Redefining what’s possible with personalized medicine
What if you could use your body’s own cells to create a custom treatment made exactly for you? What once seemed like a futuristic dream is happening now at Penn Medicine. Our cell and gene therapies don’t just treat the symptoms of an illness, they target the root cause by fixing problems at their most basic level.
Made from materials that come from living things, like your own cells and DNA, these treatments are often called “biologics.” Cell therapy works by using healthy cells to repair, replace, or destroy damaged parts of the body, while gene therapy updates the “instructions” inside your cells so they work correctly again. These new treatments offer hope for people with rare or serious diseases, making it possible to slow down or even cure illnesses that used to be untreatable.
At Penn Medicine, we don’t just use these treatments, we helped invent them. Our scientists discovered the foundation for CAR T cell therapy, a type of immunotherapy that targets cancer cells. This work led to the first FDA approval in 2017 and changed how cancer is treated. Today, our teams continue to refine these approaches and expand their use, giving you access not just to the best therapies, but to the experts who helped create them.
Cell therapy versus gene therapy
Cell therapy and gene therapy are partners that often work together. While both focus on treating the underlying cause of a disease, they each do the job in their own way.
Cell therapy uses healthy cells that come from either your body or a donor to target and eliminate cancer or abnormal cells. In some cases, cells may be changed in a lab before being returned to your body to fight disease. For example, in some cell therapies for cancer, your immune cells are “trained” to find, target, and destroy cancer cells.
Common approaches include:
- CAR T cell therapy: Reprograms T cells to find and attack cancer cells.
- T cell receptor (TCR) therapy: Modifies T cells to recognize specific cancer markers in solid tumors.
- Tumor infiltrating lymphocyte (TIL) therapy: Uses immune cells from a tumor that are grown and strengthened in a lab.
- Bone marrow or stem cell transplant: Replaces damaged or diseased blood-forming cells with healthy ones to rebuild your blood cells and immune system.
Gene therapy rewrites the “instruction manual” to change how genes work inside cells, and therefore how the cells themselves work. This can help the body make the right proteins or stop harmful ones. New genetic instructions are delivered through a modified, harmless virus or similar lab-designed material that can be picked up naturally by the body. This delivery system, called a vector, delivers a healthy new gene or removes a defective one. It allows cells to function normally or actively respond to an updated genetic code designed by scientists and clinicians to make needed changes.
Common approaches include:
- Gene addition: Adds a working copy of a gene to replace a faulty one.
- Gene editing: Changes or repairs DNA within cells to correct genetic mistakes.
- In vivo therapy: Delivers genetic material directly into the body through an injection or infusion.
- Ex vivo therapy: Removes and modifies cells outside the body and then returns them.
Some treatments use both cell therapy and gene therapy approaches together. With this method, cells are removed, genetically changed in the lab, and returned to the body. These modified cells can fight disease and help create new, healthy cells. This approach is used for health conditions like cancer and sickle cell disease.
Engineering a cure: CAR T cell therapy
Treating disease with cell and gene therapies was once thought to be impossible. CAR T cell therapy, a breakthrough that began at Penn Medicine, uses your own immune cells to fight cancer in a novel way.
Conditions treated with advanced biologics
Cell and gene therapies offer a new path forward for people facing serious, rare, or complex illnesses.
These therapies address a wide range of inherited and acquired conditions of the blood and immune system, including:
Cell therapy for cancer uses your own immune system as a powerful tool to find and destroy tumors. Care may include treatment for:
Pioneering research at Penn Medicine led to one of the first gene therapies for inherited vision loss. Luxturna, an FDA-approved treatment, can improve vision in people born with Leber congenital amaurosis (LCA), a type of early onset retinitis pigmentosa. Inherited retinal disease care specialists replace missing or faulty genes in the eye with healthy ones to help slow or stop disease progression.
Genetic alteration brings new options to people with rare neurological conditions. The Penn Neurogenetics Therapy Center leads early-phase clinical trials of gene-based therapies for neurological disorders, including:
- Spinal muscular atrophy
- Cerebral adrenoleukodystrophy
Next-generation immunotherapies at Penn Medicine
When you come to Penn Medicine, you’re choosing a team that’s spent decades outsmarting disease by looking at health in a completely new way. We helped launch a new era of medicine by developing some of the world’s first approved cell and gene therapies. Our researchers pioneered CAR T cell therapy, the first FDA-approved personalized cellular therapy for cancer and helped lead the development of the first FDA-approved gene therapy for inherited blindness. These breakthroughs changed how doctors treat disease and opened the door to entirely new forms of personalized medicine.
