Iindaba zeBluebird Bio: Iindaba ezilungileyo ezivela kwi-FDA

Ngaba uyazilandela iindaba zeBluebird Bio? Ukuba awukho, lixesha lokuba wazi kwaye uvule izaziso zakho kulo lonke uhlaziyo lwamva nje malunga nale nkampani. Kuba ibekwe ukuba ifikelele kwiindawo eziphakamileyo nangawuphi na umzuzu.

Kulindeleke ukuba izitokhwe zale nkampani zinyuke ziye phezulu njengoko ikomiti ecebisayo yoLawulo lokuTya kunye neDrugs (i-FDA) icebise ngovavanyo olubini lwale nkampani yebhayothekhi yonyango lovavanyo lofuzo.

Ke usenokuba uzibonile izitokhwe zenkampani zinyuka zinyuka kuphela. Ngolwazi lwakho, inqaku elithi 'BLUE' onokuthi ulibonile kwizikrini lelenkampani ithile. Ke ngaphandle kwemeko yentengiso iyonke, abaninizabelo bale nkampani bafumana ukuphumla okufunekayo kakhulu.

Iindaba ezibalulekileyo zeBluebird Bio

Umfanekiso weBluebird bio iindaba

Le yinkampani yaseCambridge, eMassachusetts esekwe kwi-biotechnology egxile ekuphuhliseni unyango lwemfuza yezifo ezinzima zemfuzo kunye nomhlaza. Ngaphambili, ichiza elivunyiweyo kuphela elivela kwiManyano yaseYurophu (EU) yayiyiBetigeglogene autotemcel eqhele ukuhamba ngegama (Zynteglo).

Ukukukhumbuza, eli liyeza lesibini kwawona maxabiso aphakamileyo ehlabathini axabisa i-1.8 yezigidi zeerandi. Ngokunokwenzeka okuninzi inkampani yabona izabelo zayo zinyuka kodwa bezisehla kancinci kude kube ngoku. Ngokuvunywa kweendlela ezimbini zonyango, kulindeleke ukuba ibuyisele ukuzithemba okulahlekileyo kwikamva layo kubatyali-mali.

Eminye imisebenzi eyenziwa yile nkampani iquka unyango lwemfuza yeLentiGlobin kwiSickle cell disease kunye neCerebral Adrenoleukodystrophy. I-IT ikwasebenzela ukunyanga i-Acute Myeloid leukemia, i-Merkel-cell carcinoma, i-MAGAA4 izicubu eziqinileyo, kunye ne-Diffuse B-cell lymphoma enkulu.

Ukuqala uhambo lwayo njengeGenetix Pharmaceuticals ngo-1992 ingqondo yamalungu e-MIT faculty i-Irving London kunye no-Philippe Leboulch, eli qumrhu le-biotechnology libone izabelo zalo zinyuka ukuya kuthi ga kwi-178.29 yeedola ngo-2018 kwaye emva koko, babekwindlela yokuwa ngokubanzi.

Kodwa ngale ndaba, izabelo zanda malunga ne-28.7% ukuya kwi-4.80 ngoMvulo we-14 Juni 2022. Izitokhwe zisendleleni yokunyuka kwepesenti enkulu kwiminyaka esibhozo edlulileyo, ngokwedatha evela kwiDatha ye-Dow Jones Market. Kuyafaneleka ukwazi ukuba izabelo ziphantsi ngaphezu kwe-46% kulo nyaka.

Ukutsiba kwexabiso kulindeleke kwingcebiso yonyango lwemfuza yebhayothekhi nguLawulo lokuTya kunye neChiza. Ngomhla we-9 kaJuni iKomiti yeeNgcebiso zeSelula, iTissue, kunye neGene Therapies ye-FDA icebise nge-elivadogene autotmcel okanye i-Eli-CEL gene therapy.

Olu nyango lusebenza kunyango lwesifo esidityaniswe ne-X chromosome, i-cerebral adrenoleukodystrophy yokuqala esebenzayo. NgoLwesihlanu, iqumrhu likarhulumente elifanayo licebise iBetibeglogene autotemcel okanye i-beti-cel, olu lunyango lwexesha elinye oluyilelwe ukunyanga abaguli be-beta-thalassemia.

Emva kokonyango, akuyi kubakho imfuneko yokumpontshelwa kweeseli ezibomvu zegazi kwizigulane ezichaphazelekayo zesifo, ezinokuthi zifuneke rhoqo. I-FDA kulindeleke ukuba yenze isigqibo esisemthethweni se-beti-cel ngomhla we-19 ka-Agasti kwaye umhla we-Eli-CEL ngumhla we-16 kaSeptemba kulo nyaka.

isiphelo

Ngezi ndaba ezinkulu, abantu baye baqala ukuthatha umdla kwizabelo zenkampani kwaye yingakho iindaba ze-Bluebird Bio zenza imijikelezo kwiikota zemali kwiimarike. Kungakhathaliseki ukuba ixabiso liya phi, ibluebird kulindeleke ukuba ixhamle kakhulu kwezi ngcebiso.

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