Tome Biosciences has debuted with $213 million to bring their genome-editing platform to the clinic. The technology can insert large DNA sequences into the genome in any position to correct genes in vivo. The tool, dubbed PASTE — programmable addition via site-specific targeting elements — was developed by Tome co-founders Omar Abudayyeh and Jonathan Gootenberg from the Massachusetts Institute of Technology. Investors include Andreessen Horowitz, Arch Venture Partners, Polaris and Fujifilm.

PASTE uses a CRISPR–Cas9 nickase fused with two other enzymes: a reverse transcriptase and a serine integrase. The guide RNA-directed reverse transcriptase creates landing sites for the serine integrase, which inserts large sequences of DNA. Tome has shown that its tool can insert sequences of ~36 kb in various dividing and non-dividing cell types. And, unlike other editing tools, it doesn’t rely on repair responses to the double-strand breaks that can result in insertions, deletions and off-target effects.

Tome Biosciences 斥资 2.13 亿美元首次亮相，将其基因组编辑平台引入临床。该技术可以将大的DNA序列插入到基因组的任意位置，以在体内纠正基因。该工具被称为 PASTE（通过特定位点的目标元素进行可编程添加），由来自麻省理工学院的 Tome 联合创始人 Omar Abudayyeh 和 Jonathan Gootenberg 开发。投资者包括 Andreessen Horowitz、Arch Venture Partners、Polaris 和 Fujifilm。

PASTE 使用与另外两种酶融合的 CRISPR–Cas9 切口酶：逆转录酶和丝氨酸整合酶。引导RNA引导的逆转录酶为丝氨酸整合酶创建着陆位点，丝氨酸整合酶插入大的DNA序列。 Tome 已证明其工具可以在各种分裂和非分裂细胞类型中插入约 36 kb 的序列。而且，与其他编辑工具不同，它不依赖于对双链断裂的修复反应，而双链断裂可能会导致插入、删除和脱靶效应。