First CRISPR clinical trial approved outside of China
- 5 years ago
USA / SWITZERLAND — The FDA has given approval to two pharmaceutical companies to begin a human gene-editing trial.
A joint clinical trial led by CRISPR Therapeutics and Vertex will use gene editing to target sickle cell disease and beta-thalassemia. This will be the first confirmed CRISPR gene editing clinical trial to take place outside of China.
According to a CRISPR Therapeutics news release, the gene-editing therapy, called CTX001, will be used to modify a patient's stem cells to increase hemoglobin levels in red blood cells.
The first trial of gene-editing will be tested on two patients, with each being administered a single dose of CTX001. The patients will be monitored to see when the edited cells start producing mature blood cells, according to the news release.
A joint clinical trial led by CRISPR Therapeutics and Vertex will use gene editing to target sickle cell disease and beta-thalassemia. This will be the first confirmed CRISPR gene editing clinical trial to take place outside of China.
According to a CRISPR Therapeutics news release, the gene-editing therapy, called CTX001, will be used to modify a patient's stem cells to increase hemoglobin levels in red blood cells.
The first trial of gene-editing will be tested on two patients, with each being administered a single dose of CTX001. The patients will be monitored to see when the edited cells start producing mature blood cells, according to the news release.