Vertex Pharmaceuticals to buy US firm Exonics Therapeutics

福泰制药计划10亿美元收购Exonics公司,开发遗传性神经肌肉疾病基因疗法

2019-06-10 16:02:00 PHARMACEUTICAL

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Vertex Pharmaceuticals has signed an agreement to acquire US-based Exonics Therapeutics in a deal valued up to $1bn (£787m). Under the deal, Exonics equity holders will receive $245m upfront payment from Vertex, as well as potential future payments based on the achievement of specified development and regulatory milestones for the Duchenne Muscular Dystrophy and Myotonic Dystrophy Type 1 programmes. Based in Watertown of Massachusetts, Exonics Therapeutics is involved in the development of gene editing therapies for the treatment of patients with DMD and other severe genetic neuromuscular diseases. Exonics has used SingleCut CRISPR to genetically repair and restore dystrophin in multiple small and large animal DMD preclinical models. Exonics’ technology, which is licensed from UT Southwestern Medical Center, is based on the research of Dr. Olson. Subject to certain conditions, including the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act and other customary conditions, the deal is expected to be completed in the third quarter of this year. Vertex chairman, president and CEO Dr Jeffrey Leiden said: “Through the expanded collaboration with CRISPR and the acquisition of Exonics, we are bringing together the intellectual property, technologies, and scientific expertise needed to establish a leading gene editing platform for DMD and DM1.” Vertex has also expanded its collaboration with CRISPR Therapeutics to discover and develop gene editing therapies for the treatment of DMD and DM1. As per terms of the deal, Vertex will pay $175m (£137m) upfront for the exclusive worldwide rights to CRISPR Therapeutics’ existing and future intellectual property, including foundational CRISPR/Cas9 technology, novel endonucleases, single and double cut guide RNAs, and AAV vectors for DMD and DM1 gene editing products. Vertex will take responsibility for all other research, development, manufacturing, and commercialisation costs. CRISPR Therapeutics will secure payments of up to $1bn (£787m) , including the upfront and potential future payments, based on the achievement of specified research, development, regulatory, and commercial milestones for the DMD and DM1 programmes. Vertex will also pay tiered royalties on future net sales on any products, which may result from this collaboration.
福泰(Vertex)制药签订协议,收购美国Exonics Therapeutics公司,金额高达10亿美元(7.87亿英镑)。 根据该协议, Exonics 的股权持有者将从福泰(Vertex)获得2.45亿美元的预付款,以及基于 Duchenne Music Dystrophy 和 Myotoni Dystrophy 1类项目实现特定开发和监管里程碑的潜在未来付款。 Exonics Therapeutics 位于马萨诸塞州沃特敦,致力于为 DMD 和其他严重遗传性神经肌肉疾病患者开发基因编辑疗法。 Exonics 已经使用 SingleCut CRISPR 在多个小型和大型动物 DMD 临床前模型中对肌营养不良蛋白进行基因修复和恢复。 Exonics 的技术是由 UT Southwest Medical Center 授权的,它基于 Olson 博士的研究。 根据某些条件,包括 Hart-Scott-Rodino Antitrust Improvements Act 的等待期届满和其他惯常条件,该交易预计将于今年第三季度完成。 福泰(Vertex)董事长、总裁兼首席执行官 Jeffrey Leiden 博士表示:“通过扩大与 CRISPR 的合作以及收购 Exonics ,我们将为 DMD 和 DM1建立一个领先的基因编辑平台所需的知识产权、技术和科学专业知识结合起来。” 福泰(Vertex)还扩大了与 CRISPR 疗法的合作,以发现和开发用于 DMD 和 DM1治疗的基因编辑疗法。 根据协议条款,福泰(Vertex)将支付1.75亿美元(合1.37亿英镑)的预付款,用于获得 CRISPR Therapeutics 现有和未来知识产权的全球独家权利,包括基础 CRISPR / Cas9技术、新型内切酶、单切和双切引导 RNA 以及 DMD 和 DM1基因编辑产品的 AAV 载体。 福泰(Vertex)将负责所有其他研究、开发、制造和商业化成本。 CRISPR Therapeutics 将根据 DMD 和 DM1项目的特定研究、开发、监管和商业里程碑的成就,确保支付至多10亿美元(7.87亿英镑),包括前期和潜在的未来付款。 福泰(Vertex)还将为任何产品的未来净销售额支付分层的版税,这可能是这种协作的结果。

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