Sionna Therapeutics i aso nei na faʻasalalau le faʻalauiloaina aloaia o le kamupani ma le tapunia o le $ 111 miliona Series B faʻatupeina. O le taamilosaga na taitaia e le OrbiMed faatasi ai ma le auai mai tupe na fautuaina e T. Rowe Price Associates, Inc., Q Healthcare Holdings, LLC., o se lala atoa o le QIA, o le sovereign wealth fund o Qatar, ma le aufaipisinisi muamua e aofia ai le RA Capital, TPG's Le Rise Fund, Atlas Venture, ma le Cystic Fibrosis Foundation. E tusa ma le $150 miliona ua maua e Siona e oo mai i le taimi nei.
O loʻo faʻalauteleina e Sionna se paipa o molelaʻau laiti muamua-i-vasega ua fuafuaina e toe faʻafoʻi atoatoa ai le galuega a le cystic fibrosis transmembrane conductance regulator (CFTR) porotini o loʻo faaletonu i le CF, e ala i le faʻamautuina o le CFTR muamua nucleotide-binding domain (NBD1). O le mafuaʻaga autu o le CF o le genetic mutation ΔF508 lea e aʻafia ai le mautu o le NBD1 ma le CFTR galuega.
I faʻataʻitaʻiga faʻataʻitaʻi faʻataʻitaʻiga in vitro CF, o Sionna's NBD1-faʻatatau i molelaʻau laiti, faʻatasi ma isi modulators faʻaopoopo, ua faʻaalia ai le gafatia e faʻasaʻo ai le gaugau, matua, ma le mautu o le porotini CFTR e aʻafia e le ΔF508 genetic mutation. Ole mea lea e mafai ai ona fa'atau sa'o ole CFTR ile luga ole sela ma fa'atonutonu masani ole tafega o ion ma vai. E ala i le toe faʻaleleia atoatoa o le CFTR galuega mo tagata mamaʻi ma le ΔF508 genetic mutation i tulaga o loʻo vaʻaia i sela o tagata e aunoa ma le CF, o le paipa a Sionna e mafai ona tuʻuina atu le aoga sili-i-vasega ma aoga sili ona lelei mo tagata e maua i le CF.
"NBD1 ose taʻutaʻua ma suʻesuʻeina sini, ae o loʻo manatu e le mafai ona faʻaogaina e oʻo mai i le taimi nei. Faʻavae i luga oa matou taumafaiga taulaʻi ma faʻaauau le alualu i luma i luga o le NBD1, matou te vaʻai i le gafatia e faʻasaʻo ai galuega a le CFTR i le toʻatele o tagata e iai le CF, "o le tala lea a Mike Cloonan, Peresetene ma Ofisa Sili o Pulega o Sionna Therapeutics. "O la matou misiona i Siona o le faʻaleleia atili o le soifua maloloina ma le tulaga lelei o le olaga mo tagata o loʻo faʻaauau pea ona pagatia i taunuuga o le olaga atoa ma le avega o le ola ma le CF. Faatasi ai ma lenei faʻatupeina, la matou faʻalapotopotoga malosi, ma la matou 'au faʻapitoa ma talenia, matou te fiafia e faʻalauiloa Sionna ma o loʻo taulaʻi atu i le faʻalauteleina o faʻalapotopotoga muamua mai le matou paipa eseese i totonu o le falemaʻi.
O le CF ose faama'i tuga, e ono oti ai e mafua mai i le suiga o le kenera CFTR lea e oo atu ai i le faaputuputuina o le mucus i totonu o māmā ma ala ea, faaletonu le gaioiina o le pancreatic ma isi faaletonu o totoga e mafai ona i ai se aafiaga taua ma e masani ona ogaoga i le soifua maloloina ma le olaga. fa'amoemoe. E sili atu i le 100,000 tagata o loʻo nonofo ma CF i le lalolagi atoa, ii, e tusa ma le 90 pasene oi latou o loʻo i ai le genetic mutation ΔF508 lea e tupu i totonu ole NBD1 domain ole CFTR. O lenei suiga e mafua ai le NBD1 e faʻaalia i le vevela o le tino ma faʻaleagaina ai le CFTR. E ui i le maua o togafitiga faʻamaonia i le taimi nei ma le alualu i luma taua i luga o isi sini i totonu o le CFTR, o le toʻatele o tagata e iai le ΔF508 e le ausia atoa le CFTR galuega. O le NBD1 e mana'omia e fa'amanino ai le galuega a le CFTR ma le gaosiga o le soifua maloloina, fa'afefe solo i totonu o le ea, fa'aogaina o mea'ai, ma isi totoga.
OA MEA E AVEA MAI LENEI TUSI:
- CF is a serious, potentially fatal genetic disease caused by a mutation in the CFTR gene that leads to a buildup of mucus in the lungs and airways, impaired pancreatic function and other organ dysfunction that can have a significant and often severe impact on health and life expectancy.
- In clinically predictive in vitro CF models, Sionna’s NBD1-targeted small molecules, in combination with other complementary modulators, have demonstrated the potential to normalize folding, maturation, and stability of the CFTR protein affected by the ΔF508 genetic mutation.
- By fully restoring CFTR function for patients with the ΔF508 genetic mutation to levels seen in cells of people without CF, Sionna’s pipeline has the potential to deliver best-in-class efficacy and optimal clinical benefit for people with CF.
