Julia Buntaine Hoel, “Thoughts 23” (2016–2017), digital print on aluminum, 20 x 16 inches. Neuron data acquired from neuroimaging software developed by EyeWire.

Gene Therapy for All


Making Gene Therapy Accessible
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In “Making Gene Therapy Accessible” (Issues, Fall 2022), Kevin Doxzen and Amy Lockwood highlight contentious issues around gene therapy, even as the treatment shows good results and has raised hopes for many people with incurable diseases. The authors rightly point out that unless leaders carve out appropriate policies to develop gene therapy through a collaborative process, this novel therapy will be accessed by less than 1% of the world’s population.

Treatment that is unavailable to patients in need has no value at all. Policies will need to focus on research promotion, clinical and regulatory infrastructural development, capacity-building, training, and development of an approval pathway and community adoption for success and sustainable affordability. And as the authors suggest, rather than concentrating on single diseases, efforts should be focused on establishing a platform that would be applicable to multiple diseases. This approach could help researchers working to develop therapies not only for infectious diseases such as HIV and hemoglobinopathies such as sickle cell disease and thalassemia, but also for “rare” diseases that may in fact be common in low- and middle-income countries (LMICs).

In our opinion, one of the biggest roadblocks in this regard is intellectual property rights. The successful application of patent pools in the development of antiretroviral drugs in LMICs provides a tried and tested strategy for bringing down the cost of gene therapy by sharing these rights. Moreover, lack of competition affects the cost of gene therapies, as only a very small number of companies are developing such therapies. Bringing in more players may bring down the costs markedly.

Treatment that is unavailable to patients in need has no value at all. 

Apart from encouraging research and development, the authors also rightly underline the significance of regulatory guidelines and laws to ensure execution of safe and ethical research. To begin, global clinical trials need to be encouraged and facilitated with the participation of various patient populations from countries associated with high disease burdens. There needs to be proper guidance documents for development of indigenous platforms—utilizing the current capabilities and intellectual property of researchers and clinicians of various countries—to establish self-reliant assets for LMICs. This is also necessary for local gene therapy methods and products developed through technology transfer. To encourage the best practices globally, there should be twinning programs to provide appropriate hands-on training on the platforms established elsewhere and to generate a well-trained workforce for these resource-intensive and innovative technologies. Data sharing across the globe for drafting evidence-based recommendations for treating diseases with these modalities should also be encouraged so that stakeholders may learn from each other’s experiences.

Also important are organizations such as the Global Gene Therapy Initiative, which, as the authors highlight, play a pivotal role in the development of gene therapies in LMICs. With the participation of multidisciplinary experts, such initiatives can go a long way toward preparing LMICs to maximize the impact of gene therapy.

Finally, policymakers and other authorities in government need to develop funding mechanisms and policies to prioritize long-term success and stronger health systems with respect to gene therapy, realizing the transformative potential of these technologies in improving millions of lives. Also, as emphasized above, regulatory convergence needs to be aimed for, to solve the existing bottlenecks and build the ecosystem for gene therapy methods and products in LMICs.

Indian Council of Medical Research Headquarters

New Delhi, India

Development, acceptance, and sustainability of successful health interventions require both good planning and sound policies, as well as partnerships among many stakeholders. This is particularly the case when dealing with complex and sensitive interventions such as the introduction of and equitable access to gene therapy in low- and middle-income countries (LMICs), as Kevin Doxzen and Amy Lockwood highlight. The authors point out the familiar long delays between the time that new interventions become routine in high-income countries and their accessibility in LMICs. This must change.

To accelerate this change, Doxzen and Lockwood advocate for intersectoral, cross-cutting programs rather than single-disease vertical programs. Besides the examples the authors cite, this approach has proven to be very effective by other programs such as the European and Developing Countries Clinical Trials Partnership (EDCTP) and World Health Organization-TDR, which support interventions against diseases of the poor, especially in LMICs, and have broader mandates that include capacity development, networking, and fostering co-ownership of their programs.

Capacity development, including building environments for conducting quality health research and health service delivery, has far-reaching outcomes beyond the intended primary focus, as the authors cite in the case of the President’s Emergency Plan for AIDS Relief, or PEPFAR, and its contribution to the COVID-19 response. The same can be said about the EDCTP and the World Health Organization-TDR programs, which have shown that it is most cost-effective to support cross-cutting issues that can be used generally in different settings.

Capacity development, including building environments for conducting quality health research and health service delivery, has far-reaching outcomes beyond the intended primary focus.

However, as Doxzen and Lockwood point out, for ambitious programs such as equitable global accessibility of gene therapy, it is paramount to have in place good policies, sound strategic delivery plans, and coordination of activities. The strategy should consider inputs from all major stakeholders, including health authorities, regulatory agencies, civil society, international health organizations, the scientific community, development partners, industry, and the affected communities.

Of particular importance should be the involvement of health authorities in LMICs right from the start to inculcate a sense of co-ownership of the program. This will foster acceptance, active participation, self-determination, and program sustainability. Failure to do so may lead to public resistance, as evidenced in, for example, the vaccination campaign to irradicate polio in Africa and vaccinations efforts against COVID-19 and Ebola. Polio vaccination programs were falsely accused of imposing birth control in Nigeria, and COVID-19 immunization programs using mRNA vaccines were widely associated with negative misinformation about interference with human genes. Such involvement is particularly important in dealing with a sensitive issue such as gene therapy, which is prone to misinterpretation and misinformation. The involvement of local authorities and communities will also point out areas of weakness and capacity that need strengthening, including regulatory, laboratory, and clinical services.

Since it requires many years for such services to be readily available and accessible, this planning should take place now. There is no time to waste.

Retired Vice Chancellor

Hubert Kairuki Memorial University

Dar es Salaam, Tanzania

Cite this Article

“Gene Therapy for All.” Issues in Science and Technology 39, no. 2 (Winter 2023).

Vol. XXXIX, No. 2, Winter 2023