MANAMA, Bahrain — The approval came quietly, in a regulatory filing on a Saturday. But the ripple it sent through the medical world was anything but small. On December 2, 2023, Bahrain’s National Health Regulatory Authority cleared a treatment called exagamglogene autotemcel for two devastating blood disorders. The decision made the tiny Gulf kingdom the second country on earth to approve a therapy that literally rewrites a patient’s DNA.
That therapy, sold under the brand name Casgevy, is a collaboration between Vertex Pharmaceuticals and CRISPR Therapeutics. It uses the CRISPR/Cas9 gene-editing tool — a technology that won its discoverers a Nobel Prize in 2020 — to fix the genetic errors behind sickle cell disease and transfusion-dependent beta thalassemia. The United Kingdom had already signed off on it a month earlier, in November 2023. Bahrain followed. The United States, as of that same December weekend, was still reviewing it.
Here is what makes this approval different from the standard drug-approval story. It is not just another pill or injection. Casgevy is a one-time treatment. Doctors take a patient’s own blood stem cells, edit them in a lab to produce fetal hemoglobin — a form of hemoglobin that is naturally resistant to sickling — and infuse them back. The goal: a permanent cure for a lifelong disease.
Sickle cell disease is brutal. Red blood cells, normally round and flexible, turn into rigid crescents. They clog vessels, starve organs of oxygen, and cause pain so severe that patients often end up in emergency rooms on morphine drips. Beta thalassemia is similarly punishing. Patients require blood transfusions every few weeks, which over time loads the body with excess iron, damaging the heart and liver. Both conditions are genetic. Both disproportionately affect people of African, Middle Eastern, and South Asian descent.
Bahrain sits in the heart of a region where these diseases are common. The approval there was not abstract. It was local. The NHRA’s decision means Bahraini patients — and potentially others in the Gulf — now have a path to a therapy that was previously only theoretical.
The treatment is not without risk. The most common side effects reported include low levels of platelets and white blood cells. That is a standard complication of any stem cell transplant procedure, which involves chemotherapy to clear out the bone marrow before the edited cells are infused. Still, for patients who have spent years in and out of hospitals, those risks may be acceptable.
The financial picture is another matter. Casgevy is not cheap. Gene therapies of this kind typically carry price tags in the hundreds of thousands of dollars. Vertex and CRISPR Therapeutics have not publicly set a price for the Bahrain market, but the economics of a small Gulf state paying for an expensive cell therapy will be watched closely by other countries.
What the Bahrain approval signals, though, is momentum. The UK was first. Bahrain was second. The US Food and Drug Administration is weighing a decision. If the FDA says yes, Casgevy would become the first cell-based gene therapy using CRISPR/Cas9 ever approved in the United States. That would be a historic moment — not just for the companies involved, but for the entire field of gene editing, which has spent years moving from lab benches to hospital beds.
For now, the approval in Bahrain is a concrete step. A regulatory body looked at the data and said yes. Patients in one country now have an option that did not exist before. That is the kind of fact that does not need embellishment.
