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

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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Robert Califf (Pablo Martinez Monsivais, AP Images, File)

As buzz on Califf FDA nom heats up, in­dus­try and agency in­sid­ers of­fer a strong nod for the ‘per­fect’ choice

For once in this long, dramatic road to finding a new FDA commissioner, there’s been some continuity. Both CNN and Politico reported this weekend that Rob Califf met with President Biden to discuss the permanent commish role, following earlier news broken by the Washington Post that all signs point to Califf.

Although there may be a few Democrats who continue to grandstand about the dangers of COI (Califf has worked for Verily, sits on the board of Centessa Pharmaceuticals, and has other ties to industry research), with the pandemic ongoing and the need for some kind of continuity at FDA mounting, Califf is likely to meet the same fate as when he first won Senate confirmation in 2016, by a vote of 89-4 — Bernie Sanders and 6 others didn’t vote.

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AstraZeneca CEO Pascal Soriot (Raphael Lafargue/Abaca/Sipa USA)

A com­bo of As­traZeneca's Imfinzi and chemo wins where oth­ers have failed in piv­otal bil­iary tract test

Looking to run with the big dogs in the PD-(L)1 class, AstraZeneca’s Imfinzi has a tall hill to climb to compete in an increasingly bustling market. An aggressive combo strategy for the drug has paid off so far, and now AstraZeneca is adding another notch to its belt.

A combo of Imfinzi (durvalumab) and chemotherapy significantly extended the lives of first-line patients with advanced biliary tract cancer over chemo alone, according to topline results from the Phase III TOPAZ-1 study revealed Monday.

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Sean Ianchulev, Eyenovia CEO and CMO

Re­cent court de­ci­sion push­es FDA to re­ject and re­clas­si­fy drug-de­vice com­bo, crush­ing shares

Back in April, the FDA lost a crucial court case in which its broad discretion of regulating medical products that might satisfy the legal definitions of either “drug” and/or “medical device” was sharply curtailed.

In addition to the appeals court ruling that Genus Medical Technologies’ contrast agent barium sulfate (aka Vanilla SilQ) should not be considered a drug, as the FDA had initially ruled, but as a medical device, the agency also was forced to spell out which drugs would transition to devices as a result of the ruling.

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Peter Greenleaf, Aurinia CEO

Af­ter pass­ing on Ac­celeron, Bris­tol My­ers eyes bolt-on ac­qui­si­tion of au­toim­mune spe­cial­ist — re­port

Bristol Myers Squibb is looking to beef up its autoimmune portfolio by scooping up Aurinia Pharmaceuticals, Bloomberg reported.

The recent overtures to Aurinia, relayed by anonymous insiders, came just as Bristol Myers turned down buyout talks with partners at Acceleron — which Merck ultimately struck a deal to acquire for $11.5 billion. Bristol Myers has reportedly decided to cash out on its minority stake, likely bagging $1.3 billion in the process, while keeping the royalty deals on two of Acceleron’s blood disorder drugs.

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So — that pig-to-hu­man trans­plant; Po­ten­tial di­a­betes cure reach­es pa­tient; Ac­cused MIT sci­en­tist lash­es back; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

We’re incredibly excited to welcome Beth Bulik, seasoned pharma marketing reporter, to the team. You can find much of her work in our new Marketing channel — and in her weekly newsletter, Endpoints PharmaRx, which will launch in early November. Add it to your subscriptions here.

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NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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