Work

A new frontier: FDA approvals for gene therapy in sickle cell disease

Year: 2024

Type: article

Abstract: On December 8, 2023, the US Food and Drug Administration (FDA) approved two autologous gene therapy products, Lyfgenia (lovotibeglogene autotemcel by bluebird bio) and Casgevy (exagamglogene autotemcel by Vertex Pharmaceuticals), for the treatment of individuals with sickle cell disease (SCD) ages 12 years and older with recurrent vaso-occlusive events (VOEs). 1 FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease [press Release]2023 Google Scholar Lyfgenia utilizes a lentiviral vector (LVV)-based strategy to transduce autologous hematopoietic stem cells (HSCs) with a LVV encoding βA-T87Q, an anti-sickling globin. Casgevy, the first ever FDA-approved clustered regularly interspaced short palindromic repeats (CRISPR)-based gene therapy product, utilizes CRISPR-Cas9 editing at the erythroid-specific enhancer of the BCL11A gene to enhance fetal hemoglobin (HbF) production. These approvals are the culmination of decades of basic science research, 5–10 years of clinical trial experience, and the brave, altruistic decision by over 50 individuals with SCD to advance science for the 100,000 Americans living with SCD and the millions more worldwide.

Source: Molecular Therapy

DOI: 10.1016/j.ymthe.2024.01.015

Authors: Alexis Leonard, John F. Tisdale

Institutions: St. Jude Children's Research Hospital, National Institutes of Health, National Institute of Diabetes and Digestive and Kidney Diseases, National Heart, Lung, and Blood Institute

Language: English

Topic: Hemoglobinopathies and Related Disorders

Subfield: Genetics

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: hybrid

Estimated APC paid: $3,900