CNN —
The United Kingdom has become the first country to give regulatory approval to a medical treatment involving the revolutionary CRISPR gene editing tool.
The country’s Medicines and Healthcare products Regulatory Agency said Thursday it had given a greenlight to a treatment known as Casgevy, which will be used to treat sickle cell disease and beta thalassemia. Both genetic conditions are caused by errors in the genes for hemoglobin, which is used by red blood cells to carry oxygen around the body. There is no known universally successful treatment for either disorder.
Sickle cell disease, which can result in attacks of debilitating pain, is more common in people with an African or Caribbean family background. Beta thalassemia mainly affects people of Mediterranean, South Asian, Southeast Asian and Middle Eastern origin, the statement said.
A worker from the Community Blood Center hangs a bag of blood during a transfusion for Kevin Wake at the Sickle Cell Center at University Health on March 7, 2023. (Tammy Ljungblad/The Kansas City Star/Tribune News Service via Getty Images)
FDA considers first CRISPR gene editing treatment that may cure sickle cell
“Both sickle cell disease and β-thalassemia are painful, life-long conditions that in some cases can be fatal. To date, a bone marrow transplant — which must come from a closely matched donor and carries a risk of rejection — has been the only permanent treatment option,” said Julian Beach, the interim executive director of healthcare quality and access at the MHRA, in a statement.
“I am pleased to announce that we have authorised an innovative and first-of-its-kind gene-editing treatment called Casgevy, which in trials has been found to restore healthy haemoglobin production in the majority of participants with sickle-cell disease and transfusion-dependent β-thalassaemia, relieving the symptoms of disease.”
CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a revolutionary gene-editing technology that allows scientists to modify DNA at specific locations in the genome. It uses a guide RNA to identify the target DNA sequence and the Cas9 enzyme to cut the DNA, enabling the addition, removal, or alteration of genetic material. This precise editing capability has made it a powerful tool for treating genetic disorders.
The CRISPR treatment that has been greenlit in the UK targets sickle cell disease and beta thalassemia. Both are inherited blood disorders that affect the production of hemoglobin, leading to various health complications. The treatment aims to correct the genetic mutations responsible for these conditions, potentially providing a long-term cure.
'Greenlit' refers to the approval process where a treatment receives regulatory authorization to proceed with clinical use. In this context, it means that the UK regulatory authorities have evaluated the safety and efficacy of the CRISPR treatment for sickle cell disease and beta thalassemia and have given it the go-ahead for use in patients.
While CRISPR has shown great promise, potential risks include off-target effects, where unintended parts of the genome are altered, leading to unforeseen consequences. There is also the possibility of immune reactions to the CRISPR components and long-term effects that are not yet fully understood. Ongoing research and clinical trials aim to minimize these risks and ensure the safety of the treatment.
The approval of CRISPR treatment in the UK is a significant milestone that may pave the way for further research and development of gene editing therapies for various genetic disorders. It sets a precedent for regulatory frameworks and could encourage investment and collaboration in gene therapy research, ultimately leading to new treatments for other diseases.