Mozafancogene autotemcel for treating Fanconi anaemia subtype A in children and adolescents

Mozafancogene autotemcel is in development for the treatment of Fanconi anaemia subtype A (FA-A) in children. Fanconi anaemia (FA) is a rare genetic condition caused by changes (mutations) in certain genes that help repair deoxyribonucleic acid (DNA). In people with FA-A, the affected gene is called FANCA. When DNA is not properly repaired, it can cause cells to die prematurely or grow in an uncontrolled way. This can lead to physical abnormalities, low numbers of blood cells, and an increased risk of cancer. Over time, FA can cause the bone marrow (the soft, spongy tissue inside bones that makes blood cells) to stop working properly, also known as bone marrow failure. Bone marrow makes stem cells, which produce red blood cells, white blood cells, and platelets, all of which are essential for staying healthy. There are currently limited options for treating FA.