Julie Kanter, MD, on Developing Recommendations for SCD Post Gene Therapy

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The director of the Adult Sickle Cell Clinic and associate professor at University of Alabama Birmingham discussed work the National Alliance of Sickle Cell Centers is conducting.

“We are developing some consensus recommendations on what pre and post therapy should look like for individuals with sickle cell disease, recognizing that these are not the same individuals that some transplant centers have taken care of, for, let's say, myeloma or some other autologous process, and that they need to be managed differently, and with particular caution to certain areas, including pain management, anxiety, comorbid conditions, and then the risk of seizure or other problems. So, I think it's just going to be really important that these individuals are really well managed."

In December 2023, the FDA approved bluebird bio’s lovotibeglogene autotemcel (lovo-cel), marketed as Lyfgenia, as a treatment for sickle cell disease (SCD) in patients aged 12 years and older, alongside Vertex and CRISPR Pharmaceuticals' exagamglogene autotemcel (exa-cel; Casgevy) for the same indication.

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 recently 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. Kanter also serves as president of the National Alliance of Sickle Cell Centers (NASCC). She shared some work the NASCC is conducting, including developing consensus recommendations for SCD gene therapy centers on caring for patients after receiving gene therapies. The recommendations also include utilizing hub and spoke models of care centers and follow-up.

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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