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Gene Therapy Drugs

Gene therapy drugs are used to treat leukemia, blindness, and genetic diseases. They work by adding new genes to the body to compensate for defective ones.

Why are gene therapy drugs prescribed?

  1. Sickle Cell Disease (9%)
  2. Hemophilia (9%)
  3. Inherited Metabolic Diseases (9%)
  4. Retinal Dystrophy (9%)
  5. Thalassemia (9%)
  6. DEB (9%)
  7. Melanoma (9%)
  8. Leukemia (9%)
  9. Non-Hodgkin Lymphoma (9%)
  10. Multiple Myeloma (9%)

Key takeaways

Gene therapy medications are used to treat certain types of cancer, genetic disorders, and blood conditions. These medications work by introducing new genetic material into your cells to correct or replace faulty or missing genes. These treatments are often given as a single dose, and they're administered as injections or infusions. Examples include Abecma (idecabtagene vicleucel), Breyanzi (lisocabtagene maraleucel), Luxturna (voretigene neparvovec), and more.

Prices for popular Gene Therapy Drugs medications
Viewing 16 of 16 medications
BEREMAGENE GEPERPAVEC treats wounds in people with epidermolysis bullosa, a condition that causes fragile, blistering skin. It works by helping your body make more of a protein needed to support and strengthen skin. This helps reduce blistering and heals wounds.
VALOCTOCOGENE ROXAPARVOVEC treats hemophilia A. It works by helping your body make factor VIII, a protein that helps your blood clot. This lowers the risk of bleeding.
TISAGENLECLEUCEL is a gene therapy that is made from cells in your immune system. It is used to treat acute lymphoblastic leukemia and certain types of non-Hodgkin lymphoma.
AXICABTAGINE CILOLEUCEL is a gene therapy that is made from cells in your immune system. It treats certain types of cancer. Some of the cancers treated are large B-cell lymphoma and follicular lymphoma.
LISOCABTAGENE MARALEUCEL treats leukemia and lymphoma. It works by helping your immune system slow or stop the spread of cancer cells.
Amtagvi is part of the Gene Therapy Drugs class and treats Melanoma. Gene therapy drugs are used to treat leukemia, blindness, and genetic diseases. They work by adding new genes to the body to compensate for defective ones. Amtagvi is only available as a brand name drug.
EXAGAMGLOGENE AUTOTEMCEL treats sickle cell disease. It may also be used to treat beta thalassemia. It works by helping your body make healthy red blood cells. These cells do not sickle. This can reduce symptoms of sickle cell disease, such as pain crises. Healthy red blood cells also reduce the need for blood transfusions in people with beta thalassemia.
Kebilidi is part of the Gene Therapy Drugs class and treats Inherited Metabolic Diseases. Gene therapy drugs are used to treat leukemia, blindness, and genetic diseases. They work by adding new genes to the body to compensate for defective ones. Kebilidi is only available as a brand name drug.
CILTACABTAGENE AUTOLEUCEL treats multiple myeloma, a type of bone marrow cancer. It works by helping your immune system slow or stop the spread of cancer cells.
IDECABTAGENE VICLEUCEL treats multiple myeloma, a type of bone marrow cancer. It works by helping your immune system slow or stop the spread of cancer cells.
BREXUCABTAGENE AUTOLEUCEL is a gene therapy that is made from cells in your immune system. It is used to treat mantle cell lymphoma and acute lymphoblastic leukemia.
BETIBEGLOGENE AUTOTEMCEL treats beta thalassemia. It works by helping your body make healthy red blood cells, which reduces the need for blood transfusions.
ELIVALDOGENE AUTOTEMCEL treats cerebral adrenoleukodystrophy (CALD). It works by helping your body make more of a protein needed to support the protective membrane that covers nerve cells in your brain and spinal cord. This delays worsening symptoms.
ONASEMNOGENE ABEPARVOVEC is used to treat patients with spinal muscular atrophy (SMA). It is not a cure.\n \nThis medicine may be used for other purposes; ask your health care provider or pharmacist if you have questions.
Hemgenix is part of the Gene Therapy Drugs class and treats Hemophilia. Gene therapy drugs are used to treat leukemia, blindness, and genetic diseases. They work by adding new genes to the body to compensate for defective ones. Hemgenix is only available as a brand name drug.
VORETIGENE NEPARVOVEC is a gene therapy used to treat a certain form of inherited vision loss.\n \nThis medicine may be used for other purposes; ask your health care provider or pharmacist if you have questions.
Disclaimer: Popularity is based on total prescriptions for the brand and generic versions of each drug, regardless of the condition being treated. Some drugs are prescribed for multiple conditions.

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