CRISPR trail­blaz­ers Zhang, Liu and Joung join forces to launch Beam with $87M and cut­ting-edge gene-edit­ing tech

Three of the founders be­hind the high pro­file gene-edit­ing com­pa­ny Ed­i­tas are tak­ing their sci­en­tif­ic prowess to a new ven­ture, launch­ing a start­up this week that ex­pands on the pi­o­neer­ing CRISPR tech the en­tire space is built on.

The new com­pa­ny — called Beam Ther­a­peu­tics — is step­ping out Mon­day with $87 mil­lion in launch mon­ey. In­vestors are back­ing true trail­blaz­ers in gene edit­ing, as Beam’s sci­en­tif­ic founders in­clude David Liu, now-se­r­i­al en­tre­pre­neur Feng Zhang, and J Kei­th Joung. All three are sci­en­tif­ic founders of Ed­i­tas, the CRISPR com­pa­ny launched in 2013 that now gar­ners a $1.7 bil­lion mar­ket cap.

Beam is tak­ing a to­tal­ly new ap­proach to gene edit­ing, hop­ing to tweak base pairs with­out ac­tu­al­ly cut­ting the strand of DNA or RNA. If CRISPR can be com­pared to scis­sors, then Beam’s base edit­ing tech is more like a pen­cil, Liu tells me, eras­ing a “bad” base and writ­ing in a good one.

The im­pe­tus for Beam’s launch was Liu’s re­search at Har­vard, which is now li­censed to the Cam­bridge start­up. The li­cense cov­ers two base edit­ing plat­forms: a C base ed­i­tor and an A base ed­i­tor. The C base one fea­tures Cas9 linked to a cy­ti­dine deam­i­nase to make C-to-T or G-to-A ed­its. The A base ed­i­tor has Cas9 linked to “an evolved form of adeno­sine deam­i­nase,” which can make A-to-G or T-to-C ed­its.

Liu’s DNA base edit­ing tech is just part of Beam’s pack­age, though. Zhang, the guy who just months ago helped launched a dif­fer­ent gene edit­ing start­up (Ar­bor), has joined forces with Liu, con­tribut­ing RNA base edit­ing tech­nol­o­gy from his lab that fea­tures Cas13 linked to an adeno­sine deam­i­nase that can ed­it A-to-G in RNA tran­scripts.

On top of li­cens­es to Liu and Zhang’s tech, Beam al­so has patents from Ed­i­tas. Beam’s CEO John Evans tells me the deal gives the start­up ex­clu­sive rights to IP li­censed to Ed­i­tas by Har­vard, Broad, and Mass­a­chu­setts Gen­er­al Hos­pi­tal, along with cer­tain Ed­i­tas tech­nol­o­gy. As part of that deal, Beam has an ex­clu­sive sub­li­cense to base edit­ing patents out of Liu’s lab and patents by MGH for CRISPR tech­nol­o­gy de­vel­oped in Joung’s lab.

Beam’s ap­proach to gene edit­ing is quite com­pelling for a num­ber of rea­sons. First, slic­ing DNA is a per­ma­nent change, and that isn’t al­ways a good idea.

“There are some states of dis­ease that don’t last a life­time, but are tran­sient,” Liu said. “For those cas­es, you may not want to ad­dress the dis­ease by mak­ing a per­ma­nent change to the DNA of cells.”

“Eras­ing and writ­ing in” ed­its with­out clip­ping could be in­cred­i­bly use­ful in these cas­es.  Liu said edit­ing mu­ta­tions in the nu­cle­obas­es of DNA and RNA has the po­ten­tial to “re­verse a large frac­tion — per­haps the ma­jor­i­ty — of point mu­ta­tions as­so­ci­at­ed with dis­ease.”

But what are the odds that it will work? Liu said he’s hap­py with ear­ly in­di­ca­tions. Af­ter first pub­lish­ing work on these base ed­i­tors back in 2016, dozens of re­searchers have tried their hand at the tech­nol­o­gy and saw suc­cess in bac­te­ria, fun­gi, in­sects, mice, and even hu­man em­bryos.

Beam wants to stay qui­et on which dis­eases its first tar­gets will tack­le but did men­tion that the com­pa­ny is ac­tive­ly work­ing on 10-15 tar­gets and is “en­cour­aged by ear­ly re­sults.” This Se­ries A fund­ing, which was led by F-Prime Cap­i­tal Part­ners and ARCH, should take the com­pa­ny “mul­ti­ple years,” Evans said.

“The length of that run­way gives us con­fi­dence that we can push for­ward quick­ly with lead pro­grams, but al­so take time to go deep on the sci­ence and build a broad pipeline in par­al­lel,” Evans said.

Evans has joined Beam from ARCH, where he was a part­ner. Al­though VCs of­ten sit in as tem­po­rary CEOs while a start­up hunts for a more per­ma­nent can­di­date, Evans said he’s in for the long haul at Beam. While still af­fil­i­at­ed with ARCH, Evans is join­ing Beam full time.

“At Agios, I saw the pow­er of pre­ci­sion med­i­cine to di­rect­ly cor­rect mu­ta­tions in leukemia,” Evans said. “That was a pow­er­ful ex­pe­ri­ence for me, and I want to do that again. With base edit­ing, I see a re­al po­ten­tial to do it again and again.”

