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These Gene Therapy Drugs Offer Breakthrough Results Cost Millions

Author: Candy Swift
by Candy Swift
Posted: Jun 22, 2022

Genes are made up of the nucleotide sequences required to make a polypeptide chain or functional RNA, which maintains life's fundamental structure and function by storing all of the information necessary for race, blood type, incubation, growth, apoptosis, and other activities.

Gene is regarded as the "Holy Grail" of biological research due to its great potential. Gene therapy research has sparked a major medical revolution in recent years, but its costs have put people off. This article will discuss various high-priced gene therapies that are now available.

l Zolgensma for SMA

Zolgensma is a gene therapy designed for spinal muscular atrophy (SMA), which is a serious neuromuscular disease characterized by loss of motor neurons leading to progressive muscle weakness and paralysis. The incidence of SMA is about 1 in 10,000 live births and is the main genetic cause of infant death. In the trial, 15 patients infused with Zolgensma remained alive for 24 months, and 92% of patients can sit unassisted for 5 seconds or longer.

In May 2019, the FDA approved Zolgensma with the price setting at $ 2.1 million, roughly 9 times the median sale price for a home in the U.S. and 33 times the national per capita income.

l LentiGlobin for SCD

LentiGlobin is a kind of stem cell gene therapy designed to add functional copies of a modified form of the?-globin gene, which does not work as it should in sickle cell disease (SCD) sufferers, into a patient’s own hematopoietic stem cells. When patients have copies of the functional gene, their red blood cells can produce anti-sickling hemoglobin which decreases the amount of the faulty sickled hemoglobin.

Leerink, a specialist investment bank focusing on the healthcare sector, estimates LentiGlobin's price at about $1.2 million in the U.S. and $900,000 in the EU.

l Glybera for FCS and Strimvelis for SCID

Glybera and Strimvelis are two gene therapy products approved by the European Medicines Agency (EMA). Strimvelis was launched in 2016 for the treatment of severe combined immunodeficiency (SCID) and Glybera was launched in 2012 to treat familial chylomicronemia syndrome (FCS).

SCID is a rare disease that causes newborns to almost completely lose their ability to resist viruses, bacteria, and mold. Some patients can only live in a sterile environment, so they are also called. Strimvelis involves removing stem cells from the patient's bone marrow, transducing a normal copy of the adenosine deaminase (ADA) gene into the stem cells, and then reintroducing the stem cells into the patient by intravenous infusion, after which some of the stem cells will return to the bone marrow. The therapy has been implemented in 18 children with the earliest 15 years ago, and up to now, they are still alive.

FCS is a rare autosomal recessive disorder caused by mutations in lipoprotein lipase. The patients suffer from various acute pancreatitis recurs, which is very painful and even life-threatening. Glybera uses an adeno-associated virus (AAV) to deliver genes that produce functional lipoprotein lipase to patients' skeletal muscles. After treatment, the incidence of pancreatitis is greatly reduced, and dietary restrictions can be relaxed to improve the quality of life.

Glybera is priced at about $ 1,000,000 and has only sold 1 since it went on sale in 2012. Due to a lack of market demand, it delisted in 2017. Strimvelis is priced at about $700,000. Only two copies were sold after it was launched in May 2016, and only two patients are scheduled to receive treatment.

Although gene therapy has increased the cure rate of many rare diseases, the expense of treatment has made it unaffordable for most individuals. At the currently, the US government is progressively adopting an insurance reform pilot program in order to entice insurance companies to pay gene therapy.

About the Author

Candy Swift: Focus on the cutting edge biological information around the world.

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Author: Candy Swift
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Candy Swift

Member since: Nov 06, 2019
Published articles: 131

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