Keep up with the latest research in the fast-moving field of gene therapy
August 2017: tisagenlecleucel
Initially approved for B-cell precursor acute lymphoblastic leukemia
October 2017: axicabtagene ciloleucel
Initially approved for relapsed or refractory large B-cell lymphoma
December 2017: voretigene neparvovec-rzyl
Initially approved for inherited retinal disease
May 2019: onasemnogene abeparvovec-xioi
Initially approved for spinal muscular atrophy
July 2020: brexucabtagene autoleucel
Initially approved for relapsed or refractory mantle cell lymphoma
February 2021: lisocabtagene maraleucel
Initially approved for relapsed or refractory large B-cell lymphoma
March 2021: idecabtagene vicleucel
Initially approved for relapsed or refractory multiple myeloma after four or more prior lines of therapy
February 2022: ciltacabtagene autoleucel
Initially approved for relapsed or refractory multiple myeloma
August 2022: betibeglogene autotemcel
Initially approved for β-thalassemia requiring regular red blood cell (RBC) transfusion
September 2022: elivaldogene autotemcel
Initially approved for early, active cerebral adrenoleukodystrophy (CALD)
November 2022: etranacogene dezaparvovec-drlb
Initially approved for hemophilia B (congenital Factor IX deficiency)
December 2022: nadofaragene firadenovec-vncg
April 2023: omidubicel-onlv
Initially approved for hematologic malignancies that are planned for umbilical cord blood transplantation following myeloablative conditioning
May 2023: beremagene geperpavec
Initially approved for the treatment of wounds in people with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene
June 2023: donislecel-jujn
Initially approved for type 1 diabetes
June 2023: valoctocogene roxaparvovec-rvox
Initially approved for severe hemophilia A (congenital factor VIII deficiency with factor VIII activity <1 IU/dL)
December 2023: exagamglogene autotemcel
Initially approved for people with sickle cell disease who have recurrent vaso-occlusive crises
December 2023: lovotibeglogene autotemcel
Initially approved for people with sickle cell disease who have a history of vaso-occlusive events
March 2024: atidarsagene autotemcel
Initially approved for the treatment of children with pre-symptomatic late infantile (PSLI), pre- symptomatic early juvenile (PSEJ), or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD)
April 2024: fidanacogene elaparvovec-dzkt
Initially approved for treating moderate to severe hemophilia B (congenital factor IX deficiency)
June 2024: delandistrogene moxeparvovec-rokl
Initially approved for ambulatory and non-ambulatory pediatric patients with Duchenne muscular dystrophy (DMD)
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