I-New Mechanism Ingakwazi Ukubuyisela Ngokugcwele Umsebenzi we-CFTR ku-Cystic Fibrosis

I-Sionna Therapeutics namuhla imemezele ukwethulwa ngokusemthethweni kwenkampani kanye nokuvalwa kwezimali eziyizigidi eziyi-111 ze-Series B. Umjikelezo ubuholwa yi-OrbiMed ngokubamba iqhaza kwezimali ezilulekwe ngabakwa-T. Rowe Price Associates, Inc., Q Healthcare Holdings, LLC., inkampani ephethwe ngokuphelele ye-QIA, isikhwama somnotho esizimele saseQatar, nabatshalizimali bangaphambilini okuhlanganisa i-RA Capital, i-TPG's. I-Rise Fund, i-Atlas Venture, kanye neCystic Fibrosis Foundation. USionna useqoqe cishe amaRandi ayizigidi eziyi-150 kuze kube manje.

I-Sionna ithuthukela ngepayipi lama-molecule amancane esigaba sokuqala aklanyelwe ukubuyisela ngokugcwele ukusebenza kwephrotheni ye-cystic fibrosis transmembrane conductance regulator (CFTR) enesici ku-CF, ngokuzinzisa isizinda sokuqala se-CFTR esibopha i-nucleotide (NBD1). Imbangela ehamba phambili ye-CF ukuguqulwa kofuzo i-ΔF508 ethinta ukuzinza kwe-NBD1 kanye nomsebenzi we-CFTR.

Kumamodeli e-CF e-in vitro abikezela ngokomtholampilo, ama-molecule amancane e-Sionna e-NBD1 eqondiswe ku-NBD508, ehlangene namanye amamoduli ahambisanayo, abonise amandla okwenza kujwayelekile ukugoqa, ukuvuthwa, nokuzinza kwephrotheni ye-CFTR ethintwa ukuguqulwa kofuzo kwe-ΔF508. Lokhu kuvumela ukushushumbiswa okufanele kwe-CFTR endaweni yeseli kanye nokulawulwa okuvamile kokugeleza kwama-ion namanzi. Ngokubuyisela ngokugcwele ukusebenza kwe-CFTR ezigulini ezinokuguqulwa kofuzo kwe-ΔFXNUMX kumazinga abonwa kumaseli abantu abangenayo i-CF, ipayipi le-Sionna linamandla okuletha ukusebenza kahle okusezingeni eliphezulu kanye nenzuzo yomtholampilo ephelele yabantu abane-CF.

“I-NBD1 yinto eyaziwayo futhi ecwaningiwe, kodwa ibithathwa njengento engalawuleki kuze kube manje. Ngokusekelwe emizamweni yethu egxilile kanye nenqubekelaphambili eqhubekayo ku-NBD1, sibona amandla okwenza umsebenzi we-CFTR ujwayelekile kubantu abaningi abane-CF,” kusho uMike Cloonan, uMongameli kanye neSikhulu Esiphezulu seSionna Therapeutics. “Umgomo wethu eSionna uwukuthuthukisa kakhulu impilo kanye nezinga lempilo yabantu abaqhubeka nokuhlushwa yimiphumela yempilo yonke kanye nomthwalo wokuphila ne-CF. Ngalolu xhaso lwezimali, ithimba lethu eliqinile, kanye nethimba lethu elinolwazi nelinekhono, sijabule kakhulu ukwethula iSionana futhi sigxile ekuthuthukiseni amakhompawundi okuqala asuka epayipini lethu ahlukene aye emtholampilo.”

I-CF isifo sofuzo esiyingozi, esingase sibe yingozi esibangelwa ukuguquka kwezakhi zofuzo ze-CFTR okuholela ekwakhekeni kwamafinyila emaphashini nasezindleleni zomoya, ukungasebenzi kahle kwama-pancreas nokunye ukungasebenzi kahle kwezitho ezingase zibe nomthelela omkhulu futhi ngokuvamile onzima empilweni nempilo. ukulindela. Kunabantu abangaphezu kwe-100,000 abaphila ne-CF emhlabeni jikelele,ii, amaphesenti alinganiselwa ku-90 abo anokuguqulwa kofuzo i-ΔF508 okwenzeka ngaphakathi kwesizinda se-NBD1 se-CFTR. Lokhu kuguqulwa kubangela i-NBD1 ukuthi yenzeke ekushiseni komzimba futhi iphazamise ukusebenza kwe-CFTR. Naphezu kokutholakala kokwelashwa okuvunyelwe okwamanje kanye nenqubekelaphambili ebalulekile eyenziwe kokunye okuhlosiwe ngaphakathi kwe-CFTR, abantu abaningi abane-ΔF508 abafinyeleli umsebenzi ogcwele we-CFTR. I-NBD1 ibalulekile ukwenza umsebenzi we-CFTR ube ojwayelekile kanye nokukhiqizwa kwamafinyila anempilo, agelezayo ngokukhululeka emigwaqweni yomoya, ohlelweni lokugaya ukudla, nakwezinye izitho zomzimba.