Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)

Area of Science:

• Biomedical Science
• Global Health
• Therapeutic Development

Background:

• Gene and cell therapies represent a rapidly growing sector, projected to reach $10B USD by 2025.
• A significant disparity exists in the accessibility of these advanced treatments, particularly excluding low- and middle-income countries (LMIC).
• This exclusion is critical for global health issues like human immunodeficiency virus (HIV) infection and hemoglobinopathies, which disproportionately affect populations in LMIC.

Purpose of the Study:

• To address the geographic exclusion of LMIC in gene therapy development and patient access.
• To establish the Global Gene Therapy Initiative (GGTI) to overcome barriers to LMIC inclusion.
• To report on the progress of the GGTI's goal of initiating two Phase I clinical trials in LMIC by 2024.

Main Methods:

• Formation of the Global Gene Therapy Initiative (GGTI) in 2020, comprising diverse stakeholders.
• Development of strategies to address infrastructural, regulatory, and capacity-building needs in LMIC.
• Planning and preparation for the introduction of Phase I clinical trials in Uganda and India.

Main Results:

• The GGTI has made progress towards its goal of initiating gene therapy clinical trials in LMIC.
• Key stakeholders from various sectors have been engaged to foster collaboration.
• Specific focus on advancing gene therapies for HIV and hemoglobinopathies in target LMIC.

Conclusions:

• Bridging the gap in gene therapy access requires comprehensive development of research, clinical, and regulatory infrastructure.
• Capacity-building, training, and community adoption are essential for sustainable gene therapy provision in LMIC.
• The GGTI is actively working to facilitate the inclusion of LMIC in gene therapy development and clinical application.