The United States Patent and Trademark Office (USPTO) has accepted a landmark patent from Dei BioPharma Ltd., a Ugandan biotechnology company, for a novel gene therapy platform targeting sickle cell disease—an advance that could dramatically lower the cost of treatment and expand access across low- and middle-income countries.
The patent, accepted on January 26, 2026, was announced by the company over the weekend. Dei BioPharma is led by Ugandan scientist Dr. Matthias Magoola, who founded the firm and invented the technology.
Sickle cell disease is a hereditary blood disorder that causes red blood cells to become rigid and crescent-shaped, leading to chronic pain, infections, organ damage and shortened life expectancy. Although recent gene therapies have demonstrated curative potential, their high costs—often running into millions of dollars per patient—have made them largely inaccessible, particularly in sub-Saharan Africa, where the disease burden is highest.
Dei BioPharma’s innovation takes a different approach. Instead of correcting the defective gene responsible for sickle cell disease in individual patients, the company’s scientists developed a method to sustain the production of fetal hemoglobin—a form of hemoglobin naturally produced during pregnancy and early infancy that does not cause red blood cells to sickle.
In most people, fetal hemoglobin production stops around six months after birth as the body transitions to adult hemoglobin. For individuals with sickle cell disease, symptoms typically begin after this switch. Using CRISPR-based gene-editing technology, Dei BioPharma’s platform targets a genetic control mechanism responsible for shutting down fetal hemoglobin production. By disabling this switch, the therapy allows the body to continue producing fetal hemoglobin, preventing the red blood cells from deforming.
Crucially, the genetic switch is the same in all humans, meaning the treatment can be standardized rather than tailored to each patient—a key factor in reducing costs.
“This invention was designed from the outset to address not only the biology of sickle cell disease, but also the issue of access,” Dr. Magoola said. “By focusing on a universal genetic switch instead of the sickle mutation itself, we can develop a single treatment applicable to all patients.”
Current curative options, such as bone marrow transplants or personalized gene therapies, often require complex laboratory procedures, highly specialized facilities and extensive customization. Dei BioPharma says its approach eliminates many of these barriers, enabling large-scale manufacturing, storage and distribution across diverse health systems.
The company estimates that its platform could reduce the cost of gene therapy by more than 95 percent, potentially bringing curative treatment within reach of public health systems in Africa, the Middle East and parts of Asia.
According to Dei BioPharma, the therapy could be effective across all major forms of sickle cell disease, including HbSS, HbSC and sickle beta-thalassemia, making it one of the first gene therapies designed for broad, scalable use against a single-gene disorder.
Globally, an estimated 20 million people live with sickle cell disease, the majority of them in sub-Saharan Africa. The company compares its model to that of generic medicines, where standardized production significantly lowers costs and improves access.
The newly accepted patent covers the gene-editing tools, delivery systems and therapeutic processes required to sustain fetal hemoglobin production. Dei BioPharma is currently conducting preclinical studies to evaluate safety and effectiveness ahead of planned human trials.
Dr. Magoola said the breakthrough reflects a broader mission to ensure advanced medical innovations benefit populations that have historically been left behind.
“Our goal has always been to ensure that cutting-edge biological medicines are not limited to a small fraction of the world’s population, but are accessible to the more than 90 percent who currently cannot afford them,” he said.
