Success rate of more than 90 percent: first therapy with genetic scissors approved

class=”sc-29f61514-0 fwWrRV”>

A drug based on the genetic scissor technology Crispr has been approved in Great Britain for the first time in the world. The Medicines and Healthcare products Regulatory Agency (MHRA) announced this on Thursday.

The drug called ‘Casgevy’ is approved for the treatment of sickle cell disease and beta-thalassemia, two genetic blood diseases, in patients aged twelve years and older. Both diseases are caused by errors in the gene for hemoglobin. This is an iron-containing protein complex found in red blood cells and used to transport oxygen.

The so-called Crispr/Cas gene scissors can be specifically targeted at individual genes. The developers of the method, Emmanuelle Charpentier and Jennifer A. Doudna, received the Nobel Prize for this in 2020. “Casgevy” is used to change the faulty genes in patients’ bone marrow stem cells so that they can produce functioning hemoglobin, the MHRA said in a statement.

The treatment can last lifelong

For this purpose, stem cells are extracted from the bone marrow, processed in the laboratory and then reintroduced into the patient. The therapy requires a hospital stay of several weeks. But the result has the potential to last a lifetime, they said.

Both sickle cell disease and beta thalassemia are painful, lifelong conditions that can be fatal in some cases, says Julian Beach, interim director of the MHRA’s Healthcare Quality and Access department. He added: “Until now, a bone marrow transplant, which must come from a well-matched donor and carries the risk of rejection, has been the only permanent treatment option.”

Sickle cell disease can cause severe bouts of pain, serious and life-threatening infections, and anemia, a lack of oxygen in the blood, also called anemia. Patients with beta thalassemia also suffer from anemia and often require blood transfusions at three to five week intervals.

More than 90 percent of patients showed success

Of 28 patients with sickle cell disease treated with the drug in a clinical trial, 97 percent were free of severe pain attacks for at least a year.

In the clinical trial of beta-thalassemia patients dependent on blood transfusions, 93 percent (39 of 42 patients) did not require a red blood cell transfusion for at least twelve months. In the remaining three patients, a reduction in the number of blood transfusions by at least 70 percent was possible.

The drug is manufactured in a collaboration between Vertex and CRISPR Therapeutics, headquartered in the US and Switzerland. According to a statement from the two companies, around 2,000 people in Britain are currently eligible for treatment with “Casgevy”.

Vertex director Reshma Kewalramani spoke of a “historic day in science and medicine”. CRISPR Therapeutics boss Samarth Kulkarni said he hoped this was just the first of many applications of the method that could benefit people with serious diseases, the statement said. (SDA)

Source: Blick