Prime editing has made its medical debut by treating a teenager suffering from chronic granulomatous disease, aiming to correct mutations affecting immune cells. Researchers reported that the treatment restored function in two-thirds of the patient's neutrophils without serious side effects a month post-treatment. Although results are promising, significant assessment will take time, as confirmed by experts in the field. Prime Medicine, the company behind the therapy, plans not to pursue its further development independently, highlighting the challenges in progressing gene-editing therapies for rare diseases.
Despite the promising results of prime editing in treating a rare immune disorder, Prime Medicine announced it will not develop the therapy further on its own.
Annarita Miccio noted that while it will take time to assess the success of the treatment, it represents a promising approach to a challenging disease.
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