CRISPR pi­o­neer Feng Zhang co-founds a 'lim­it­less' biotech up­start with big plans for speed­ing new drug de­vel­op­ment

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.

David Scott

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.

Win­ston Yan

“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.


Im­age: Feng Zhang. MIT

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

Ron DePinho (file photo)

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“If it doesn’t come out of MGH or MIT or Harvard, probably not that interesting,” he said of the mindset.

Now, he and some well-known industry players are looking to change that, and they’ve reeled in just over $38 million to do it.

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As­traZeneca caps PD-L1/CT­LA-4/chemo com­bo come­back with OS win. Is treme­li­mum­ab fi­nal­ly ready for ap­proval?

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Combining Imfinzi and tremelimumab with physicians’ choice of chemotherapy helped patients with stage IV non-small cell lung cancer live longer, the company reported — marking the first time the still-experimental tremelimumab has demonstrated an OS benefit.

For AstraZeneca and CEO Pascal Soriot, the positive readout — which is devoid of numbers — offers much-needed validation for the big bet they made on Imfinzi plus tremelimumab, after the PD-L1/CTLA-4 regimen failed multiple trials in head and neck cancer as well as lung cancer.

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Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

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Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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Brent Saunders (Richard Drew, AP Images)

OcuWho? Star deal­mak­er turned aes­thet­ics czar Brent Saun­ders flips back in­to biotech. But who’s he team­ing up with now?

Brent Saunders went on a tear of headline-blazing deals building Allergan, merging and rearranging a variety of big companies into one before an M&A pact with Pfizer blew up and sent him on a bout of biotech drug deals. That didn’t work so well, so under pressure, he got his buyout at AbbVie — which needed a big franchise like Botox. And it was no big surprise to see him riding the SPAC wave into a recent $1 billion-plus deal that left him in the executive chairman’s seat at an aesthetics outfit — now redubbed The Beauty Health Company — holding a big chunk of the equity.

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'Chang­ing the whole game of drug dis­cov­ery': Leg­endary R&D vet Roger Perl­mut­ter leaps back in­to work as a biotech CEO

Roger Perlmutter needs no introduction to anyone remotely involved in biopharma. As the R&D chief first at Amgen and then Merck, he’s built a stellar reputation and a prolific career steering new drugs toward the market for everything from cancer to infectious diseases.

But for years, he’s also held a less known title: science partner at The Column Group, where he’s regularly consulted about the various ideas the VCs had for new startups.

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Ad­comm splits slight­ly in fa­vor of FDA ap­prov­ing Chemo­Cen­tryx’s rare dis­ease drug

The FDA’s Arthritis Advisory Committee on Thursday voted 10 for and 8 against the approval of ChemoCentryx’s $CCXI investigational drug avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

The vote on whether the FDA should approve the drug was preceded by a split vote of 9 to 9 on whether the efficacy data support approval, and 10 to 8 that the safety profile of avacopan is adequate enough to support approval.

Gold­man Sachs jumps aboard Bain-backed 503(b) com­pound­ing phar­ma­cy with a $275M debt loan to sup­ply hos­pi­tals

Long the bane of the FDA’s existence, compounding pharmacies have seen a minor resurgence in the past year as short-term saviors for hospital drug shortages. Now, a 503(b) company specializing in hospital meds has earned a big backer to keep expanding its 200-drug strong portfolio.

Goldman Sachs and Owl Rock Capital Partners have doled out a $275 million debt loan to QuVa Pharma, a 503(b)-certified outsourcing facility providing compounded drugs to hospitals, the company said Thursday.