Tsis ntev los no, peb cov tshuaj kho noob tau pom zoo rau kev lag luam, xws li: (1) Thaum Lub Xya Hli 21, 2022, PTC Therapeutics, Inc. (NASDAQ: PTCT) tshaj tawm tias nws AAV gene therapy Upstaza ™ tau pom zoo los ntawm European Commission Nws yog thawj cov tshuaj kho cov noob ncaj qha txhaj rau hauv lub hlwb (saib cov ntawv dhau los hauv Gene 'The Mile's therapy. hlwb raug pom zoo rau kev lag luam).(2) Lub Yim Hli 17, 2022, US Food and Drug Administration (FDA) tau pom zoo Bluebird Bio's gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) rau kev kho mob ntawm beta thalassemia.Qhov kev pom zoo ntawm txoj kev kho mob hauv Tebchaws Meskas yog qhov tsis txaus ntseeg "kev pab hauv cov daus" rau Bluebird Bio, uas yog qhov teebmeem nyiaj txiag.(3) Thaum Lub Yim Hli 24, 2022, BioMarin Pharmaceutical (BioMarin) tshaj tawm tias European Commission tau pom zoo rau kev lag luam ntawm ROCTAVIAN ™ (valoctocogene roxaparvovec), kev kho noob rau hemophilia A, rau kev kho mob ntawm cov neeg mob uas tsis muaj keeb kwm ntawm FVIII factor inhibitors thiab tsis zoo AAV5 antibodies hemophilia yav dhau los cov neeg mob uas muaj cov kab mob hemophilia. Ib txoj kev kho noob tau pom zoo rau kev lag luam).Txog tam sim no, 41 cov tshuaj kho noob tau pom zoo rau kev lag luam thoob ntiaj teb.

Gene yog lub hauv paus genetic unit uas tswj cov yam ntxwv.Tsuas yog cov noob ntawm qee cov kab mob, uas yog tsim los ntawm RNA, cov noob ntawm cov kab mob feem ntau yog tsim los ntawm DNA.Feem ntau cov kab mob ntawm cov kab mob yog tshwm sim los ntawm kev sib cuam tshuam ntawm cov noob thiab ib puag ncig, thiab ntau yam kab mob tuaj yeem kho los yog txo qis hauv cov ntsiab lus los ntawm kev kho noob.Gene therapy yog suav tias yog lub kiv puag ncig hauv kev kho mob thiab chaw muag tshuaj.Cov tshuaj kho mob dav dav suav nrog cov tshuaj raws li DNA hloov kho DNA cov tshuaj (xws li cov kab mob vector-raws li hauv vivo gene therapy tshuaj, hauv vitro gene therapy, tshuaj liab qab plasmid, thiab lwm yam) thiab cov tshuaj RNA (xws li tshuaj antisense oligonucleotide tshuaj, siRNA tshuaj, thiab mRNA gene therapy, thiab lwm yam);Cov tshuaj kho cov noob caj noob ces feem ntau suav nrog cov tshuaj plasmid DNA, cov tshuaj kho noob raws li cov kab mob kab mob, cov tshuaj kho noob raws li cov kab mob vectors, gene kho tshuab thiab cov tshuaj kho cell uas hloov kho hauv vitro.Tom qab xyoo ntawm kev txhim kho tortuous, cov tshuaj kho noob tau ua tiav cov txiaj ntsig kev kho mob.(tsis suav cov tshuaj tiv thaiv DNA thiab cov tshuaj tiv thaiv mRNA), 41 cov tshuaj kho noob tau pom zoo rau kev lag luam hauv ntiaj teb.Nrog rau kev tshaj tawm cov khoom lag luam thiab kev txhim kho sai ntawm cov thev naus laus zis thev naus laus zis, gene therapy yog hais txog mus rau lub sijhawm ntawm kev loj hlob sai.

