Ugandan Biotech Breakthrough Promises Affordable, Scalable Gene Therapy for Sickle Cell Disease

Uganda’s biotechnology firm, Dei BioPharma Ltd, has unveiled a major scientific breakthrough that could transform the treatment of sickle cell disease globally, particularly in regions where the burden of the disease is highest.

On January 26, 2026, the United States Patent and Trademark Office (USPTO) accepted a groundbreaking patent developed by Dei BioPharma for an advanced gene therapy platform targeting sickle cell disease. The innovation was invented by the company’s founder and Managing Director, Dr. Matthias Magoola, a Ugandan scientist whose work is increasingly gaining global recognition.

Dei BioPharma announced the development over the weekend, describing it as a potentially game-changing approach that addresses not only the biology of sickle cell disease but also the long-standing challenge of access to curative treatments. Sickle cell disease is an inherited blood disorder in which red blood cells become rigid and misshapen, leading to severe pain, frequent infections, organ damage, and reduced life expectancy. An estimated 20 million people worldwide live with the condition, the vast majority of them in sub-Saharan Africa.

Although recent advances in gene therapy have demonstrated that sickle cell disease can be cured, existing treatments are extremely expensive—often costing millions of dollars per patient. These therapies typically rely on personalised genetic modification, complex laboratory processes, donor matching, and highly specialised medical facilities, placing them far beyond the reach of most patients in low- and middle-income countries.

Dei BioPharma’s innovation takes a fundamentally different approach. Rather than attempting to repair the defective sickle cell gene in each individual patient, the company has developed a gene-editing platform that keeps the body producing fetal haemoglobin—the form of haemoglobin naturally present during pregnancy and early infancy.

All humans are born producing fetal haemoglobin, which does not cause red blood cells to sickle. Normally, the body switches from fetal to adult haemoglobin about six months after birth. In people with sickle cell disease, symptoms begin when this switch introduces the faulty adult haemoglobin that drives the condition.

Using CRISPR gene-editing technology, Dei BioPharma’s platform targets a universal genetic “control switch” responsible for this transition. By disabling the switch, fetal haemoglobin production continues throughout life, preventing red blood cells from becoming rigid and distorted.

Because this genetic switch is shared by all humans, the treatment can be standardised rather than customised for individual patients. This allows the same gene-editing product to be manufactured at scale, stored, distributed, and administered across different populations and health systems.

“This invention was designed from the beginning to solve not only the biology of sickle cell disease, but also the access problem,” Dr. Magoola said. “By targeting a universal genetic switch rather than the sickle mutation itself, we can develop a single, standardised treatment that works for all patients.”

The company estimates that its approach could reduce the cost of gene therapy for sickle cell disease by more than 95 percent, potentially making curative treatment affordable for public health systems in Africa, the Middle East, and parts of Asia. The therapy could be applied across all major forms of the disease, including HbSS, HbSC, and sickle beta-thalassemia.

Dei BioPharma describes the platform as a new model for gene therapy, inspired by the principles behind generic medicines—standardised products that enable scale, lower costs, and wider access once regulatory approvals are obtained.

“This opens the door to what could become the first scalable, broadly applicable gene therapy for a single-gene disease,” Dr. Magoola said. “Sickle cell disease disproportionately affects populations that have historically been last to benefit from medical innovation. Our objective is to reverse that pattern.”

The patent covers the gene-editing tools, delivery methods, and therapeutic processes required to activate and sustain fetal haemoglobin production. Dei BioPharma is currently conducting preclinical studies to assess safety, durability, and effectiveness, with plans to progress to human clinical trials. The company says it will work closely with regulators, research institutions, and strategic partners as development advances.

For Dr. Magoola, the breakthrough reflects a broader mission to democratise access to cutting-edge biological medicines.

“Our commitment has always been to make advanced biological drugs accessible to the more than 90 percent of people who currently cannot afford them,” he said. “This innovation brings that goal closer to reality.”

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