One of the key scientific players involved in the emergence of the incredibly buzzy gene editing tech CRISPR/Cas9 is backing a biotech startup called Arbor Biotechnologies in Cambridge, MA. which just unveiled what it believes is a new, more versatile CRISPR tech.
In a paper published today in Molecular Cell, two former members of Feng Zhang’s lab — David Scott and Winston Yan — outlined their discovery of an enzyme called Cas13d, which they say is considerably smaller and better than the rest of the Cas13 family of enzymes, giving it greater potential in RNA surgery. And their paper was published on the same day as a separate study out of Salk which centered on the exact same enzyme.
The biotech came out of stealth mode today with a $15.6 million round, which we’ve been tracking, and a plan to employ the new CRISPR system with a platform drug development tech built around artificial intelligence, genome sequencing, gene synthesis and screening efforts to go about the business of finding new molecules. Using the new CRISPR system to characterize proteins, the group plans to play a role in the ongoing integration of computational science in drug discovery, looking to shorten development timelines and improve on some incredibly bad failure rates.
In a call with me late Thursday, Scott and David Cheng, founder of the search engine at Arbor, emphasized that the new paper on Cas13d was done entirely in-house.
“All of this is wholly an effort at Arbor,” says Scott. “Feng is a co-founder, he is involved, but he is not an author of the study, that was wholly performed in-house” at the 2-year-old biotech.
From the summary of the new paper:
The small size, minimal targeting constraints, and modular regulation of Cas13d effectors further expands the CRISPR toolkit for RNA manipulation and detection.
At the same time, researchers at the Salk Institute also unveiled their scientific work on Cas13d, using it to tailor RNA rather than DNA, where CRISPR made its rep.
“CRISPR has revolutionized genome engineering, and we wanted to expand the toolbox from DNA to RNA,” said Salk’s Patrick Hsu. And like Feng Zhang, he believes that Cas13d enzymes are perfectly suited for the job, with 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 worked in cells grown from a patients with neurodegenerative disorder frontotemporal dementia, rebalancing tau levels.
It will likely take years to prove if these young revolutionaries — and a host of colleagues operating in AI — are right, but they already have the rapt attention of every drug development organization on the planet.
David Walt of Harvard and the Wyss Institute and co-founder of Illumina and Quanterix is credited as a co-founder with Zhang along with Scott and Yan, who both worked in Zhang’s MIT lab. And they are pumped about the scientific potential.
“Arbor’s revolutionary platform accelerates the rate of discovery and characterization of new biomolecules by orders of magnitude,” said Scott in a statement.
“We are now at the cusp of being able to convert sequence data into a catalog of protein functions. The possibilities are limitless,” noted Yan.
Zhang was the principal scientist behind the emergence of Editas, one of the original gene editing startups to hit following the emergence of CRISPR/Cas9, a tech that several biotechs are using to edit out disease in genes.
Keith Crandell of ARCH Venture Partners, Annie Hazlehurst of Faridan Ventures, and several private investors are credited with the round.
It was probably coordinated by Cell Press editors but I didn’t know about the Arbor team’s work until this morning. We submitted last year, so food for thought on #ASAPbio. Super exciting company though, and always glad to see independent work from multiple groups!
— Patrick Hsu (@pdhsu) March 15, 2018
Image: Feng Zhang.MIT