Recently USFDA approved for use two breakthrough gene therapies for the management of Sickle Cell Disease in children aged 12 and above.

Casgevy is the first FDA-approved drug to use a revolutionary genome editing method, signifying a milestone in the area of cell-based gene therapy.

On the other hand, Lyfgenia involves genetically modifying the patient's blood stem cells to create HbAT87Q, a gene-therapy-produced haemoglobin that works similarly to haemoglobin A.

HbAT87Q-containing red blood cells have a decreased risk of sickling and occluding blood flow.

Both products are created from patients' own blood stem cells that have been changed and are given back as a one-time, single-dose infusion transplant.