BBM-H901 o le uluai suʻesuʻega fou o vailaʻau (IND) faʻamaonia i Saina mo togafitiga faʻasolosolo e ala i le auala e tuʻuina atu ai IV mo faʻamaʻi faʻamaʻi, ma faʻapitoa, mo le hemophilia B. I le taimi nei, e leai se oloa faʻamaʻi faʻamaʻi AAV ua faʻatagaina mo le maketiina mo le hemophilia. i Saina ma le lalolagi atoa.
O lenei su'esu'ega falema'i resitalaina (CTR20212816) o le tele-center, tasi-lima, tatala-igoa ma tasi-togafitiga su'esu'ega. O lona faʻamoemoe o le iloilo lea o le saogalemu, faʻapalepale, pharmacokinetics, faʻapea foʻi ma le aoga umi ma le saogalemu o se tui faʻapipiʻi e tasi o le BBM-H901 i tagata mamaʻi hemophilia B ≥18 tausaga le matua ma le faʻaogaina o le toto IX (FIX) gaioiga ≤ 2 IU/dL (≤2%).
“I le avea ai ma oloa tele muamua a le kamupani na faia i totonu o la matou fale cGMP faʻaonaponei, matou te fiafia ona ua ausia e le Belief BioMed se mataʻina taua i suʻesuʻega faʻafomaʻi ina ua maeʻa ona faʻaogaina le mataupu muamua i le suʻesuʻega faʻapitoa. BBM-H901 ua faʻaalia muamua le saogalemu lelei ma le aoga umi i se suʻesuʻega na amataina suʻesuʻega faʻapitoa (IIT). I lena suʻesuʻega na matua faʻaititia le fua faatatau o le toto (ABR), ma o le maualuga o mea faʻamaʻi FIX na matua faʻateleina ma faʻaauau pea i tagata mamaʻi uma ina ua uma le tui IV o le BBM-901. Leai se fa'alavelave matuia na tupu (SAE) na lipotia mai, "o le tala lea a Dr. Xiao Xiao, fa'avae fa'atasi, Ta'ita'ifono ma Ofisa Sili Saienisi (CSO) o le Belief BioMed.
"Matou te faʻafetai i a matou fomaʻi galulue, matou tagata gasegase ma le matou 'au atoa na faia lenei mea," o le faʻaopoopo mai lea a Dr. Xiao. Talitonu BioMed, i le avea ai o se kamupani ta'uta'ua mo togafitiga o kenera i Saina, o le a fa'aauau pea ona laasia i su'esu'ega fa'apitoa ma su'esu'ega fa'afoma'i e aumai nisi vaila'au fou i tagata mama'i le tagolima. E le gata i lea, o lenei mataʻina ua faʻavaeina se faavae mautu mo le faʻaogaina o le falemaʻi o a tatou vailaʻau faʻamaʻi faʻamaʻi ma saunia le ala mo le atinaʻeina o falemaʻi o nei vailaʻau mo le tele o manaʻoga faʻapitoa ma le le ausia i le lumanaʻi. O la matou sini o le faʻaavanoaina o vailaʻau faʻafomaʻi faʻafomaʻi faʻamaʻi ma taugofie i tagata mamaʻi, ma i le taimi lava e tasi, e faʻafaigofie ai le tuputupu aʻe o le gaosiga o vailaʻau.
OA MEA E AVEA MAI LENEI TUSI:
- In addition, this milestone has laid a solid foundation for the clinical application of our gene therapy drugs and paved the way for the clinical development of these drugs for a variety of major and unmet clinical needs in the future.
- Its objective is to evaluate the safety, tolerability, pharmacokinetics, as well as long-term efficacy and safety of a single intravenous infusion of BBM-H901 in hemophilia B patients ≥18 years of age with endogenous blood coagulation factor IX (FIX) activity ≤2 IU/dL (≤2%).
- Our goal is to make innovative gene therapy drugs available and affordable to the patients, and at the same time, to facilitate the growth of gene therapy industry.
