Genome editing: decoding the technical challenges and balancing precision, safety, and global regulations

“AI and machine learning will play an increasingly central role in research and development activities, from predicting the best targets for editing to enhancing precision and efficiency to reducing off-target effects.”

Houria Bachtarzi is a gene and cell therapy consultant operating at the forefront of technical CMC, regulatory science, and strategic development. In an industry where no product or technology is ever the same, her role is to provide a tailored scientific and regulatory insight to address these complexities effectively. Here, Houria explores the current landscape and future direction of gene-edited therapies, with a focus on technical challenges, regulatory development, and emerging platform technologies. She examines the scientific and policy milestones shaping clinical success, long-term safety, and reaching the global stage where genetic medicines have no borders.

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