This article contains a list of commercially available gene therapies.

Gene therapies

Alipogene tiparvovec Alipogene tiparvovec, sold under the brand name Glybera, is a gene therapy treatment designed to reverse lipoprotein lipase deficiency (LPLD), a rare recessive disorder, due to mutations in LPL, which can cause severe pancreatitis. It was reco ...

(Glybera): AAV-based treatment for lipoprotein lipase deficiency (no longer commercially available) *

Axicabtagene ciloleucel Axicabtagene ciloleucel, sold under the brand name Yescarta, is a medication used for the treatment for large B-cell lymphoma that has failed conventional treatment. T cells are removed from a person with lymphoma and genetically engineered to p ...

(Yescarta): treatment for large

B-cell lymphoma The B-cell lymphomas are types of lymphoma affecting B cells. Lymphomas are "blood cancers" in the lymph nodes. They develop more frequently in older adults and in immunocompromised individuals. B-cell lymphomas include both Hodgkin's lympho ...

* Betibeglogene autotemcel (Zynteglo): treatment for

beta thalassemia Beta thalassemias (β thalassemias) are a group of inherited blood disorders. They are forms of thalassemia caused by reduced or absent synthesis of the beta chains of hemoglobin that result in variable outcomes ranging from severe anemia to cl ...

* Brexucabtagene autoleucel (Tecartus): treatment for

mantle cell lymphoma Mantle cell lymphoma (MCL) is a type of non-Hodgkin's lymphoma (NHL), comprising about 6% of NHL cases. There are only about 15,000 patients presently in the United States with mantle cell lymphoma. It is named for the mantle zone of the lymph n ...

and

acute lymphoblastic leukemia Acute lymphoblastic leukemia (ALL) is a cancer of the lymphoid line of blood cells characterized by the development of large numbers of immature lymphocytes. Symptoms may include feeling tired, pale skin color, fever, easy bleeding or bruisin ...

* Cambiogenplasmid (Neovasculgen): treatment for vascular endothelial growth factor

peripheral artery disease Peripheral artery disease (PAD) is an abnormal narrowing of arteries other than those that supply the heart or brain. When narrowing occurs in the heart, it is called coronary artery disease, and in the brain, it is called cerebrovascular dis ...

Ciltacabtagene autoleucel Ciltacabtagene autoleucel, sold under the brand name Carvykti, is an anti-cancer medication used to treat multiple myeloma. The most common adverse reactions include pyrexia, cytokine release syndrome, hypogammaglobulinemia, musculoskeletal ...

(Carvykti): treatment for

multiple myeloma Multiple myeloma (MM), also known as plasma cell myeloma and simply myeloma, is a cancer of plasma cells, a type of white blood cell that normally produces antibodies. Often, no symptoms are noticed initially. As it progresses, bone pain, an ...

* Elivaldogene autotemcel (Skysona): treatment for cerebral

adrenoleukodystrophy Adrenoleukodystrophy (ALD) is a disease linked to the X chromosome. It is a result of fatty acid buildup caused by peroxisomal fatty acid beta oxidation which results in the accumulation of very long chain fatty acids in tissues throughout the b ...

* Etranacogene dezaparvovec (Hemgenix): treatment for

hemophilia B Haemophilia B, also spelled hemophilia B, is a blood clotting disorder causing easy bruising and bleeding due to an inherited mutation of the gene for factor IX, and resulting in a deficiency of factor IX. It is less common than factor VIII defi ...

Gendicine Gendicine is a recombinant adenovirus engineered to express wildtype-p53 (rAd-p53). This virus is designed to treat patients with tumors which have mutated p53 genes. __TOC__ History Gendicine is the first gene therapy product approved for clin ...

: treatment for head and neck squamous cell carcinoma *

Idecabtagene vicleucel Idecabtagene vicleucel, sold under the brand name Abecma, is a cell-based gene therapy to treat multiple myeloma. The most common side effects include cytokine release syndrome (CRS), infections, fatigue, musculoskeletal pain, and a weakened ...

(Abecma): treatment for multiple myeloma * Nadofaragene firadenovec (Adstiladrin): treatment for bladder cancer *

Onasemnogene abeparvovec Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy medication used to treat spinal muscular atrophy (SMA). It is used as a one-time infusion into a vein. Onasemnogene abeparvovec works by providing a new copy o ...

(Zolgensma): AAV-based treatment for spinal muscular atrophy * Strimvelis: treatment for adenosine deaminase deficiency (ADA-SCID) *

Talimogene laherparepvec Talimogene laherparepvec, sold under the brand name Imlygic, is a biopharmaceutical medication used to treat melanoma that cannot be operated on; it is injected directly into a subset of lesions which generates a systemic immune response against ...

(Imlygic): treatment for

melanoma Melanoma, also redundantly known as malignant melanoma, is a type of skin cancer that develops from the pigment-producing cells known as melanocytes. Melanomas typically occur in the skin, but may rarely occur in the mouth, intestines, or eye ( ...

in patients who have recurring skin lesions *

Tisagenlecleucel Tisagenlecleucel, sold under the brand name Kymriah, is a CAR T cells medication for the treatment of B-cell acute lymphoblastic leukemia (ALL) which uses the body's own T cells to fight cancer (adoptive cell transfer). Serious side effects occu ...

(Kymriah): treatment for B cell lymphoblastic leukemia * Valoctocogene roxaparvovec (Roctavian): treatment for

hemophilia A Haemophilia A (or hemophilia A) is a genetic deficiency in clotting factor VIII, which causes increased bleeding and usually affects males. In the majority of cases it is inherited as an X-linked recessive trait, though there are cases which aris ...

Voretigene neparvovec Voretigene neparvovec, sold under the brand name Luxturna, is a gene therapy medication for the treatment of Leber congenital amaurosis. Leber's congenital amaurosis, or biallelic RPE65-mediated inherited retinal disease, is an inherited diso ...

(Luxturna): AAV-based treatment for

Leber congenital amaurosis Leber congenital amaurosis (LCA) is a rare inherited eye disease that appears at birth or in the first few months of life. It affects about 1 in 40,000 newborns. LCA was first described by Theodor Leber in the 19th century. It should not be co ...

References

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Gene therapies

See also

References

External links