Genome Editing

Genome Editing designates precise modification of DNA sequences within living cells using programmable nucleases that create targeted double-strand breaks, with cellular repair mechanisms subsequently introducing desired changes. This revolutionary technology encompasses CRISPR-Cas systems offering unprecedented accessibility, efficiency, and versatility enabling correction of disease-causing mutations, enhancement of cellular therapies, and creation of disease models.

The biopharmaceutical industry rapidly adopted genome editing across research, development, and emerging therapeutic applications. Clinical successes include sickle cell disease treatments editing haematopoietic stem cells, with ongoing trials addressing beta-thalassaemia, inherited blindness, and genetic liver diseases. Cell line engineering employs genome editing creating high-producing biomanufacturing cell lines. Safety considerations address off-target editing at unintended sites, large deletions or chromosomal rearrangements, and immunogenicity against bacterial Cas proteins. Regulatory frameworks continue evolving, with agencies developing guidelines addressing editing characterisation and long-term safety monitoring. As base editors and prime editors advance, genome editing applications expand addressing genetic diseases and enhancing cellular therapies.