Feng Zhang, heavy­weight col­lab­o­ra­tors un­veil lat­est CRISPR up­start — a di­ag­nos­tic com­pa­ny

Im­age: David Walt, Todd Gol­ub, Rahul Dhan­da, Feng Zhang, Deb­o­rah Hung. SHER­LOCK

One of the more dra­mat­ic dy­nam­ics in the aca­d­e­m­ic CRISPR world in­volves the Broad In­sti­tute and the Uni­ver­si­ty of Cal­i­for­nia, where Feng Zhang and Jen­nifer Doud­na, two pi­o­neers of the field, have their labs re­spec­tive­ly. As the dust starts to set­tle on their patent dis­pute re­gard­ing ther­a­peu­tic us­es of the gene edit­ing tech, a new storm is brew­ing in the di­ag­nos­tic ap­pli­ca­tions of CRISPR.

Zhang and eight oth­er lead­ing ex­perts in CRISPR, syn­thet­ic bi­ol­o­gy, di­ag­nos­tics and in­fec­tious dis­eases have pulled back the cur­tain on Sher­lock Bio­sciences, a start­up aimed at pro­vid­ing “bet­ter, faster and more af­ford­able” ways to de­tect ge­net­ic sig­na­tures of dis­ease.

The com­pa­ny stands on two foun­da­tion­al tech­nolo­gies: SHER­LOCK, or Spe­cif­ic High-sen­si­tiv­i­ty En­zy­mat­ic Re­porter un­LOCK­ing, was de­vel­oped by Zhang and col­lab­o­ra­tors in­clud­ing co-founders Jonathan Gooten­berg and Omar Abu­dayyeh. Pair­ing CRISPR with the Cas13 en­zyme, the tech­nol­o­gy uti­lizes a guide RNA to find a ge­net­ic se­quence, where Cas13 not on­ly cuts the tar­get but be­gin shriv­el­ing oth­er RNA near­by, cre­at­ing a sig­nal that can then be pre­sent­ed in ob­serv­able forms — such as a line on a pa­per strip.

“Any one tech­nol­o­gy out there had some re­al­ly valu­able at­trib­ut­es in mol­e­c­u­lar di­ag­nos­tics but SHER­LOCK brought all of those to­geth­er,” Rahul Dhan­da, a co-founder and di­ag­nos­tics vet­er­an who took on the CEO role, told me. “It had speed, so you didn’t have to wait days for an in­fec­tious dis­ease re­sult; it had af­ford­abil­i­ty, which meant that you can do on­col­o­gy test­ing in a way that let every­body par­tic­i­pate in those re­sults; and it had sim­plic­i­ty, which means that you can reach peo­ple to do test­ing that couldn’t do it be­fore in de­cen­tral­ized set­tings.”

First in­vent­ed in 2017, an en­hanced ver­sion of SHER­LOCK was de­scribed in a pa­per pub­lished last Feb­ru­ary along­side an­oth­er ex­plain­ing DE­TEC­TR, a sim­i­lar tool that de­ploys Cas12 and a flu­o­res­cent mol­e­cule for de­tec­tion.

Doud­na of UC Berke­ley, who led the work on DE­TEC­TR, de­buted Mam­moth Bio­sciences short­ly there­after with a group of grad stu­dents, some of them from her lab. When we last heard from them, Mam­moth was work­ing with a $23 mil­lion round and back­ing from May­field, NFX, 8VC as well as Ap­ple’s Tim Cook and Grail found­ing CEO Jeff Hu­ber.

Im­age: Omar Abu­dayyeh, Jonathan Gooten­berg, Rahul Dhan­da, James Collins, Par­dis Sa­beti. SHER­LOCK

While com­par­isons with Mam­moth might be in­evitable, Dhan­da says he’s “very con­fi­dent” on the patent side and has faith in Sher­lock’s mul­ti-plat­form ap­proach to en­gi­neer­ing bi­ol­o­gy.

“Now I’ve been in the in­dus­try 20 years, I know how to nav­i­gate this field; it’s a nu­anced enough field that ex­pe­ri­ence mat­ters,” he added. “I would say that when I think about who we should be fo­cused on as com­pe­ti­tion, those are the large play­ers, the es­tab­lished mul­ti-bil­lion dol­lar di­ag­nos­tic com­pa­nies out there, and those are the com­pa­nies that we in­tend to build strong part­ner­ships with if we can find the right com­ple­ment to our goals and theirs.”

Sher­lock now has $35 mil­lion in the bank — half of it in the form of a grant from the Open Phil­an­thropy Pro­ject, which al­so pro­vid­ed an ad­di­tion­al in­vest­ment. As it ex­plores ap­pli­ca­tions in in­fec­tious dis­eases, pre­ci­sion on­col­o­gy and food in­spec­tion, it is on the look­out for “se­lec­tive part­ner­ing” while ad­vanc­ing de­vel­op­ment pro­grams and de­sign­ing new as­says on its own, the com­pa­ny said. So far, that has in­volved every­thing from phar­ma­ceu­ti­cal com­pa­nies look­ing for com­pan­ion di­ag­nos­tics to an­timi­cro­bial de­vel­op­ers seek­ing help with clin­i­cal tri­als to con­sumer health play­ers in search of at-home test­ing. Agri­cul­ture and bio­process test­ing are among the pos­si­bil­i­ties, too.

Dhan­da is still rais­ing cash for Sher­lock’s Se­ries A, which will al­so help fund the sec­ond plat­form in Sher­lock’s ar­se­nal: IN­SPEC­TR (IN­ter­nal Splint-Pair­ing Ex­pres­sion Cas­sette Trans­la­tion Re­ac­tion). James Collins at the Wyss In­sti­tute was cred­it­ed for the tech, which com­pris­es two halves of a cod­ing re­gion that fuse to cre­ate a sig­nalling pro­tein when the tar­get is present, ba­si­cal­ly repli­cat­ing how a cell would gen­er­ate a pro­tein.

In the com­ing year, Dhan­da is al­so plan­ning to hire 6 to 10 peo­ple to ramp up the R&D work cur­rent­ly han­dled by 10 staffers.

Oth­er co-founders in­clude Todd Gol­ub, Deb­o­rah Hung, Par­dis Sa­beti and David Walt — il­lus­tri­ous aca­d­e­mics who make up a team “like none oth­er.”

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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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.

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.

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.

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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Ab­b­Vie touts new da­ta for Hu­mi­ra suc­ces­sor; Gilead inks dis­cov­ery deal

→ Ab­b­Vie is tout­ing new pos­i­tive da­ta com­par­ing their ag­ing block­buster Hu­mi­ra with their hoped-for block­buster upadac­i­tinib. Over 48 weeks a larg­er pro­por­tion of pa­tients tak­ing the ex­per­i­men­tal drug ex­pe­ri­enced clin­i­cal re­mis­sion than in the con­trol arm with Hu­mi­ra. Their drug brought in $20 bil­lion last year, top­ping the scales in the num­ber 1 slot.

→ Gilead has turned to Van­cou­ver-based Ab­Cellera for its lat­est dis­cov­ery deal. Ab­Cellera will use its know-how in “sin­gle-cell screen­ing of nat­ur­al im­mune sources” to find an­ti­body can­di­dates for Gilead to pur­sue in the in­fec­tious dis­ease field. The deal in­cludes an up­front and mile­stones.