Beam has plans to set up an of­fice in the heart of Cam­bridge’s Cen­tral Square neigh­bor­hood. It em­ploys 15 peo­ple to­day, and plans on hir­ing “sev­er­al more” in the near fu­ture.


Top Im­age: Feng Zhang, David Liu and J Kei­th Joung. BEAM THER­A­PEU­TICS

Nick Galakatos, Blackstone global head of life sciences

Nick Galakatos and the Black­stone team now have a record $4.6B to in­vest in bio­phar­ma, with a big fo­cus on push­ing com­pa­nies over the top

Nick Galakatos and his team at Blackstone Life Sciences have seen their biggest opportunities swell up in mostly established players who don’t have all the money they need to accomplish everything on the to-do list. And right now, with the industry booming, that’s a long list with some hefty needs.

The Blackstone team has neatly tied up the largest private fund ever raised in life sciences for making big dreams come true in biopharma. Late Thursday, Blackstone put out word that they had closed their highly anticipated fund with the projected $4.6 billion all in.

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UP­DAT­ED: Bio­gen shares spike as ex­ecs com­plete a de­layed pitch for their con­tro­ver­sial Alzheimer's drug — the next move be­longs to the FDA

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimer’s drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agency’s decision-making time to a mere 6 months.

The news drove a 10% spike in Biogen’s stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

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Gilead boasts of pos­i­tive remde­sivir da­ta on mor­tal­i­ty — but their analy­sis pro­vokes the skep­tics

Gilead is surging again off data that suggest its antiviral remdesivir might improve survival.

The new data come from an analysis Gilead conducted comparing the death rate and recovery time of patients in one of its remdesivir trials to a group of 800 patients “with similar baseline characteristics and disease severity” who received only standard-of-care around the same time. The result, they said, suggested that patients who received remdesivir had a 62% better chance at surviving than those who did not.

Hal Barron, GSK

Win or lose on the mar­ket­ing OK, the FDA just gunned down GSK’s bright hopes for their BC­MA ther­a­py

The FDA’s ODAC — the Oncologic Drugs Advisory Committee — has a well-known bias in favor of adding new cancer drugs to the market, even if efficacy is at best marginal and serious safety issues demand careful management.

Doctors want as many arrows in their quiver as they can get. And when patients are dying after failing multiple drugs, why not give it a go one more time?

GlaxoSmithKline, though, is about to test out how their new BCMA antibody drug conjugate belantamab mafodotin can do after being mauled in an in-house FDA review, ahead of the Tuesday expert panel discussion. Even if the agency goes ahead with an expected green light, this drug will likely be constrained to a small niche — icing any plans they may have for making waves in oncology anytime soon.

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Covid-19 roundup: BioN­Tech go­ing head-to-head with Mod­er­na as PhI­II mR­NA launch looms; Tri­al on Shin­zo Abe’s once-fa­vorite an­tivi­ral is in­con­clu­sive

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BioNTech chief Ugur Sahin told the Wall Street Journal that his company will start Phase III testing of their vaccine later this month, setting them up to lateral the data to regulators before the end of this year.

That puts them essentially on the exact same schedule as Moderna is dedicated to. The Massachusetts rival to BioNTech also expects to launch Phase III this month. Lots of rumors have circulated about delays and conflict among the scientists advancing the Moderna jab, but the biotech has consistently stuck to its plan to start a late-stage pivotal this month.

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Stephan Christgau, Amanda Hayward, Andreas Segerros and Magnus Persson (Eir Ventures)

A new ven­ture fund amid a pan­dem­ic? In the Nordics? Eir Ven­tures brings it on with €76M first close

From Pharmacia and Lundbeck to Novo Nordisk and AstraZeneca, the Nordic countries have been the birthplace for some legacy pharma companies. But for all that history and reputation, Stephan Christgau counts only five specialized life science investors backing biotechs today.

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Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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Andrew Kruegel, Kures president and co-founder (Columbia Tech Ventures via Vimeo)

Af­ter psilo­cy­bin and ke­t­a­mine, a new biotech comes along de­vel­op­ing a drug Scott Got­tlieb fought

Andrew Kruegel was six years into his chemistry work at Columbia University, when, one day in August 2016, he learned he might have only 30 days before the government made him destroy his research.

Kruegel had been studying kratom, a leaf long used in Southeast Asia as a stimulant or for pain. It had opioid-like properties, he found, but seemed to offer pain relief without the addictive potential or respiratory side effects of traditional opioids — a riddle that might help illuminate how human opioid receptors work.

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Atul Deshpande, Harbour BioMed chief strategy officer & head, US operations (Harbour BioMed)

An­oth­er biotech IPO set-up? Multi­na­tion­al biotech leaps from round to round, scoop­ing up cash at a blis­ter­ing pace

A short four months after announcing a $75 million haul in Series B+ fundraising, the multinational biotech Harbour BioMed pulled in another round of investments and eclipsed the nine-digit mark in the process.

Harbour completed its Series C financing, the company announced Thursday morning, raising $102.8 million and bringing its total investment sum to over $300 million since its founding in late 2016. The biotech plans to use the money to transition early-stage candidates from the discovery phase, fund candidates already in the clinic, and prep late-stage candidates for commercialization.

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