In an­oth­er CRISPR first, Penn re­searchers dose US can­cer pa­tients with gene-edit­ed T cell ther­a­py

CRISPR has been test­ed in US pa­tients for the first time — but per­haps not in the man­ner you imag­ined.

The Uni­ver­si­ty of Penn­syl­va­nia has led the way dos­ing two pa­tients with re­lapsed can­cers — one with mul­ti­ple myelo­ma and one with sar­co­ma — with a T cell ther­a­py in which the T cell re­cep­tor and PD-1 pro­tein are edit­ed out.

Us­man “Oz” Azam

Fund­ing for the tri­al came part­ly from Tmu­ni­ty, a biotech co-found­ed by Penn’s CAR-T vi­sion­ary Carl June and helmed by No­var­tis vet Us­man “Oz” Azam. Park­er In­sti­tute for Can­cer Im­munother­a­py, an ear­ly in­vestor in Tmu­ni­ty, is al­so bankrolling the tri­al. A Penn spokesper­son con­firmed the news, which was first re­port­ed by NPR.

The ba­sic idea here is not un­like CAR-T, ex­cept with dif­fer­ent el­e­ments and tech­nolo­gies: Take a pa­tient’s T cells, en­gi­neer it to ex­press NY-ESO-1 TCR, while elim­i­nat­ing TCR and PD-1 orig­i­nal­ly on the sur­face us­ing CRISPR.

A group of UCLA and UCSF sci­en­tists backed by the Park­er In­sti­tute has been study­ing new tech­niques to get new genes in­to T cells — not just elim­i­nat­ing them — with CRISPR.

That’s not the most straight­for­ward way of ap­ply­ing the gene edit­ing tech, which has en­ticed oth­er drug de­vel­op­ers with the po­ten­tial to el­e­gant­ly fix ge­net­ic er­rors lead­ing to dis­ease. But it does speak to the ver­sa­til­i­ty of CRISPR, now a pop­u­lar­ized tool that’s spawned up­starts rang­ing from di­ag­nos­tics to tar­get­ed bac­te­ria erad­i­ca­tion.

Carl June

Nev­er­the­less, the trio of CRISPR pi­o­neers as­so­ci­at­ed — Ed­i­tas Med­i­cines, In­tel­lia Ther­a­peu­tics and CRISPR Ther­a­peu­tics — re­main the ones to watch for first-in-hu­man test­ing. In fact, just two months ago CRISPR Ther­a­peu­tics dosed the first pa­tient with its Ver­tex-part­nered be­ta tha­lassemia drug. It is al­so plan­ning to treat a sick­le cell dis­ease pa­tient with the same drug in mid-2019 for a sep­a­rate tri­al.

Of course, that’s as­sum­ing we’re talk­ing out­side of Chi­na, where in­fu­sions of CRISPR-edit­ed T cells in can­cer pa­tients have been tak­ing place for more than a year. In a stun­ner, Chi­nese sci­en­tist Jiankui He claimed the birth of ba­by twins who were ge­net­i­cal­ly mod­i­fied as fe­tus­es, stir­ring up wide­spread con­dem­na­tion and soul search­ing in the sci­en­tif­ic com­mu­ni­ty.

“2019 is the year when the train­ing wheels come off and the world gets to see what CRISPR can re­al­ly do for the world in the most pos­i­tive sense,” Fy­o­dor Urnov, a gene-edit­ing sci­en­tist at the Al­tius In­sti­tute for Bio­med­ical Sci­ences in Seat­tle and the Uni­ver­si­ty of Cal­i­for­nia, Berke­ley, told NPR.

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.

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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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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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.

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.