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

Lessons for biotech and phar­ma from a doc­tor who chased his own cure

After being struck by a rare disease as a healthy third year medical student, David Fajgenbaum began an arduous journey chasing his own cure. Amidst the hustle of this year’s JP Morgan conference, the digital trials platform Medable partnered with Endpoints Studio to share Dr. Fajgenbaum’s story with the drug development industry.

What follows is an edited transcript of the conversation between Medable CEO Dr. Michelle Longmire and Dr. Fajgenbaum, and it is full of lessons for biotech executives charged with bringing the next generation of medicines to patients.

Kathy High (file photo)

Gene ther­a­py pi­o­neer Kathy High has left Spark af­ter com­plet­ing $4.3B union with Roche

Kathy High dedicated the past seven years of her life shepherding experimental gene therapies she’s developed at Children’s Hospital of Philadelphia toward the market as president and head of R&D at Spark Therapeutics. Now that the biotech startup is fully absorbed into Roche — with an FDA approval, a $4.3 billion buyout and a promising hemophilia program to boast — she’s ready to move on.

Roche confirmed her departure with Endpoints News and noted “she will take some well-deserved time off and then will begin a new chapter in a sabbatical at a university.”

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Tim Mayleben (file photo)

Es­pe­ri­on's goldilocks cho­les­terol fight­er wins FDA ap­proval — will its 'tra­di­tion­al' pric­ing ap­proach spur adop­tion?

It’s more effective than decades-old statins but not as good as the injectable PCSK9 — the goldilocks treatment for cholesterol-lowering, bempedoic acid, has secured FDA approval.

Its maker, Esperion Therapeutics, is betting that their pricing strategy — a planned list price of between $10 to $11 a day — will help it skirt the pushback the PCSK9 cholesterol fighters, Repatha and Praluent, got from payers for their high sticker prices.

The sky-high expectations for the pair of PCSK9 drugs that were first approved in 2015 quickly simmered — and despite a 60% price cut, coupled with data showing the therapies also significantly cut cardiovascular risk, sales have not really perked up.

Esperion is convinced that by virtue of being a cheaper oral therapy, bempedoic acid will hit that sweet spot in terms of adoption.

“We’re kind of like the old comfortable shoe,” Esperion’s chief commercial officer Mark Glickman remarked in an interview with Endpoints News ahead of the decision date. “It’s an oral product, once-daily and nontitratable — these are things that just resonate so true with patients and physicians and I think we’ve kind of forgotten about that.”

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James Collins, Broad Institute via Youtube

UP­DAT­ED: A space odyssey for new an­tibi­otics: MIT's ma­chine learn­ing ap­proach

Drug development is complex, expensive and comes with lousy odds of success — but in most cases, if you make it across the finish line brandishing a product with an edge (and play your cards right) it can be a lucrative endeavor.

As it stands, the antibiotic market is cursed — it harbors the stink of multiple bankruptcies, a dearth of innovation, and is consequently barely whetting the voracious appetites of big pharma or venture capitalists. Enter artificial intelligence — the biopharma industry’s cure-all for the pesky process of making a therapeutic, including data mining, drug discovery, optimal drug delivery, and addressable patient population.

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Gilead los­es two more patent chal­lenges on HIV pill, set­ting up court­room fight in Delaware

Gilead sustained two more losses in their efforts to rid themselves of an activist-backed patent lawsuit from the US government over a best-selling HIV pill.

Urged on by activists seeking to divert a portion of Gilead’s revenue to clinics and prevention programs, the Department of Health and Human Services made a claim to some of the patents for the best-selling HIV prevention drug, Truvada, also known as PrEP. Gilead responded by arguing in court that HHS’s patents were invalid.

Today, the US Patent and Trademark Office ruled that Gilead was likely to lose the last two of those challenges as well. The USPTO ruled against Gilead on the first two patents earlier this month.

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Tal Zaks (Moderna via YouTube)

For two decades, a new vac­cine tech­nol­o­gy has been slow­ly ap­proach­ing prime time. Now, can it stop a pan­dem­ic?

Two months before the outbreak, Moderna CMO Tal Zaks traveled from Cambridge, MA to Washington DC to meet with Anthony Fauci and the leaders of the National Institutes of Health.

For two years, Moderna had worked closely with NIH researchers to build a new kind of vaccine for MERS, one of the deadliest new viruses to emerge in the 21st century. The program was one test for a new technology designed to be faster, cheaper and more precise than the ways vaccines had been made for over a century. They had gathered evidence the technology could work in principle, and Fauci, the longtime head of the National Institute of Allergy and Infectious Diseases and a longtime advocate for better epidemic preparedness, wanted to see if it, along with a couple of other approaches, could work in a worst-case scenario: A pandemic.

