Sabon Injiniyanci Zai Iya Dawo da Cikakkun Ayyukan CFTR a cikin Cystic Fibrosis

Sionna Therapeutics a yau ta sanar da ƙaddamar da kamfanin a hukumance da kuma rufe tallafin dala miliyan 111 na Series B. OrbiMed ne ya jagoranci zagayen tare da halartar kuɗaɗen da T. Rowe Price Associates, Inc., Q Healthcare Holdings, LLC ya ba da shawara, wani reshen QIA gabaɗaya, asusun dukiyar Qatar, da masu saka hannun jari na baya ciki har da RA Capital, TPG's Asusun Rise, Atlas Venture, da Gidauniyar Cystic Fibrosis. Sionna ta tara kusan dala miliyan 150 zuwa yau.

Sionna tana haɓaka bututun ƙananan ƙwayoyin cuta na farko da aka tsara don cikakken dawo da aikin furotin na cystic fibrosis transmembrane conductance regulator (CFTR) wanda ke da lahani a cikin CF, ta hanyar daidaita yankin farko na nucleotide-binding na CFTR (NBD1). Babban dalilin CF shine maye gurbi na ΔF508 wanda ke shafar kwanciyar hankali na NBD1 da aikin CFTR.

A cikin tsinkayar asibiti a cikin samfuran CF na vitro, ƙananan ƙwayoyin Sionna na NBD1 da aka yi niyya, a hade tare da sauran masu daidaitawa, sun nuna yuwuwar daidaita folding, maturation, da kwanciyar hankali na furotin CFTR wanda ΔF508 ke haifar da maye gurbi. Wannan yana ba da damar fataucin CFTR da ya dace zuwa saman tantanin halitta da tsarin al'ada na kwararar ions da ruwa. Ta hanyar cikakken maido da aikin CFTR ga marasa lafiya tare da maye gurbi na ΔF508 zuwa matakan da aka gani a cikin sel na mutane ba tare da CF ba, bututun Siona yana da yuwuwar isar da ingantaccen inganci a cikin aji da fa'idar asibiti mafi kyau ga mutanen da ke da CF.

“NBD1 sanannen manufa ce kuma aka yi bincike, amma an yi la’akari da shi ba za a iya lalacewa ba har zuwa yanzu. Bisa ga kokarin da muka mayar da hankali da kuma ci gaba da ci gaba a kan NBD1, muna ganin yiwuwar daidaita aikin CFTR a yawancin mutanen da ke da CF, "in ji Mike Cloonan, Shugaba da Babban Jami'in Harkokin Kiwon Lafiya na Sionna Therapeutics. "Manufarmu a Sionna ita ce inganta lafiya da ingancin rayuwa ga mutanen da ke ci gaba da shan wahala daga sakamakon rayuwa da nauyin rayuwa tare da CF. Tare da wannan tallafin, ƙungiyarmu mai ƙarfi, da ƙwararrun ƙwararrun ƙwararrun ƙwararrunmu, muna farin cikin ƙaddamar da Sionna kuma muna mai da hankali kan haɓaka mahaɗan farko daga bututunmu na daban zuwa asibitin.

CF cuta ce mai tsanani, mai yuwuwar kisa wanda ya haifar da maye gurbi a cikin kwayar halittar CFTR wanda ke haifar da tarin gamsai a cikin huhu da hanyoyin iska, rashin aikin pancreatic da sauran raunin gabobin da zai iya yin tasiri mai mahimmanci kuma sau da yawa akan lafiya da rayuwa. tsammani. Akwai fiye da mutane 100,000 da ke zaune tare da CF a duniya,ii, an kiyasta kashi 90 cikin dari waɗanda ke da maye gurbin kwayoyin halitta ΔF508 wanda ke faruwa a cikin yankin NBD1 na CFTR. Wannan maye gurbin yana haifar da NBD1 don bayyanawa a zafin jiki kuma yana lalata aikin CFTR. Duk da samuwan jiyya da aka yarda da su a halin yanzu da kuma ci gaba mai mahimmanci da aka samu a kan wasu manufofi a cikin CFTR, yawancin mutanen da ke da ΔF508 ba su cimma cikakken aikin CFTR ba. NBD1 yana da mahimmanci don daidaita aikin CFTR da kuma samar da lafiya, ƙura da ƙura a cikin iska, tsarin narkewa, da sauran gabobin.