Top News and Expert Insights for World Sickle Cell Day 2025
In honor of World Sickle Cell Day, observed annually on June 19, we took a look back at news and expert insights from the past year in cell and gene therapy for SCD.
The landscape of care for sickle cell disease (SCD) is rapidly changing. On a single day in 2023—December 8—the field of hematology was shaken by the FDA’s simultaneous approval of 2 gene therapy products for the treatment of SCD: Vertex Pharmaceuticals' and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel; marketed as Casgevy) and bluebird bio’s lovotibeglogene autotemcel (lovo-cel; marketed as Lyfgenia). While these products continue to be rolled out to patient populations, a number of other advanced therapeutics are in clinical-stage development for SCD.
In honor of World Sickle Cell Day, held annually on June 19, CGTLive® is taking a look back at some of our major news stories and expert insights in SCD cell and gene therapy from the past year. Click the "READ MORE" buttons for more details and information about each item.
Beam’s Base-Editing HSC Therapy BEAM-101 Raises HbF Levels in Patients With Sickle Cell Disease
June 16, 2025 — At the European Hematology Association (EHA) 2025 Congress, held June 12 to 15, both virtually and in Milan, Italy, Beam Therapeutics presented new data from its phase 1/2 BEACON clinical trial (NCT05456880), which is evaluating the company’s investigational base-edited autologous hematopoietic stem cell (HSC) therapy BEAM-101 for the treatment of SCD. During the conference, CGTLive reached out to Beam Therapeutics' team to learn more about the new data.
Experts from Beam Therapeutics, including chief executive officer John Evans, MBA, weighed in on the updated results. They emphasized the durable increases in fetal hemoglobin (HbF) seen in the data and explained how BEAM-101 may differentiate itself from other gene therapy products for SCD.
Sickle Cell Disease Gene Therapy Exa-Cel's Ability to Prevent VOCs
December 12, 2024 — At at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition, held December 7-10, 2024, in San Diego, California, 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, presented updated data from the ongoing phase 3 CLIMB SCD-121 clinical trial (NCT03745287) evaluating exa-cel, a nonviral CRISPR/Cas9-based gene editing cell therapy intended to treat SCD. CGTLive®'s sister site HCPLive® interviewed Frangoul at the conference to learn more.
Frangoul went over the key points from the new data, which included over 90% of participants remaining vasoocculsive event (VOC)-free and an over 90% hospitalization-prevention rate. He also discussed the broader impact of exa-cel as the first CRISPR-based therapy and future plans for further research.
Canada Approves Vertex and CRISPR Therapeutics’ Gene Therapy Casgevy for Sickle Cell Disease and TDT
September 27, 2024 — Health Canada has granted marketing authorization to Vertex Pharmaceuticals' and CRISPR Therapeutics’ autologous gene-edited cell therapy exa-cel for the treatment of SCD in patients aged 12 years and older with recurrent VOCs and patients aged 12 years and older with transfusion-dependent beta thalassemia (TDT).
"Casgevy's approval is an exciting moment for 2 patient communities that have long awaited an innovative therapy that brings new hope and possibilities for those in need," Kevin Kuo, MD, hematologist and clinician investigator in the Red Blood Cell Disorders Clinic at University Health Network, and principal investigator for the CLIMB-131 clinical program (NCT04208529).
The approvals were based on data from the ongoing phase 1/2/3 CLIMB-121 clinical trial (NCT03745287), which is being conducted exclusively in patients with SCD, and the ongoing phase 1/2/3 CLIMB-111 clinical trial (NCT03655678),which is in patients with TDT; both trials are taking place at sites in multiple countries, including Canada. The primary end point for CLIMB-121, freedom from severe VOCs for 12 consecutive months, was met. Likewise, the primary end point for CLIMB-111, transfusion independence for 12 consecutive months, was also met. Vertex characterized the safety profile of the treatment as generally consistent with myeloablative conditioning using busulfan and hematopoietic stem cell transplant.
Uptake of Nononcology Gene Therapy Remains Slow in Hematology
August 19, 2024 — Patient use of the 2 FDA-approved gene therapy products for SCD, exa-cel and lovo-cel, in the commercial setting has remained low.
Despite the approval of both gene therapy products by the FDA occurring simultaneously on December 8, 2023, only 4 patients have begun treatment with lovo-cel in the commercial setting, while 20 patients are reported to have begun treatment with exa-cel. Alongside the number for lovo-cel, bluebird also pointed out that this year 4 patients have started on elivaldogene autotemcel (Lenti-D, Skysona), its marketed gene therapy for cerebral adrenoleukodystrophy, and 19 patients have started on betibeglogene autotemcel (beti-cel, Zynteglo), its marketed gene therapy for TDT. As such, bluebird bio has reported 23 total cell collections for SCD/TDT gene therapies lovo-cel and beti-cel. The company expects that patient uptake of the 3 aforementioned therapies will continue to increase, with 85 patient starts expected across the therapies by the end of the year.
“We are seeing clear evidence that our commercial launch is accelerating, with over 20 cell collections completed in SCD and TDT to date in 2024, and more than 40 additional patients already scheduled to initiate the treatment journey for a bluebird gene therapy by the end of this year,” Andrew Obenshain, MBA, the chief executive officer of bluebird bio, said in a statement. “We are further encouraged by the commitment to provide patient access across both commercial and government payers, most recently conveyed through multiple positive Medicaid decisions and the growing number of published coverage policies for Lyfgenia, and we expect approximately 85 patient starts across our portfolio this year.”
Actinium’s Antibody Radiation Conjugate Iomab-ACT Cleared for US Trial as Conditioning Therapy for SCD Allo-BMT
July 30, 2024 — Actinium’s Iomab-ACT, an investigational antibody radiation conjugate (ARC) intended to serve as an alternative to chemotherapy conditioning agents, has received clearance of an investigational new drug application from the FDA for trial evaluating it as a conditioning agent for patients planning to receive allogeneic bone marrow transplant (allo-BMT) for the treatment of SCD.
Unlike the nontargeted chemotherapy and total body irradiation currently used for SCD patients receiving allo-BMT, Iomab-ACT is directed at CD45, an antigen expressed on immune cells. Iomab-ACT is intended to broaden eligibility for patients with SCD to receive allo-BMT to some patients who cannot receive current chemotherapy conditioning. Pending positive results in the trial, which is to be carried out in a collaboration with the Columbia University, Actinium may later evaluate Iomab-ACT as an alternative to current chemotherapy conditioning for patients planning to receive either of the 2 FDA-approved gene therapy options for SCD: exa-cel and lovo-cel.
"Undergoing chemotherapy- or total body irradiation-based conditioning for curative allo-BMT or gene therapy often brings severe side effects for patients with SCD,” Markus Mapara, MD, PhD, a professor of medicine at Columbia University Irving Medical Center, and director of the Bone Marrow Transplantation and Cell Therapy Program at Vagelos College of Physicians and Surgeons, said in a statement. “These toxicities include organ damage, infections, infertility, and the potential for secondary malignancies. Leveraging extensive data from CD45 ARC conditioning in allo-BMTs, I am thrilled to lead this pioneering study using Iomab-ACT, a nonchemotherapeutic targeted radiotherapy conditioning, for patients with SCD. This innovative approach aims to minimize toxicity while ensuring complete donor hematopoiesis engraftment. Success in this trial could revolutionize treatment, enabling the use of a low-toxicity method for the engraftment of genetically engineered autologous stem cells in SCD patients."
