Gene edit­ing up­start backed by Broad star Feng Zhang nabs $135M, blue­print­ing a plat­form biotech for the long haul

Im­age: John Evans. BEAM

Last May, when Beam Ther­a­peu­tics had its com­ing out par­ty, com­plete with $87 mil­lion in in­vest­ment back­ing, a sol­id core of 15 staffers and a dream team of sci­en­tif­ic co-founders, CEO John Evans was sure they were set for sev­er­al years of R&D work.

But that’s not the way it played out. 

At a time when the mon­ey spig­ot is wide open for the right biotech plans and teams, Beam is an­nounc­ing that it went back to the ven­ture well to flesh out its syn­di­cate and added a whop­ping $135 mil­lion mon­ster round to the bank. That core team has swelled to 70, with more hires on the way as the ex­ec­u­tive team pre­pares to host a staff of 130. And Evans says they are well on their way to cre­at­ing a pipeline of pro­grams be­gin­ning with the 10 pre­clin­i­cal ef­forts they have un­der­way in new­ly opened labs.

“We de­cid­ed to go broad,” says Evans. “There are like­ly a lot of places for this be­ing the best op­tion for pa­tients.”

The big idea at Beam is that in­stead of us­ing a spe­cial gene edit­ing cut­ting tool to do per­ma­nent sur­gi­cal al­ter­ations of DNA, they’re pen­cilling in their ed­its with base edit­ing en­zymes that mod­i­fy nu­cle­obas­es  — cor­rect­ing or mod­i­fy­ing dis­ease caus­ing genes or writ­ing in code for ge­net­ic vari­a­tions that can pre­vent dis­ease. It’s one of a group of next-wave gene edit­ing out­fits that have come along in the wake of pi­o­neers like Ed­i­tas, In­tel­lia or CRISPR Ther­a­peu­tics. 

The Broad In­sti­tute’s Feng Zhang, who has backed Ed­i­tas and oth­er up­starts in the field, joined with Broad col­league David Liu and Har­vard Med’s Kei­th Joung to pro­vide the sci­en­tif­ic in­spi­ra­tion.


Im­age: David Liu, Kei­th Joung, Feng Zhang. BEAM

At this point in Beam’s ex­is­tence, go­ing broad means stick­ing with gen­er­al­i­ties and avoid­ing specifics about the work they’re do­ing in the lab. That’s stan­dard op­er­at­ing pro­ce­dure in start­up land. The de­tails can start to come lat­er as they be­gin to pub­lish their work and line up INDs for the first clin­i­cal pro­grams now in the works.

The plan now is to tran­si­tion from their plat­form con­cept in­to a pipeline sto­ry, says Evans. And they’re adding AAV vec­tors, lipid nanopar­ti­cles and oth­er kinds of tech that can get their ed­its wher­ev­er they need to go in the body.

“We’re not go­ing to throw this over the wall to a phar­ma com­pa­ny,” adds the CEO, adding that the team at Beam in­tends to build it all in house from end-to-end — much like they did at Agios, where he was SVP of cor­po­rate de­vel­op­ment.

Go­ing all the way means in­vest­ing heav­i­ly in man­u­fac­tur­ing ear­ly on and lay­ing the foun­da­tion for an even­tu­al shift to com­mer­cial­iza­tion. And that means plan­ning for the long haul, with lots of cap­i­tal re­quired to make it a re­al­i­ty.

That takes a syn­di­cate of back­ers ready for the long haul as well. And here’s who’s back­ing the B round: new in­vestors Red­mile Group, LLC; Cor­morant As­set Man­age­ment; GV; Al­ti­tude Life Sci­ence Ven­tures, and “ad­di­tion­al undis­closed in­vestors.” Then there’s al­so new mon­ey from ex­ist­ing in­vestors: F-Prime Cap­i­tal, ARCH Ven­ture Part­ners, Eight Roads Ven­tures, and Omega Funds.

Nick Leschly via Getty

UP­DAT­ED: Blue­bird shares sink as an­a­lysts puz­zle out $1.8M stick­er shock and an un­ex­pect­ed de­lay

Blue­bird bio $BLUE has un­veiled its price for the new­ly ap­proved gene ther­a­py Zyn­te­glo (Lenti­Glo­bin), which came as a big sur­prise. And it wasn’t the on­ly un­ex­pect­ed twist in to­day’s sto­ry.

With some an­a­lysts bet­ting on a $900,000 price for the β-tha­lassemia treat­ment in Eu­rope, where reg­u­la­tors pro­vid­ed a con­di­tion­al ear­ly OK, blue­bird CEO Nick Leschly said Fri­day morn­ing that the pa­tients who are suc­cess­ful­ly treat­ed with their drug over 5 years will be charged twice that — $1.8 mil­lion — on the con­ti­nent. That makes this drug the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ new­ly ap­proved Zol­gens­ma at $2.1 mil­lion, with an­a­lysts still wait­ing to see what kind of pre­mi­um can be had in the US.

Neil Woodford, Woodford Investment Management via YouTube

Un­der siege, in­vest­ment man­ag­er Wood­ford faces an­oth­er in­vest­ment shock

Em­bat­tled UK fund man­ag­er Neil Wood­ford — who has con­tro­ver­sial­ly blocked in­vestors from pulling out from his flag­ship fund to stem the blood­let­ting, af­ter a slew of dis­ap­point­ed in­vestors fled fol­low­ing a se­ries of sour bets — is now pay­ing the price for his ac­tions via an in­vestor ex­o­dus on an­oth­er fund.

