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."

OTQ923, a CRISPR/Cas9-edited, autologous, CD34+ stem cell therapy, induced durable expression of fetal hemoglobin in 2 participants with sickle cell disease enrolled in a phase 1/2 trial (NCT04443907). Updated data from the trial were presented at the 64th American Society of Hematology (ASH) Annual Meeting, held December 10-12, 2022, in New Orleans, Louisiana, by Akshay Sharma, MBBS, assistant member, bone marrow transplant department, St. Jude Children’s Research Hospital.

CGTLive spoke with Sharma to learn more about the data seen in the first 2 patients treated with OTQ923. He also discussed trends in data being presented at the ASH meeting, including multiple presentations assessing non-genotoxic based conditioning approaches for autologous cell transplantation.

Click here to read more coverage of the ASH 2022 meeting.

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