Rayne H. Rouce is an associate professor at the Center for Cell and Gene Therapy, Dan L. Duncan Comprehensive Cancer Center, Texas Children's Hospital, Baylor College of Medicine, Houston, TX, USA.
Matthew H. Porteus is a professor in the Department of Pediatrics and the Center for Definitive and Curative Medicine, Stanford University School of Medicine, Stanford, CA, USA.
Science. 2024 Aug 2;385(6708):475. doi: 10.1126/science.ads0252. Epub 2024 Aug 1.
Patients with devastating illnesses demonstrate incredible courage in battling their disease. Innovative cell and gene therapies (CGTs), built on decades of research, are changing the lives of those who suffer from conditions ranging from cancer to sickle cell disease to neurologic diseases. Although hailed for their promise and recognized for benefits that will exceed the costs, the high prices of CGTs ($300 thousand to $4 million per dose) leave these therapies out of reach for many. This accessibility problem will only be solved if academia, industry, investors, funders, regulators, and advocacy groups work together to put CGT breakthroughs in the hands of all who stand to benefit.
患有绝症的患者在与疾病作斗争时表现出令人难以置信的勇气。建立在几十年研究基础上的创新细胞和基因疗法（CGTs）正在改变那些患有从癌症到镰状细胞病到神经疾病等各种疾病的人的生活。虽然 CGT 因其前景而受到赞誉，并因其将超过成本的收益而得到认可，但 CGT 的高昂价格（每剂 30 万至 400 万美元）使许多人无法获得这些疗法。只有学术界、工业界、投资者、资助者、监管机构和宣传团体共同努力，将 CGT 突破成果应用于所有受益人群，才能解决这种可及性问题。
