Global Speakers Address Developing Gene Therapies in Low- and Middle-Income Countries

In the first Annual Meeting session planned by ASGCT’s Global Outreach Committee, speakers from three countries with middle-income economies discussed solutions to improve access to gene therapies in low- and middle-income countries (LMICs). According to Rahul Purwar, Ph.D., associate professor at the Indian Institute of Technology Bombay, challenges include the high level of technical and clinical training needed to provide the complex processes involved in gene therapies. Additional barriers are high manufacturing costs and infrastructural limitations in building facilities with the necessary GMP and quality control requirements. Another component required to conduct clinical trials is a robust and comprehensive regulatory framework for gene therapy, which India has developed, enabling the initiation of clinical trials for CAR T-cell therapy there.

Brazil also has a regulatory framework for advanced cell therapy, tissue engineering, and gene therapy products, according to Renata Parca, MS, from ANVISA, the Brazilian regulatory agency. Parca noted that the complexity of these products requires innovative regulatory approaches to assessing and controlling risks. She indicated ANVISA’s development and implementation of the Brazilian regulatory framework relied on the expertise of local scientists and international models of evaluation. The Agency’s strategy was to utilize reliance on, and convergence between, other national regulatory authorities, as well as following ICH guidelines. She indicated that marketing authorization in Brazil follows one of three paths—standard approval, priority approval, and conditional approval, which is used only for severe debilitating conditions. Use of real-world data and long-term follow-up are both common for advanced therapies, especially for gene therapies. The two approvals of gene therapies in Brazil last year (for Luxturna and Zolgensma) has attracted more companies to the region.

Connecting commercial developers to local investigators focused on starting trials is an important component to increasing indigenous development of gene therapies in LMICs, according to Johnny Mahlangu, M.B.Ch.B., M.Med., personal professor in haematology and head of the school of pathology in the faculty of health sciences of the University of Witwatersrand. Mahlangu indicated another solution, which addresses infrastructural and financial limitations in the context of hemophilia, is a “hub and spoke approach” in which a single infusion center develops the infrastructure to prepare cells and/or vectors, and then sends the final product to regional hemophilia treatment centers for infusion and follow-up. In Africa, the hub could serve several countries. All speakers agreed that educational opportunities are also beneficial, especially hands-on training for researchers and clinicians.

ASGCT’s Global Outreach Committee appreciated the opportunity to bring together these speakers from middle-income countries because several diseases that would largely benefit from gene therapy development and application are more prevalent in these countries. An excellent example of this disparity is sickle cell disease, with most of the development for gene therapies being held in North America although countries such as India and those of sub-Saharan Africa pose millions of unattended patients. A similar landscape can be found for cancer incidence and access to cutting-edge treatment in the next years, including gene therapy-based strategies. Scientific societies such as ASGCT can contribute to continued dialog on these challenges.

Martín Bonamino, Ph.D., is a researcher at the Brazilian National Cancer Institute (INCA) and a member of ASGCT's Global Outreach Committee.

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