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EU stamps his­toric OK on blue­bird’s gene ther­a­py for β-tha­lassemia — now sit back and wait for the price

It’s of­fi­cial: blue­bird bio has scored its first drug ap­proval with an EU mar­ket­ing au­tho­riza­tion for its gene ther­a­py for trans­fu­sion-de­pen­dent β-tha­lassemia.

Zyn­te­glo is the new name for a treat­ment once known as Lenti­Glo­bin, a process that takes CD34+ cells from a pa­tient’s hematopoi­et­ic stem cells, in­serts func­tion­al copies of the βA-T87Q-glo­bin gene, then re­in­fus­es the mod­i­fied HSCs. Pa­tients are giv­en chemother­a­py to prime their bone mar­rows be­fore the re­in­fu­sion.

Atara brings on No­var­tis vet to re­place founder Ciechanover as chief

Af­ter found­ing T-cell im­munother­a­py com­pa­ny Atara Bio­ther­a­peu­tics us­ing Am­gen $AMGN drug as­sets in 2012, Isaac Ciechanover made a sur­prise de­ci­sion to leave the com­pa­ny this Jan­u­ary. No­var­tis vet­er­an Pas­cal Tou­chon is set to re­place him as chief, the South San Fran­cis­co biotech said on Tues­day.

Ex­ist­ing CAR-T ther­a­pies — No­var­tis’ $GILD Kym­ri­ah and Gilead’s $GILD Yescar­ta — tar­get a sin­gle anti­gen called CD19. Atara — akin to oth­er play­ers in pur­suit of next-gen CAR-T drugs — is work­ing on en­gi­neer­ing T cells (a type of white blood cell) to tack­le mul­ti­ple tar­gets for ad­dress­ing can­cers with di­verse cell types that of­ten be­come re­sis­tant to treat­ment, such as acute myel­oge­nous leukemia and B cell ma­lig­nan­cies.

No­var­tis rolls out its next big drug at a record-set­ting price of $2.12M — let the gene ther­a­py pric­ing de­bate be­gin

Let the next big debate over gene therapy pricing begin now.

Novartis announced midday Friday that the FDA had stamped its OK on Zolgensma, a groundbreaking gene therapy for lethal cases of spinal muscular atrophy that will test the market’s acceptance of 7-figure prices.

After years of discussion and debate, Novartis set the price at $2.12 million spread out over a 5-year installment plan.

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An­oth­er gene ther­a­py com­pa­ny launch­es with VC cash and a CRISPR tech to tar­get tox­ic RNA

A San Diego-based biotech has at­tract­ed sub­stan­tial ven­ture sup­port for its clin­i­cal for­ay in­to gene ther­a­pies that can tar­get tox­ic brands of RNA — sparked by DNA mu­ta­tions — that trig­ger ge­net­ic dis­eases. And they’ve wooed a well-known West Coast en­tre­pre­neur to the helm.

The biotech is called Lo­cana, which is work­ing on the sci­en­tif­ic ex­plo­rations done by UC San Diego pro­fes­sor Gene Yeo. Yeo’s pub­lished work in­cludes lab ex­per­i­ments that used CRISPR Cas9 tech to mod­i­fy repet­i­tive RNA —an ap­proach with im­pli­ca­tions for some tough dis­eases like my­oton­ic dy­s­tro­phy type 1 and 2, Hunt­ing­ton’s dis­ease, and C9orf72-linked amy­otroph­ic lat­er­al scle­ro­sis. That work was pub­lished in Cell.

David Hal­lal brings his first cell ther­a­py com­pa­ny in­to the fold, and it's tied to a ma­jor league fi­nanc­ing deal

David Hal­lal wasn’t kid­ding around when he said that his start­up El­e­vate­Bio was ready to do busi­ness.

The ex-Alex­ion CEO is un­veil­ing his first port­fo­lio deal for his $150 mil­lion cell and gene ther­a­py start­up El­e­vate­Bio this morn­ing. And it’s tied in­to a ma­jor league fi­nanc­ing round that puts Hal­lal’s team right on the thresh­old of a Phase III cam­paign, with an im­me­di­ate need to ramp up man­u­fac­tur­ing and start lay­ing the foun­da­tion for po­ten­tial com­mer­cial work.

