Arbor Biotechnologies Comes Out of Stealth Mode

Arbor Biotechnologies, Inc., headquartered in Cambridge, Massachusetts, came out of stealth mode yesterday with a $15.6 million Series A financing round, which closed in June 2017. The round was led by Keith Crandell of ARCH Venture Partners and Annie Hazlehurst of Faridan Ventures, with the participation of several private investors. Today, two researchers with the company, David Scott and Winston Yan, published a paper describing their new CRISPR technology involving an enzyme called Cas13d in the journal Molecular Cell.

One of the company’s founders is Feng Zhang of the Broad Institute and MIT. Zhang’s work is fundamental to CRISPR/Cas9, and has been behind the founding of several companies in the field, including Editas Medicine. Other founders include Scott and Yan, as well as David Walt of Harvard and the Wyss Institute, who is also the co-founder of Illumina, Inc. and Quanterix. 

CRISPR is a DNA gene editing tool that allows researchers to precisely clip out sections of DNA and insert whatever type of DNA they want in its place. The technology utilizes an enzyme called Cas9. The company’s technology, described in the Molecular Cell article, utilizes a new molecule from the CRISPR-Cas13 enzyme family called Cas13d. This molecule is much smaller than other members of the family of enzymes and has implications for RNA manipulation.

Simultaneously, in the same journal, and apparently independently, scientists at the Salk Institute also published a paper on their own work on Cas13d. "CRISPR has revolutionized genome engineering, and we wanted to expand the toolbox from DNA to RNA," said Patrick Hsui with Salk, and one of the authors of the paper.

John Carroll, with Endpoints News, writes, "And like Feng Zhang, he believes that Cas13d enzymes are perfectly suited for the job, with the implications for disease caused by toxic RNA or improperly spliced RNA, the messengers that translate DNA into proteins. Dubbed CasRx, they packaged it in a virus and used it to target the toxic tau known to cluster in the brains of Alzheimer’s patients. Aimed at neurons, it works in cells grown from patients with the neurodegenerative disorder frontotemporal dementia, rebalancing tau levels."

Arbor Biotechnologies was founded in 2016, but came out of stealth mode yesterday with the announcement. It utilizes artificial intelligence to build a genetic search engine that gives it the ability to identify new proteins and enzymes for drug development.

One of the key differences in working with RNA as opposed to DNA, is that edits to RNA would be fixable. The Boston Business Journal notes, "Edits to DNA, including those made by CRISPR/Cas9, are permanent. That’s a good thing if the edit cures a disease, but worrisome when the genetic change is ‘off-target,’ or unintended. Edits to RNA, on the other hand, are only temporary—one major reason why the approach is gaining traction among scientists."

Yan told Business Insider, "We are now at the cusp of being able to convert sequence data into a catalog of protein functions. The possibilities are limitless."

Register as Visitor to CPhI China 2018!