Akshay Sharma, MBBS, on Inducing Fetal Hemoglobin in Sickle Cell With CRISPR/Cas9-Edited Stem Cells

"We are editing the promoters of the HBG1 and HBG2 gamma globin genes. By editing the promoter, we are hoping that it'll be a more specialized, more targeted approach to actually disrupt the binding site of the transcription factors, which are responsible for the gamma- to betaglobin switch. And so, we believe that it's a more targeted and more elegant approach, and the data on the first 2 patients looks quite promising."

REFERENCE

Treatment of individuals with severe sickle cell disease with OTQ923, an autologous, ex vivo, CRISPR/Cas9-edited, CD34+ hematopoietic stem and progenitor cell product, leads to durable engraftment and fetal hemoglobin induction. Presented at: 64th ASH Annual Meeting, December 10-12, New Orleans, Louisiana. Abstract #786