Every new parent dreams of holding a healthy baby, but for some, that dream comes with a heavy burden. Around 1 in 58,000 newborns are diagnosed with Severe Combined Immune Deficiency (SCID), a rare genetic disorder that leaves them without a functioning immune system. For parents of children with SCID, daily life is filled with anxiety; even a gentle touch can expose their child to infections, and something as simple as a cold can become life-threatening. Without treatment, most of these children do not live past the age of two.

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Currently, the only effective treatment is stem cell transplantation, which requires finding amatching donor. Even if a donor is found, the transplantation process is often stressful. Families may have to travel long distances to specialised hospitals, which can be especially challenging with a vulnerable child.

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One of the most common causes of SCID is a mutation in the RAG1 gene, which is essential for producing immune cells that fight infections. Fortunately, a new gene therapy offers hope for those with RAG1-SCID. This innovative treatment repairs the child’s own stem cells, eliminating the need for a donor and patient travel. In this process, the patient’s blood is sent to a lab, where a healthy version of the RAG1 gene is added to the isolated cells. After thorough testing, the improved cells are returned to the local hospital for treatment. This allows families to receive life-changing care closer to home, with the child effectively being their own donor.

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So far, five patients have received this therapy, and the results have been extraordinary. The first two patients in the trial are now attending school, getting vaccinated, and living normal lives, cured of what was once a deadly disease.

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At Genewity, we are dedicated to improving the lives of those affected by SCID, giving them the chance to live healthy lives and allowing parents to embrace their children without fear.

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