Ubambiswano luzothuthukisa inkundla ye-Skyline Therapeutics' edidiyelwe yokwelashwa kofuzo ye-AAV ngokusekelwe kubuchwepheshe bayo be-vector nobunjiniyela bokuklama kanye nekhono lokukhiqiza ukuze kuthuthukiswe izindlela zokwelapha ezintsha zofuzo ngokugxila ku-genetic dilated cardiomyopathies (DCM), iqembu lezifo ezithuthukayo, ezibhubhisayo ezingenawo amandla. izinketho zokwelashwa ezihlosiwe.
Ngaphansi kwesivumelwano, i-BioMarin kanye ne-Skyline Therapeutics zizosebenzisana ekutholakaleni nasekucwaningeni kuze kufike ku-Investigational New Drug Application (IND). I-BioMarin iletha ulwazi ekuthuthukisweni kokwelashwa kwezakhi zofuzo, isayensi yezinto eziphilayo zenhliziyo nemithambo yegazi kanye nemininingwane ngesisekelo sofuzo sezifo, futhi i-Skyline inikela ngobuchule bayo ekuthuthukiseni imikhiqizo yokwelapha ngezakhi zofuzo okuhlanganisa ubunjiniyela be-vector nobuchwepheshe bokuklama namandla okwenza kulokhu kubambisana. Inkampani ngayinye izothuthukisa izinhlelo ngokuthuthukiswa komtholampilo ezindaweni ezichazwe ngaphambilini.
Ukuze isekele imizamo yayo ye-R&D yamaphrojekthi okusebenzisana, i-Skyline Therapeutics izothola inkokhelo engadalulwanga ehambisana nokusayina, ehlanganisa inkokhelo yangaphambili kanye nokutshalwa kwezimali kwe-equity evela kwa-BioMarin, futhi iyafaneleka ukuthola izinkokhelo ezishiwo kusengaphambili ze-R&D, izilinganiso zokulawula nezohwebo.
I-BioMarin izoba namalungelo okudayisa ngemikhiqizo yokwelapha ewumphumela wokubambisana ezindaweni zayo, okuhlanganisa i-United States, i-Europe, ne-Latin America, kanti i-Skyline Therapeutics izoba nesibopho sokuthengisa esifundeni sase-Asia-Pacific. Ngaphezu kwalokho, i-Skyline Therapeutics izofaneleka ukuthola izinkokhelo ze-royalty ekuthengisweni kwesikhathi esizayo okuvela ku-BioMarin ezindaweni zayo.
“Sijabule kakhulu ukumemezela esikulindele kuzoba ukubambisana okunezithelo ekuxhumaneni phakathi kwendlela entsha ye-Skyline yobunjiniyela be-AAV vector kanye nokuklama kanye nekhono lethimba lethu elifakazelwe ekudaleni nasekuthuthukiseni izindlela zokwelapha ngofuzo,” kusho uKevin Eggan, iPhini Likamongameli Weqembu, iNhloko yoMnyango. Ucwaningo kanye Nokuthuthukiswa Kwangaphambi kwesikhathi, okuvela ku-BioMarin.
“Sijabule ukusebenzisana ne-Skyline Therapeutics ukuze sibhekane nalezi zinhlobo zofuzo ze-dilated cardiomyopathy. Lokhu kubambisana kuqinisa ubuholi bethu ekwelashweni kwezakhi zofuzo zenhliziyo futhi kwandisa ukusebenzisana kwethu kwe-R&D e-Asia, lapho inani elikhulu leziguli lihlushwa yilezi zifo ezibhubhisayo,” kusho uBrinda Balakrishnan, iPhini Likamongameli Weqembu, Ukuthuthukiswa Kwebhizinisi Nebhizinisi e-BioMarin. "Sibheke ngabomvu ukukhuthaza lokhu kubambisana nokuletha imithi eguqulayo ezigulini emhlabeni wonke."
“I-Dilated cardiomyopathy yisifo esibucayi senhliziyo lapho ukungahambi kahle kwesakhiwo noma ukusebenza kwemisipha yenhliziyo kungaholela ezinkingeni ezinjengokwehluleka kwenhliziyo nokuhluleka kwenhliziyo, okuholela ekuguleni okukhulu nokufa. Ukuguqulwa kwezakhi zofuzo eziningi kuhlotshaniswa nokuthuthukiswa kwe-DCM, phakathi kwezinye izimbangela zesifo, "kusho u-Jay Hou, Isikhulu Esiyinhloko Sesayensi kwa-Skyline Therapeutics. “Kanye nethimba le-BioMarin sihlonze inqwaba yezakhi zofuzo ezibalulekile ezihlobene ne-DCM. Siyajabula ukusebenzisana eduze ne-BioMarin futhi sisebenzise ubuchwepheshe bethu be-AAV ukuze siphenye lezi zinhloso ezintsha futhi sithuthukise izindlela zokwelapha ezintsha zeziguli ze-DCM.”
“Ukubambisana ne-BioMarin kusebenzisa amandla ezinkampani zombili ekuthuthukisweni kokwelapha ngezakhi zofuzo. Nethimba le-BioMarin, sihlanganyela umgomo wokusebenza ekhonsathini ukuthuthukisa imithi yofuzo yesifo senhliziyo nemithambo yegazi ebhekana nezidingo zezokwelapha ezingafinyeleleki,” kusho u-Amber Cai, oyi-CEO ye-Skyline Therapeutics. "Ngokubambisana, sizosebenzisa ukwelashwa kwezakhi zofuzo ukubhekana nezifo zenhliziyo ngendlela eshintsha isifo engashintsha i-paradigm yokwelashwa kulezi zimo."
LOKHO ONGAKUTHATHE KULESI SIHLOKO:
- BioMarin will have the rights to commercialize therapeutic products resulting from the collaboration in its territories, including the United States, Europe, and Latin America, and Skyline Therapeutics will be responsible for commercialization in the Asia-Pacific region.
- Ukuze isekele imizamo yayo ye-R&D yamaphrojekthi okusebenzisana, i-Skyline Therapeutics izothola inkokhelo engadalulwanga ehambisana nokusayina, ehlanganisa inkokhelo yangaphambili kanye nokutshalwa kwezimali kwe-equity evela kwa-BioMarin, futhi iyafaneleka ukuthola izinkokhelo ezishiwo kusengaphambili ze-R&D, izilinganiso zokulawula nezohwebo.
- “We are thrilled to announce what we anticipate will be a fruitful collaboration at the interface between Skyline’s innovative approach to AAV vector engineering and design and our team’s proven expertise in creating and developing gene therapies,”.
