Haydar Frangoul, MD, Details CLIMB-121 Data on Exa-Cel in Sickle Cell Disease

Haydar Frangoul, MD, a pediatric hematologist and oncologist at the Sarah Cannon Research Institute in Nashville, Tennessee, met with HemeToday to share findings from the CLIMB-121 trial on exagamglogene autotemcel (exa-cel) in patients with sickle cell disease (SCD).

The data had recently been presented at the 65th American Society of Hematology Annual Meeting & Exposition in San Diego, California.

“The primary objective of the trial was to increase fetal hemoglobin above 20%, because there are individuals with sickle cell disease [who] are relatively asymptomatic because they have persistent elevated fetal hemoglobin. What we did in this trial is we emulated what nature has already shown us for the last 60 to 70 years by editing the BC11A and the erythroid enhancer region. What we were able to achieve is fetal hemoglobin at or above 40% in our subjects, which allowed them to become [vaso-occlusive crisis] free and hospitalization free.”

Dr. Frangoul also described the ongoing long-term outcome studies and his hopes for the availability of exa-cel overall.

“We are super excited because I think this is a transformative therapy, and it has the potential to be the first-ever gene-editing functional cure for patients with sickle cell disease,” he suggested.