A new gene therapy is offering fresh hope to Kenyans living with sickle cell disease, a lifelong genetic condition that has left many grappling with chronic pain and limited treatment options.
The Exagamglogene Autotemcel (Exa-cel) gene therapy, approved by the United Kingdom (UK) in January this year, is a one-off treatment designed for patients with sickle cell disease (SCD) and beta-thalassemia, with recommendation from the National Institute for Health and Care Excellence.
Beta-thalassemia is a genetic blood disorder characterized by reduced or absent production of beta-globin, a component of haemoglobin, which is the protein in red blood cells that carries oxygen throughout the body. In Kenya, prevalence of the disease is high in the coastal region, affecting about 0.3% of the population.
However, sickle cell disease is more prevalent, affecting thousands of individuals. According to the Ministry of Health, at least 14,000 children are born with sickle cell disease in Kenya annually, with the highest burden in Western, Nyanza, and Coastal regions, where about 4.5% of children are born with the disease and 18% with the sickle cell trait.
Limited treatment options leave families struggling with pain, hospitalisations, and a reduced quality of life.
But with gene therapy, patients now have the chance to get a one-time treatment that has the potential for a long-term cure. Exa-cel is a type of gene-edited therapy that involves modifying a patient’s own cells to correct genetic defects that cause blood disorders such as sickle cell.
First, stem cells are collected from a patient’s bone marrow or blood. These cells are then modified in a laboratory using a CRISPR Cas9 gene editing technique to correct genetic mutations that cause sickle cell disease. The goal is to activate the production of healthy haemoglobin.
After editing, the cells are grown and expanded in a laboratory before being infused back into the patient’s body. These modified cells are designed to produce healthy red blood cells, which can significantly reduce or even eliminate the symptoms associated with blood disorders.
According to Amanda Pritchard, Chief Executive of the National Health Service (NHS) in England, the introduction of gene editing therapy was a ‘leap in the right direction’ in the fight against sickle cell disease.
Amanda noted that the innovative gene editing therapy is part of a series of revolutionary gene therapies introduced by the NHS England, which offer hope for a cure for those with severe sickle cell disease.
Kenya stands at the forefront of embracing this revolutionary therapy, which could significantly improve the quality of life for thousands
Clinical trials indicate that exa-cel may prevent painful and unpredictable sickle cell crises, the primary symptom of the disease, by blocking blood vessel obstructions that cause intense pain. Experts believe the therapy offers patients a potential for a life free of the disease, with 96.6% of participants showing a “functional cure”.
Between 2023-2024, there were over 32,000 hospital admissions in England for sickle cell disorders, with nearly 14,000 related to sickle cell anaemia crises. A survey from the Sickle Cell Society revealed that 24% of individuals with sickle cell spent 1-2 weeks in the hospital in the past two years.
With the introduction of exa-cel, eligible patients aged 12 and older who experience recurrent crises can now access it at specialist NHS centres in London, Manchester, and Birmingham.
Kenya stands at the forefront of embracing this revolutionary therapy, which could significantly improve the quality of life for thousands living with sickle cell disease in the country.
Suvy Amadi, 36, a sickle cell patient who thought she would not live past 18 years, welcomed the invention but expressed fears that Kenya is still far from embracing the new gene therapy technology.
“The gene editing therapy brings a beacon of hope to those who have long suffered from sickle cell disease like me. It’s a breakthrough that promises not just to treat the symptoms, but to potentially eliminate the root cause of the disease, offering a brighter future to countless families,” says Amadi.
She however, notes that while the revolutionary treatment stirs hope, it is not only distant but also prohibitively expensive, making it out of reach for many patients in the country.
In the UK, gene editing therapy costs £1.65 million pounds (about Ksh284 million) per person, and $2.2 million (about Ksh284 million) per person in the US. Not only that, but there is also a combination of legal, ethical, and health expertise for its administration.
We don’t yet know the long-term effects, as we are altering a person’s genetic makeup
Phionah Obandah, a Genomics Scientist and advocate for sickle cell disease, says gene therapy is a cutting-edge treatment that could be a game changer for sickle cell patients globally.
