Genetic medicines

Genetic medicines use nucleic acids to reprogram cellular function, correcting disease at the molecular source rather than managing systemic symptoms.

Genetic medicine has transitioned from theoretical research to a clinical standard, evidenced by the 2023 FDA approval of Casgevy for sickle cell disease. This sector encompasses diverse modalities including viral vector gene replacement, mRNA-based protein synthesis, and CRISPR-mediated base editing. By delivering functional DNA or RNA directly into a patient’s cells, these therapies address the root cause of over 7,000 known rare diseases and increasingly target common conditions like cardiovascular disease. Current industry momentum is driven by 4,469 therapies in the global pipeline, with 49% focused on gene therapy and 29% on RNA technologies. As manufacturing scales and delivery vectors such as lipid nanoparticles (LNPs) improve, the focus is shifting toward one-time curative treatments that eliminate the need for lifelong chronic care.

https://www.asgct.org/education/more-resources/gene-and-cell-therapy-faqs