalating trial designed to assess the safety and efficacy of VNX-101 in patients with relapsed or refractory CD19-positive hematologic malignancies.

Learn more about this promising study

Please contact us at [email protected] for more information regarding eligibility for gene therapies that are FDA-approved or in clinical trials.