I-BBM-H901 iwumuthi omusha ophenyayo (IND) wokuqala ogunyazwe e-China ukuze welashwe ngezakhi zofuzo ngomzila we-IV wokulethwa kwezifo zofuzo, futhi ikakhulukazi, we-hemophilia B. Okwamanje, awukho umkhiqizo wokwelapha wofuzo we-AAV ovunyelwe ukumakethwa i-hemophilia. e-China nasemhlabeni jikelele.
Lolu cwaningo lokubhalisa lomtholampilo (CTR20212816) luyizikhungo eziningi, olungalo olulodwa, olunelebula evulekile kanye nocwaningo lokwelashwa okukodwa. Inhloso yawo ukuhlola ukuphepha, ukubekezelelana, i-pharmacokinetics, kanye nokusebenza kwesikhathi eside kanye nokuphepha kokufakwa kwe-BBM-H901 emthanjeni okukodwa ezigulini ezine-hemophilia B ≥iminyaka engu-18 ubudala nomsebenzi we-endogenous blood coagulation factor IX (FIX) ≤ 2 IU/dL (≤2%).
“Njengomkhiqizo wokuqala omkhulu wenkampani owenziwe endaweni yethu ye-cGMP esezingeni eliphezulu, siyajabula ukuthi i-Belief BioMed ifinyelele ingqopha-mlando ocwaningweni lomtholampilo ngemva kokudosa isihloko sokuqala ocwaningweni lomtholampilo olubalulekile. I-BBM-H901 ngaphambilini ikhombise ukuphepha okuhle nempumelelo yesikhathi eside kumseshi oqaliswe uhlolo lomtholampilo (IIT). Kulolo cwaningo izinga lokopha kwaminyaka yonke (ABR) lehla kakhulu, futhi izinga le-coagulation factor FIX lenyuke kakhulu futhi laphikelela kuzo zonke iziguli ngemva komjovo we-IV we-BBM-901. Asikho isigameko esibi esibi (i-SAE) esibikiwe,” kusho uDkt. Xiao Xiao, umsunguli, uSihlalo kanye neSikhulu Esiphezulu Sesayensi (CSO) se-Belief BioMed.
“Sibonga kakhulu ababambisene nathi emitholampilo, umphakathi wethu onesineke kanye nethimba lethu lonke elenze lokhu kwenzeke,” kwengeza uDkt. Xiao. I-Belief BioMed, njengenkampani ehamba phambili yokwelapha ngezakhi zofuzo e-China, izoqhubeka nokuthatha igxathu kuzo zombili izifundo zangaphambi komtholampilo nezomtholampilo ukuze ilethe eminye imithi emisha ezigulini eziswele. Ngaphezu kwalokho, lesi senzakalo esiyingqopha-mlando siye sabeka isisekelo esiqinile sokusetshenziswa emtholampilo kwemithi yethu yokwelapha isakhi sofuzo futhi savula indlela yokuthuthukiswa komtholampilo kwale mithi yezinhlobonhlobo zezidingo zomtholampilo ezinkulu nezingafinyeleli esikhathini esizayo. Umgomo wethu uwukwenza imishanguzo emisha yokwelapha izakhi zofuzo itholakale futhi ithengeke ezigulini, ngesikhathi esifanayo, ukwenza kube lula ukukhula kwemboni yokwelapha ngofuzo.
LOKHO ONGAKUTHATHE KULESI SIHLOKO:
- 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.
