Julie Kanter, MD, on Highest Priorities for Sickle Cell After Gene Therapy Approvals

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The director of the Adult Sickle Cell Clinic and associate professor at University of Alabama Birmingham pointed out access to a sickle cell specialist as one such priority.

“I think it's most important to recognize that this will change the field slowly. So, we need to keep doing everything we're doing now, for individuals with sickle cell disease, recognizing they face horrible stigma and health disparities. And we need to continue to address those and ensure not just that people have access to cell and gene therapy, but that they have access to a sickle cell specialist... I think what's most important right now is that we get every individual living with SCD to a sickle cell specialist to be evaluated for what types of all kinds of therapies that might help them.”

The FDA approved the first gene therapies for treating sickle cell disease (SCD) in December 2023: bluebird bio’s lovotibeglogene autotemcel (lovo-cel), marketed as Lyfgenia, and Vertex and CRISPR Pharmaceuticals' exagamglogene autotemcel (exa-cel; Casgevy) in patients aged 12 years and older with SCD.

Lovo-cel consists of autologous CD34+ hematopoietic stem cells collected by plerixafor mobilization and apheresis, transduced with BB305 lentiviral vector (LVV) encoding the human beta-A-T87Q globin gene.

CGTLive spoke with Julie Kanter, MD, director, Adult Sickle Cell Clinic and associate professor, hematology and oncology, University of Alabama Birmingham, who served as investigator on multiple of lovo-cel's clinical trials, including the HGB-206 (NCT02140554) and HGB-210 (NCT04293185) trials, to learn more about what she considers the highest priorities in the field after the gene therapy approvals. She stressed that it will be important to continue to watch long-term follow-up data on lovo-cel and exa-cel after approval, with special attention paid to exa-cel, as the first approved CRISPR-edited gene therapy.

REFERENCE
FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease. News release. FDA. December 8, 2023. Accessed December 8, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease
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