Kev faib tawm ntawm kev kho noob (Duab qhov chaw: Biological Jingwei)

Kab lus no teev 41 hom kev kho mob uas tau pom zoo rau kev lag luam (tsis suav cov tshuaj tiv thaiv DNA thiab tshuaj tiv thaiv mRNA).

1. Kev kho hauv vitro noob

(1) Strimvelis

Lub tuam txhab: Tsim los ntawm GlaxoSmithKline (GSK).

Sijhawm rau kev ua lag luam: Pom zoo los ntawm European Union thaum lub Tsib Hlis 2016.

Cov Lus Qhia: Rau kev kho mob hnyav ua ke tiv thaiv kab mob (SCID).

Lus Cim: Cov txheej txheem dav dav ntawm txoj kev kho no yog thawj zaug tau txais tus neeg mob tus kheej hematopoietic qia hlwb, nthuav dav thiab kab lis kev cai hauv vitro, thiab tom qab ntawd siv cov kab mob retrovirus los qhia ib daim qauv ntawm kev ua haujlwm ADA (adenosine deaminase) noob rau hauv lawv cov hematopoietic qia hlwb, thiab thaum kawg hloov pauv cov kab mob hematopoietic qia hlwb.Hematopoietic qia hlwb yog infused rov qab rau hauv lub cev.Cov txiaj ntsig kho mob tau pom tias 3-xyoo txoj sia nyob ntawm ADA-SCID cov neeg mob kho nrog Strimvelis yog 100%.

(2) Zalmoxis

Lub tuam txhab: Tsim los ntawm MolMed, Ltalis.

Sijhawm rau kev ua lag luam: Tau txais kev tso cai ua lag luam EU hauv 2016.

Cov lus qhia: Nws yog siv rau kev kho mob ntxiv ntawm kev tiv thaiv kab mob ntawm cov neeg mob tom qab hematopoietic qia cell transplantation.

Lus Cim: Zalmoxis yog ib qho allogeneic T cell suicide gene immunotherapy hloov los ntawm cov kab mob retroviral.1NGFR thiab HSV-TK Mut2 tua tus kheej noob tso cai rau tib neeg siv ganciclovir txhua lub sijhawm los tua T hlwb uas ua rau muaj kev tiv thaiv kab mob tsis zoo, tiv thaiv kev tsis zoo ntxiv ntawm GVHD uas yuav tshwm sim, thiab rov ua kom lub cev tsis muaj zog hauv cov neeg mob haploidentical HSCT tom qab phais Escort.

(3) Invossa-K

Lub tuam txhab: Tsim los ntawm TissueGene (KolonTissueGene) tuam txhab.

Sijhawm mus ua lag luam: Pom zoo rau npe hauv South Kauslim thaum Lub Xya Hli 2017.

Indications: Rau kev kho mob ntawm degenerative hauv caug mob caj dab.

Lus Cim: Invossa-K yog ib qho kev kho mob ntawm cov noob caj noob ces uas cuam tshuam nrog tib neeg chondrocytes.Allogeneic hlwb yog hloov kho cov noob caj noob ces hauv vitro, thiab cov hlwb hloov tau tuaj yeem nthuav qhia thiab zais qhov hloov pauv kev loj hlob β1 (TGF-β1) tom qab txhaj tshuaj intra-articular.β1), yog li txhim kho cov tsos mob ntawm osteoarthritis.Cov txiaj ntsig kho mob qhia tau tias Invossa-K tuaj yeem txhim kho hauv caug mob caj dab.Daim ntawv tso cai raug tshem tawm los ntawm Kaus Lim Qab Teb tus tswj hwm tshuaj hauv xyoo 2019 vim tias cov chaw tsim khoom tsis raug sau npe cov khoom xyaw siv.

(4) Kev lag luam

Lub tuam txhab: Tsim los ntawm American bluebird bio (bluebird bio) tuam txhab.

Lub sijhawm ua lag luam: Pom zoo los ntawm European Union hauv 2019, thiab pom zoo los ntawm FDA thaum Lub Yim Hli 2022.

