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BALTIMORE — Gene and cell therapies have transformed a handful of devastating disorders in the U.S. and Europe, with more treatments on the way. But those technologies have largely failed to reach most of the world.

These lags are ubiquitous in the current drug development system, where new therapies are approved in wealthy countries and then slowly leak out to low- and middle-income countries, many years later. In the case of gene therapy, that means countries most affected by a disease such as sickle cell may wait decades to access a curative therapy.

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For the last few years, a handful of researchers in the U.S. and around the world have tried to find ways of ensuring global access to these technologies. In 2020, they formed the Global Gene Therapy Initiative, with an initial goal of setting up a gene therapy clinical trial for a hemoglobinopathy — such as a disease like sickle cell or beta thalassemia — or HIV in two different low- and middle-income countries by 2024.

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