First CRISPR Drug Coming Soon
July 6, 2022| |
Vertex and CRISPR Therapeutics presented impressive results of clinical trials for exagamglogene autotemcel (exa-cel), which can help patients with beta thalassemia and sickle cell disease (SCD). Exa-cel is the first gene editing therapy of its kind and will be submitted for regulatory approval in the US, UK, and Europe by the end of 2022.
Beta thalassemia affects 1 in 100,000 people globally. It is characterized by damages or missing genes that cause the body to produce less hemoglobin, which can lead to the enlargement of vital organs. Regular blood transfusion is necessary for patients with this disease. On the other hand, SCD affects millions globally and the sickle-shaped hemoglobin blocks the healthy blood cells from transporting oxygen around the body.
The latest results of the clinical trials of exa-cel were revealed at the European Hematology Association Congress in Switzerland. It was reported that 75 patients with beta thalassemia or SCD given who underwent the gene editing therapy had very minimal to zero blood transfusions or incidences of life-threatening blockages.
Read the original story in Fast Company.
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