Alexis Kuhn, PharmD, BCOP, on Hemoglobinopathy Gene Therapies From a Pharmacist’s Perspective


The pediatric oncology pharmacist at Mayo Clinic discussed the incorporation of the recently FDA-approved gene therapies for SCD and TDT into the work of pharmacists.

“These therapies are obviously very new, very exciting, and very complicated. As pharmacists, there are a lot of areas that we do need to pay attention to and be aware of the nuances [of] not only for the individual agents, but for all of the concomitant therapies that our patients may be on prior to receiving these therapies.”

Over just the past few months, the clinical community has seen multiple FDA approvals of gene therapy products for hemoglobinopathies. In December 2023, Vertex Pharmaceuticals' and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel, marketed as Casgevy) and bluebird bio’s lovotibeglogene autotemcel (lovo-cel, marketed as Lyfgenia) were both approved for sickle cell disease indications.1 Just over a month later, in January 2024, the FDA approved exa-cel for an additional indication in patients with transfusion-dependent beta thalassemia (TDT).2 These new products, the first gene therapies to be approved for hemoglobinopathies, are rapidly changing the treatment landscape for patients with SCD and TDT. As such, it is critically important for doctors and clinical pharmacists to stay up to date with the latest information regarding these therapies in order to prescribe them safely.

At the 2024 Tandem Meetings |Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, held in San Antonio, Texas, February 21-24, 2024, Alexis Kuhn, PharmD, BCOP, a pediatric oncology pharmacist at Mayo Clinic, gave a talk focused on considering these new gene therapies from a pharmacist’s perspective.3 Shortly after the talk, CGTLive® spoke with Kuhn to learn more. She emphasized that because these gene therapies are given to patients in a multistep process that involves apheresis, preconditioning, and administration of the gene therapy itself, providers need to be mindful of other medications and therapies the patient may be on during each step of the process, determine whether a hold is necessary, and continuously monitor patients closely. Kuhn also encouraged doctors to work with their clinical pharmacy support if they have it, and to integrate this support early and often into their treatment plans for patients.

Click here for more coverage of Tandem 2024.

1. FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease. News release. FDA. December 7, 2023. Accessed February 23, 2024.
2. Vertex Announces US FDA Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Transfusion-Dependent Beta Thalassemia. News release. January 16, 2024. Accessed February 23, 2024.
3. Kuhn A. Geneie in a bottle: Gene therapy for hemoglobinopathies. Presented at: 2024 Tandem Meetings, February 21-24, San Antonio, Texas.

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