Global Gene Therapy Initiative Aims to Bring Gene Therapy Trials to India and Uganda

Jennifer Adair, PhD, and Lindsay Androski, JD, MBA - September 15, 2021

The Global Gene Therapy Initiative (GGTI) recently published its first report in Gene Therapy, which highlights the need for cost-effective gene therapy development strategies in low-and-middle-income countries.

The cell and gene therapy field is burgeoning with technology breakthroughs, and new research has enabled the development of potentially curative therapies for debilitating genetic and malignant conditions. Despite this, low-and-middle-income countries (LMICs) are increasingly excluded from realizing the benefits of gene therapies due to the high cost and sophisticated infrastructure required for their development and manufacture.

The Global Gene Therapy Initiative (GGTI) was formed in 2020 to stimulate greater inclusion of LMICs in gene therapy research, manufacturing improvements, and infrastructure development and to open sustainable pathways to global gene therapy access. The working group recently published its first report in Gene Therapy, which highlights progress towards its goal of introducing two gene therapy phase I clinical trials in Uganda and India by 2024.

Key insights from the report include the immediate need to develop regionally appropriate, cost-effective strategies for developing gene therapies, particularly for human immunodeficiency virus (HIV) and hemoglobinopathies that disproportionately burden patients in LMICs. Through continued work with clinical partners in India and Uganda, the GGTI aims to address key cost and access challenges, including clinical readiness, technology development, regulation, and community outreach. The GGTI also aims to conduct economic studies to support the business case for introducing gene therapies in LMICs as an effective population health strategy.

The GGTI’s efforts represent a critical first step towards equitable and sustained gene therapy access. Though great challenges remain for researchers, clinicians, and communities, the group’s work serves as a valuable mechanism to empower LMICs and ensure all vital stakeholders are included in the gene therapy development ecosystem.

Read the full article in Gene Therapy here.

Dr. Adair is a former member of ASGCT's Board of Directors. She is also the Fleischauer Family Endowed Chair in Gene Therapy and an associate professor at Fred Hutchinson Cancer Research Center. Ms. Androski is president and CEO of Roivant Social Ventures.

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