Today, around 320 million population is diagnosed with rare genetic diseases. In fact, according to NIH (National Institutes of Health), 30 million, or 10% population of the United States, have one of the rare diseases. Further, there are 7,000+ known rare diseases, and 95% currently have no treatment. But the solution to all these problems lies in the hands of gene therapy in the coming days. Gene therapy could help patients suffering from a rare disease to live a life without the need for ongoing treatments or the inconvenience of daily disease management. 


In August 2022, the FDA approved the first-ever cell-based gene therapy drug called Zynteglo, a treatment for patients with a rare genetic blood disease such as beta-thalassemia. This single treatment will cost nearly USD 2.8 million, creating the history of the most expensive medicine. The bluebird bio's gene therapy is branded under the name Zynteglo. Zynteglo is a one-time gene therapy product administered as a single dose. Each dose of Zynteglo is a customized treatment created using the patient’s own cells (bone marrow stem cells) that are genetically modified to produce functional beta-globin (a hemoglobin component).


Gene therapy is the method that uses genes to correct the underlying genes problems; it is used to prevent, treat or cure the disease by modifying or manipulating the gene. In simple words, gene therapy will help replace a faulty gene or add new genes to cure or improve the body’s immune system to fight the disease. 

The breakthrough in medical science can be witnessed by one of the early gene therapy examples that has a cure for the Severe Combined Immune Deficiency (SCID) disease. In 1990, the four-year-old Ashanthi de Silva became one of the first gene therapy success stories. She was suffering from severe combined immunodeficiency (a disorder called "bubble boy disease") which is caused by insufficient levels of the enzyme adenosine deaminase (ADA). Without treatment, patients rarely survive toddlerhood. In addition to hematopoietic cell transplantation, standard treatment at that time consisted of lifelong replacement with synthetic ADA given via intramuscular injections once or twice weekly. Turning to a cutting-edge treatment, scientists were able to deliver to Ashanthi a healthy version of the gene that produces ADA using a viral vector and the success of this operation opened the door to many new trials after 1990. The significant credit behind the operation's success goes to W. French Anderson and his colleagues at the NIH, who successfully performed the procedures. Further, W. French Anderson's critical contributions to gene therapy have helped him be termed the “father of gene therapy.” 


The critical challenges of gene therapy are immunogenicity, mutagenicity, and lack of sustainable therapeutic benefits. Despite these constraints, gene therapy offers various applications listed as: 

  • It is used in the replacement of the genes that cause medical ill-health. 
  • The gene therapy method primarily destroys the problem-causing genes in the body. 
  • It helps the human body to fight against diseases by adding genes to the body to support the immune system.  
  • This method treats diseases like ADA deficiency, cystic fibrosis, cancer, etc. 


The gene therapy market holds a very promising future. Currently, in the U.S., gene therapy is still available only as a part of a clinical trial. Still, it has a bright future for treating various diseases like cancer, heart disease, diabetes, cystic fibrosis, and AIDS. Researchers are investigating several ways of performing successful gene therapy procedures, including replacing mutated genes, fixing mutated genes, and making diseased cells more evident to the immune system. 

According to the recent research report by Arizton Advisory Intelligence, the U.S. gene therapy market expects to grow with a compound annual growth rate (CAGR) of over 43% from 2022-2027. One of the significant reasons behind the growth can be attributed to the rising number of gene therapy approvals; it is estimated that by the year 2025, the U.S. is expected to approve 10–20 gene therapies yearly. 

Know more about the report inclusions:  


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