The road from clinical trials to regulatory green light now appears to be downhill for the treatment for sickle cell anemia developed by CRISPR Therapeutics, the company co-founded by Emmanuelle Charpentier. We knew it as CTX001 but it has changed its name to exa-cel (which stands for exagamglogene autotemcel). It was one of the first CRISPR-based gene therapies to enter clinical trials, in 2019. It changed the lives of Victoria Gray and dozens of sickle cell anemia and thalassemia patients enrolled in several countries. Now it also leads the way in the late stage of the regulatory process, both in Europe and the United States, and could come to market first, in 2023. For more information see the press-release by Vertex, that collaborates at exa-cel manufacturing, regulatory and commercialization.
Covid19 is affecting everyone, but it has hit the sickle cell (SCD) community particularly hard. According to STAT News the pandemic has temporarily stopped clinical trials and the introduction of new drugs, besides directly impacting SCD patients who are at high risk for severe complications from Sars-Cov2 infection and may need hospital assistance for SCD pain crises.Continue reading