Ke hahai nei ʻoe i ka nūhou Bluebird Bio? Inā ʻaʻole ʻoe, ʻo ia ka manawa e komo i ka ʻike a hoʻohuli i kāu mau leka no nā mea hou loa e pili ana i kēia hui. No ka mea, hiki ke kiʻekiʻe hou i kēlā me kēia manawa.
Ke manaʻo ʻia nei e piʻi aʻe ana nā kumukūʻai o kēia hui ma ke ʻano he komite ʻōlelo aʻoaʻo o ka Food and Drug Administration (FDA) i ʻōlelo i ʻelua mau hoʻāʻo ʻana o kēia hui biotech i nā lāʻau hoʻokolohua hoʻokolohua.
No laila ua ʻike paha ʻoe i ka piʻi a piʻi ʻana o nā waihona o ka hui. No kou ʻike, ʻo ka māka 'BLUE' āu i ʻike ai ma nā pale no kēia hui. No laila ʻoiai ke kūlana mākeke holoʻokoʻa, ke loaʻa nei nā mea kuleana o kēia ʻoihana i kahi hoʻomaha koʻikoʻi.
Nūhou Bio Bluebird Pono
ʻO kēia kahi hui biotechnology ma Cambridge, Massachusetts e kālele ana i ka hoʻomohala ʻana i nā lāʻau lapaʻau no nā maʻi maʻi genetic koʻikoʻi a me ka maʻi kanesa. Ma mua, ʻo kāna lāʻau lapaʻau i ʻae ʻia mai ka European Union (EU) ʻo Betigeglogene autotemcel ka mea maʻamau i ka inoa (Zynteglo).
No ka hoʻomanaʻo ʻana iā ʻoe, ʻo kēia ka lua o ka lāʻau kūʻai nui loa o ka honua ma ke kumukūʻai he $1.8 miliona. Me ka nui o ka hiki ke ʻike ka hui i ka piʻi ʻana o kāna mau ʻāpana akā ua emi mau lākou a hiki i kēia manawa. Me ka ʻae ʻia o nā lāʻau lapaʻau ʻelua, manaʻo ʻia e hoʻihoʻi i ka hilinaʻi nalowale i kona wā e hiki mai ana mai nā mea hoʻopukapuka.
ʻO nā hana pipeline ʻē aʻe o ka ʻoihana ʻo LentiGlobin gene therapy no ka maʻi Sickle cell a me ka Cerebral Adrenoleukodystrophy. Ke hana pū nei ʻo IT no ka mālama ʻana i ka maʻi leukemia Myeloid Acute, Merkel-cell carcinoma, MAGEA4 solid tumors, a me Diffuse large B-cell lymphoma.
E hoʻomaka ana i kāna huakaʻi ma Genetix Pharmaceuticals ma 1992 he brainchild o nā lālā kumu kula MIT ʻo Irving London lāua ʻo Philippe Leboulch, ua ʻike kēia ʻano biotechnology i kāna mau ʻāpana a hiki i $ 178.29 i 2018 a ma hope o kēlā, aia lākou ma kahi ʻano hāʻule holoʻokoʻa.
Akā me kēia nūhou, ua piʻi nā ʻāpana ma kahi o 28.7% a i 4.80 ma ka Pōʻakahi 14 Iune 2022. Aia nā waihona ma ke ala no ka piʻi nui loa o ka pākēneka i nā makahiki ʻewalu i hala, e like me ka ʻikepili mai Dow Jones Market Data. He mea pono ke ʻike ua emi nā ʻāpana ma luna o 46% i kēia makahiki.
Manaʻo ʻia ka lele ʻana o ka waiwai mai ka ʻōlelo aʻoaʻo o ka biotech's gene therapies e ka Food and Drug Administration. Ma ka lā 9 o Iune, ua ʻōlelo ke Kōmike Kūkākūkā Cellular, Tissue, a me Gene Therapies o ka FDA i ka elivadogene autotmcel a i ʻole Eli-CEL gene therapy.
Hoʻohana ʻia kēia lāʻau lapaʻau i ka mālama ʻana i kahi maʻi i hoʻopili ʻia i ka chromosome X, ka adrenoleukodystrophy cerebral active mua. Ma ka Pōʻalima, ua ʻōlelo ke kino aupuni hoʻokahi iā Betibeglogene autotemcel a i ʻole beti-cel, he lāʻau lapaʻau hoʻokahi kēia i hoʻolālā ʻia e mālama i nā maʻi beta-thalassemia.
Ma hope o ka hoʻomaʻamaʻa ʻana, ʻaʻohe pono o ka hoʻololi ʻana i ke koko ʻulaʻula i nā poʻe maʻi i hoʻopilikia ʻia o ka maʻi, a i ʻole ke koi ʻia i kēlā me kēia manawa. Manaʻo ka FDA e hoʻoholo i ka manaʻo no ka beti-cel ma ka lā 19 o ʻAukake a ʻo ka lā no Eli-CEL ʻo ka 16 o Kepakemapa o kēia makahiki.
Panina
Me kēia nūhou maikaʻi, ua hoʻomaka ka poʻe e hoihoi i nā ʻāpana o ka hui a ʻo ia ke kumu e holo ai ka nūhou Bluebird Bio i nā wahi kālā ma nā mākeke. Ma kahi e hele ai ke kumukūʻai, manaʻo ʻia e pōmaikaʻi ka bluebird i kēia mau ʻōlelo aʻoaʻo.