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Trial Expansions

Trials examining CRISPR-based treatments for sickle cell disease and beta-thalassemia are expanding, according to New Scientist.

It adds that, in early studies, the exa-cel treatment from CRISPR Therapeutics and Vertex Pharmaceuticals has had promising results. The treatment involves the removal of patients' hematopoietic stem cells, which then undergo editing to produce high levels of fetal hemoglobin before being returned.

The companies announced at a European Hematology Association meeting that of the 44 individuals in their beta-thalassemia trial, 42 no longer need transfusions and that none of the 31 individuals with severe sickle cell disease have had a crisis event since being treated, New Scientist adds.

Following these results, the companies are now going to include children under the age of 12 in their trials, in the hopes that earlier treatment will prevent lasting damage, it notes.

It adds that the companies are additionally at an early stage of exploring whether this gene-editing step could be conducted within the body.