A systematic review of clinical trials for gene therapies for ,B-hemoglobinopathy around the world

Author(s):Show less -

Ros, Felipe Augusto ; Couto, Samuel Campanelli Freitas ; Milhomens, Jonathan ; Ovider, Ian ; Maio, Karina Tozatto ; Jennifer, Viviane ; Ramos, Rodrigo Nalio ; Picanco-Castro, Virginia ; Kashima, Simone ; Calado, Rodrigo T. ; Barros, Luciana Rodrigues Carvalho ; Rocha, Vanderson

• Document type: Journal article
• Source: CYTOTHERAPY; v. 25, n. 12, p. 7-pg., 2023-11-17.

Abstract

Background aims: Amidst the success of cell therapy for the treatment of onco-hematological diseases, the first recently Food and Drug Administration-approved gene therapy product for patients with transfusion-dependent ,B-thalassemia (TDT) indicates the feasibility of gene therapy as curative for genetic hematologic disorders. This work analyzed the current-world scenario of clinical trials involving gene therapy for ,B-hemoglobinopathies.Methods: Eighteen trials for patients with sickle cell disease (SCD) and 24 for patients with TDT were analyzed.Results: Most are phase 1 and 2 trials, funded by the industry and are currently recruiting volunteers. Treatment strategies for both diseases are fetal hemoglobin induction (52.4%); addition of wild-type or therapeutic ,B-globin gene (38.1%) and correction of mutations (9,5%). Gene editing (52.4%) and gene addition (40.5%) are the two most used techniques. The United States and France are the countries with the greatest number of clinical trials centers for SCD, with 83.1% and 4.2%, respectively. The United States (41.1%), China (26%) and Italy (6.8%) lead TDT trials centers.Conclusions: Geographic trial concentration indicates the high costs of this technology, logistical issues and social challenges that need to be overcome for gene therapy to reach low-and middle-income countries where SCD and TDT are prevalent and where they most impact the patient's health.(c) 2023 International Society for Cell & Gene Therapy. Published by Elsevier Inc. All rights reserved. (AU)

• FAPESP's process: 13/08135-2 - CTC - Center for Cell-Based Therapy
• Grantee: Dimas Tadeu Covas
• Support Opportunities: Research Grants - Research, Innovation and Dissemination Centers - RIDC
• FAPESP's process: 20/07055-9 - Nucleus of Cellular Therapy - NuTeC
• Grantee: Rodrigo do Tocantins Calado de Saloma Rodrigues
• Support Opportunities: Research Grants - Problem-Oriented Research Centers in São Paulo
• FAPESP's process: 22/07503-7 - Fetal hemoglobin induction as a treatment strategy for sickle cell anemia using gene therapy
• Grantee: Felipe Augusto Rós
• Support Opportunities: Scholarships in Brazil - Doctorate