Accelerating Global Access to Gene Therapies: Case Studies from Low- and Middle-Income Countries

Gene therapy is at the forefront of modern medicine. By making precise changes to the human genome, these sophisticated technologies can potentially lead to one-time lifelong cures. As of mid-2022, more than 2,000 gene therapies were in development worldwide, contributing to a global market value that is expected to reach nearly $20 billion by 2027.

Researchers are applying gene therapies to infectious and non-communicable diseases (e.g. HIV, sickle cell disease) that affect tens of millions of people around the globe, most of whom live in low- and middle-income countries (LMICs). Without concerted efforts to build gene therapy capacity in low-resource settings, these transformative treatments are likely to remain out of reach for communities carrying the highest disease burdens, further widening the global health divide.

This white paper developed in collaboration with Thunderbird School of Global Management and Sandra Day O’Connor College of Law at Arizona State University, provides an overview of what is required to sustainably deliver gene therapies in LMICs, from preliminary R&D through clinical trials all the way to market access. Expert insight was contributed by clinical leaders across LMICs who are actively engaged in gene therapy research, offering case studies comparing struggles and successes across multiple political economies. As part of a growing movement seeking to disrupt global health paradigms, this publication aims to motivate and guide action by policy-makers, health-systems leaders, pharmaceutical companies, funders and other stakeholders.