However, she emphasized that while gene therapy holds promise, there are significant challenges ahead, especially in terms of infrastructure and accessibility in Kenya and across Africa.
“Gene editing therapy involves modifying a patient’s genetic material to correct the abnormality that causes the disease. Despite its potential, the treatment requires highly specialised infrastructure, resources that are currently more accessible in countries like the United Kingdom and the United States,” said Obandah.
“This technology is not yet available in Kenya, and it’s important to understand that it is still under clinical trials. We don’t yet know the long-term effects, as we are altering a person’s genetic makeup.”
Besides the regimen that was approved in the UK, the United States Food and Drug Administration (FDA) in 2023 approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older.
Casgevy, a cell-based gene therapy, is approved for treating sickle cell disease in patients aged 12 and older with recurrent crises. It is the first FDA-approved therapy utilising CRISPR/Cas9, a genome editing technology.
Lyfgenia is another cell-based gene therapy that modifies blood stem cells to produce HbAT87Q, a gene-therapy-derived haemoglobin that mimics normal adult haemoglobin. This reduces the risk of red blood cells sickling and blocking blood flow, offering a promising treatment for patients.
However, as is the case with Exa-cel gene therapy, Casgevy and Lyfgenia also have prohibitive costs of $2.2 million (Ksh285 million) and $3.1 million (Ksh402 million) per patient, respectively.
Government must allocate more resources to awareness campaigns and, ultimately, for treatment options like gene therapy
According to Prof Gordon Nguka, a Medical Dietetics and Paediatric Physiology consultant, while the potential for gene therapy is enormous, there is need for early screening in areas with high prevalence of sickle cell disease, to ensure that those who need treatment most can access it.
“The government must allocate more resources to awareness campaigns and, ultimately, for treatment options like gene therapy,” Prof Nguka said. “While it’s a significant investment, the benefits for sickle cell patients could be life-changing.”
Prof Nguka, who is also a lecturer in the Department of Nutritional Sciences at Masinde Muliro University of Science and Technology, said that in addition to gene therapy, bone marrow transplants also offer curative options for sickle cell disease, although they rely on donor availability.
According to Academic Model Providing Access to Healthcare (AMPATH), which is a part of a collaboration between Moi Teaching and Referral Hospital, Moi University, and Indiana University (including the Indiana Haemophilia and Thrombosis Centre), Sickle cell disease (SCD) is the most common hemoglobinopathy encountered in Kenya.
AMPATH data shows that In the western part of the country, about 4.5% of children are born with SCD, and 18% of children are born with sickle cell trait and that it has contributed significantly to the mortality rate in children younger than age 5 years, primarily because of late diagnosis, educational gaps among service providers, and lack of access to appropriate treatment.
As of 2021, AMPATH has provided care to over 3,000 patients of all ages with SCD across western Kenya and offered medications such as Hydroxyurea, Penicillin V, Folic Acid, Proguanil, analgesics, and vaccines through the Revolving Fund Pharmacy to Sickle cell warriors.
Since 2012, AMPATH has trained over 5,000 healthcare workers on SCD diagnosis, treatment, and supportive care and screened over 25,000 children, with treatment initiation to 75% for most identified cases and examined more than 2,800 patients in haematology clinics
On March 13, 2025, the Jaramogi Oginga Odinga Teaching and Referral Hospital (JOOTRH) launched a dedicated SCD care unit, one of the first of its kind in the region.
In Kisumu, 32 out of every 100 children born annually are affected by SCD, which represents 3.2% of newborns diagnosed with SCD, and 13.9% of them carry sickle cell trait (139 per 1,000). About 1,440 of 44,000 annual newborns have sickle cell traits
To combat the high prevalence of SCD, the Ministry of Health in partnership with Novartis and other stakeholders in September 2023, launched the Afya Dhabiti Project aimed at strengthening SCD care through early diagnosis, reducing the treatment costs and increase the availability of hydroxyurea, a critical medication for SCD.
