Clinical trials have shown promising results, with patients experiencing reduced symptoms.
The treatment, known as Casgevy, works by knocking out a gene that naturally turns down the production of foetal haemoglobin.
The treatment's price is speculated to be around $3 million, excluding hospital stays.
The UK has approved the world's first treatment using the gene-editing tool CRISPR for two blood disorders: sickle cell anaemia and beta thalassemias.
The United Kingdom has approved the world's first treatment using the gene-editing tool CRISPR for two blood disorders: sickle cell anaemia and beta thalassemias. The treatment, known as Casgevy, has been developed by Vertex Pharmaceuticals and CRISPR Therapeutics. It works by knocking out a gene that naturally turns down the production of foetal haemoglobin, thereby compensating for the genetic error that causes these disorders.
Clinical trials have shown promising results, with patients experiencing reduced symptoms. One such patient, Victoria Gray, who has suffered from sickle cell disease her whole life, volunteered for the trial in hopes of improving her quality of life. After undergoing the CRISPR therapy, Gray experienced a significant reduction in pain and has not had a pain crisis, hospitalization, or blood transfusion since.
However, the treatment is expected to be expensive and may not be widely available in low-income countries. The treatment's price is speculated to be around $3 million, excluding hospital stays. Over $1 billion has been spent on manufacturing and trials. Despite the high costs, more CRISPR therapies are expected to follow as clinical trials progress.
The treatment is also pending approval in the United States, with the FDA expected to give its approval soon.
The therapy, called Exa-cel, has shown promising results in clinical trials, including for the first patient, Victoria Gray.
Gray, who has suffered from sickle cell disease her whole life, volunteered for the trial in hopes of improving her quality of life.
After undergoing the CRISPR therapy, Gray experienced a significant reduction in pain and has not had a pain crisis, hospitalization, or blood transfusion since.
