Sickle cell disease: experts recommend the adoption of gene therapy.

Official data shows that approximately 4 million Nigerians, or about 2% of the population, live with this disease.

Professor Jennifer Adair, co-founder of the Global Gene Therapy Initiative (GGTI) and faculty member at the University of Massachusetts Chan School of Medicine, stressed the urgent need to introduce curative treatments such as gene therapy and bone marrow transplants in countries most affected by sickle cell disease.

She made this appeal in Abuja during a press conference at the 5th World Congress on Sickle Cell Disease , alongside Dr. Alexis Thompson, a physician-scientist at the Children's Hospital of Philadelphia and principal investigator in gene therapy trials in the United States, and Jimmy Olaghere, a Nigerian-born beneficiary of the groundbreaking gene therapy.

Stressing the need for Nigeria to formally adopt gene therapy with appropriate administrative structures and legislative support, she noted that the adoption of this therapy is essential not only for its many benefits, but also to advance science in the country.

Gene therapy is a medical approach that treats or prevents diseases by modifying a person's genes. It typically involves replacing, repairing, or regulating faulty genes—often by introducing a healthy copy to replace a defective or missing one—thus addressing the root cause of the disease.

“Nigeria has the largest number of sickle cell patients in the world. It is essential that those affected, the ‘warriors’ as they are often called, understand that curative solutions are not science fiction, but are very real and increasingly accessible,” she said.

She noted that gene therapy is not entirely new to Nigeria, as the country is already part of the GGTI, a coalition of nations committed to strengthening local capacities for research, regulation and access to gene-based treatments.

According to her, among the first six countries in the network, Uganda has already adopted legislation to support bone marrow transplants and gene editing.

Adair noted that Nigeria has the necessary foundations, scientists, clinicians, regulators and patient advocates, but needs to align these elements into a coherent national strategy.

She stated that Nigeria would not be alone in this process, as the GGTI also supports knowledge transfer, including training programs between Nigeria, India and the United States, and promotes access to technical information in local languages.

"We are working to support clinicians who want to learn how to manufacture and administer these therapies locally," she said.

Addressing concerns about affordability in countries where healthcare funding is limited, Adair acknowledged the high cost of gene therapy in the United States, which currently stands between $2 million and $3 million.

She noted, however, that the figures are based on value-based pricing models rather than actual manufacturing costs. She cited the example of Uganda, where the same therapy could be profitable at around $40,000, adding: "While $40,000 is still a high cost, it represents a starting point for investment."