The FDA on Tues­day ap­proved a gene ther­a­py for a rare im­mune dis­ease called Wiskott-Aldrich syn­drome, for which pa­tients have lit­tle op­tions be­sides a bone mar­row trans­plant.

The gene ther­a­py will be mar­ket­ed as Waskyra, and was de­vel­oped by Fon­dazione Telethon, an Ital­ian char­i­ty fo­cused on rare dis­ease re­search. It marks the first ap­proved cell and gene ther­a­py from a non­prof­it ap­pli­cant, ac­cord­ing to FDA.

The gene ther­a­py was pre­vi­ous­ly li­censed by Or­chard Ther­a­peu­tics, which stopped work on the treat­ment in 2022 in an ef­fort to save cash amid lay­offs. It’s one of the many gene ther­a­pies aban­doned by in­dus­try even with promis­ing clin­i­cal da­ta be­cause they may not be prof­itable.

Pa­tients who were giv­en Roche’s ex­per­i­men­tal breast can­cer pill di­rect­ly af­ter hav­ing surgery were 30% less like­ly to have in­va­sive dis­ease re­cur­rence than those who were giv­en stan­dard of care, ac­cord­ing to in­ter­im da­ta pre­sent­ed Wednes­day.

The da­ta should al­low Roche to seek ap­proval of its drug in the ad­ju­vant set­ting, set­ting it up to be­come the first in its class to be ap­proved for post­sur­gi­cal use. But the re­sults are al­so im­por­tant as a cur­tain-rais­er for the read­out of an­oth­er tri­al of the drug, the hot­ly-await­ed per­se­vERA.

The lid­ERA study test­ed giredestrant, a se­lec­tive es­tro­gen re­cep­tor de­grad­er or SERD, in more than 4,000 pa­tients with ear­ly-stage, es­tro­gen re­cep­tor-pos­i­tive, HER2-neg­a­tive breast can­cer. Half the sub­jects were giv­en Roche’s drug and the oth­ers re­ceived their doc­tor’s choice of one of four hor­mone ther­a­pies. They took the med­i­cine for a me­di­an of around two years and eight months.

Plus, news about Sanofi, InduPro Ther­a­peu­tics, De­nali and Bio­cy­to­gen:

📊 John­son & John­son's menin in­hibitor da­ta at #ASH25: Ku­ra and Syn­dax weren’t the on­ly com­pa­nies to present menin in­hibitor com­bi­na­tions in first-line acute myeloid leukemia. J&J said its ex­per­i­men­tal drug blexi­menib elicit­ed high re­sponse rates when giv­en in com­bi­na­tion with in­ten­sive chemother­a­py. Of 24 evalu­able pa­tients in the Phase 1b study, about 80% achieved a com­plete re­sponse with ei­ther full or par­tial hema­to­log­ic re­cov­ery. As ri­vals in the menin space eye ear­ly use of their treat­ments, Jef­feries an­a­lysts wrote that “the abil­i­ty to com­bine safe­ly, min­i­mize mon­i­tor­ing bur­den, and sup­port long-term ad­min­is­tra­tion will like­ly dri­ve mar­ket lead­er­ship in the menin par­ty.” — Nicole De­Feud­is

Since Take­da paid bil­lions for Nim­bus Ther­a­peu­tic­s' pro­gram i