New Mechanism Inogona Kudzoreredza Zvakakwana Basa reCFTR muCystic Fibrosis

Sionna Therapeutics nhasi yakazivisa kuvhurwa zviri pamutemo kwekambani uye kuvharwa kwemari inosvika zviuru zana negumi zvemadhora Series B. Kutenderera kwacho kwakatungamirwa neOrbiMed nekutora chikamu kubva kumari yakarairwa neT. Rowe Price Associates, Inc., Q Healthcare Holdings, LLC., chikamu chese cheQIA, homwe yehupfumi yeQatar, nevaimboisa mari vanosanganisira RA Capital, TPG's. Iyo Rise Fund, Atlas Venture, uye Cystic Fibrosis Foundation. Sionna yakwidza mari inosvika zana nemakumi mashanu emamiriyoni emadhora kusvika parizvino.

Sionna iri kufambisira mberi pombi yemamorekuru madiki ekutanga-mu-kirasi akagadzirirwa kudzoreredza zvizere basa recystic fibrosis transmembrane conductance regulator (CFTR) protein ine hurema muCF, nekudzikamisa CFTR yekutanga nucleotide-binding domain (NBD1). Chikonzero chikuru cheCF ndiyo genetic mutation ΔF508 inobata NBD1 kugadzikana uye CFTR basa.

Mumakiriniki ekufungidzira mu vitro CF modhi, Sionna's NBD1-yakanangwa mamorekuru madiki, pamwe chete nemamwe anoenderana modulators, yakaratidza kugona kugadzirisa kupeta, kukura, uye kugadzikana kweCFTR protein yakakanganiswa neAF508 genetic mutation. Izvi zvinobvumira kutengesa kwakakodzera kweCFTR kune sero pamusoro uye yakajairwa mutemo wekuyerera kwemaion nemvura. Nekudzoreredza zvizere basa reCFTR kune varwere vane ΔF508 genetic mutation kusvika kumatanho anoonekwa mumasero evanhu vasina CF, pombi yeSionna ine mukana wekuunza zvakanakisa-mu-kirasi kushanda uye yakakwana yekiriniki inobatsira kune vanhu vane CF.

“NBD1 inzvimbo inozivikanwa uye yakaongororwa, asi yave kunzi haigoneke kusvika parizvino. Zvichienderana nekuedza kwedu kwakanangana nekufambira mberi paNBD1, tinoona mukana wekugadzirisa kushanda kweCFTR muhuwandu hwevanhu vane CF, "akadaro Mike Cloonan, Mutungamiriri uye Chief Executive Officer weSionna Therapeutics. “Basa redu kuSiona nderekusimudzira zvakanyanya hutano nemhando yehupenyu kune vanhu vanoramba vachitambura nemhedzisiro yehupenyu hwese uye mutoro wekugara neCF. Nerubatsiro urwu rwemari, sangano redu rakasimba, uye timu yedu ine ruzivo uye ine tarenda, tinofara kuburitsa Sionna uye takatarisana nekusimudzira macompounds ekutanga kubva papiipi yedu yakapatsanurwa kupinda mukiriniki.

CF chirwere chakakomba, chinogona kuuraya chinokonzerwa nekuchinja kweiyo CFTR gene iyo inotungamira mukuungana kwemukosi mumapapu nemhepo, kukanganiswa kwepancreatic basa uye kumwe kusashanda kwenhengo kunogona kuve kwakakomba uye kazhinji kwakanyanya kukanganisa hutano nehupenyu. kutarisira. Kune vanopfuura 100,000 vanhu vanogara neCF pasi rose, ii, inofungidzirwa makumi mapfumbamwe kubva muzana avo vane genetic mutation ΔF90 inoitika mukati meNBD508 domain yeCFTR. Kuchinja uku kunoita kuti NBD1 ibude patembiricha yemuviri uye inokanganisa kushanda kweCFTR. Pasinei nekuvapo kwemishonga inobvumirwa iye zvino uye kufambira mberi kukuru kwakaitwa pane zvimwe zvinangwa mukati meCFTR, vanhu vazhinji vane ΔF1 havaiti basa rakazara reCFTR. NBD508 yakakosha kugadziridza basa reCFTR uye kugadzirwa kweane hutano, anoyerera akasununguka mucus mumhepo, digestive system, uye dzimwe nhengo.