Unyango lwe-Genetic cardiovascular disease luya kuphuhliso olutsha

Ubambiswano luya kuphucula iqonga le-Skyline Therapeutics 'elihlangeneyo le-AAV yonyango yofuzo esekwe kubunjineli bayo be-vector yobunini kunye noyilo lwetekhnoloji kunye nokukwazi ukuvelisa ukuphuhlisa unyango lwemfuza olunolutsha olugxile kwi-genetic dilated cardiomyopathies (DCM), iqela lezifo eziqhubela phambili, ezitshabalalisayo ezingenazo. iinketho zonyango ekujoliswe kuzo.

Ngaphantsi kwesivumelwano, i-BioMarin kunye ne-Skyline Therapeutics iya kusebenzisana ekufumaneni kunye nophando ukuya kwi-Investigational New Drug Application (IND). I-BioMarin izisa amava kuphuhliso lonyango lwemfuza, ibhayoloji yentliziyo kunye nokuqonda kwisiseko sofuzo sezifo, kwaye i-Skyline inegalelo lobuchwephesha bayo ekuphuhliseni iimveliso zonyango lwemfuza kubandakanya ubunjineli beVector kunye noyilo lobuchwephesha kunye nobuchule bokuvelisa kule ntsebenziswano. Inkampani nganye iya kuqhubela phambili iinkqubo ngophuhliso lweklinikhi kwiindawo ezichazwe kwangaphambili.  

Ukuxhasa iinzame zayo ze-R&D kwiiprojekthi zentsebenziswano, i-Skyline Therapeutics iya kufumana intlawulo engachazwanga ehambelana nokusayina, equka intlawulo yangaphambili kunye notyalo-mali olulinganayo oluvela kwi-BioMarin, kwaye ifanelekile ukufumana iintlawulo ezichazwe kwangaphambili ze-R & D, imilinganiselo yokulawula kunye nezorhwebo.

I-BioMarin iya kuba namalungelo okuthengisa iimveliso zonyango ezibangelwa yintsebenziswano kwimimandla yayo, kuquka i-United States, iYurophu, kunye neLatin America, kunye ne-Skyline Therapeutics iya kuba noxanduva lokuthengisa kummandla we-Asia-Pacific. Ukongeza, i-Skyline Therapeutics iya kufaneleka ukufumana iintlawulo ze-royalty kwiintengiso zexesha elizayo ezivela kwi-BioMarin kwimimandla yayo.

“Siyavuya ukwazisa into esiyilindeleyo ukuba iya kuba yintsebenziswano eneziqhamo kunxibelelwano phakathi kwendlela entsha ye-Skyline yobunjineli beVector ye-AAV kunye noyilo kunye nobuchule obuqinisekisiweyo beqela lethu ekudaleni nasekuphuhliseni unyango lwemfuza,” utshilo uKevin Eggan, uSekela Mongameli weQela, iNtloko yeSebe. Uphando kunye noPhuhliso lwakwangoko, ukusuka kwi-BioMarin.

“Siyavuya ukusebenzisana ne-Skyline Therapeutics ukujongana nezi ntlobo zemfuzo ze-dilated cardiomyopathy. Le ntsebenziswano yomeleza ubunkokeli bethu kunyango lwemfuza yentliziyo kwaye yandisa intsebenziswano yethu ye-R&D ukuya e-Asia, apho inani elikhulu lezigulana zithwaxwa zezi zifo zitshabalalisayo, utshilo uBrinda Balakrishnan, uSekela Mongameli weQela, uPhuhliso lweShishini kunye noPhuhliso lweShishini kwi-BioMarin. "Sijonge phambili ekukhuthazeni le ntsebenziswano kunye nokuzisa amayeza aguqulayo kwizigulana kwihlabathi liphela."

“I-Dilated cardiomyopathy sisifo esimandundu sentliziyo apho ukungahambi kakuhle kwesakhiwo okanye ukusebenza kwesihlunu sentliziyo kunokukhokelela kwiingxaki ezinjenge-arrhythmia kunye nokusilela kwentliziyo, okukhokelela ekuguleni okukhulu kunye nokufa. Ukuguqulwa kwezakhi zofuzo ezininzi kuhambelana nokuphuhliswa kwe-DCM, phakathi kwezinye ii-etiologies zesifo, "utshilo uJay Hou, iGosa eliyiNtloko lezeNzululwazi kwi-Skyline Therapeutics. “Kunye neqela le-BioMarin sichonge inani lemizila yemfuza ebalulekileyo eyayanyaniswa ne-DCM. Kuyasivuyisa ukusebenza ngokusondeleyo ne-BioMarin kwaye sisebenzise itekhnoloji yethu yeVector ye-AAV ukugocagoca ezi thagethi zintsha kunye nokuphuhlisa unyango olunoveli lwezigulane ze-DCM.

“Intsebenziswano neBioMarin ikhulisa amandla eenkampani zombini kuphuhliso lonyango lwemfuza. Ngeqela le-BioMarin, sabelana nenjongo yokusebenza kwikhonsathi yokuphuhlisa unyango lwe-genetic cardiovascular disease ejongene neemfuno zonyango ezingafezekanga, "kusho u-Amber Cai, i-CEO ye-Skyline Therapeutics. "Sisonke, siza kusebenzisa unyango lwemfuza ukujongana nezifo zentliziyo kunye nesifo sokuguqula indlela enokuthi itshintshe i-paradigm yonyango kwezi meko."