Much Needed Gene Editing Therapy Now Available

Landmark gene editing therapy for sickle cell disease patients will soon be available to people with severe illness across the United Kingdom.

“The approval of exa-cel today marks a significant shift in the treatment landscape of sickle cell disease in the UK. It is the beginning of re-addressing the inequalities in care experienced by so many with the condition,” says Funmi Dasaolu, a sickle cell disease patient herself.

Few treatments are currently available for sickle cell disease, an inherited blood disorder that causes severe pain, infections, anemia, and other issues. The UK’s National Institute for Health and Care Excellence (NICE) approved the CRISPR gene-editing therapy. It involves taking a patient’s blood stem cells, editing them in a lab, and replacing them in the patient. Even though it has been approved, questions remain about its long-term effectiveness and safety. Data will continue to be collected on patients who receive it this go around and continually reevaluated by NICE. The newly approved treatment will only be available for severe forms of the disease in patients 12 and older in the United Kingdom.