Cell & Gene Therapy

CAR-T engineering modifies T cells with chimeric antigen receptors targeting cancer markers. CRISPR gene editing makes precise genomic modifications. AAV vectors deliver gene therapy payloads to target tissues. Lentiviral vectors integrate therapeutic genes into cell genomes. Base editing and prime editing enable precise modifications without double-strand DNA breaks. Lipid nanoparticles deliver mRNA and gene editing components.

CAR-T treats blood cancers (lymphoma, leukemia, myeloma) with 40-80% complete remission rates. Gene therapy corrects single-gene disorders (SMA, hemophilia, sickle cell). Gene editing treats genetic blood disorders. Engineered cell therapies are being developed for solid tumors, autoimmune diseases, and organ transplant rejection.

The global cell and gene therapy market exceeded $20 billion in 2025, with projections to reach $50+ billion by 2030. Over 2,000 clinical trials are active globally. Approved therapies include CAR-T (Kymriah, Yescarta, Tecartus, Breyanzi, Abecma) and gene therapies (Zolgensma, Luxturna, Casgevy).