Pharma,,,,BioPharma

旨在开/关基因开关,色度畜栏1.25亿美元用于新浪潮

Chroma Medicine正在开发表观遗传疗法,该疗法打开或关闭基因。这家Biotech初创公司来自隐形模式,并获得了1.25亿美元的A轮资金的支持。

细胞具有相同的DNA,但是身体具有实现任何给定细胞变成的内置方式,无论是肝细胞还是神经元。Biotech初创公司Chroma Medicine首席执行官Catherine Stehman-Breen解释说,该系统称为表观基因组,采用了DNA中的内容并有意义。她说,如果您将DNA视为硬件,那么表观基因组就是告诉硬件该怎么做的软件。

Genetic medicines research is marching forward with therapies that treat disease by cutting and editing the genome, or by introducing genetic material intended to fix faulty genetic code. But Stehman-Breen said tapping into the epigenome offers an alternative approach, and one with potential advantages. Instead of trying to correct a mutated gene, Chroma is targeting the software that controls it in order to regulate its expression.

Stehman-Breen说:“利用这种机制使我们在治疗应用方面具有巨大的广度。”“我们认为这最终是一个非常强大的工具。”

剑桥,,,,马萨诸塞州-based Chroma is building a pipeline of epigenetic medicines that turn genes on or off and the startup hasraised $125 millionto support its research. The Series A round of funding announced Wednesday was led by Cormorant Asset Management.

Chroma’s approach mimics what the body already does, said Vic Myer, the company’s president and chief scientific officer. There are some genes, such as retroviruses in the genome, that remain dormant for life because the epigenome turns them off. Similarly, a Chroma drug would silence a toxic or mutant gene, allowing a healthy gene to do its job. For haploinsufficiencies, diseases in which one gene of a gene pair is not functioning properly, Chroma would take the opposite approach and turn on gene expression.

The drugs themselves are large molecules engineered to go to a specific location in the genome, Myer said. The goal is for these therapies to be one-time treatments. After the drug does its work, when the cell divides, the epigenomic system understands that a particular gene is supposed to be turned off. The daughter cells that follow retain the gene silencing. Myer, a former executive at gene-editing biotech Editas Medicine, said he left that company after nearly five years wanting to do something different. He was persuaded to return to genomic medicines by Chroma’s technology.

“It’s an elegant approach, and sort of a natural, normal approach to turn off a gene if you want to turn off a gene, and turn on a gene if you want to turn on a gene,” Myer said.

Chroma基于其六个科学创始人的研究:Luke Gilbert,Keith Joung,David Liu,Angelo Lombardo,Luigi Nadine和Jonathan Weissman。根据Stehman-Breen的说法,Chroma的基础是Weissman,Gilbert等人的研究,publishedin the journal Cell in 2016, showing how a “CRISPRoff” fusion protein could turn off gene expression. That change was maintained as cells divided and as stem cells differentiated into neurons.

Chroma, which formed last year, recently acquired Milan, Italy-based Epsilen Bio, whose co-founders include Lombardo and Naldini. Stehman-Breen said Chroma’s founders knew Lombardo and Naldini, and ongoing conversations led the founders to conclude that the two companies are complementary, and it made sense to pursue epigenetic medicines together.

基因沉默是Chroma技术平台中最先进的部分。迈尔说,那是因为沉默是绝对的 - 当您将基因放下时,很难将其倒下。打开基因可以具有不同的基因表达的设定点。迈尔说,从治疗的角度来看,Chroma对开发基因的研究“有点远”。

沉默基因的想法可能是RNA干扰(RNAi)采用的方法。这些核酸疗法激活已经在细胞中已经阻止基因产生引起疾病的蛋白质的机制。Alnylam Pharmaceuticals获得了RNAi疗法的首次FDA批准。但是RNAi的治疗作用并不能持续下去,因此必须长期服用这些治疗方法。

There are other approaches to regulating gene expression. Constellation Pharma’s clinical-stage epigenetic medicines are small molecules that target enzymes that activate or inactivate disease-causing genes. That biotech, whose research focuses on cancer, was今年早些时候被Morphosys以17亿美元的价格收购。CAMP4 Therapeutics is developing programmable RNA therapies intended to upregulate gene expression to address haploinsufficiencies. Cambridge-basedCAMP4在6月筹集了4500万美元

Omega Therapeutics,旗舰店开创性的生物技术that went public in July with a$126 million IPO,正在开发称为“表观基因组控制器”的疗法。这些融合蛋白被设计为靶向DNA上的绝基因组域。但是,剑桥公司不是打开或关闭基因的二元方法,而是更精确地“调谐”了一种疗法,以至于基因表达的正确水平,以及正确的持续时间。欧米茄的铅治疗候选者调节了一个难以靶向的基因,其活性与许多癌症的进展有关。调低而不是关闭基因是最好的方法,因为健康细胞也依赖于基因,首席执行官Mahesh Karande今年早些时候向Medcit万博互联网怎么样了y News解释了

Chroma尚未透露其旨在治疗的疾病。Stehman-Breen表示,1.25亿美元的款项代表了该公司自成立以来所筹集的总数。她估计,随着初创公司继续建立技术平台并开发新疗法,现金将持续到未来两年。

融资的其它投资者包括Casdin Capital, Janus Henderson Investors, Omega Funds, funds and accounts advised by T. Rowe Price Associates, and Wellington Management, along with seed investors Atlas Venture, Newpath Partners, and Sofinnova Partners.

Photo by Flickr userDean Hochman通过创意共享license

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