Today, our scientists and physicians continue building on that work. Our Center for Cellular Immunotherapies is a global leader in the field, anchoring its research with one of the world's largest portfolios of early-phase clinical trials. Teams across Penn Medicine are studying new ways to improve immunotherapies, make treatments safer and more effective, and expand their use to more cancers, genetic diseases, and immune disorders.
By bringing together experts in genetics, immunology, cancer care, and research, we move discoveries from the laboratory to patient care as quickly as possible. For many people, this means access to innovative therapies and clinical trials that may not be widely available elsewhere.
Approved cell and gene therapy products
There are currently more than 40 FDA-approved cell and gene therapy products available in the United States, and that number is growing quickly.
Penn Medicine offers access to many approved therapies, including:
- Abecma for multiple myeloma
- Amtagvi for advanced melanoma
- Aucatzyl for acute lymphoblastic leukemia
- Breyanzi for large B-cell lymphomas, mantle cell lymphoma, and chronic lymphocytic leukemia/small lymphocytic lymphoma
- Carvykti for multiple myeloma
- Hemgenix for hemophilia B
- Kymriah for large B-cell lymphomas and acute lymphoblastic leukemia
- Luxturna for inherited vision loss
- Lyfgenia and Zynteglo for sickle cell disease and beta thalassemia
- Omisirge for blood and immune system diseases
- Skysona and Zolgensma for rare neurological conditions in children
- Tecartus for acute lymphoblastic leukemia and mantle cell lymphoma
- Tecelra for synovial sarcoma
- Yescarta for large B-cell lymphomas and follicular lymphoma
Navigating your cell or gene therapy treatment
At Penn Medicine, care is coordinated from start to finish by a team of specialists with expertise in cell and gene therapies. Your specific experience depends on the condition being treated and the type of therapy used.
If you’re receiving an “autologous” (self) cell-based treatment, blood is collected through a process called apheresis. Similar to dialysis, you’re connected to a machine through an IV line that draws blood, separates specific cells (like T cells), and returns the rest of your blood to your body. This process typically takes several hours. The removed cells are then sent to a lab, where they’re modified with new genetic instructions. Manufacturing your personalized cells can take a few days or up to three to six weeks. Once ready, the cells are returned to your body through an infusion.
Some gene therapies follow a similar process, while others are delivered more simply—directly into your bloodstream or to a specific organ through an injection or infusion. Many gene therapies are designed to be a one-time treatment.
Treatment takes place in infusion suites designed for comfort and close monitoring. From cell collection to infusion and follow-up visits, every step is carefully managed. Your care may also be supported by the Center for Cellular Immunotherapies, a world-class hub for developing and advancing these therapies, for certain clinical trials.
Long-term monitoring and immune recovery
Because cell and gene therapies affect how your immune system works, recovery takes time and is different for everyone. Your experience depends on the condition being treated, the type of therapy used, and how your body responds.
For therapies that use modified blood or stem cells, the first few weeks after infusion focus on a process called engraftment. This is the critical period when the new, modified cells travel to the bone marrow and begin creating healthy red blood cells, white blood cells, and platelets. Your care team closely monitors your blood counts to make sure the cells are working properly.
Depending on the therapy, recovery may involve a hospital stay lasting several days or weeks. Other treatments are done in an outpatient setting with frequent follow-up visits. Some people may need to stay close to the medical center for a period of time, so side effects can be managed quickly and recovery can be closely monitored.
As your body adjusts to treatment, temporary side effects like fatigue, nausea, fever, or flu-like symptoms are common.
Cell and gene therapies can temporarily weaken your immune system while your body recovers. During this time, you may be more likely to get infections and may need to avoid crowds or people who are sick. Some people also take medications to help prevent infection and support immune recovery.
Your care team monitors your immune function, blood counts, and overall health through regular follow-up visits and testing. As your body begins producing healthy new cells, immune function and energy levels often improve over time.
Recovery continues after you leave the hospital or infusion center. It’s normal to feel tired as your body uses energy to heal and build new cells. Depending on your treatment and condition, you may gradually return to work, exercise, school, and other daily activities over several weeks or months.
Your care team will explain any temporary physical limitations and help guide your recovery. Rehabilitation, which can include physical therapy and nutrition support, may also be part of your care plan.
Cell and gene therapies often require long-term follow-up to monitor effectiveness and immune recovery. At Penn Medicine, we continue tracking your progress through regular visits, blood tests, imaging, and other evaluations based on your condition and treatment.
Some gene therapies are designed to create long-lasting genetic changes inside cells. Because these therapies are still relatively new, the FDA may recommend monitoring for many years after treatment, sometimes up to 15 years. This long-term follow-up helps track how well the therapy is working and watch for delayed side effects.
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