Haydar Frangoul, MD, on Sickle Cell Disease Gene Therapy Exa-Cel's Ability to Prevent VOCs

This interview originally appeared on our sister site, HCPLive®.

“I keep telling people that our therapy was CRISPR gene editing 1.0 and I'm pretty sure as the field evolves, we are going to potentially use safer conditioning regimen for patients that does not cause hair loss and infertility. But we had to start somewhere… and I think that's why it's so groundbreaking, is what we did is we established the bar, and now it can only get better from here."

Vertex Pharmaceuticals' and CRISPR Therapeutics' exagamglogene autotemcel (exa-cel, marketed as Casgevy), a nonviral CRISPR/Cas9-based gene editing cell therapy intended to treat sickle cell disease (SCD), functions by reactivating fetal hemoglobin (HbF). The product has been approved by the FDA as one-time treatment for patients aged at least 12 years with severe SCD since late 2023. Notably, the ongoing phase 3 CLIMB SCD-121 clinical trial (NCT03745287) continues to evaluate the safety and efficacy of exa-cel in people with SCD.

Haydar Frangoul, MD, the medical director of pediatric hematology/oncology at Sarah Cannon Research Institute and Pediatric Transplant and Cellular Therapy Program at TriStar Centennial, recently presented the most up to date data from this trial at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition, held December 7-10, 2024, in San Diego, California. CGTLive®'s sister site, HCPLive®, interviewed Frangoul on the conference floor to learn more.

Frangoul shared highlights from the data update, which included over 90% of participants remaining vasoocculsive event (VOC)-free and an over 90% hospitalization-prevention rate. He also pointed out that participants maintained fetal hemoglobin levels of over 40% sustained for up to 5 years.

Click here for more coverage of ASH 2024

REFERENCE
Frangoul H, Locatelli F, Sharma A, et al. Durable Clinical Benefits with Exagamglogene Autotemcel for Severe Sickle Cell Disease. Presented at: Presented at: ASH Annual meeting; December 7-10; San Diego, California. Abstract 4954.