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UG Standard - Latest News

Dr. Magoola unveils gene therapy breakthrough to transform sickle cell treatment

The platform targets universal genetic switch to enable scalable gene therapy

by Julian Ninsiima | Reporter
02/02/2026
in Business, News
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Dr. Magoola, who is leading Dei BioPharma to develop a manufacturing facility with atleast 26 different units, says the biotech firm will boost Africa’s quest for self sufficiency.

KAMPALA, UGANDA – January 30, 2026 – The United States Patent & Trademark Office (UPTO) has on January 26, 2026 accepted the Dei BioPharma Ltd advanced ground-breaking patent for the treatment of sickle cell disease.

Dei BioPharma, the Ugandan biotechnology company led by scientist Dr. Matthias Magoola has announced the scientific breakthrough over the weekend, a development that could significantly reshape the treatment of sickle cell disease, offering new hope to millions of patients worldwide, the majority of whom live in Sub-Saharan Africa.

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The Ugandan-based global biotech firm said in a statement, that her innovation, has developed through a gene therapy platform could make curative treatment for sickle cell disease far more affordable and accessible than existing options.

The innovation was invented by the company’s founder and chief executive, Dr. Matthias Magoola, and has been accepted for patenting by the United States Patent and Trademark Office (USPTO).

Sickle cell disease is an inherited blood disorder in which red blood cells become misshapen, leading to severe pain, infections, organ damage and reduced life expectancy.

While recent gene therapies have shown the disease can be cured, their extremely high cost—often running into millions of dollars per patient—has placed them beyond the reach of most people, particularly in low- and middle-income countries.

Dei Biopharma’s approach seeks to address not only the medical challenge, but also the long-standing access barriers that have defined sickle cell care.

All humans are born producing fetal haemoglobin, the oxygen-carrying protein present during pregnancy and early infancy that does not cause red blood cells to sickle.

Fetal haemoglobin is produced from early pregnancy until about six months after birth, after which the body naturally switches to adult haemoglobin.

In people with sickle cell disease, symptoms typically begin when this switch occurs, introducing the defective adult haemoglobin that drives the condition.

Rather than attempting to repair the faulty gene in each patient, Dei Biopharma’s scientists have developed a way to keep fetal haemoglobin production switched on.

Using CRISPR gene-editing technology, the platform targets a genetic “control switch” that regulates the transition from fetal to adult haemoglobin. By disabling this switch, the body continues producing the protective fetal form, 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 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.”

Current curative treatments for sickle cell disease often rely on bone marrow transplants or personalised gene therapies that require donor matching, complex laboratory processes and highly specialised medical facilities. These requirements make them impractical for most countries where the disease burden is highest.

Dei Biopharma says its platform removes many of these barriers. The same gene-editing product could be manufactured at scale, stored, distributed and administered across different populations and health systems.

The company estimates that the approach could reduce the cost of gene therapy for sickle cell disease by more than 95 percent, potentially placing it within reach of public health systems in Africa, the Middle East and parts of Asia.

By focusing on a shared genetic mechanism, the therapy could be applied across all major forms of the disease, including HbSS, HbSC and sickle beta-thalassemia.

“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 affects an estimated 20 million people globally, with the vast majority living in sub-Saharan Africa. Yet most advanced treatments have been developed with high-income markets in mind, reinforcing global health inequalities.

Dei Biopharma describes its innovation as a new model for gene therapy, drawing on the logic of generic medicines, where standardised products enable scale, lower costs and wider access once regulatory approvals are secured.

“Sickle cell disease disproportionately affects populations that have historically been last to benefit from medical innovation,” Dr. Magoola said.

“Our objective is to reverse that pattern by making advanced gene therapies manufacturable and affordable at global scale.”

The patent covers the gene-editing tools, delivery methods and therapeutic processes required to activate fetal haemoglobin. The company is currently conducting preclinical studies to assess safety, durability and effectiveness before progressing to human trials.

Dei Biopharma says it plans to work with regulators, research institutions and strategic partners as it advances the platform toward clinical development.

For Dr. Magoola, the breakthrough reflects a broader ambition to ensure that cutting-edge biological medicines are not reserved for a small share of the world’s population.

“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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Tags: affordable medicinebiotechnologyCRISPRDei BioPharmaDr. Matthias Magoolafetal hemoglobingene therapygenetic engineeringGlobal Healthhealthcare accesshematologymedical innovationSickle Cell DiseaseUgandaUSPTO

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