Gene Therapy

Gene Therapy encompasses therapeutic strategies introducing, replacing, or modifying genetic material in patient cells to treat or prevent disease, representing a transformative approach addressing root genetic causes rather than managing symptoms. These interventions employ diverse vectors including viral vectors like adeno-associated virus or lentivirus, non-viral methods using plasmids or mRNA, and ex vivo approaches modifying cells outside the body before reinfusion.

The biopharmaceutical industry has transformed gene therapy from experimental concept to approved therapeutics with expanding pipelines. Vector development optimises tropism for target tissues, payload capacity, immunogenicity profiles, and manufacturing scalability. Manufacturing challenges include achieving sufficient vector yields, ensuring product purity and safety, and validating complex processes. Safety considerations address insertional mutagenesis risks, immune responses against viral capsids, and off-target effects. Regulatory pathways require comprehensive preclinical packages, manufacturing validation, and clinical programmes with long-term follow-up. As technology matures through improved vectors, enhanced delivery, and better manufacturing, gene therapy applications expand from rare monogenic disorders to common diseases.