Mus­tang Bio ven­tures in­to gene ther­a­py, li­cens­ing 'bub­ble boy dis­ease' treat­ment from St. Jude

When Mus­tang Bio $MBIO inked the lease deal for its Worces­ter, MA man­u­fac­tur­ing fa­cil­i­ty last year, CEO Man­ny Litch­man said a more cost ef­fec­tive man­u­fac­tur­ing ap­proach would lend a com­pet­i­tive edge to their ear­ly-stage CAR-T pro­grams. As it turns out, gene ther­a­py will al­so be part of that game plan.

Li­cens­ing from St. Jude Chil­dren’s Re­search Hos­pi­tal in Mem­phis, Mus­tang adds an ex vi­vo lentivi­ral gene ther­a­py to for one form of “bub­ble boy dis­ease” to its pipeline, which has up till now been stocked with can­cer-fight­ing cell ther­a­pies.

Manuel Litch­man

“With our team’s ex­ten­sive ex­per­tise in vi­ral vec­tor de­sign, man­u­fac­tur­ing and trans­duc­tion, we are build­ing a ful­ly in­te­grat­ed cell and gene ther­a­py com­pa­ny, with the goal of lever­ag­ing the trans­for­ma­tive po­ten­tial of these tech­nolo­gies to bring life-sav­ing treat­ments to pa­tients in need,” Litch­man said in a state­ment.

For­mal­ly called se­vere com­bined im­mun­od­e­fi­cien­cy (SCID), “bub­ble boy syn­drome” comes in sev­er­al vari­a­tions based on the ex­act cause for the ab­nor­mal func­tions of T and B cells. St. Jude’s ther­a­py, de­vel­oped in the lab of its di­rec­tor of ex­per­i­men­tal hema­tol­ogy Bri­an Sor­renti­no, tar­gets the most com­mon form: X-linked SCID.

Bri­an Sor­renti­no

It works by ge­net­i­cal­ly mod­i­fy­ing a pa­tient’s own blood stem cells and re­in­fus­ing them, in a process that starts with giv­ing the pa­tients a low dose of the can­cer drug busul­fan. Ul­ti­mate­ly, the treat­ment is de­signed to en­able “im­mune sys­tem re­con­sti­tu­tion and the res­o­lu­tion of dis­sem­i­nat­ed in­fec­tions” so that the pa­tients not on­ly live be­yond in­fan­cy but do so with­out reg­u­lar­ly re­ceiv­ing in­tra­venous im­munoglob­u­lin.

The idea is not ex­act­ly new. Glax­o­SmithK­line had been mar­ket­ing Strimvelis, its own gene ther­a­py aimed at a less com­mon vari­a­tion of the dis­ease called ADA-SCID, for more than a year be­fore hand­ing it off to Or­chard Ther­a­peu­tics, a UK biotech up­start run by some of its for­mer staffers who helped de­vel­op it.

Just this morn­ing, Or­chard un­veiled its $150 mil­lion mega-round to bring that treat­ment, and its oth­er ex­per­i­men­tal ther­a­pies, to a broad­er mar­ket faster.

Mean­while, the new part­ners at Mus­tang and St. Jude will fo­cus on push­ing through their own Phase I/II clin­i­cal tri­als. In the one in­volv­ing pa­tients un­der age 2, eight have al­ready been treat­ed to en­cour­ag­ing ef­fects.

“Our ther­a­py has been well tol­er­at­ed thus far, and none of the in­fants re­quired any blood prod­uct sup­port af­ter low dose of busul­fan,” said Eweli­na Mam­carz, who’s lead­ing the study. “Most im­por­tant­ly, we ob­serve re­cov­ery of all cells of the im­mune sys­tem, which is tru­ly an achieve­ment over pri­or gene ther­a­py tri­als, where B cell re­con­sti­tu­tion did not oc­cur, and pa­tients re­quired in­tra­venous im­munoglob­u­lin for life.”

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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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.

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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J&J gains an en­thu­si­as­tic en­dorse­ment from Pres­i­dent Don­ald Trump for their big new drug Spra­va­to

Pres­i­dent Don­ald Trump has lit­tle love for Big Phar­ma, but there’s at least one new drug that just hit the mar­ket which he is en­am­ored with.

Trump, ev­i­dent­ly, has been read­ing up on J&J’s new an­ti-de­pres­sion drug, Spra­va­to. And the pres­i­dent — who of­ten likes to break out in­to a full-throat­ed at­tack on greedy drug­mak­ers — ap­par­ent­ly en­thused about the ther­a­py in a meet­ing with of­fi­cials of Vet­er­ans Af­fairs, which has long grap­pled with de­pres­sion among vet­er­ans.

Savara shares are crushed as PhI­II tri­al flunks pri­ma­ry, key sec­on­daries — but they can’t stop be­liev­ing

In­vestors are in no mood to hear biotechs tout the suc­cess of a “key” sec­ondary end­point when the piv­otal Phase III flunks the pri­ma­ry goal. Just ask Savara. 

The Texas biotech $SVRA went look­ing for a sil­ver lin­ing as com­pa­ny ex­ecs blunt­ly con­ced­ed that Mol­gradex, an in­haled for­mu­la­tion of re­com­bi­nant hu­man gran­u­lo­cyte-macrophage colony-stim­u­lat­ing fac­tor (GM-CSF), failed to spur sig­nif­i­cant­ly im­proved treat­ment out­comes for pa­tients with a rare res­pi­ra­to­ry dis­ease called au­toim­mune pul­monary alve­o­lar pro­teinosis, or aPAP.

As an­oth­er an­tibi­otics biotech sinks in­to a cri­sis, warn­ings of a sec­tor ‘col­lapse’

Another antibiotics company is scrambling to survive today, forcing the company’s founding CEO to exit in a reorganization that eliminates its research capabilities as the survivors look to improve on minuscule sales of their newly approved treatment. And the news — on top of an alarming series of failures — spurred at least one figure in the field to warn of a looming collapse of the antimicrobial resistance research field.

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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.