Har­g­reaves Lans­down, which has in the past sold and pro­mot­ed the Wood­ford funds via its re­tail in­vest­ment plat­form, has re­port­ed­ly with­drawn £45 mil­lion — its en­tire po­si­tion — from the in­vest­ment man­ag­er’s In­come Fo­cus Fund.

News­mak­ers at #EHA19: Re­gen­eron, Ar­Qule track progress on re­sponse rates

Re­gen­eron’s close­ly-watched bis­pe­cif­ic con­tin­ues to ring up high re­sponse rates

Re­gen­eron’s high-pro­file bis­pe­cif­ic REGN1979 is back in the spot­light at the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion sci­en­tif­ic con­fab. And while the stel­lar num­bers we saw at ASH have erod­ed some­what as more blood can­cer pa­tients are eval­u­at­ed, the re­sponse rates for this CD3/CD20 drug re­main high.

A to­tal of 13 out of 14 fol­lic­u­lar lym­phomas re­spond­ed to the drug, a 93% ORR, down from 100% at the last read­out. In 10 out of 14, there was a com­plete re­sponse. In dif­fuse large B-cell lym­phoma the re­sponse rate was 57% among pa­tients treat­ed at the 80 mg to 160 mg dose range. They were all com­plete re­spons­es. And 2 of these Cars were for pa­tients who had failed CAR-T ther­a­py.

Bain’s biotech team has cre­at­ed a $1B-plus fund — with an eye to more Big Phar­ma spin­outs

One of the biggest investors to burst onto the biotech scene in recent years has re-upped with more than a billion dollars flowing into its second fund. And this next wave of bets will likely include more of the Big Pharma spinouts that highlighted their first 3 years in action.

Adam Koppel and Jeff Schwartz got the new life sciences fund at Bain Capital into gear in the spring of 2016, as they were putting together a $720 million fund with $600 million flowing in from external investors and the rest drawn from the Bain side of the equation. This time the external investors chipped in $900 million, with Bain coming in for roughly $180 million more.

They’re not done with Fund I, with plans to add a couple more deals to the 15 they’ve already posted. And once again, they’re estimating another 15 to 20 investments over a 3- to 5-year time horizon for Fund II.

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Ted Love. HAVERFORD COLLEGE

Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

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Gene ther­a­pies seize the top of the list of the most ex­pen­sive drugs on the plan­et — and that trend has just be­gun

Anyone looking for a few simple reasons why the gene therapy field has caught fire with the pharma giants need only look at the new list of the 10 most expensive therapies from GoodRx.

Two recently approved gene therapies sit atop this list, with Novartis’ Zolgensma crowned the king of the priciest drugs at $2.1 million. Right below is Luxturna, the $850,000 pioneer from Spark, which Roche is pushing hard to acquire as it adds a gene therapy group to the global mix.

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Adding mar­quee in­vestors, Black­Thorn bags $76M to back an AI-dri­ven strat­e­gy for pre­ci­sion neu­ro med­i­cine

As ar­ti­fi­cial in­tel­li­gence and ma­chine learn­ing loom ever larg­er in drug dis­cov­ery and de­vel­op­ment, a biotech op­er­at­ing at the “nexus” of tech­nol­o­gy and neu­ro­sciences has cashed in with $76 mil­lion in fresh fi­nanc­ing.

The big idea at Black­Thorn Ther­a­peu­tics is to do for neu­robe­hav­ioral dis­or­ders what ge­net­i­cal­ly tar­get­ed ther­a­py has done for on­col­o­gy: Re­de­fine pa­tient pop­u­la­tions by the un­der­ly­ing bi­ol­o­gy — dys­reg­u­lat­ed brain cir­cuits, or neu­rotypes — in­stead of symp­toms, there­by find­ing the pa­tients who are most like­ly to ben­e­fit at en­roll­ment phase.

Fol­low­ing CAR-T pi­o­neer­s' foot­steps, Tes­sa launch­es Chi­na JV in $120M deal

These days just about every biotech se­ri­ous about glob­al de­vel­op­ment — and not just com­mer­cial­iza­tion — has a Chi­na strat­e­gy. Tes­sa Ther­a­peu­tics, a Bay­lor as­so­ci­at­ed out­fit based out of Sin­ga­pore, is no ex­cep­tion.

Tak­ing a page out of the CAR-T pi­o­neers’ play­book, Tes­sa is es­tab­lish­ing a joint ven­ture with Chi­na-Sin­ga­pore Guangzhou Knowl­edge City, which is ini­tial­ly putting down $40 mil­lion for a 13% stake with $40 mil­lion more to come in a sec­ond stage. The biotech, which now re­tains an 87% con­trol, is al­so rolling out its own con­tri­bu­tions in two phas­es, start­ing with $20 mil­lion and all its tech­nol­o­gy li­cense rights for Chi­na.

Search­ing for the next block­buster to fol­low Darza­lex, J&J finds a $150M an­ti-CD38 drug from part­ner Gen­mab

Now that J&J and Genmab have thrust Darzalex onto the regulatory orbit for first-line use in multiple myeloma, the partners are lining up a deal for a next-gen follow-on to the leading CD38 drug.


Janssen — J&J’s biotech unit — has its eyes on HexaBody-CD38, a preclinical compound generated on Genmab’s tech platform designed to make drugs more potent via hexamerization.


Genmab is footing the bill on studies in multiple myeloma and diffuse large B-cell lymphoma; once it completes clinical proof of concept, Janssen has the option to license the drug for a $150 million exercise fee. There’s also $125 million worth of milestones in play.

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