Jennifer Doudna and Jonathan Weissman. BARBARA RIES for UCSF

GSK's Bar­ron joins hands with Doud­na, Weiss­man to de­ploy CRISPR tech in drug dis­cov­ery

When Hal Bar­ron un­veiled his grand plan to turn Glax­o­SmithK­line’s R&D group around, he cham­pi­oned ge­net­ics as one of two key fo­cus­es for the UK phar­ma gi­ant, an­nounc­ing a dis­cov­ery deal with 23andme and pledg­ing to fo­cus more on CRISPR tech as well as ma­chine learn­ing. A year lat­er, the R&D chief is build­ing on that promise as he seals a new pact with two promi­nent CRISPR re­searchers in his neigh­bor­hood in San Fran­cis­co.

Jen­nifer Doud­na and Jonathan Weiss­man, both in­ves­ti­ga­tors at Howard Hugh­es Med­ical In­sti­tute, are throw­ing their weight be­hind a new col­lab­o­ra­tion space dubbed the Lab­o­ra­to­ry for Ge­nomics Re­search.

“LGR is about build­ing that space where cre­ative sci­ence is part­nered with the de­vel­op­ment of ro­bust tech­nol­o­gy that will help de­vel­op to­mor­row’s drugs,” Doud­na, who pi­o­neered the use of CRISPR gene edit­ing tool in hu­mans, said in a state­ment. “I think we’re go­ing to be able to do sci­ence that none of us can even imag­ine to­day.”

Hal Bar­ron GSK

Click on the im­age to see the full-sized ver­sion

Un­like the CRISPR biotechs that Doud­na has helped found, though, LGR will use the tools not to cre­ate ther­a­peu­tics or di­ag­nos­tics, but to un­cov­er how ex­act­ly gene mu­ta­tions cause dis­ease. By tin­ker­ing with one gene at a time and then com­par­ing the da­ta on a mas­sive scale, the goal is to iden­ti­fy po­ten­tial tar­gets for ge­net­ic dis­eases.

In par­tic­u­lar, the lab will fo­cus on im­munol­o­gy, on­col­o­gy and neu­ro­science.

This marks the sec­ond ini­tia­tive that Weiss­man — a func­tion­al ge­nomics ex­pert at UCSF— is di­rect­ing with fel­low Howard Hugh­es Med­ical In­sti­tute in­ves­ti­ga­tor Doud­na, who’s based out of UC Berke­ley. The duo are co-di­rec­tors of the In­no­v­a­tive Ge­nomics In­sti­tute, which has a broad mis­sion to im­prove pub­lic health with CRISPR.

Be­tween the two UC in­sti­tu­tions 24 full-time staffers will be hired at the LGR, work­ing along­side 14 GSK em­ploy­ees in a space near UCSF’s Mis­sion Bay cam­pus and run­ning on a $67 mil­lion bud­get over five years. They will get a hand from GSK’s ar­ti­fi­cial in­tel­li­gence and ma­chine learn­ing group on build­ing the tools need­ed to an­a­lyze all the da­ta. Chris Miller, the new head of func­tion­al ge­nomics GSK scooped from Ab­b­Vie, will al­so help steer the ship.

The an­nounce­ment to­day is a cul­mi­na­tion of dis­cus­sions and ne­go­ti­a­tions be­tween GSK and the two uni­ver­si­ties for over a year, Bar­ron told STAT. Not long af­ter be­gin­ning his cur­rent CSO role, he sat down with Doud­na about har­ness­ing the lat­est CRISPR ad­vances for drug dis­cov­ery, and with­in min­utes there was “in­cred­i­ble ex­cite­ment” around the project.

For the drug­mak­er, it is an op­por­tu­ni­ty to test a hy­brid mod­el bring­ing in­dus­try and acad­e­mia un­der one roof, in­stead of sim­ply li­cens­ing tech or fos­ter­ing biotech spin­outs. Aside from cer­tain ex­clu­sive li­cens­es for GSK, the tools cre­at­ed in the lab will be ac­ces­si­ble to oth­er re­searchers.

“One of our key goals is to ad­vance the field over­all and make these tools as broad­ly avail­able as pos­si­ble,” Weiss­man said. “The LGR screen­ing cen­ter will en­able labs at UCSF and Berke­ley. Hav­ing ac­cess to it will give our sci­en­tists op­por­tu­ni­ties to ad­vance their re­search in ways that would be very hard for them to do in their own labs.”

Im­age: Jen­nifer Doud­na Jonathan Weiss­man by Bar­bara Ries for UCSF

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.


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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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.

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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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.

Plagued by de­lays, As­traZeneca HQ costs soar to £750M as it edges to­ward 2020 com­ple­tion

In the lat­est up­date on As­traZeneca’s de­lay-prone HQ project, the phar­ma gi­ant re­vealed that the cost of con­struc­tion has swelled to £750 mil­lion ($956 mil­lion) — more than dou­ble the orig­i­nal es­ti­mate in 2013.

The move-in date is still in 2020, a spokesper­son con­firmed, af­ter As­traZeneca pushed pro­ject­ed com­ple­tion from 2016 to 2017, and then to the spring of 2019. While the ini­tial plan called for a £330 mil­lion (then $500 mil­lion) in­vest­ment, the cost bal­looned to £500 mil­lion ($650 mil­lion), and more in the most re­cent up­date.

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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Russ­ian sci­en­tist plans to one-up Jiankui He in cre­at­ing his own CRISPR ba­bies — Na­ture

If De­nis Re­brikov has his way, the world could be ex­pect­ing more CRISPR ba­bies soon.

The Russ­ian sci­en­tist has told Na­ture he is con­sid­er­ing fol­low­ing Jiankui He’s ex­am­ple in knock­ing out the CCR5 gene in em­bryos and im­plant­i­ng them in­to women — ex­cept do­ing it in a bet­ter way. It marks the first de­c­la­ra­tion of in­ter­est in con­tin­u­ing the work when re­searchers around the world are call­ing for sus­pen­sion of hu­man germline edit­ing and stricter stan­dards, fol­low­ing a glob­al back­lash against He’s claims that he fa­cil­i­tat­ed the birth of twin girls who had been CRISPR-ed as em­bryos.