1亿美金，Intrexon与Ziopharm为CAR-T研究再添一把火！

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来源：生物谷
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& e, i2 G5 m  C. s  c, T2015年1月15日讯 /生物谷BIOON/ --随着医疗技术的提高，科学家们日渐意识到人体的免疫系统可能才是人们对抗病魔最有力的武器。因此，自去年以来，免疫疗法在医药研发领域中渐趋白热化。许多生物医药巨头都在这一领域中投入了巨大的人力物力。可以说，2015年如果一个医药公司不做点肿瘤免疫疗法的研究，你都不好意思跟同行打招呼。而在众多的免疫疗法中，CAR-T疗法最为广大产业人士所看好。
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最近，两家制药公司Intrexon和Ziopharm联手投资一亿美元为这一领域再添了一把火。公司宣布与德州大学的安德森癌症研究中心签订了价值一亿美元的合作协议共同开发相应的CAR-T疗法。与目前Juno和诺华等公司的传统方法不同，安德森癌症研究中心建立了一种非病毒载体系统来进行T细胞的免疫。这样就避免了许多潜在风险。协议规定，安德森癌症研究中心今年将向两家公司提供五种候选药物完成临床前研究。
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7 Z, _  K3 D" f3 ~CAR-T疗法是通过分离患者的T细胞，在体外利用嵌合抗原对其进行免疫，使这些T细胞成为靶向杀灭特定肿瘤细胞的"导弹"后再扩大培养并回输至患者体内。由于这些细胞本身就源于患者本人，因而其造成个体差异和副作用的风险也会大大降低。
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目前在这一领域中跑在最前列的是诺华公司。Juno公司和Kite公司紧随其后。另一方面，强生公司和辉瑞公司也砸入巨资希望实现华丽的逆袭。让我们拭目以待，看看谁才能成为这场竞争中最后的赢家。
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详细英文报道：, C1 `' J/ U% _( L
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Partners Intrexon ($XON) and Ziopharm ($ZIOP) are cutting in on the fervor over a new class of immunotherapies for cancer, striking a deal with the University of Texas' MD Anderson Cancer Center.
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# P$ ?# m$ g3 CUnder the deal, the pair will give MD Anderson $100 million for a CAR-T technology, made through a process in which scientists remove T cells from a patient's blood and equip them with targeting mechanisms called chimeric antigen receptors (CARs), which seek out and bind to proteins expressed by cancer cells. The resulting cells are reinjected into the patient, at which point they track down malignancies and attack them as they would any commonplace infection.& j  b/ p% a$ _: i0 x
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MD Anderson's approach, dubbed Sleeping Beauty, uses a nonviral gene transfer system to get those modified T cells to their targets, in contrast to leading CAR-T therapies from Novartis ($NVS), Juno Therapeutics ($JUNO), Kite Pharma ($KITE) and others. That presents some safety advantages, researchers say, as MD Anderson's approach has thus far avoided some of the dangerous immune responses that have afflicted rival CAR-T programs.
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: Y7 `' n: L) X: ~# @Through the deal, Intrexon and Ziopharma expect to get 5 CAR-T candidates into the clinic this year, and the companies are pursuing both patient-derived immunotherapies and off-the-shelf treatments based on donor T cells.+ |% E+ U, o8 g

2 X% t) [. d( Y% I, s8 `! t* K% i& T"Genetically engineering our patients' immune-system T cells to efficiently attack and destroy cancer cells represents one of the most exciting approaches with curative potential in oncology today," MD Anderson President Ron DePinho said in a statement. "We believe coupling MD Anderson's unique CAR-T cell approach with the powerful technologies of Ziopharm and Intrexon will allow us to build T cells that hit cancer harder, with greater precision, under tighter control and with potentially fewer side effects for patients."1 E/ g  y, _' z) }: k* d: |

. G# O9 y3 `  |' T, P) Y1 T4 h: t! ]" `CAR-T therapies have generated stellar results in early clinical trials, galvanizing widespread interest around the industry. Accordingly, Ziopharm's shares shot up more than 50% on the announcement of the MD Anderson deal, while Intrexon rose about 14%. The news is particularly promising for Ziopharm, which is still on the mend after the costly Phase III failure of its cancer treatment palifosfamide back in 2013.0 t4 a: }( J+ o/ C$ V$ f5 x
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Meanwhile, leading the way in CAR-T is Novartis ($NVS), which is working alongside the University of Pennsylvania. The fast-crowding space also includes Kite, which grossed more than $140 million in a June IPO on the strength of its approach to T cell modification, and Juno, which pulled of 2014's biggest biotech IPO thanks to the promise of its clinical candidates. Oncology heavyweight Celgene ($CELG) has jumped into the field with gene therapy pioneer bluebird bio ($BLUE), while latecomers Johnson & Johnson ($JNJ) and Pfizer ($PFE) have shouldered into the space with with high-dollar deals of their own, and Bellicum Pharmaceuticals ($BLCM) is wending toward the clinic with a novel approach to the field.