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Phekolo ea lefu la pelo le methapo ea lefutso e ea nts'etsopeleng e ncha

ngotsoeng ke mohlophisi

BioMarin Pharmaceutical Inc. le Skyline Therapeutics (eo pele e neng e le Geneception), k'hamphani ea liphatsa tsa lefutso le lisele tse tsepamisitseng maikutlo ho nts'etsopele ea liphekolo tse ncha bakeng sa litlhoko tse sa finyelloang tsa bongaka, kajeno li phatlalalitse tšebelisano-'moho ea leano la lilemo tse ngata bakeng sa ho sibolloa, nts'etsopele le khoebo ea Adeno-Associated Virus. AAV) kalafo ea liphatsa tsa lefutso ho phekola mafu a lefu la pelo.

Tšebelisano-'moho e tla matlafatsa sethala sa kalafo se kopaneng sa AAV sa Skyline Therapeutics se ipapisitseng le boenjiniere ba eona ba vector le theknoloji ea moralo le bokhoni ba tlhahiso ea ho nts'etsapele liphekolo tse ncha tsa lefutso tse shebaneng le lefutso la cardiomyopathies (DCM), sehlopha sa mafu a ntseng a tsoela pele, a senyang a se nang letho. mekhoa ea phekolo e lebisitsoeng.

Tlas'a tumellano, BioMarin le Skyline Therapeutics li tla sebelisana 'moho mabapi le ho sibolloa le ho etsa lipatlisiso ho ea ho Investigational New Drug Application (IND). BioMarin e tlisa boiphihlelo ho nts'etsopele ea kalafo ea liphatsa tsa lefutso, baeloji ea pelo le methapo ea mali le temohisiso mabapi le liphatsa tsa lefutso tsa mafu, 'me Skyline e kenya letsoho bokhabane ba eona ba ho nts'etsapele lihlahisoa tsa kalafo ea liphatsa tsa lefutso ho kenyeletsoa boenjiniere ba vector le thekenoloji ea moralo le bokhoni ba tlhahiso tšebelisanong ena. Khampani ka 'ngoe e tla ntšetsa pele mananeo ka nts'etsopele ea bongaka libakeng tse boletsoeng esale pele.  

E le ho tšehetsa boiteko ba eona ba R&D bakeng sa merero ea tšebelisano-'moho, Skyline Therapeutics e tla fumana tefo e sa boleloang e amanang le ho saena, e kenyelletsang tefo ea pele le letsete la tekano ho tsoa ho BioMarin, 'me e lokeloa ke ho fumana litefo tse boletsoeng esale pele bakeng sa R&D, melaoana ea taolo le khoebo.

BioMarin e tla ba le litokelo tsa ho rekisa lihlahisoa tsa phekolo tse bakoang ke tšebelisano-'moho libakeng tsa eona, ho kenyeletsa United States, Europe, le Latin America, 'me Skyline Therapeutics e tla ikarabella bakeng sa khoebo sebakeng sa Asia-Pacific. Ho feta moo, Skyline Therapeutics e tla tšoaneleha ho fumana litefo tsa borena thekisong ea nako e tlang ho tsoa ho BioMarin libakeng tsa eona.

"Re thabetse ho phatlalatsa seo re se lebelletseng hore e tla ba tšebelisano e ntle lipakeng tsa mokhoa o mocha oa Skyline oa boenjiniere le boqapi ba li-vector tsa AAV le boiphihlelo bo netefalitsoeng ba sehlopha sa rona sa ho theha le ho nts'etsapele liphekolo tsa lefutso," ho boletse Kevin Eggan, Motlatsi oa Motlatsi oa Sehlopha, Hlooho ea Lefapha. Patlisiso le Nts'etsopele ea Pele, ho tsoa ho BioMarin.

"Re thabetse ho sebelisana le Skyline Therapeutics ho sebetsana le mefuta ena ea lefutso ea lefu la pelo le atileng. Tšebelisano ena e matlafatsa boeta-pele ba rona kalafong ea lefutso la pelo le ho atolosa tšebelisano ea rona ea R&D ho ea Asia, moo bakuli ba bangata ba tšoeroeng ke mafu ana a senyang, "ho boletse Brinda Balakrishnan, Motlatsi oa Motlatsi oa Sehlopha, Nts'etsopele ea Khoebo le Khoebo ho BioMarin. "Re labalabela ho khothaletsa tšebelisano ena le ho tlisa meriana e fetolang bakuli lefatšeng ka bophara."

"Dilated cardiomyopathy ke lefu le tebileng la pelo leo ho lona ho se sebetse hantle ha mesifa ea pelo ho ka bakang mathata a kang arrhythmia le ho hloleha ha pelo, ho bakang mafu a mangata le lefu. Liphetoho liphatseng tsa lefutso tse ngata li amahanngoa le nts'etsopele ea DCM, har'a mabaka a mang a lefu lena, "ho boletse Jay Hou, Ofisiri e ka Sehloohong ea Saense Skyline Therapeutics. "Mmoho le sehlopha sa BioMarin re hloaile mefuta e mengata ea bohlokoa e amanang le DCM. Re thabetse ho sebetsa haufi-ufi le BioMarin le ho sebelisa theknoloji ea rona ea li-vector ea AAV ho hlahloba lipakane tsena tse ncha le ho nts'etsapele liphekolo tse ncha bakeng sa bakuli ba DCM.

Tšebelisano 'moho le BioMarin e phahamisa bokhoni ba lik'hamphani ka bobeli ho nts'etsopele ea liphekolo tsa lefutso. Le sehlopha sa BioMarin, re arolelana sepheo sa ho sebetsa ka konsarete ho nts'etsapele liphekolo tsa lefutso la lefu la pelo le methapo e sebetsanang le litlhoko tse phahameng tsa bongaka tse sa fihlelleheng, "ho boletse Amber Cai, CEO oa Skyline Therapeutics. "Ka kopanelo, re tla sebelisa kalafo ea liphatsa tsa lefutso ho sebetsana le mafu a pelo le methapo ka mokhoa o fetolang lefu o ka fetolang paradigm ea kalafo maemong ana."

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mohlophisi

Mohlophisi e moholo oa eTurboNew ke Linda Hohnholz. O lula ntlong e kholo ea eTN e Honolulu, Hawaii.

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