Casgevy gene-editing therapy now approved in Saudi Arabia for SCD

Saudi Arabia’s regulatory authorities have approved Casgevy (exagamglogene autotemcel), a one-time gene-editing therapy, to treat sickle cell disease (SCD) in adults and adolescents, ages 12 and older. The approval by the Saudi Food and Drug Authority (SFDA), the country’s equivalent to the U.S. Food and Drug Administration, also…

RoslinCT to manufacture CRISPR-based gene therapy Casgevy

RoslinCT will manufacture the groundbreaking new gene therapy Casgevy (exagamglogene autotemcel) for sickle cell disease (SCD) patients in the U.S. and U.K. RoslinCT, a cell and gene therapy contract development and manufacturing company, has facilities in Edinburgh, Scotland and Hopkinton, Massachusetts. The company worked with Vertex…

Researchers Detect DNA Modifications Created by Gene Editing

Researchers have developed a method to detect and measure large unintended DNA modifications created by CRISPR-Cas9 gene-editing methods currently being investigated as treatments for sickle cell disease (SCD). High levels of unintended gene modifications that occurred at selected target sites demonstrate the need for a more careful evaluation of…