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The first CRISPR Cas9 gene edit** was officially approved yesterday.
This is the first FDA-approved gene edit** in the U.S. F.D., and it is significant.
In November, Vertex and CRISPR Therapeutics announced that they had licensed their products for sickle cell disease and transfusion-dependent thalassemia – Casgevy.
Although there may be only 2,000 patients in the UK who meet the criteria, the significance behind it is significant because it gives a glimpse of the regulator's attitude towards innovation**. Sure enough, one month later, the US FDA also approved the **.
Gene editing in thalassemia** may be the first time, and more CRISPR-based gene editing** will be available in the future.
On Friday, the U.S. FDA approved for the first time the treatment of sickle cell disease and transfusion-dependent thalassemia — casgevy. This means that CRISPR-based gene editing** will be more available in the future. 
However, it is estimated that only 2000 patients in the UK are eligible for CASGEVY. "Today is a historic day in science and medicine: Casgevy in Great Britain is the world's first CRISPR-based regulatory authorization. Reshma Kewalramani, MD, CEO and President of Vertex. "I hope this is the first of many applications of this Nobel Prize-winning technology that can benefit eligible patients with serious diseases," said Samarth Kulkarni, Chairman and CEO of CRISPR Therapeutics. 
How effective is casgevy?In two global clinical trials of Casgevy**SCD and TDT, the trials met their respective primary focus – i.e., freedom from serious VOCs for at least 12 consecutive months or independent of blood transfusion. Once realized, these benefits are expected to last a lifetime. In these ongoing studies, the safety profile of 97 patients with SCD and TDT who have received Casgevy** to date is generally consistent with medullary conditioning of busulfan and hematopoietic stem cell transplantation. 
This authorisation provides a new option for eligible patients who are waiting for an innovation**, and I look forward to patients receiving this as soon as possible, said Jose de la Fuente, a professor at Imperial College London who is the principal investigator of the Climb-111 and Climb-121 studies. About sickle cell diseaseSickle cell disease (SCD) is an inherited blood disorder that affects red blood cells, which are essential for carrying oxygen to all organs and tissues in the body. Due to malformations or "sickle" blood cells, SCD can cause severe pain, organ damage, and shortened lifespan. People with SCD may experience painful blockages of blood vessels, also known as vaso-occlusive crises (VOCs), which can lead to acute chest syndrome, stroke, jaundice, and heart failure symptoms. Individuals may also develop anemia, which can lead to end-organ damage and premature death. Vaso-occlusive crisis is a hallmark of SCD and often causes severe pain. Currently, the standard protocol for SCD is primarily symptomatic and does not adequately address the burden of disease or alleviate the need for chronic care. Most often, the focus is on relieving pain, minimizing organ damage, staying hydrated, and reducing fever, requiring medications, sometimes monthly blood transfusions, and frequent hospital visits. The only** method for SCD is a stem cell transplant from a matched donor, but this option is only available to a small percentage of patients with SCD. SCD requires lifelong** and heavy use of health care resources, ultimately leading to reduced life expectancy, lifetime earnings, and reduced productivity. In the UK, the average age of death for people with SCD is around 40 years. About thalassemiaThalassemia is an inherited blood disorder that affects red blood cells, which are essential for carrying oxygen to all organs and tissues in the body. Erythrocytosis, also known as anemia, is the main manifestation of thalassemia. As a result of this anemia, people with thalassemia may experience fatigue and shortness of breath, and babies may experience problems such as developmental delays, jaundice, etc. Complications of thalassemia also include enlargement of the spleen, liver, and/or heartSkeletal deformities;and delayed puberty. Thalassemia is individualized and depends on the severity of the disease each person is experiencing. Many people have to have regular blood transfusions to get healthy donated blood into their bodies. This requires multiple hospital visits and can also lead to an unhealthy iron buildup. Today, stem cell transplantation from a matched donor is an option, but only for a small percentage of thalassemia patients. Thalassemia requires lifelong** and heavy use of health care resources, ultimately leading to reduced life expectancy, reduced quality of life, and reduced lifetime income and productivity. In the UK, the average age of death for people with TDT is around 55 years. About Casgevy**
How Exa-Cel works Casgevy is a transgenic autologous CD34+ cell-rich cluster containing human hematopoietic stem and progenitor cells that have been edited ex vivo by CRISPR Cas9 in the erythroid specific enhancer region of the BCL11A gene. The latest data from the ongoing pivotal trials were published at the European Haematology Association Congress in June 2023. EXA-CEL is also under review by the European Medicines Agency, the Saudi Food and Drug Administration, and the U.S. Food and Drug Administration (FDA). The FDA has granted the SCD Priority Review and the TDT Standards Review and will formally approve both of them by the end of this year or early next year**.