Gene therapy

Gene therapy engineers a patient's own biology to treat or cure disease by delivering functional genetic material directly into cells.

Gene therapy has transitioned from experimental science to a clinical standard for previously untreatable conditions. By using viral vectors (like AAV) or non-viral lipid nanoparticles to deliver corrective DNA, the technology addresses the root cause of genetic disorders rather than just managing symptoms. In 2026, the field is defined by the global rollout of CRISPR-based treatments like Casgevy for sickle cell disease and the expansion of in vivo editing for common conditions like hypertension. With over 4,000 therapies currently in the global pipeline and a market valuation exceeding $18 billion, the focus has shifted to industrializing manufacturing and reducing the multi-million dollar price tags that currently limit patient access.

https://asgct.org