The gene and cell therapy field saw its first approved treatments in Europe in 2012 and the United States in 2017 and is projected to be at least a $10B USD industry by 2025. Despite this success, a massive gap exists between the companies, clinics, and researchers developing these therapeutic approaches, and their availability to the patients who need them. The unacceptable reality is a geographic exclusion of low-and middle-income countries (LMIC) in gene therapy development and ultimately the provision of gene therapies to patients in LMIC. This is particularly relevant for gene therapies to treat human immunodeficiency virus infection and hemoglobinopathies, global health crises impacting tens of millions of people primarily located in LMIC. Bridging this divide will require research, clinical and regulatory infrastructural development, capacity-building, training, an approval pathway and community adoption for success and sustainable affordability. In 2020, the Global Gene Therapy Initiative was formed to tackle the barriers to LMIC inclusion in gene therapy development. This working group includes diverse stakeholders from all sectors and has set a goal of introducing two gene therapy Phase I clinical trials in two LMIC, Uganda and India, by 2024. Here we report on progress to date for this initiative.
Non-viral genetic therapy strategies for the treatment of ataxia telangiectasia
James Grey received the ESGCT-sponsored prize for best poster presentation at the British Society for Gene and Cell Therapy (BSGCT) Annual Conference, which took place from 19-21 June 2019. Read about his winning presentation here!
Gene therapy for thrombotic thrombocytopaenic purpura
Robyn Bell received the ESGCT-sponsored prize for best oral presentation at the British Society for Gene and Cell Therapy (BSGCT) Annual Conference, which took place from 19-21 June 2019. Read about her winning presentation here!
CRISPR-Cas9 genome editing - molecular scissors a bit too sharp?
Grégoire Cullot received the ESGCT-sponsored prize for best oral presentation at the Société Française de Thérapie Cellulaire et Génique (SFTCG) Thematic Day on Viral Vectors, which took place on 17 May 2019 in Paris. Read about his winning presentation here!
Dgkk as a new therapeutic target in the Fmr1-KO murine model of Fragile X
Karima Habbas received the ESGCT-sponsored prize for best poster at the Société Française de Thérapie Cellulaire et Génique (SFTCG) Thematic Day on Viral Vectors, which took place on 17 May 2019 in Paris. Read about her winning presentation here!