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

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Roivant par­lays a $450M chunk of eq­ui­ty in biotech buy­out, grab­bing a com­pu­ta­tion­al group to dri­ve dis­cov­ery work

New Roivant CEO Matt Gline has crafted an all-equity upfront deal to buy out a Boston-based biotech that has been toiling for several years now at building a supercomputing-based computational platform to design new drugs. And he’s adding it to the Erector set of science operations that are being built up to support their network of biotech subsidiaries with an eye to growing the pipeline in a play to create a new kind of pharma company.

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Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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Per­cep­tive's fourth — yes, fourth — SPAC jumps to Nas­daq as the blank check tree con­tin­ues to ripen

The biotech SPAC boom has gone almost hand-in-hand with the industry’s IPO gold rush, and this week saw more blank check companies hop aboard the train.

Leading the way is Perceptive Advisors’ fourth SPAC, appropriately named Arya Sciences Acquisition IV, which priced Friday morning after raising $130 million. And on top of that, new Ziopharm executive chair James Huang is launching his own SPAC with MSD Partners and Panacea Venture, filing S-1 paperwork Thursday with plans to raise $200 million.

CEO Fred Aslan (Artiva)

NK cell ther­a­py play­er Arti­va makes some more noise, pulling in $120M Se­ries B less than a month af­ter Mer­ck deal

Not even one month after Big Pharma took notice of Artiva when Merck signed a collaboration worth nearly $2 billion in milestones, the off-the-shelf NK cell biotech already has its next big fundraise.

Artiva returns from the venture well Friday with a $120 million Series B round, money they will use to get their first program into the clinic and to file INDs for another two candidates. The raise marks the latest development in a rapidly expanding footprint for Artiva, which, in addition to the Merck deal last month, has now raised almost $200 million since its Series A last June.

With dust set­tled on ac­tivist at­tack, Lau­rence Coop­er leaves Zio­pharm to a new board

Laurence Cooper has done his part.

In the five years since he left a tenured position at Houston’s MD Anderson Cancer Center to become CEO of Boston-based Ziopharm, he’s steered the small-cap immunotherapy player through patient deaths in trials, clinical holds, short attacks and, most recently, an activist attack on the board.

So when the company has “fantastic news” like an IND clearance for a TCR T cell therapy program, he’s ready to pass on the baton.

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Doug Ingram (file photo)

Why not? Sarep­ta’s third Duchenne MD drug sails to ac­cel­er­at­ed ap­proval

Sarepta may be running into some trouble with its next-gen gene therapy approach to Duchenne muscular dystrophy. But when it comes to antisense oligonucleotides, the well-trodden regulatory path is still leading straight to an accelerated approval for casimersen, now christened Amondys 45.

We just have to wait until 2024 to find out if it works.

Amondys 45’s approval was unceremonious, compared to its two older siblings. There was no controversy within the FDA over approving a drug based on a biomarker rather than clinical benefit, setting up a powerful precedent that still haunts acting FDA commissioner Janet Woodcock as biotech insiders weighed her potential permanent appointment; no drama like the FDA issuing a stunning rejection only to reverse its decision and hand out an OK four months later, which got more complicated after the scathing complete response letter was published; no anxious tea leaf reading or heated arguments from drug developers and patient advocates who were tired of having corticosteroids as their loved ones’ only (sometimes expensive) option.

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Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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