Cov lus qhia: Rau kev kho mob ntawm cov ntshav-dependent β-thalassemia.

Lus Cim: Zynteglo yog ib qho kev kho mob lentiviral hauv vitro noob, uas siv cov kab mob lentiviral los qhia txog kev ua haujlwm ntawm β-globin gene (βA-T87Q-globin gene) rau hauv hematopoietic qia hlwb tshem tawm ntawm cov neeg mob., thiab tom qab ntawd infuse cov genetic hloov kho autologous hematopoietic qia hlwb rov qab rau hauv tus neeg mob.Thaum tus neeg mob muaj cov noob βA-T87Q-globin ib txwm, lawv tuaj yeem tsim cov protein HbAT87Q ib txwm, uas tuaj yeem txo lossis tshem tawm qhov xav tau ntawm cov ntshav.Nws yog ib qho kev kho ib zaug tsim los hloov cov ntshav mus tas li thiab cov tshuaj noj tas mus li rau cov neeg mob hnub nyoog 12 xyoos thiab laus dua.

(5) Skysona

Lub tuam txhab: Tsim los ntawm American bluebird bio (bluebird bio) tuam txhab.

Sijhawm rau kev ua lag luam: Pom zoo los ntawm EU rau kev lag luam thaum Lub Xya Hli 2021.

Cov Lus Qhia: Rau kev kho mob thaum ntxov cerebral adrenoleukodystrophy (CALD).

Lus Ceeb Toom: Skysona gene therapy yog tib txoj kev kho noob ib zaug tau pom zoo rau kev kho mob thaum ntxov cerebral adrenoleukodystrophy (CALD).Skysona (elivaldogene autotemcel, Lenti-D) yog ib qho hematopoietic qia cell lentiviral hauv vitro gene therapy Lenti-D.Cov txheej txheem dav dav ntawm kev kho yog raws li hauv qab no: autologous hematopoietic qia hlwb raug tshem tawm ntawm tus neeg mob, hloov kho hauv vitro los ntawm lentivirus nqa tib neeg ABCD1 noob, thiab tom qab ntawd rov qab mus rau hauv tus neeg mob.Rau kev kho mob ntawm cov neeg mob hnub nyoog qis dua 18 xyoo nrog ABCD1 gene mutation thiab CALD.

(6) Kymriah

Lub tuam txhab: Tsim los ntawm Novartis.

Sijhawm mus ua lag luam: Pom zoo los ntawm FDA thaum Lub Yim Hli 2017.

Indications: Kev kho mob ntawm precursor B-cell mob lymphoblastic leukemia (TAG NRHO) thiab relapsed thiab refractory DLBCL.

Cov lus ceeb toom: Kymriah yog cov tshuaj lentiviral hauv vitro gene therapy, lub ntiaj teb thawj qhov kev pom zoo CAR-T kho, tsom rau CD19 thiab siv cov co-stimulatory factor 4-1BB.Tus nqi yog $ 475,000 hauv Asmeskas thiab $ 313,000 hauv Nyiv.

(7) Yescarta

Lub tuam txhab: Tsim los ntawm Kite Pharma, ib lub koom haum ntawm Gilead.

Sijhawm mus ua lag luam: Pom zoo los ntawm FDA thaum Lub Kaum Hli 2017.

Indications: Rau kev kho mob ntawm relapsed los yog refractory loj B-cell lymphoma.

Lus Cim: Yescarta yog ib qho kev kho mob hauv vitro gene therapy.Nws yog qhov thib ob txoj kev kho CAR-T pom zoo hauv ntiaj teb.Nws tsom rau CD19 thiab siv cov costimulatory yam ntawm CD28.Tus nqi hauv Asmeskas yog $ 373,000.

(8) Tecartus

Lub tuam txhab: Tsim los ntawm Gilead (GILD).

Sijhawm mus ua lag luam: Pom zoo los ntawm FDA thaum Lub Xya Hli 2020.

Indications: Rau relapsed los yog refractory mantle cell lymphoma.

