Groundbreaking CRISPR Treatment for Sickle-Cell Disease Receives UK Approval

Regulators in the United Kingdom have given the green light to Casgevy, the first treatment developed through CRISPR, a groundbreaking gene-editing technique. Manufactured by Boston-based Vertex Pharmaceuticals and Switzerland-based CRISPR Therapeutics, Casgevy aims to cure sickle-cell disease and its related condition, beta-thalassemia.

Around 2,000 patients in the UK suffering from sickle-cell disease or beta thalassemia are expected to qualify for this innovative treatment. The companies anticipate that the Food and Drug Administration (FDA) in the United States will grant approval for Casgevy for sickle-cell patients in early December, with a decision on beta-thalassemia expected next year.

Another sickle-cell gene therapy by Bluebird Bio, based in Somerville, Mass., is also anticipated to receive FDA approval in late December. Unlike Casgevy, Bluebird’s treatment does not rely on gene editing; instead, it uses a method that inserts new DNA into the genome.

Sickle-cell disease, affecting mostly Black and Hispanic populations, is caused by a faulty gene resulting in abnormal hemoglobin production, deforming red blood cells, and causing severe pain episodes. About 100,000 Americans are believed to have this illness.

In beta thalassemia, a rare condition, the defective gene leads to insufficient levels of hemoglobin in red blood cells.

Casgevy uses CRISPR to edit the DNA, activating a gene that produces an alternative form of hemoglobin. In the UK, patients eligible for the sickle-cell treatment must be at least 12 years old and have a history of repeated extreme pain episodes.

There is no upper age limit, and patients with organ damage from sickle-cell disease are not excluded. However, patients must have no other treatment options, as compatible bone marrow donors for transplants are scarce.

For patients battling sickle-cell disease, the Vertex and Bluebird treatments offer a long-awaited ray of hope. The disease not only causes excruciating pain but also leads to bone and organ damage and strokes. The misshapen blood cells result in anemia as they have a shorter lifespan.

While these gene-editing treatments hold promise, they are demanding and require specialized expertise not available in most hospitals. Patients undergo intensive chemotherapy to clear their bone marrow of abnormal stem cells, making room for genetically altered cells. Following this, patients spend a month or more in the hospital while their marrow regrows.

Despite the promising outcomes, gene editing is a costly process. Vertex and CRISPR Therapeutics have yet to set a price in the UK, and the cost in the United States is expected to reach millions of dollars per patient. However, considering the annual $3 billion cost of sickle-cell disease to the U.S. health system, the investment may prove worthwhile.

Vertex is currently testing its sickle-cell treatment in children aged 5 to 11, aiming to prevent the irreversible organ damage that occurs over time. Stuart Arbuckle, executive vice president and chief operating officer at Vertex, notes that pricing discussions will involve stakeholders responsible for covering the costs.

Victoria Gray, Vertex’s first sickle-cell patient, attests to the life-changing impact of the treatment. Diagnosed with sickle-cell disease at three months old, Gray faced frequent hospitalizations due to pain crises. After receiving the gene-editing treatment in 2019, she reports that all her symptoms have vanished, heralding a new beginning beyond her dreams.

As the world witnesses the evolution of gene-editing technologies, these advancements in CRISPR treatments represent a monumental leap toward more effective and targeted therapies for genetic disorders. With regulatory approvals, these treatments offer renewed hope for patients grappling with the complexities of sickle-cell disease and beta-thalassemia.