FDA Closer to Approving CRISPR Gene Editing Treatment for Sickle Cell Disease

Proposed Gene Therapy Uses CRISPR Technology to Alter Patient's Stem Cells, Showing Promising Results in Trials and Could Offer Lifelong Relief from Debilitating Disease

The FDA is nearing approval for the first CRISPR gene editing treatment for sickle cell disease, developed by Boston-based Vertex Pharmaceuticals and Swiss company CRISPR Therapeutics. This treatment is considered a groundbreaking advancement, offering hope to those with the debilitating disease.
The therapy, named exa-cel, uses CRISPR (clustered regularly interspaced short palindromic repeats) technology to selectively modify the patient's DNA and potentially cure sickle cell disease. If approved, this would be the first treatment using such genetic modification.
The treatment involves using the patient’s own stem cells, which are altered using CRISPR to correct the genetic abnormalities causing the disease. The edited stem cells are then returned to the patient via a one-time infusion. Preliminary tests showed promising results, with reduced vaso-occlusive crises in patients - the primary cause of hospitalization in sickle cell patients.
Sickle cell disease disproportionately impacts African Americans, with approximately 100,000 people in the US diagnosed with the disease. The current standard treatment involves a risky stem cell or bone marrow transplant, with fewer than 20% of patients having a suitable donor.
While experts acknowledge potential risks such as unintended genetic alterations, they assert the benefits outweigh the risks. Furthermore, the treatments are expected to be expensive, costing an estimated $1-2 million per patient. However, this would likely be offset by reducing the lifelong care costs for patients with sickle cell disease.