Gene and Cell Therapies Enter a New Era: From Revolutionary Treatments to Market Realities
The Great Cell and Gene Therapy Reckoning: From Revolutionary Promise to Industry Crossroads As personalized CRISPR therapies arrive and CAR-T cells target solid tumors, the field confronts manufacturing challenges and soaring costs The gene and cell therapy revolution that scientists have promised for decades is finally materializing—but not quite in the way many had envisioned. In 2025, the field stands at a pivotal crossroads, celebrating historic medical breakthroughs while grappling with formidable economic and manufacturing obstacles that threaten to limit patient access to these potentially life-saving treatments. A Landmark Year for Personalized Medicine In February 2025, an infant known as KJ became the world's first patient to receive a fully personalized CRISPR gene editing therapy at Children's Hospital of Philadelphia. Born with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, a rare metabolic disease, KJ received a customized base editing therapy del...