Gene editing technology empowers rare disease drug development
Release date:
2023-03-02
As gene-editing technology continues to mature, its application in the development of drugs for rare diseases is steadily deepening.
As gene-editing technology continues to mature, its application in the development of drugs for rare diseases is steadily deepening. Research teams are leveraging CRISPR-Cas9 gene-editing technology to conduct precise repair studies targeting disease-causing genes associated with various rare disorders—and have successfully demonstrated therapeutic efficacy in animal models. Building on these breakthroughs, several pharmaceutical companies have already initiated relevant clinical trials, aiming to translate this cutting-edge gene-editing technology into viable drug products. For patients suffering from rare diseases, this advancement holds immense significance, offering the potential to transform the current landscape where effective treatments remain scarce for most rare conditions, and paving the way for entirely new approaches in the quest for innovative therapies.
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