11 Dec 2023 • The U.S. FDA has approved two milestone treatments, Casgevy and Lyfgenia, marking the first cell-based gene therapies for treating SCD in patients aged 12 years and older.

Casgevy, a cell-based gene therapy, is approved for treating SCD in patients who experience recurrent vaso-occlusive crises. Casgevy stands out as the first FDA-approved therapy utilizing CRISPR/Cas9, a type of genome editing technology.

CRISPR/Cas9 can be directed to cut DNA in targeted areas, enabling the ability to accurately edit (remove, add, or replace) DNA where it was cut. The modified blood stem cells are transplanted back into the patient where they engraft (attach and multiply) within the bone marrow and increase the production of fetal hemoglobin (HbF), a type of hemoglobin that facilitates oxygen delivery. In patients with sickle cell disease, increased levels of HbF prevent the sickling of red blood cells.

Source: FDA | Read full story