“[We were] trying to find a test case for how to demonstrate if our technology could rapidly prepare,” Zaks told Endpoints News.

Zaks and Fauci, of course, wouldn’t have to wait to develop a new test. By year’s end, an outbreak in China would short circuit the need for one and throw them into 24/7 work on a real-world emergency. They also weren’t the only ones with new technology who saw a chance to help in a crisis.

An ocean away, Lidia Oostvogels was still on vacation and relaxing at her mother’s house in Belgium when her Facebook started changing. It was days after Christmas and on most people’s feeds, the news that China had reported a novel virus to the World Health Organization blurred into the stream of holiday sweaters and fir trees. But on Oostvogels’s feed, full of vaccine researchers and virus experts, speculation boiled: There was a virus in China, something contained to the country, but “exotic,” “weird,” and maybe having to do with animals. Maybe a coronavirus.

Lidia Oostvogels

“I was immediately thinking like, ‘Hey, this is something that if needed, we can play a role,'” Oostvogels told Endpoints.

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Christos Kyratsous (via LinkedIn)

He built a MERS treat­ment in 6 months and then the best Ebo­la drug. Now Chris­tos Kyrat­sous turns his sights on Covid-19

TARRYTOWN, NY — In 2015, as the Ebola epidemic raged through swaths of West Africa, Kristen Pascal’s roommates sat her down on their couch and staged an intervention.

“Are you sure this is what you want to be doing with your life?” she recalls them asking her.

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Pascal, a research associate for Regeneron, had been coming home at 2 am and leaving at 6 am. At one point, she didn’t see her roommate for a week. For months, that was life in Christos Kyratsous’ lab as the pair led a company-wide race to develop the first drug that could effectively treat Ebola before the outbreak ended. For Pascal, that was worth it.

“I’m ok, I don’t have Ebola,” Pascal told them. “I see that death toll rising and I can’t not do something about it.”

Last August, Regeneron learned they had succeeded: In a large trial across West Africa, their drug, REGN-EB3, was vastly more effective than the standard treatments. It was surprise news for the company, coming just 10 months into a trial they thought would take several years and a major victory in the global fight against a deadly virus that killed over 2,000 in 2019 and can carry a mortality rate of up to 90%.

For Kyratsous and Pascal, though, it brought only fleeting reprieve. Just four months after the NIH informed them REGN-EB3 worked, Kyratsous was back in his office reading the New York Times for updates on a new outbreak on another continent, and wondering alongside Pascal and senior management whether it was time to pull the trigger again.

In late January, as the death toll swelled and the first confirmed cases outside China broke double digits, they made a decision. Soon they were back on the phone with the multiple government agencies and their coronavirus partners at the University of Maryland’s Level 3 bio lab. The question was simple: Can Kyratsous and his team use a process honed over two previous outbreaks, and create a treatment before the newest epidemic ends? Or worse, if, as world health experts fear, it doesn’t vanish but becomes a recurrent virus like the flu?

“Christos likes things immediately,” Matt Frieman, Regeneron’s coronavirus collaborator at the University of Maryland, told Endpoints. “That’s what makes us good collaborators: We push each other to develop things faster and faster.”

Kristen Pascal (Regeneron)

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The first time Regeneron tried to respond to a global outbreak, it was something of a systems test, Kyratsous explains from his office at Regeneron’s Tarrytown headquarters. Kyratsous, newly promoted, has crammed it with photos of his family, sketches of viral vectors and a shark he drew for his 3-year-old son. He speaks rapidly – an idiosyncrasy his press person says has only been aggravated this afternoon by the contents of his “Regeneron Infectious Diseases”-minted espresso glass – and he gesticulates with similar fluidity, tumbling through antibodies, MERS, the novel coronavirus, Ebola-infected monkeys.

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Jim Scholefield via PR Newswire

Mer­ck los­es its chief dig­i­tal of­fi­cer, spot­light­ing tal­ent hunt for the hottest ti­tle in Big Phar­ma

Over the last few years we’ve seen the chief digital officer title become one of the hottest commodities in Big Pharma as global organizations hunt the best talent to sharpen the cutting edge of their tech platforms.

But Merck just discovered how hard it may be to keep them focused on pharma.

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Don't let Ab­b­Vie fool FTC with an easy di­vesti­ture, plead crit­ics in lat­est at­tack on $63B Al­ler­gan buy­out

If the FTC must let AbbVie and Allergan go ahead with their merger, at least make them divest their latest blockbuster on the market, a chorus of unions, consumer groups and public interest organizations plead in a new attempt to rein in the megamerger.

There’s a second part to their argument: If the antitrust watchdog does greenlight the divestiture AbbVie wants, then at least ensure the pharma giant cannot corner its future rivals with its exclusionary tactics.

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