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How gene therapy is used

Targeting disease at the genetic level represents a new era in medicine. Gene therapy may offer the potential for life-changing treatment for patients with devastating illnesses. In addition, by restoring normal function in cells, gene therapy has the potential to treat the underlying cause of disease rather than just the symptoms. In the future, addressing the underlying cause of disease could give patients access to transformative treatments that may alleviate the burden of chronic disease management.

What are the different diseases treated by gene therapy?

Single-gene diseases have been a primary focus of gene therapy research and development. These developments fulfill an urgent need. Gene therapy offers the possibility of going beyond managing symptoms to address the underlying causes of diseases.1

Select genetic diseases with ongoing gene therapy research and development

The US Food and Drug Administration (FDA) has approved several gene therapies. Other candidates are still in clinical development or are under review for approval.

Learn more about how Astellas Gene Therapies is developing investigational gene therapy treatments for Pompe disease, X-linked myotubular myopathy (XLMTM) and myotonic dystrophy type 1 (MDT1).

Note: The table below does not contain an exhaustive list for gene therapy research, as this area continues to evolve. Except where FDA approval is noted in the table, other research programs are investigational treatments for which safety and efficacy are not yet established and there is no guarantee that they will be approved by the FDA.

Gene therapies under development. Click to see available therapies.

Blood disorders

Sickle cell disease

Hemophilia A and B, other clotting disorders

Etranacogene dezaparvovec-drlb, AAV, FDA approved 2022

Betibeglogene autotemcel, LV, FDA approved 2022

Severe anemias


Nadofaragene firadenovec-vncg, AV, FDA approved 2022

Some lymphomas

Axicabtagene ciloleucel, CAR T cell therapy, FDA approved 2017

Brexucabtagene autoleucel, CAR T cell therapy, FDA approved 2020

Lisocabtagene maraleucel, CAR T cell therapy, FDA approved 2022

Tisagenlecleucel, CAR T cell therapy, FDA approved 2017

Some leukemias

Brexucabtagene autoleucel, CAR T cell therapy, FDA approved 2020

Talimogene laherparepvec, genetically modified viral therapy, FDA approved 2015

Ciltacabtagene autoleucel, CAR T cell therapy, FDA approved 2022

Idecabtagene vicleucel, CAR T cell therapy, FDA approved 2021

Eye disease

Voretigene neparvovec-rzyl, AAV, FDA approved 2017

Inherited immunological and metabolic disorders

Hereditary angioedema

Pompe disease

Neurodegenerative disorders

Huntington's disease

Frontotemporal dementia

Parkinson's disease

Alzheimer's disease

Elivaldogene autotemcel, LV, FDA approved 2022

Neuromuscular diseases

Duchenne muscular dystrophy

Myotonic dystrophy

Onasemnogene abeparvovec-xioi, AAV gene replacement therapy, FDA approved 2019

X-linked myotubular myopathy (XLMTM)

Who can use gene therapy?

Who is eligible to receive gene therapy depends on many different factors. Gene therapies approved by the FDA have a statement indicating when and how they can be used. Some of the variables affecting who can use a particular drug include:

  • Diagnosis
  • Disease severity
  • Disease progression
  • Co-existing conditions
  • Age or weight
  • A patient’s previous treatments and their response to them

How do you test for a genetic disease?

A genetic test may identify a genetic disease by detecting gene variants. A genetic test can help confirm a genetic disease or uncover a person’s chance of developing or passing on a genetic disease.

Types of genetic testing

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Newborn screening

  • Standardized testing in the first days of life
  • Not all genetic conditions are included; screening varies by state
  • Most newborns in the US undergo some newborn screening
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Carrier testing

  • Voluntary screening for people with a family history or risk factor for an inherited disease
  • Determines if an individual carries a copy of a gene variant known to cause disease
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Genomic sequencing

  • Changes in genes can be detected through genomic sequencing to identify variants that contribute to or cause disease
  • Testing can help diagnose diseases and identify treatments
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