Medical director, pediatric hematology/oncology, Sarah Cannon Pediatric Transplant and Cellular Therapy Program, TriStar Centennial, and investigator, Sarah Cannon Research Institute

Haydar Frangoul, MD, MS

Frangoul discussed highlights from the latest data update from the CLIMB SCD-121 trial of Casgevy.

Haydar Frangoul, MD: Preventing VOCs in People With Sickle Cell Disease With Exa-Cel Gene Editing Therapy

Exagamglogene autotemcel (exa-cel; Casgevy, Vertex Pharma and CRISPR Therapeutics) is a non-viral CRISPR-Cas9 gene editing cell therapy that reactivates fetal hemoglobin (HbF), approved as a one-time treatment for patients aged at least 12 years with severe sickle cell disease (SCD). The CLIMB SCD-121 phase 3 study is ongoing and continues to assess the safety and efficacy of exa-cel in people with SCD.

The latest data from the CLIMB SCD-121 study are being presented at the

 66th American Society of Hematology (ASH) Annual Meeting and Exposition

, held December 7-10, 2024, in San Diego, California, by Haydar Frangoul, MD, MS, medical director, pediatric hematology/oncology, Sarah Cannon Pediatric Transplant and Cellular Therapy Program, TriStar Centennial, and investigator, Sarah Cannon Research Institute.

“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 will 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,” Frangoul told 

He shared highlights from the data update, including an over 90% rate of participants remaining vaso-occulsive event (VOC)-free, and an over 90% hospitalization prevention rate. He also noted that participants maintained fetal hemoglobin levels of over 40% sustained for up to 5 years.

“The big question that yet to be answered is, yes, we are preventing VOCs, but how is that going to impact the end organ damage that we know SCD patients will develop years and years and years down the road. So, I am very excited to see where the field goes,” Frangoul said.

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.

Haydar Frangoul, MD, MS

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