UK approves gene editing treatment for children as young as five

British health regulators have expanded access to a gene editing treatment, approving its use for children as young as five years old who have severe sickle cell disease or transfusion dependent beta thalassemia. Streamlinefeed and Mirage News both reported that the Medicines and Healthcare products Regulatory Agency expanded the authorization for the treatment, known as Casgevy or exagamglogene autotemcel. [1][2]

According to streamlinefeed, the therapy was previously restricted in the United Kingdom to patients aged 12 and over. Regulators expanded the approval to offer a potential functional cure for younger children facing lifelong genetic conditions. [1]

Casgevy is the first approved therapy to utilize CRISPR gene editing. CRISPR is a genetic engineering technique based on a bacterial defense system that uses a Cas9 protein and guide RNA to cut DNA at specific locations, allowing existing genes to be removed or new ones added. [1][3]

Victoria Gray was the first patient ever treated with the CRISPR gene editing tool for sickle cell disease. As HEROIC BREAKING NEWS reported earlier, researchers have also been working on early detection tools, including an affordable point of care device developed at IIT Bombay by a team led by Professor Mahesh Tirumkudulu to screen for sickle cell disease using a single drop of blood. [4][6][7]