Cell and gene therapy encompasses the technologies that treat diseases by modifying patients' cells or delivering genetic material to correct or replace defective genes. CAR-T cell therapy (engineering a patient's immune cells to attack cancer) has produced breakthrough treatments: Kymriah and Yescarta deliver complete remission rates of 40-80% in certain blood cancers that had no effective treatment. Gene therapy using viral vectors (AAV, lentivirus) delivers functional copies of genes to correct single-gene disorders, with Zolgensma ($2.1 million per dose for spinal muscular atrophy) and Casgevy (the first CRISPR-based therapy for sickle cell disease) representing landmark approvals. The sector's primary challenge is manufacturing: each CAR-T treatment is manufactured individually from a patient's own cells, creating a production bottleneck that limits access and drives costs above $400,000 per treatment. Allogeneic (off-the-shelf) approaches that use donor cells rather than patient cells could solve the manufacturing challenge but face scientific hurdles around immune rejection.

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