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

Ven­ture Cap­i­tal as a Strate­gic Part­ner: Fu­el­ing In­no­va­tion be­yond Fi­nance

The average level of investment required for a biotech start-up to succeed is increasing every year, elevating the pressure even further on venture capital to make smart financial investments. Financial investment alone, however, does not always guarantee that exciting innovations can be transformed into real businesses that make a meaningful difference to patients.

Beyond just capital

At Astellas Venture Management (AVM) – a wholly-owned venture capital organization within Astellas, headquartered in the San Francisco Bay Area – capital is just one of the ingredients we offer to add value to our biotechnology investments and partnerships. We generally take a strategic investor approach for companies in our invested portfolio, providing access to expertise, technology and/or resources in addition to the injection of finance. An equity investment from AVM can include access to Astellas’ research and development (R&D) capabilities and expertise, and a global network of partner academic institutions and biotechnology companies, to help advance and accelerate the start-up’s innovation.

UP­DAT­ED: Ver­tex joins Mer­ck, Pfiz­er — re­vamp­ing multi­bil­lion-dol­lar tri­al strat­e­gy as biotech R&D crum­bles

You can add Pfizer, Merck and — as we found out Friday morning — Vertex to the growing list of pharma giants hitting the pause button on a range of clinical trials. But not everyone in R&D is getting a red light.

Vertex says that it’s doing its best to keep working its pipeline strategy, coming up with a plan “to enable virtual clinic visits and home delivery of study drug to ensure study continuity and medical monitoring, and to facilitate study procedures.”

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Covid-19 roundup: In­ter­cept, blue­bird and a grow­ing list of biotechs feel the pain as pan­dem­ic man­gles FDA, R&D sched­ules

Around 100 staffers at Boston area hospitals have now tested positive for Covid-19, spotlighting the growing risk that the pandemic will sideline many of the most essential workers in healthcare as caseloads peak in the US and around the globe. With more than 3,400 deaths, Spain has become the latest country to surpass the official death count attributed to the new coronavirus in China, where the outbreak originated. As of Thursday morning, confirmed global cases had crossed 470,000 and the death count eclipsed 21,000.

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Af­ter crit­ics lam­bast­ed Gilead for grab­bing the FDA's spe­cial rare drug sta­tus on remde­sivir, they're giv­ing it back

Two days after Gilead won orphan drug status for remdesivir as a potential treatment for Covid-19, they’re handing it back.

The company was slammed from several sides after Gilead reported that the FDA had come through with the special status, which comes with 7 years of market exclusivity, the waiver of FDA fees and some tax credits as well. Typically, everyone who can get orphan status lands it without much of a fuss, but Democratic presidential candidate Bernie Sanders, Public Citizen and other consumer groups were outraged.

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Mod­er­na CEO Stéphane Ban­cel out­lines a short path for emer­gency use of a coro­n­avirus vac­cine

NIAID director Anthony Fauci has left no doubts that it takes 12 to 18 months to get a new vaccine tested and in commercial use, in the best of circumstances. But in times of a global emergency — like these — maybe there’s another, faster route to follow.

In an SEC filing on Tuesday, Moderna $MRNA staked out a record-setting pathway to getting their mRNA vaccine into the frontline of the healthcare response as early as this fall. The SEC filing notes that CEO Stéphane Bancel told Goldman Sachs that an emergency use approval could allow the vaccine to go to healthcare workers and certain individuals in a matter of months — presumably provided the NIH sees the safety and efficacy data they would need from the Phase I.

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Caught in a Covid-19 mael­strom, Eli Lil­ly locks down clin­i­cal tri­als as multi­bil­lion-dol­lar R&D ops de­rail

The Covid-19 pandemic has derailed Eli Lilly’s $6 billion R&D operations.

The pharma giant reported Monday morning that it has decided to hit the brakes on most new study starts and pause enrollment for most ongoing studies. Lilly adds that it is continuing dosing for ongoing studies, “but with study-by-study consideration.”

The pandemic has severely disrupted healthcare systems around the globe, says Lilly, making it difficult or impossible to conduct studies at many research sites. And there’s no timeline for when it expects to get back on track.

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As share buy­backs come un­der scruti­ny, what's in store for the bio­phar­ma in­dus­try?

Stock buybacks are not to be permitted for companies that will be bailed out in the coronavirus stimulus package, Congressional leaders have signaled. To what degree the biopharma industry has relied on buybacks for earnings growth in recent years, and if the trend continues, are the big questions as scrutiny into the practice heightens and balance sheets weaken with the coronavirus pandemic wreaking havoc on global economies.

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A Sin­ga­pore VC rais­es $200M for a new round, but will Covid-19 pre­vent it from rais­ing the rest?

A top Singaporean biotech venture fund is nearly halfway toward its largest ever fund, but in a sign of what could be in store for VCs amid a global economic freeze, said they could face headwinds raising the other half.

Vickers Venture Partners has secured $200 million out of a targeted $500 million for its 6th fund, first announced in early 2018. They’ve given themselves 13 months to complete the financing, Vickers founder Finian Tan told Deal Street Asia, but the financial frost settling amid the Covid-19 pandemic could slow efforts.

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Strug­gling Unum ex­ecs are ready to con­sid­er a sale, merg­er or any deal that comes its way

Unum $UMRX is working its way through a survival plan of sorts.

After getting hit with a trio of FDA holds in its brief public history and triggering its second pivot to a new lead drug program while laying off 60% of the staff, the troubled penny stock biotech Unum Therapeutics has hatched new plans to secure financial backing while lining up a go-forward strategy for the company.

First, Lincoln Park Capital Fund has agreed to buy up to $25 million of the long-suffering stock, as Unum directs. And the executive team — led by CEO Chuck Wilson — has put everything on the table for consideration: a sale, acquisition, merger, licensing deal, you name it. The ACTR707 program, meanwhile, is being formally wrapped up — their second failed lead program.