Mechanism e Ncha e ka Khutlisa Mosebetsi oa CFTR ka Botlalo ho Cystic Fibrosis

Sionna Therapeutics kajeno e phatlalalitse phatlalatso ea semmuso ea k'hamphani le ho koaloa ha lichelete tsa $ 111 milione Series B. Potoloho ena e ne e etelletsoe pele ke OrbiMed ka seabo ho tsoa licheleteng tse neng li elelitsoe ke T. Rowe Price Associates, Inc., Q Healthcare Holdings, LLC., e leng lekala le felletseng la QIA, letlole le ikemetseng la leruo la Qatar, le batseteli ba nakong e fetileng ho kenyeletsoa RA Capital, TPG's. The Rise Fund, Atlas Venture, le Cystic Fibrosis Foundation. Sionna e bokelletse chelete e ka bang $ 150 milione ho fihlela joale.

Sionna e ntse e tsoela pele ka phaephe ea limolek'hule tse nyane tsa boemo ba pele tse etselitsoeng ho khutlisetsa ka botlalo tšebetso ea protheine ea cystic fibrosis transmembrane conductance regulator (CFTR) e nang le bothata ho CF, ka ho tsitsisa sebaka sa pele sa CFTR se tlamang nucleotide (NBD1). Lebaka le ka sehloohong la CF ke phetoho ea liphatsa tsa lefutso ΔF508 e amang botsitso ba NBD1 le mosebetsi oa CFTR.

Mefuteng ea CF e lebelloang ka mokhoa oa bongaka, limolek'hule tse nyane tse lebisitsoeng ho Sionna's NBD1, hammoho le li-modulator tse ling tse tlatselletsang, li bonts'itse bokhoni ba ho etsa hore protheine ea CFTR e be maemong a tloaelehileng e anngoeng ke phetoho ea liphatsa tsa lefutso tsa ΔF508. Sena se lumella khoebo e nepahetseng ea CFTR sebakeng sa sele le taolo e tloaelehileng ea phallo ea li-ion le metsi. Ka ho khutlisetsa ts'ebetso ea CFTR ka botlalo bakeng sa bakuli ba nang le phetoho ea liphatsa tsa lefutso tsa ΔF508 maemong a bonoang liseleng tsa batho ba se nang CF, pipeline ea Sionna e na le monyetla oa ho fana ka katleho ea maemo a holimo le melemo e nepahetseng ea bongaka bakeng sa batho ba nang le CF.

"NBD1 ke sepheo se tsebahalang le se entsoeng lipatlisiso, empa se nkuoe e le ntho e sa rarolleheng ho fihlela joale. Ho itšetlehile ka boiteko ba rona bo tsepamisitsoeng le tsoelo-pele e tsoelang pele ho NBD1, re bona monyetla oa ho etsa hore mosebetsi oa CFTR o tloaelehe ho bongata bo boholo ba batho ba nang le CF," ho boletse Mike Cloonan, Mopresidente le Mohlanka e Moholo oa Sionna Therapeutics. "Morero oa rona ho Sionna ke ho ntlafatsa haholo bophelo bo botle le boleng ba bophelo ho batho ba ntseng ba tsoela pele ho utloa bohloko ka lebaka la litlamorao tsa bophelo bohle le moroalo oa ho phela le CF. Ka thuso ena ea lichelete, mokhatlo oa rona o matla, le sehlopha sa rona se nang le boiphihlelo ebile se na le talenta, re thabetse ho tsebisa Sionna, 'me re tsepamisitse maikutlo ho ntlafatseng metsoako ea pele ho tsoa ho phaephe ea rona e fapaneng ho ea tleliniking.

CF ke lefu le tebileng, le ka 'nang la bolaea liphatsa tsa lefutso le bakoang ke phetoho ea liphatsa tsa lefutso tsa CFTR tse lebisang ho bokellaneng ha mucus matšoafong le litsela tsa moea, ts'ebetso ea pancreatic e holofetseng le ho se sebetse ha litho tse ling tse ka bang le phello e kholo le hangata e matla bophelong le bophelo. tebello. Ho na le batho ba fetang 100,000 ba phelang le CF lefatšeng ka bophara,ii, hoo e ka bang liperesente tse 90 tsa bona ba nang le phetoho ea lefutso ΔF508 e hlahang sebakeng sa NBD1 sa CFTR. Phetoho ena e etsa hore NBD1 e hlahe ka mocheso oa 'mele mme e sitise ts'ebetso ea CFTR. Ho sa tsotellehe boteng ba liphekolo tse amohetsoeng ha joale le tsoelo-pele e kholo e entsoeng lipehelong tse ling ka har'a CFTR, batho ba bangata ba nang le ΔF508 ha ba fihlele mosebetsi o feletseng oa CFTR. NBD1 e bohlokoa ho tloaeleha ho sebetsa ha CFTR le tlhahiso ea mucus e phetseng hantle, e phallang ka bolokolohi moeeng, tsamaisong ea tšilo ea lijo le litho tse ling.