Beam Ther­a­peu­tics has pro­vid­ed a much-await­ed up­date on its ther­a­py that us­es a form of CRISPR called base edit­ing to pre­cise­ly fix a sin­gle ge­net­ic ty­po re­spon­si­ble for a rare ge­net­ic dis­ease.

On Wednes­day, Beam said it has now treat­ed 29 pa­tients with al­pha-1 an­tit­rypsin de­fi­cien­cy (AATD), a con­di­tion caused by a bro­ken en­zyme, AAT, that is nor­mal­ly made in the liv­er and is vi­tal for pro­tect­ing the lungs.

In pa­tients who got the sec­ond-high­est dose of Beam’s treat­ment, the cor­rect­ed form of the en­zyme made up 94% of the to­tal en­zyme cir­cu­lat­ing in their blood, and lev­els of the bro­ken en­zyme dropped by 84%.

Boehringer In­gel­heim said it plans to keep up high lev­els of R&D spend­ing, and could look at Chi­na-based com­pa­nies for deal­mak­ing, as key planks of its strate­gic ap­proach this year, board chair­man Shashank Desh­pande said Wednes­day.

Desh­pande was dis­cussing the com­pa­ny's 2025 per­for­mance on a call with mem­bers of the me­dia. Last year, the Ger­man bio­phar­ma’s hu­man drug sales grew 7.4% on a con­stant cur­ren­cy ba­sis to €22.7 bil­lion ($26.3 bil­lion), and the com­pa­ny ex­pects a sim­i­lar trend this year.

“We ex­pect to grow this year on a com­pa­ra­ble ba­sis, ad­just­ed for cur­ren­cy and ex­tra­or­di­nary ef­fects,” said Desh­pande, who al­so heads Boehringer’s hu­man phar­ma busi­ness unit.

Four­teen years ago, a group of Har­vard Busi­ness School stu­dents launched a start­up called Vaxess Tech­nolo­gies with a goal to dis­rupt the vac­cine in­dus­try by re­plac­ing nee­dles with a sim­ple skin patch.

Get­ting the tech­nol­o­gy to work was nev­er easy. And just as the start­up was be­gin­ning to make progress, times got tough for vac­cine de­vel­op­ers. As­traZeneca, which was eval­u­at­ing how mR­NA vac­cines worked in the start­up’s skin patch­es, qui­et­ly ter­mi­nat­ed its part­ner­ship last year. But Vaxess had an­oth­er trick up its sleeve.

The Boston-based com­pa­ny be­gan work to see if it could use the patch­es — based on dis­solv­ing mi­cronee­dles that slip be­neath the sur­face of the skin — to de­liv­er semaglu­tide, the GLP-1 ag­o­nist in Ozem­pic and We­govy.

Sarep­ta Ther­a­peu­tics re­port­ed promise in ini­tial find­ings from clin­i­cal stud­ies of two RNA mus­cle dis­ease treat­ments, as the com­pa­ny maps out a fu­ture be­yond its con­tro­ver­sial Duchenne mus­cu­lar dy­s­tro­phy ther­a­pies.

Sarep­ta is de­vel­op­ing RNA treat­ments for two forms of mus­cu­lar dy­s­tro­phy: fa­cioscapu­lo­humer­al mus­cu­lar dy­s­tro­phy (FSHD) and my­oton­ic dy­s­tro­phy (DM1). Both pro­grams stem from Sarep­ta’s part­ner­ship with Ar­row­head Phar­ma­ceu­ti­cals, which has be­come cru­cial for Sarep­ta’s pipeline of ex­per­i­men­tal treat­ments.

On Wednes­day, Sarep­ta re­port­ed drug con­cen­tra­tion da­ta with an ini­tial dose. In both dis­eases, the re­sults showed a dose re­sponse, with high­er lev­els of the drug in pa­tients' blood with high­er dos­es. The com­pa­ny al­so said that it saw far high­er con­cen­tra­tion of its treat­ments in the mus­cle com­pared to oth­er RNA treat­ments un­der de­vel­op­ment, though cross-tri­al com­par­isons come with sev­er­al caveats.