Can­cer re­search groups urge FDA to over­haul stan­dards for ear­ly cell ther­a­py tri­als, cut­ting time and cost. But will 'pre-com­pet­i­tive' al­liances fly?

Two influential nonprofits focused on cancer drug R&D are urging the FDA to take a more flexible approach to clinical work to help triage an influx of new cell and gene cancer therapies making their way to human studies. By changing current IND and manufacturing standards, they say, the agency can cut the time and cost of early-stage work, accelerating the advance of the most promising therapies in the pipeline. 

But can they get the industry to agree on data sharing and pre-competitive alliances?

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Reg­u­la­to­ry in­tel­li­gence: Up­date on re­gen­er­a­tive med­i­cine ad­vanced ther­a­pies des­ig­na­tions

This ar­ti­cle dis­cuss­es the scope and pur­pose of the spe­cial des­ig­na­tion for Re­gen­er­a­tive Med­i­cine Ad­vanced Ther­a­pies (RMAT) cre­at­ed by the pas­sage of the 21st Cen­tu­ry Cures Act. The au­thors ex­plain the ben­e­fits ex­pect­ed to be re­al­ized with RMAT, such as keep­ing the US glob­al­ly com­pet­i­tive in the field. They pro­vide a tal­ly of prod­ucts re­ceiv­ing the spe­cial des­ig­na­tion to date and a cur­rent count, by year, of prod­ucts for which RMAT des­ig­na­tion has been re­quest­ed.

Sol­id Bio’s high­er gene ther­a­py dose for Duchenne MD runs smack in­to a fresh set of safe­ty is­sues -- shares tum­ble

Solid Biosciences has just ricocheted into a new setback.A few months after reporting dismal responses from a small cohort of patients receiving its gene therapy for Duchenne muscular dystrophy, the biotech had a chance to increase the dose to what it felt would be an ideal level — and immediately ran into a serious adverse event that had to be reported to the FDA.

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Bay­er kicks off a $150M add-on for its cam­pus in Berke­ley

Bay­er is blue­print­ing a new pro­duc­tion fa­cil­i­ty to add to its cam­pus in the Bay Area with a $150 mil­lion bud­get to com­plete the task.

On Thurs­day the com­pa­ny an­nounced that it will build a be­spoke 40,000-square-foot fa­cil­i­ty on its Berke­ley cam­pus, where they al­ready pro­duce he­mo­phil­ia A prod­ucts. The plan is to add on clin­i­cal pro­duc­tion op­er­a­tions at the new Cell Cul­ture Tech­nol­o­gy Cen­ter to sup­port their drug re­search work in on­col­o­gy, car­di­ol­o­gy and some oth­er spe­cial­ty health en­deav­ors.

Sarep­ta goes back to the gene ther­a­py well at Na­tion­wide Chil­dren’s for its lat­est mus­cle-wast­ing pro­gram

Sarep­ta $SRPT is adding a pre­clin­i­cal gene ther­a­py pro­gram to the pipeline, pick­ing it up from their close con­tacts at Na­tion­wide Chil­dren’s Hos­pi­tal. The treat­ment is cal­pain 3 (CAPN-3), for Limb-gir­dle mus­cu­lar dy­s­tro­phy (LGMD) type 2A, which trig­gers the dis­ease as­so­ci­at­ed with weak­ness in mus­cles in the hips and shoul­ders.


This is the biotech’s 6th LGMD ef­fort, which us­es an AAVrh74 vec­tor to tar­get skele­tal mus­cle. The work so far has been led by Zarife Sa­henk, di­rec­tor of Clin­i­cal and Ex­per­i­men­tal Neu­ro­mus­cu­lar Pathol­o­gy at the Re­search In­sti­tute at Na­tion­wide Chil­dren’s in Ohio.