Vertex Pharma Shells Out $420M to Move Into Muscular Dystrophy

福泰将投资4.2亿美元,扩大肌肉萎缩症研究范围

2019-06-10 07:37:00 Xconomy

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 Vertex Pharmaceuticals is committing $420 million in a pair of deals that expand the company’s research scope to experimental genetic treatments for two forms of muscular dystrophy. Boston-based Vertex (NASDAQ: VRTX) announced late Thursday that it reached an agreement to pay $245 million up front to acquire Exonics, a company using gene editing techniques to develop a treatment for Duchenne muscular dystrophy and myotonic dystrophy type 1. Vertex also said it is paying CRISPR Therapeutics (NASDAQ: CRSP) $175 million up front to expand an existing research collaboration to include Duchenne and myotonic dystrophy type 1. Watertown, MA-based Exonics Therapeutics is developing treatments intended to use CRISPR-Cas9 gene editing technology to repair the genetic mutations that cause Duchenne and other neuromuscular diseases. Vertex says Exonics’ preclinical research has demonstrated the ability to genetically repair and restore dystrophin, the key missing protein that leads to Duchenne. Exonics licensed its technology from the University of Texas Southwestern Medical Center, where it was developed in the laboratory of molecular biologist Eric Olson. Vertex says Olson will continue in his role as Exonics’ chief science advisor. Under the acquisition terms, Exonics will operate as a Vertex subsidiary. Exonics shareholders could earn up to $755 million in additional payments, depending on the progress of its Duchenne and myotonic dystrophy type 1 programs. Vertex has been working with Switzerland-based CRISPR Therapeutics since 2015. The collaboration initially focused on cystic fibrosis, a core therapeutic focus of Vertex. But the partners have since gone on to advance to the clinic a genetically engineered therapy for sickle cell disease and beta thalassemia, an inherited disorder affecting hemoglobin in the blood. The expanded collaboration gives Vertex exclusive global rights to the CRISPR Therapeutics’ gene editing technology in Duchenne and myotonic dystrophy type 1. In Duchenne, Vertex will be responsible for all research and development, manufacturing, and if approved, commercialization. In myotonic dystrophy type 1, Vertex and CRISPR Therapeutics will share the research costs for RNA research that will be conducted by the Swiss company. Vertex will be responsible for additional R&D, as well as manufacturing, and commercialization expenses. Depending on the progress of the Duchenne and myotonic dystrophy programs, CRISPR Therapeutics could earn up to $825 million in additional payments. If the experimental therapies reach the market, Vertex would pay CRISPR Therapeutics royalties from sales. Vertex expects to close both deals in the third quarter of this year.
福泰(Vertex)制药公司承诺将投资4.2亿美元用于两笔交易,这两笔交易将使该公司的研究范围扩大到实验基因治疗两种形式的肌肉萎缩症。 总部位于波士顿的福泰(Vertex)( NASDAQ : VRTX )周四晚间宣布,该公司达成协议,将预先支付2.45亿美元收购 Exonics 。 Exonics 是一家利用基因编辑技术开发治疗 Duchenne 肌营养不良和1型肌营养不良的公司。福泰(Vertex)还表示,将向 CRISPR Therapeutics ( NASDAQ : CRSP )支付1.75亿美元,以扩大现有的研究合作,包括 Duchenne 和肌张力障碍1型。 位于马萨诸塞州沃特敦的 Exonics Therapeutics 公司正在开发利用 CRISPR-Cas9基因编辑技术来修复导致 Duchenne 和其他神经肌肉疾病的基因突变的治疗方法。福泰(Vertex)说 Exonics 的临床前研究已经证明了基因修复和恢复肌营养不良蛋白的能力,这是导致 Duchenne 的关键缺失蛋白。 Exonics 公司从德克萨斯大学西南医学中心获得了该技术的许可,该技术是在分子生物学家埃里克·奥尔森的实验室中开发的。福泰(Vertex)说 Olson 将继续担任 Exonics 的首席科学顾问。根据收购条款, Exonics 将作为福泰(Vertex)子公司运营。根据 Duchenne 和肌营养不良1型项目的进展情况, Exonics 股东可以获得高达7.55亿美元的额外付款。 福泰(Vertex)自2015年开始与瑞士 CRISPR Therapeutics 合作。合作最初集中在囊性纤维化,一个核心治疗的重点福泰(Vertex)。但此后,这些合作伙伴继续向临床推进镰状细胞病和β地中海贫血的基因工程治疗,这是一种遗传性疾病,影响血液中的血红蛋白。 扩大的合作使福泰(Vertex)独家拥有 CRISPR Therapeutics 在 Duchenne 和肌张力障碍1型的基因编辑技术。在 Duchenne ,福泰(Vertex)将负责所有的研究和开发,生产,如果批准,商业化。在肌张力障碍1型,福泰(Vertex)和 CRISPR 疗法将分担研究费用的 RNA 研究,将由瑞士公司进行。福泰(Vertex)将负责额外的研发、制造和商业化费用。 根据 Duchenne 和肌营养不良项目的进展情况, CRISPR 疗法可获得高达8.25亿美元的额外付款。如果实验疗法进入市场,福泰(Vertex)将从销售中支付 CRISPR 疗法专利使用费。 福泰(Vertex)预计将在今年第三季度完成这两笔交易。

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