Professor Cavazzana…provided an update on subject 1003, a patient with beta-thalassemia major treated by Professor Cavazzana in 2007 in the LG001 study using the first-generation HPV569 lentiviral vector.
Following approximately seven years of transfusion independence, subject 1003 has recently required two blood transfusions after experiencing clinical symptoms of anemia. Importantly, both the expression of HbAT87Q and the vector copy number in peripheral blood leukocytes, a measure of the persistence of the gene therapy, have remained largely unchanged.