Lus Ceeb Toom: Tecartus yog autologous CAR-T cell therapy targeting CD19, thiab yog qhov thib peb CAR-T kho tau pom zoo rau kev lag luam hauv ntiaj teb.

(9) Breyanzi

Lub tuam txhab: Tsim los ntawm Bristol-Myers Squibb (BMS).

Sijhawm mus ua lag luam: Pom zoo los ntawm FDA thaum Lub Ob Hlis 2021.

Indications: Relapsed los yog refractory (R / R) loj B-cell lymphoma (LBCL).

Lus Ceeb Toom: Breyanzi yog ib qho kev kho hauv vitro noob raws li lentivirus, thiab plaub txoj kev kho CAR-T tau pom zoo rau kev lag luam hauv ntiaj teb, tsom rau CD19.Qhov kev pom zoo ntawm Breyanzi yog ib qho tseem ceeb rau Bristol-Myers Squibb hauv kev kho mob ntawm tes, uas Bristol-Myers tau txais thaum nws tau Celgene rau $ 74 billion hauv 2019.

(10) Abecma

Lub tuam txhab: Co-tsim los ntawm Bristol-Myers Squibb (BMS) thiab bluebird bio.

Sijhawm mus ua lag luam: Pom zoo los ntawm FDA thaum Lub Peb Hlis 2021.

Indications: Relapsed los yog refractory ntau myeloma.

Lus Ceeb Toom: Abecma yog lentivirus-raws li hauv vitro gene therapy, lub ntiaj teb thawj CAR-T cell therapy aiming BCMA, thiab thib tsib CAR-T kho tau pom zoo los ntawm FDA.Lub hauv paus ntsiab lus ntawm cov tshuaj yog qhia cov chimeric BCMA receptor ntawm tus neeg mob lub autologous T hlwb los ntawm lentivirus-mediated genetic hloov kho hauv vitro.Ua ntej kev txhaj tshuaj ntawm cov tshuaj gene, tus neeg mob tau txais ob lub tebchaw ntawm cyclophosphamide thiab fludarabine rau kev kho mob ua ntej.Kev kho kom tshem tawm T hlwb tsis hloov pauv ntawm tus neeg mob, thiab tom qab ntawd muab cov T cells hloov pauv rov qab rau hauv tus neeg mob lub cev txhawm rau nrhiav thiab tua BCMA-qhia cov qog nqaij hlav cancer.

(11) Libmeldy

Lub tuam txhab: Tsim los ntawm Orchard Therapeutics.

Lub Sijhawm rau kev ua lag luam: Pom zoo los ntawm European Union rau npe rau lub Kaum Ob Hlis 2020.

Cov Lus Qhia: Rau kev kho mob ntawm metachromatic leukodystrophy (MLD).

Lus Ceeb Toom: Libmeldy yog ib txoj kev kho noob raws li kev hloov pauv hauv vitro noob hloov ntawm autologous CD34+ hlwb.Cov ntaub ntawv kho mob qhia tau hais tias ib qho kev txhaj tshuaj ntawm Libmeldy muaj txiaj ntsig zoo hauv kev hloov kho ntawm MLD thaum ntxov thiab lub cev muaj zog thiab kev paub tsis meej hauv cov neeg mob uas tsis muaj hnub nyoog tib yam.

(12) Benoda

Lub tuam txhab: Tsim los ntawm WuXi Junuo.

Sijhawm rau kev ua lag luam: Kev pom zoo los ntawm NMPA thaum lub Cuaj Hlis 2021.

Indications: Kev kho mob ntawm relapsed los yog refractory loj B-cell lymphoma (r / r LBCL) nyob rau hauv cov neeg laus tom qab ob kab los yog ntau tshaj kev kho mob.

