Nkhani Yoyamba Ikulandila Chithandizo Chatsopano Chamankhwala cha Hemophilia B

BBM-H901 ndi mankhwala oyamba ofufuza (IND) omwe amavomerezedwa ku China kuti athandizidwe ndi majini pogwiritsa ntchito njira ya IV yoperekera matenda a chibadwa, komanso makamaka, a hemophilia B. Pakali pano, palibe mankhwala amtundu wa AAV omwe adavomerezedwa kuti azitha kutsatsa kwa hemophilia. ku China komanso padziko lonse lapansi.

Phunziro lachipatala lolembetsa (CTR20212816) ndi kafukufuku wapakati, mkono umodzi, otseguka komanso chithandizo chamankhwala amodzi. Cholinga chake ndikuwunika chitetezo, kulolerana, pharmacokinetics, komanso mphamvu yayitali komanso chitetezo cha kulowetsedwa kwa BBM-H901 m'mitsempha mwa odwala omwe ali ndi hemophilia B ≥zaka 18 zakubadwa ndi endogenous blood coagulation factor IX (FIX) ≤ 2 IU/dL (≤2%).

"Monga chinthu choyambirira cha kampani chomwe chidapangidwa m'malo athu apamwamba kwambiri a cGMP, ndife okondwa kuti Belief BioMed yafika pachimake pa kafukufuku wazachipatala atamwa mutu woyamba pa kafukufuku wofunikira kwambiri wachipatala. BBM-H901 yawonetsa kale chitetezo chabwino komanso kuthandizira kwanthawi yayitali mu kafukufuku yemwe adayambitsa mayeso azachipatala (IIT). Mu phunziro limenelo mlingo wa kutulutsa magazi kwa pachaka (ABR) unachepetsedwa kwambiri, ndipo mlingo wa coagulation factor FIX unawonjezeka kwambiri ndikupitirirabe kwa odwala onse pambuyo pa jekeseni wa IV wa BBM-901. Palibe vuto lalikulu (SAE) lomwe linanenedwa, "anatero Dr. Xiao Xiao, woyambitsa nawo, Wapampando ndi Chief Science Officer (CSO) wa Belief BioMed.

"Ndife othokoza kwa othandizira athu azachipatala, gulu lathu la odwala komanso gulu lathu lonse lomwe lidapanga izi," adawonjezera Dr. Xiao. Belief BioMed, monga kampani yotsogola ya gene therapy ku China, ipitiliza kuchita bwino m'maphunziro azachipatala komanso azachipatala kuti abweretse mankhwala otsogola kwa odwala osowa. Kuonjezera apo, chochitika ichi chakhazikitsa maziko olimba a kagwiritsidwe ntchito kachipatala ka mankhwala athu a jini ndipo adatsegula njira yopangira chithandizo chamankhwala chamankhwalawa pazifukwa zosiyanasiyana zazikulu komanso zosakwaniritsidwa zachipatala m'tsogolomu. Cholinga chathu ndikupanga mankhwala opangira ma gene kuti azitha kupezeka komanso otsika mtengo kwa odwala, komanso nthawi yomweyo, kuti athandizire kukula kwamakampani opanga ma gene.