In the USA, gene therapy from Nobel laureates was approved for the treatment of a rare blood disease

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The American regulator approved gene therapy, based on a method from Nobel laureates, for the treatment of a rare hereditary blood disease.

This is Casgevy therapy, which has been finally approved for treatment beta thalassemiareports Reuters.

In this method, doctors use “molecular scissors” to cut out defective parts of genes, which can then be “turned off” or replaced with normal DNA.

Casgevy therapy is indicated for the treatment of people aged 12+ years who are in constant need of blood transfusions (have transfusion-dependent beta-thalassemia or TDT).

Casgevy is a therapy from Vertex Pharmaceuticals and CRISPR Therapeutics based on CRISPR/Cas9 gene editing technology.

The inventors of “genetic scissors” Emmanuel Charpentier and Jennifer A. Doudna received the Nobel Prize in 2020.

Illustrative photo: Syda_Productions/Depositphotos

The FDA’s decision allowed Casgevy to receive a second approval in the US. Earlier, in December 2023, the drug was allowed to treat sickle cell anemia, another hereditary blood disease.

Casgevy will be available early this year with a list price of US$2.2 million.

It can be used only by medical institutions with experience in stem cell transplantation.

In 2022, the US regulator already approved Zynteglo cell gene therapy from competitor Bluebird Bio to treat people with TDT.

We will remind you that β-thalassemia is a hereditary blood disease characterized by a violation of the synthesis of beta-globin chains. Carriers of this disease are 1.5% of the planet’s population.

According to the National Institute of Health of Great Britain, about 23,000 children are born with a severe form of β-thalassemia every year. Their lives depend on blood transfusions.

Read also: Drugs against lymphoma: a drug against a rare type of blood cancer has been approved in the USA

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