FDA Approves A CRISPR-Based Therapy for Sickle Cell Disease

After CASGEVY (exagamglogene autotemcel) passed the U.S. Food and Drug Administrations (FDA) Biologics License Application (BLA) in June this year (2023), Vertex Pharmaceuticals and CRISPR Therapeutics announced its FDA approval on December 8. CASGEVY is the first FDA-approved treatment utilizing the novel genome-editing technology CRISPR, marking an innovative advance in gene therapy and offering a glimmer of hope for patients with severe sickle cell disease (SCD). Approximately 16,000 patients aged 12 years and older with recurrent Vaso-Occlusive Crisis (VOC) are now eligible to receive this innovative one-time treatment.

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The administration of CASGEVY requires specialized knowledge in stem cell transplantation. Vertex is actively collaborating with proficient hospitals to establish Authorized Treatment Centers (ATCs) throughout the United States. Notable institutions involved in this initiative include Boston Medical Center, Childrens National Hospital, and City of Hope Childrens Cancer Center, among others. These ATCs are set to play a pivotal role in facilitating the delivery of CASGEVY to eligible patients. The companies have outlined plans to introduce additional ATCs in the forthcoming weeks, expanding accessibility for patients in need.

CASGEVY, a genome-editing cell therapy, leverages CRISPR/Cas9 technology to edit autologous CD34+ hematopoietic stem cells. This targeted approach aims to diminish BCL11A expression, leading to an increased production of fetal hemoglobin (HbF). Through the reduction or elimination of vaso-occlusive crises, CASGEVY holds the promise of significantly enhancing the quality of life for individuals affected by sickle cell disease (SCD). The therapy has obtained conditional marketing authorization in the United Kingdom and Bahrain. Regulatory agencies in Europe and Saudi Arabia are actively reviewing CASGEVY, with ongoing assessments for its investigational use in treating transfusion-dependent thalassemia (TDT).

The approval marks a significant milestone, not just in the landscape of sickle cell disease (SCD) treatments but also in advancing the frontier of CRISPR-based therapies. The success of CASGEVY represents a noteworthy leap forward in the realm of genetic medicine, offering hope and potential therapeutic avenues for those who require them.

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