Njira Yatsopano Ikhoza Kubwezeretsa Mokwanira Ntchito ya CFTR mu Cystic Fibrosis

A GWIRITSANI KwaulereKutulutsidwa 3 | eTurboNews | | eTN
Avatar ya Linda Hohnholz
Written by Linda Hohnholz

Sionna Therapeutics lero yalengeza kukhazikitsidwa kovomerezeka kwa kampaniyo komanso kutseka kwandalama za $ 111 miliyoni Series B. Zozungulirazo zidatsogozedwa ndi OrbiMed ndikutenga nawo gawo kuchokera kundalama zomwe zidalangizidwa ndi T. Rowe Price Associates, Inc., Q Healthcare Holdings, LLC., gulu lathunthu la QIA, thumba lachuma la Qatar, komanso osunga ndalama m'mbuyomu kuphatikiza RA Capital, TPG's. The Rise Fund, Atlas Venture, ndi Cystic Fibrosis Foundation. Sionna adakweza pafupifupi $150 miliyoni mpaka pano.

Sionna ikupita patsogolo paipi ya mamolekyu ang'onoang'ono amtundu woyamba omwe adapangidwa kuti abwezeretse bwino ntchito ya cystic fibrosis transmembrane conductance regulator (CFTR) protein yomwe ili ndi vuto mu CF, pokhazikitsa CFTR's first nucleotide-binding domain (NBD1). Chifukwa chachikulu cha CF ndi kusintha kwa majini ΔF508 komwe kumakhudza kukhazikika kwa NBD1 ndi CFTR ntchito.

M'mitundu yolosera mu vitro CF, tinthu tating'ono ta Sionna's NBD1, kuphatikiza ndi ma modulators ena owonjezera, awonetsa kuthekera kopindika, kukhwima, ndi kukhazikika kwa mapuloteni a CFTR omwe amakhudzidwa ndi kusintha kwa chibadwa kwa ΔF508. Izi zimalola kugulitsa koyenera kwa CFTR kupita ku selo pamwamba ndi kuwongolera bwino kwa ma ayoni ndi madzi. Pobwezeretsa mokwanira ntchito ya CFTR kwa odwala omwe ali ndi kusintha kwa majini kwa ΔF508 kumagulu omwe amawonekera m'maselo a anthu opanda CF, payipi ya Sionna ili ndi kuthekera kopereka kuthandizira bwino kwambiri komanso kupindula kwachipatala kwa anthu omwe ali ndi CF.

"NBD1 ndi chandamale chodziwika bwino komanso chofufuzidwa, koma chimawonedwa ngati chosasunthika mpaka pano. Kutengera kuyesetsa kwathu komanso kupita patsogolo kwa NBD1, tikuwona kuthekera kopangitsa kuti CFTR igwire ntchito mwa anthu ambiri omwe ali ndi CF, "anatero Mike Cloonan, Purezidenti ndi Chief Executive Officer wa Sionna Therapeutics. "Ntchito yathu ku Sionna ndikupititsa patsogolo thanzi ndi moyo wabwino kwa anthu omwe akupitirizabe kuvutika ndi zotsatira za moyo wonse komanso kulemedwa ndi CF. Ndi ndalama izi, gulu lathu lolimba, komanso gulu lathu lodziwa zambiri komanso laluso, tili okondwa kuyambitsa Sionna ndipo tikuyang'ana kwambiri kupititsa patsogolo mankhwala oyambira kuchokera ku mapaipi athu osiyanasiyana kupita kuchipatala. ”

CF ndi vuto lalikulu, lomwe lingathe kupha majini chifukwa cha kusintha kwa jini ya CFTR yomwe imatsogolera ku ntchofu m'mapapo ndi airways, kuwonongeka kwa kapamba ndi kuwonongeka kwa ziwalo zina zomwe zimatha kukhala ndi vuto lalikulu komanso nthawi zambiri paumoyo ndi moyo. chiyembekezo. Pali anthu opitilira 100,000 omwe amakhala ndi CF padziko lonse lapansi, ii, pafupifupi 90 peresenti ya omwe ali ndi masinthidwe amtundu wa ΔF508 omwe amapezeka mkati mwa NBD1 domain ya CFTR. Kusintha kumeneku kumapangitsa kuti NBD1 iwoneke pa kutentha kwa thupi ndikusokoneza ntchito ya CFTR. Ngakhale kupezeka kwa mankhwala ovomerezeka panopo komanso kupita patsogolo kwakukulu komwe kumachitika pazifukwa zina mkati mwa CFTR, anthu ambiri omwe ali ndi ΔF508 samakwaniritsa ntchito zonse za CFTR. NBD1 ndiyofunikira kuti ntchito ya CFTR ikhale yathanzi komanso kuti ikhale yathanzi, yoyenda momasuka mumayendedwe a mpweya, m'mimba, ndi ziwalo zina.

Ponena za wolemba

Avatar ya Linda Hohnholz

Linda Hohnholz

Mkonzi wamkulu kwa eTurboNews zochokera ku eTN HQ.

Amamvera
Dziwani za
mlendo
0 Comments
Zolowetsa Pamakina
Onani ndemanga zonse
0
Mukufuna malingaliro anu, chonde yankhani.x
Gawani ku...