一.全球首款上市CAR-T:
1.Tisagenlecleucel, marketed as Kymriah.
2.It was invented and initially developed at the University of Pennsylvania; Novartis completed development, obtained FDA approval, and markets the treatment. In August 30 2017, it became the first FDA-approved treatment that included a gene therapy step in the United States.
The FDA approved Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL).
“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA Commissioner Scott Gottlieb, M.D. “New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses. At the FDA, we’re committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving.”
Kymriah, a cell-based gene therapy, is approved in the United States for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse.
3. What is the KYMRIAH therapy process?
4.What makes KYMRIAH different from other therapies?
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KYMRIAH is a one-time treatment unlike other treatments delivered more than once
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KYMRIAH targets only your B cells rather than all of your body’s cells
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KYMRIAH uses a short course (4 days) of chemotherapy to prepare your body for infusion
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Even if you aren’t in remission, you can start KYMRIAH therapy
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Because KYMRIAH uses your own cells, you do not need a donor
5.诺华Kymriah的业绩，2018H1 2800万美元 ，Q2业绩 1600万美元。单个患者费用在47.5万美元。
6.目前在美获批两大适应症。https://www.pharma.us.novartis.com/sites/www.pharma.us.novartis.com/files/kymriah.pdf，说明书链接如上。
二.Gilead &Kite，Yecsrta：
（axicabtagene ciloleucel）
1.2018年H1销售额 1.08亿美元 ，Q2 6800万美元。在美国市场，Kite的Yescarta定价为37.3万美元。
2.It was developed by California-based Kite Pharma.
Axi-cel was awarded US FDA breakthrough therapy designation on October 18, 2017 for diffuse large B-cell lymphoma, transformed follicular lymphoma, and primary mediastinal B-cell lymphoma. It also received priority review and Orphan drug designation.
三.国内CAR-T：
1.复星凯特生物科技有限公司产业化项目:
租赁上海复盛医药科技发展有限公司厂房，在中试楼1层新建生产单元，并利用已建成的中试楼现有实验单元进行产业化生产，相加总规模为年产3000例CAR-T免疫细胞疗法中所需的细胞。
CART 名：益基利仑赛（拟定），按3类生物新药报。KTE-C19，axicabtagene ciloleucel。国内在2018年4月递交了临床申请。FKC876。
2.#科济制药#有限公司#免疫细胞治疗产品#商业化生产基地(Ⅰ期)。#做CART先在国内有GMP车间#。本项目在上海市金山工业区豪业路518号上海张江金山生物医药标准产业园3号厂房开设免疫细胞治疗产品商业化生产基地，年产嵌合抗原受体修饰的T细胞（CAR-T）3000例。
3.药明巨诺：
药明巨诺今年3月完成9000万美元A轮融资，Temasek淡马锡、元明资本、红杉资本中国联合领投。
6月27日，药明巨诺及其关联公司上海明聚生物宣布，明聚生物CAR-T产品JWCAR029的IND申请获得国家药品监督管理局批准。
4.传奇生物：
另外，2018年5月30日，传奇生物宣布美国食品药品监督管理局（FDA）授权传奇生物的合作伙伴——Janssen Biotech, Inc. (“Janssen”)就其针对复发/难治性多发性骨髓瘤患者的CAR-T细胞疗法LCAR-B38M (JNJ-68284528)开展1b/2期临床试验，以评估该产品的安全性和有效性。
5.已申报IND的细胞治疗：
药审中心生物制品临床部细胞治疗和基因治疗适应症小组介绍了国内外CAR-T治疗R/R B-ALL临床试验方案中受试人群、入组标准、主要疗效终点和风险控制等方面的基本情况和存在的一些共性问题。经讨论，与会专家针对不同年龄段患者的试验分组、复发难治患者的入组标准、主要疗效终点、风险控制原则等达成一致意见。后续，药审中心将参考以上意见组织起草相关的技术指南。
更多信息参考：
https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm574058.htm；
https://www.us.kymriah.com/acute-lymphoblastic-leukemia-children/about-treatment/understanding-treatment/；
https://www.pharma.us.novartis.com/sites/www.pharma.us.novartis.com/files/kymriah.pdf；
https://www.novartis.com/sites/www.novartis.com/files/q2-2018-ir-presentation.pdf；
https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm581216.htm；
https://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/ucm581222.htm;
https://www.fda.gov/downloads/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/UCM581226.pdf；
CAR T Cells: Engineering Patients’ Immune Cells to Treat Their Cancers，https://www.cancer.gov/about-cancer/treatment/research/car-t-cells；
https://www.cancer.gov/news-events/cancer-currents-blog/2017/car-t-cell-multiple-myeloma；
https://mp.weixin.qq.com/s/MD3iYX2O4ZDmZjKZA6dL7g，等。
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