2014年11月25日讯 /生物谷BIOON/ --随着医疗技术的发展，人类已经征服了无数种过去认为是不治之症的疾病，天花等疾病也已经从人类社会彻底销声匿迹。然而癌症仍然是横亘在人类改善健康状况提高生活质量的道路上的庞然大物。
近些年来，相关癌症治疗方向已经由最早的化疗和放疗方法发展为利用人体自身免疫系统识别来杀灭肿瘤细胞。今年以来，癌症免疫领域最令人瞩目的就是诺华等公司率先提出的嵌合抗原受体-T细胞（CAR-T）疗法。
而最近强生公司终于决定涉足这一领域。公司已经和Transposagen生物医药公司达成合作研究协议共同开发CAR-T疗法作为强生进入这一领域的突破口。在接下来三年内，双方的研究人员将共同开发基于Transposagen公司新一代基因技术的CAR-T抗体药物。强生公司将向Transposagen公司提供每种疗法2亿9千万美元的里程碑资金。而作为回报，强生公司将获得这些药物的全球版权。
目前在CAR-T这一研究领域中，诺华公司、Kite公司和Juno公司呈现三足鼎立的局面。其中Juno公司开发的抗CD19 CAR-T疗法刚刚获得FDA的突破性药物认证。而葛兰素史克公司也在CAR-T疗法方兴未艾时积极进入。如此看来，强生此次进入CAR-T研究领域似乎为时已晚。
不过，强生公司此次选择Transposagen公司作为合作对象，显然也是经过深思熟虑。Transposagen公司将基因编辑技术和CAR-T技术结合起来，使人体免疫系统识别异源抗体，为开发下一代CAR-T药物打下基础。或许强生此举在未来数年内能使其在CAR-T肿瘤疗法领域中后来居上。
详细英文报道：
Over the past year, new CAR-T cancer therapies have turned into a sizzling new development field in biopharma--and now Johnson & Johnson ($JNJ) wants in. The pharma giant is following in the footsteps of pioneers like Novartis, Celgene and GlaxoSmithKline, inking a discovery-stage development deal--but turning to the little-known Transposagen Biopharmaceuticals to make its entry. And J&J says the broad-ranging deal will include the use of the biotech's gene editing tech in developing new gene and cell therapies.
J&J's Janssen and the little biotech, which is based far from the madding biotech crowd in Lexington, KY, will collaborate over the next three years on new CAR-T drugs; reengineering T cells to include a chimeric antigen receptor so they can zero in on cancer cells. Along with an unspecified upfront--typically fairly small in discovery-stage deals--J&J will pay up to $292 million per treatment in milestones.
In return, J&J will control all worldwide commercial rights.
"We feel like we found the diamond in the rough," says Barry Springer, who handles the partnership side of the business for the Janssen Biotechnology Center of Excellence. The link-up was inspired, he adds, by a 5-year strategy review to get a fix on the next big wave of opportunities in R&D. Gene editing tech and gene therapies have been on their radar for awhile, and it's no secret that autologous T cell engineering--adapting cells extracted from patients--has attracted a huge amount of attention of late.
One of the reasons why Janssen zeroed in on Transposagen, he added, is that their technology has the potential to develop allogeneic T cell therapies--off-the-shelf therapies that can be instantly used to treat and potentially cure cancer. And they'll be looking beyond where the pioneers started to see what other cancer types can be targeted with this technology.
"We view it as the next-generation version of what we're after," says Springer, who says he's pumped to combine J&J's considerable development expertise with the tool kit that's been fine-tuned at Transposagen.
Novartis ($NVS), Kite Pharma ($KITE) and Juno Therapeutics--which just won breakthrough drug status at the FDA--each have their own anti-CD19 CAR T programs in development. Celgene ($CELG), in collaboration with bluebird bio ($BLUE), poses a competitive threat using technology from Baylor College of Medicine, while GlaxoSmithKline ($GSK) has entered into a $350 million T-cell reengineering deal with Adaptimmune in the U.K.
"The research collaboration with Janssen will pair Transposagen's cutting-edge gene editing and gene delivery technology and expertise with Janssen industry-leading technologies in the antibody and antibody alternative areas to create what may be the ideal CAR-T therapy," said Eric Ostertag, CEO of Transposagen.
Transposagen originally specialized in marketing genetically modified animals for R&D, but more recently it's been marketing its talents in CAR-T and CRISPR, another gene editing tech that's been the subject of growing interest in the biotech field. This new deal with J&J raises its profile in the industry considerably.