Vertex Mempersembahkan Data Jangka Panjang Positif Mengenai CASGEVY™ (exagamglogene autotemcel) di Pertemuan dan Pameran Tahunan American Society of Hematology (ASH) dan Memberikan Pembaruan Program

of CASGEVY in patients with TDT and SCD, respectively. The CLIMB-131 trial is also assessing the safety and efficacy of CASGEVY in patients with TDT. These trials have shown promising results in reducing or eliminating the need for transfusions in TDT patients and reducing vaso-occlusive crises in SCD patients.

The safety profile of CASGEVY remains consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant. Patients in the trials have demonstrated stable levels of fetal hemoglobin and allelic editing across all ages and genotypes.

Vertex is committed to bringing CASGEVY to patients around the world and has already received approvals in multiple countries. Vertex is working with reimbursement authorities to ensure sustainable access for patients and has agreements in place in various countries. Vertex has also secured a third manufacturing facility for CASGEVY to meet the growing demand for the therapy.

Overall, the data presented at the ASH Annual Meeting continue to demonstrate the transformative and durable benefits of CASGEVY for patients with SCD and TDT. Vertex is dedicated to advancing access to this groundbreaking therapy and improving the lives of patients living with these debilitating diseases.

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