Lus Cim: Benoda yog ib qho tshuaj tiv thaiv CD19 CAR-T noob kho, thiab nws tseem yog cov khoom tseem ceeb ntawm WuXi Junuo.Nws yog qhov thib ob CAR-T khoom pom zoo nyob rau hauv Suav teb, tshwj tsis yog rau relapsed / refractory loj B-cell lymphoma.Tsis tas li ntawd, WuXi Junuo tseem npaj yuav tsim Ruiki Orenza txhaj tshuaj rau kev kho mob ntawm ntau yam kev qhia, suav nrog follicular lymphoma (FL), mantle cell lymphoma (MCL), chronic lymphocytic leukemia (CLL), kab thib ob diffuse loj B-cell lymphoma (DLBCL) thiab mob lymphoblastic leukemia (ALL).

(13) TXOJ CAI

Lub tuam txhab: Legend Bio thawj cov khoom pom zoo.

Sijhawm mus ua lag luam: Pom zoo los ntawm FDA thaum Lub Ob Hlis 2022.

Indications: Kev kho mob ntawm relapsed los yog refractory ntau myeloma (R / R MM).

Lus Cim: CARVYKTI (ciltacabtagene autoleucel, hu ua Cilta-cel) yog CAR-T cell immune gene therapy with two single-domain antibodies targeting B cell maturation antigen (BCMA).Cov ntaub ntawv qhia tau hais tias CARVYKTI tau nthuav tawm tag nrho cov lus teb txog li 98% hauv cov neeg mob uas rov qab los lossis rov qab ntau yam myeloma uas tau txais plaub lossis ntau dua kev kho mob ua ntej, suav nrog cov tshuaj tiv thaiv proteasome, immunomodulators, thiab anti-CD38 monoclonal antibodies.

2. Hauv vivo gene kho raws li tus kab mob vectors

(1) Gendicine/yug dua

Lub tuam txhab: Tsim los ntawm Shenzhen Saibainuo Company.

Sijhawm rau kev ua lag luam: Pom zoo rau npe hauv Suav teb xyoo 2003.

Indications: Rau kev kho mob ntawm squamous cell carcinoma ntawm lub taub hau thiab caj dab.

Lus Cim: Recombinant human p53 adenovirus txhaj Gendicine/Jinshengsheng yog ib qho tshuaj adenovirus vector gene therapy nrog cov tswv cuab ywj pheej ywj pheej los ntawm Shenzhen Saibainuo Company.Cov tshuaj no yog tsim los ntawm tib neeg qog nqaij hlav suppressor gene p53 thiab cov khoom siv hloov kho recombinant replication-deficient Human adenovirus type 5 yog tsim los ntawm tib neeg adenovirus hom 5. Cov qub yog cov qauv tseem ceeb ntawm cov tshuaj kom ua rau cov qog nqaij hlav ua haujlwm, thiab tom kawg feem ntau ua raws li tus neeg nqa khoom.Tus kab mob adenovirus yog nqa cov tshuaj gene p53 mus rau hauv lub hom phiaj cell, thiab nthuav tawm cov qog suppressor noob p53 hauv lub hom phiaj ntawm tes.Cov khoom lag luam tuaj yeem tswj hwm ntau yam tshuaj tiv thaiv kab mob qog noj ntshav thiab txo qis cov haujlwm ntawm ntau yam oncogenes, yog li txhim kho cov nyhuv tiv thaiv qog ntawm lub cev thiab ua tiav lub hom phiaj ntawm kev tua cov qog.

(2) Rigvir

Lub tuam txhab: Tsim los ntawm Latvian tuam txhab Latima.

Lub sijhawm ua lag luam: Pom zoo hauv Latvia hauv xyoo 2004.

Indications: Rau kev kho mob ntawm melanoma.

