New Sickle Cell Cure from Ugandan Biotech Could Reach Millions

Updated by Faith Barbara N Ruhinda at 1712 EAT on Wednesday 4 February 2026

KAMPALA — Over the weekend, news emerged that could transform the future of one of the world’s most neglected diseases.

The United States Patent and Trademark Office (USPTO) has granted a groundbreaking patent to Dei BioPharma Ltd, a Ugandan biotechnology company led by scientist Dr. Matthias Magoola. The patent, approved on January 26, 2026, represents a radically different approach to treating sickle cell disease — one designed not only to cure the condition but also to make that cure affordable and accessible to the millions who need it most.

Sickle cell disease, an inherited blood disorder, causes red blood cells to become rigid and crescent-shaped. These malformed cells block blood flow, triggering severe pain, infections, organ damage, and often shortened lifespans. The condition affects an estimated 20 million people worldwide, with the majority living in low- and middle-income countries, particularly in sub-Saharan Africa.

While recent advances in gene therapy have demonstrated that sickle cell disease can be cured, these treatments are prohibitively expensive, often costing millions of dollars per patient. They also require highly specialised laboratories, personalised genetic engineering, and advanced hospital infrastructure, placing them out of reach for most families and public health systems.

Dr. Magoola said his team deliberately designed the new therapy to address both the biological and accessibility challenges.
“This invention was designed from the beginning to solve not only the biology of sickle cell disease, but also the access problem,” he said.

If successful, the innovation could mark a turning point in global efforts to tackle sickle cell disease, offering hope to millions who have long lacked effective, affordable treatment.

The science behind the breakthrough is rooted in a trait all humans share. Before birth — and for several months afterward — babies produce a form of oxygen-carrying protein called fetal haemoglobin.

Unlike adult haemoglobin, fetal haemoglobin does not cause red blood cells to sickle. Symptoms of sickle cell disease only appear when the body naturally switches to adult haemoglobin, usually around six months of age.

Rather than repairing the defective gene responsible for sickle cell disease in each individual patient, Dei BioPharma’s approach aims to prevent that switch from ever occurring.

Using CRISPR gene-editing technology, the company targets what Dr. Matthias Magoola describes as a universal genetic “control switch” — a segment of DNA that tells the body when to stop producing fetal haemoglobin and start making adult haemoglobin. By disabling this switch, the body continues producing fetal haemoglobin, preventing red blood cells from becoming rigid and misshapen. Crucially, this control switch is the same in all humans.

“By targeting a universal genetic switch rather than the sickle mutation itself, we can develop a single, standardised treatment that works for all patients,” Dr. Magoola explained.

The distinction is significant. Most existing gene therapies must be customised for each patient, driving up cost and complexity. Dei BioPharma’s platform, by contrast, could be produced at scale — manufactured, stored, distributed, and administered in the same way across different populations and health systems.

Dei BioPharma estimates its approach could cut the cost of gene therapy for sickle cell disease by more than 95 per cent.

If confirmed to be safe and effective, the therapy could make curative treatment accessible to public health systems across Africa, the Middle East, and parts of Asia — where the disease is most common.
The treatment is designed to work across all major forms of sickle cell disease, including HbSS, HbSC, and sickle beta-thalassemia.

“This could become the first scalable, widely applicable gene therapy for a single-gene disease,” said Dr. Matthias Magoola, the company’s lead scientist.

Dei BioPharma likens its model to that of generic medicines: standardised products that lower costs and broaden access once regulatory approvals are obtained. It is a deliberate shift away from pharmaceutical industry practices that have often focused on high-income markets.

“Sickle cell disease affects populations that have historically been last to benefit from medical advances,” Dr. Magoola said. “Our goal is to change that by making advanced gene therapies manufacturable and affordable on a global scale.”

Source: Observer

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