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.

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.

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 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.

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.

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 $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.