Akshay Sharma, MBBS, on Investigating CRISPR/Cas9 Edited Cells for Sickle Cell Disease

“By editing the gamma globin promoter, we are creating mutations which are very similar to hereditary persistence of fetal hemoglobin. There are many of these either small or large deletions and mutations, which exist in individuals naturally... Nature has already done this natural experiment for us, and people already have these mutations. So, by recreating those mutations, hopefully we can take advantage of what nature has offered us and treat patients with sickle cell as well.”

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

CGTLive spoke with Sharma to learn more about OTQ923 and the potential advantages it has to offer in SCD. He discussed more data to come and research that remains to be done with the therapy and disease.

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