2015年1月9日讯/生物谷BIOON/--作为基因编辑技术CRISPR/Cas9的领跑者，Intellia Therapeutics近日宣布与诺华展开一项长达5年的研发合作计划，主要致力于加速发展CRISPR/Cas9技术在CAR-T细胞治疗和造血干细胞中的应用。此次合作仅仅在Intellia与Atlas Venture和Caribou Biosciences合作3个月之后，再次证明了Intellia的团队和研发能力。
以CRISPR/Cas9为基础的基因编辑技术在一系列基因治疗的应用领域都展现出极大的应用前景，如血液病、肿瘤和其他遗传疾病。CRISPR/Cas9在多种类型的细胞和组织中都具有高效精确的基因编辑能力，在CAR-T、造血干细胞等体外治疗手段中是一个非常理想的操作平台，在体内也可适用。
根据合作协议，诺华将享有此次合作研发项目中CAR-T的专利权，而诺华会和Intellia共同推进关于造血干细胞治疗的多个项目，并且双方同意共同分配研发和所有权，这将会使Intellia独立发展造血干细胞生产管线内部的所有权。
此外，为了增加Intellia持有的股权，诺华向Intellia支付了预付款，并且为未来5年的合作提供了技术支持和研发费用。Intellia也将有资格获得后续研发成功的报酬和专利费。除此之外，Intellia还将在一些自己的生产管线中获得某些诺华的知识产权和技术。Intellia还在其他治疗领域享有与其他合作伙伴合作的权利。
Intellia的CEO和联合创始人Nessan Bermingham博士表示，Intellia与诺华的合作将会极大地推动将CRISPR/Cas9转化为实际治疗手段的应用，CAR-T和造血干细胞治疗是CRISPR/Cas9技术极具代表性的应用领域和契机，而诺华毫无疑问是这两项治疗领域的领跑者，与诺华合作可以加速发展产品的研发。
关于Intellia Therapeutics
Intellia Therapeutics是2014年成立的生物技术公司，主要致力于CRISPR/Cas9技术在治疗领域的发展，Intellia 独家享有一项在CRISPR/Cas9技术治疗领域极为重要的知识产权，2014年，Intellia Therapeutics获得了Atlas Venture和诺华的首轮融资。？
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Intellia Therapeutics， Novartis Form Partnership to Develop New Cell Therapies Using CRISPR/Cas9 Technology
Intellia Therapeutics， a leader in the development of therapeutic products using CRISPR/Cas9 technology for gene editing and repair， today announced a five-year research and development collaboration with Novartis to accelerate the ex vivo development of new CRISPR/Cas9-based therapies using chimeric antigen receptor T cells (CARTs) and hematopoetic stem cells (HSCs). This collaboration comes only three months after Intellia was launched by Atlas Venture and Caribou Biosciences， providing an important validation of Intellia's team and capabilities.
CRISPR/Cas9-based gene editing holds promise across a range of gene therapy applications， including blood disorders， cancer and other genetic based diseases. It has been shown to be an efficient and precise method for gene editing across multiple cell and tissue types， making it an ideal platform for ex vivo applications， such as CART- and HSC-based therapies， as well as in vivo applications.
Under the terms of the agreement， Novartis receives exclusive rights to develop all collaboration programs focused on engineered CARTs. Within HSCs， Novartis and Intellia will jointly advance multiple programs， and have agreed to a process for assigning development and ownership rights， which will enable Intellia to develop its own proprietary internal HSC pipeline.
In addition to increasing its equity holding in Intellia， Novartis is making an upfront payment， and providing technology access fees and funding for R&D programs during the five-year term of the collaboration. Intellia is also eligible to receive downstream success-based milestones and royalties. Intellia will gain access to certain Novartis intellectual property and technology for the development of its own product pipeline. Intellia also reserves the right to pursue additional enabling partnerships in other areas of therapeutic interest.
"Our collaboration with Novartis is an important building block for Intellia that will greatly accelerate our effort to translate the promise of CRISPR/Cas9 into meaningful advances for patients，" said Nessan Bermingham， Ph.D.， Chief Executive Officer and co-founder of Intellia. "CARTs and HSCs represent two of the most immediate opportunities for CRISPR therapeutic development， and Novartis， as a leader in this space， is the ideal partner with which to develop strong product pipelines in these areas.."
About Intellia Therapeutics
Intellia Therapeutics was formed in 2014 to lead the industry in one of the most promising new areas of therapeutic development： gene editing and repair using CRISPR/Cas9 technology. Intellia holds exclusive access to one of the most comprehensive intellectual property platforms available for the therapeutic use of CRISPR/Cas9. Intellia closed a Series A round in 2014 led by Atlas Venture and Novartis.