lovotibeglogene autotemcel (Lyfgenia)
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Indications
- gene-editing for treatment of patients with sickle cell disease >= 12 years
Adverse effects
- anemia, leukopenia, thromocytopenia, febrile neutropenia
- stomatitis
- hematologic malignancy
Procedure
- myeloablative conditioning, bone marrow ablation (high-dose chemotherapy)
- single infusion of modified hematopoietic stem cells
Mechanism of action
- uses a lentiviral vector as a gene delivery vehicle
- hematopoietic stem cells are genetically modified to produce HbA[T87Q] that functions similarly to hemoglobin A
- modified hematopoietic stem cells are transplanted back into the patient
- the stem cells engraft within the bone marrow & produce HbA[T87Q] rather than hemoglobin S
More general terms
References
- ↑ AMA Morning Rounds. Dec 11, 2023 American Medical Association
Lou N Milestone Gene Therapies for Sickle Cell Disease Greenlit by FDA. Casgevy and Lyfgenia approved for one-time treatment of sickle cell disease. MedPage Today December 8, 2023 https://www.medpagetoday.com/hematologyoncology/hematology/107751
Brooks PJ, Clay JP A Closer Look at the Approval of CRISPR/Cas9 Gene Therapy for Sickle Cell Disease. This landmark event brings up broader considerations and implications for other diseases. MedPage Today. Dec 11, 2023 https://www.medpagetoday.com/opinion/second-opinions/107793
FDA News Release, Dec 8, 2023 FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease