FDA Expected to Approve Groundbreaking CRISPR Gene-Editing Treatment for Sickle Cell Disease by December 8

FDA Advisory Committee Shares Optimism Over CRISPR Gene-Editing Treatment for Sickle Cell Disease, Despite Concerns about Access, Cost and Off-Target Genetic Modifications.

The US FDA appears poised to approve a groundbreaking CRISPR gene-editing treatment, developed jointly by Vertex Pharmaceuticals and CRISPR Therapeutics, for sickle cell disease by December 8. This would be the first time that a CRISPR-based treatment gets FDA approval.
This new gene therapy referred to as 'exagamglogene autotemcel' (exa-cel), involves modifying a patient’s own stem cells to rectify the genetic errors causing the sickle cell disease, thereby stimulating the production of fetal hemoglobin, which can reduce the disease’s severity.
Despite positive results from initial trials, the advisory committee has raised concerns over the potential off-target editing that may lead to unintended genetic modifications. However, both Vertex and CRISPR reported that they hadn't seen any such side effects during the clinical trials.
Presently, the only cure for sickle cell disease is a bone marrow transplant, which has a success rate of only 1 in 4 among full siblings. However, FDA's potential approval of this new method can give new hope to the approximately 100,000 US and 20 million worldwide patients suffering from sickle cell disease.
Although promising, the treatment faces several challenges including high costs of up to $2 million per patient, invasive procedures like blood transfusions and chemotherapy, along with potentially lengthy hospital stays. Inequality in access to the new treatment also remains a concern, as those most in need may not be able to afford it.