AAV gene therapy company AskBio secures $235m investment

AAV基因治疗公司AskBio获2.35亿美元投资,开发治疗遗传病的变革性疗法

2019-04-15 18:25:13 PHARMACEUTICAL

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标签: 医疗及健康服务
The investment in AskBio includes an amount of $225m committed by global alternative asset firm TPG Capital and life sciences investment firm Vida Ventures in exchange of a minority stake in the North Carolina-based AAV gene therapy company. AskBio’s founders and board members are investing $10m in the company as well. The new capital is expected to help advance and expand clinical trials of the company, while improving its manufacturing capabilities and capacity, and helping it generate long-term growth. AskBio CEO and co-founder Sheila Mikhail said: “With the funding from TPG and Vida, we will be able to accelerate our development of a broad range of transformative therapies for those affected by serious and oftentimes incurable genetic diseases. “We look forward to advancing our approaches for repeat administration and avoidance of neutralizing antibodies into the clinic to maximize the number of patients who benefit from AAV therapies.” Founded in 2001, AskBio has more than 500 patents in areas like AAV production, self-complementary DNA and chimeric vectors. The company’s engineered viral vectors are said to be capable of targeting particular tissues, de-target other tissues, and minimize the impact of neutralizing antibodies. Recently, the company advanced a gene therapy for Pompe Disease into clinic development. In addition, it is developing therapies for heart failure, Myotonic Dystrophy, Limb Girdle 2i along with other muscular and neurological disorders. The company’s gene therapy platform includes a cell line manufacturing process called Pro10 in addition to having an extensive AAV capsid library. Pro10 is used by Pfizer, Viralgen, Takeda and Vector Core, said AskBio, which recently entered into a partnership with Touchlight Genetics for the commercialization of a safer, more efficient and cheaper alternative to plasmid DNA. TPG Capital partner John Schilling said: “AskBio is a very special company, operating in one of the most innovative and strategically important sectors of the pharmaceutical industry today. “In addition to its leading clinical programs in several therapeutic areas, AskBio has a broad base of technologies that we believe can support quantum leaps in therapies for many partners in the market.”
对 AskBio 的投资包括全球另类资产公司 TPG Capital 和生命科学投资公司 Vida Ventures 承诺的2.25亿美元,以换取总部位于北卡罗来纳州的 AAV 基因治疗公司的少数股权。 AskBio 的创始人和董事会成员也在该公司投资1000万美元。本次新增资本预计有助于推进和扩大公司临床试验,同时提高其制造能力和产能,有助于其产生长期增长。 AskBio 首席执行官兼联合创始人希拉•米哈伊尔( Sheila Mikhail )表示:“在 TPG 和 Vida 的资助下,我们将能够加快为那些患有严重且通常无法治愈的遗传病的人开发一系列广泛的变革性疗法。 “我们期待着推进我们的方法,重复给药和避免中和抗体进入临床,以最大限度地增加患者受益于 AAV 疗法。” AskBio 成立于2001年,在 AAV 生产、自互补 DNA 和嵌合载体等领域拥有500多项专利。据说,该公司设计的病毒载体能够针对特定组织,去除其他组织的靶向性,并将中和抗体的影响降至最低。 最近,公司将蓬佩病的基因治疗发展到临床。此外,它正在开发治疗心力衰竭,肌萎缩障碍, LimbGirdle 2i 以及其他肌肉和神经障碍。 该公司的基因治疗平台除了拥有广泛的 AAV 衣壳库外,还包括一个名为 Pro10的细胞系制造过程。辉瑞(Pfizer)、 Viralgen 、武田(Takeda)和 Vector Core 使用 Pro10, AskBio 最近与 Touchlight Genetics 建立了合作关系,将一种更安全、更高效、更便宜的质粒 DNA 替代品商业化。 TPG Capital 合伙人约翰•希林( John Schilling )表示:“ AskBio 是一家非常特殊的公司,在当今医药行业最具创新性和战略重要性的领域之一经营。 “除了在几个治疗领域的领先临床项目外, AskBio 还拥有广泛的技术基础,我们相信这些技术可以支持市场上许多合作伙伴在治疗方面的量子飞跃。”