Cov Lus Qhia: Rigvir yog kev kho noob raws li cov noob hloov pauv ECHO-7 enterovirus vector, uas tau siv hauv Latvia, Estonia, Poland, Armenia, Belarus thiab lwm qhov chaw, thiab tseem tau sau npe nrog EMA ntawm European Union..Cov kws kho mob hauv kaum xyoo dhau los tau ua pov thawj tias tus kab mob Rigvir oncolytic muaj kev nyab xeeb thiab siv tau zoo, thiab tuaj yeem txhim kho kev ciaj sia ntawm cov neeg mob melanoma los ntawm 4-6 zaug.Tsis tas li ntawd, txoj kev kho kuj tseem tsim nyog rau ntau yam mob qog noj ntshav, nrog rau mob qog nqaij hlav hauv plab, mob qog noj ntshav, mob qog noj ntshav.mob qog noj ntshav, mob raum, mob qog nqaij hlav prostate, mob ntsws cancer, mob qog noj ntshav hauv tsev menyuam, lymphosarcoma, thiab lwm yam.

(3) Oncorine/Ankerui

Lub tuam txhab: Tsim los ntawm Shanghai Sunway Biotechnology Co., Ltd.

Sijhawm rau kev ua lag luam: Pom zoo rau npe hauv Suav teb xyoo 2005.

Cov Lus Qhia: Kev kho mob ntawm lub taub hau thiab caj dab, mob qog noj ntshav, mob qog nqaij hlav pancreatic, mob qog noj ntshav thiab lwm yam qog noj ntshav.

Lus Cim: Oncorine yog ib qho kab mob oncolytic gene kho cov khoom siv adenovirus ua vector.Cov tau txais oncolytic adenovirus tuaj yeem rov ua dua tshwj xeeb hauv cov qog tsis muaj lossis txawv txav p53 noob, ua rau qog cell lysis, yog li tua cov qog hlwb.tsis ua kom puas cov hlwb.Cov txiaj ntsig kho mob pom tau tias Anke Rui muaj kev nyab xeeb zoo thiab ua haujlwm tau zoo rau ntau yam qog nqaij hlav.

(4) Glybera

Lub tuam txhab: Tsim los ntawm uniQure.

Sijhawm rau kev ua lag luam: Pom zoo hauv Tebchaws Europe xyoo 2012.

Cov Lus Qhia: Kev kho mob ntawm lipoprotein lipase deficiency (LPLD) nrog rau qhov mob hnyav lossis rov tshwm sim ntawm tus mob pancreatitis txawm tias muaj kev txwv nruj nruj.

Lus Cim: Glybera (alipogene tiparvovec) yog cov tshuaj kho noob raws li AAV ua vector.Txoj kev kho no siv AAV los ua tus vector los hloov cov tshuaj gene LPL rau hauv cov leeg nqaij, kom cov hlwb sib txuas tuaj yeem tsim cov lipoprotein lipase, nws ua lub luag haujlwm hauv kev txo cov kab mob, thiab qhov kev kho no siv tau ntev heev tom qab ib qho kev tswj hwm (cov nyhuv tuaj yeem kav ntau xyoo).Cov tshuaj raug tshem tawm hauv xyoo 2017, thiab vim li cas rau nws qhov kev tshem tawm yuav cuam tshuam rau ob yam: tus nqi siab dhau thiab txwv kev lag luam xav tau.Tus nqi nruab nrab ntawm ib qho kev kho mob ntawm cov tshuaj yog siab li 1 lab US las, thiab tsuas yog ib tus neeg mob tau yuav thiab siv nws txog tam sim no.Txawm hais tias lub tuam txhab tuav pov hwm kho mob tau them rov qab rau 900,000 US las, nws kuj yog lub nra hnyav rau lub tuam txhab pov hwm.Tsis tas li ntawd, cov lus qhia rau cov tshuaj yog tsawg heev, nrog rau qhov tshwm sim ntawm kwv yees li 1 ntawm 1 lab thiab tus nqi siab ntawm misdiagnosis.

(5) Kev ntseeg

Lub tuam txhab: Tsim los ntawm Amgen.

Sijhawm rau kev ua lag luam: Hauv 2015, nws tau pom zoo rau npe hauv Tebchaws Meskas thiab European Union.

Cov lus qhia: Kev kho mob ntawm melanoma qhov txhab uas tsis tuaj yeem tshem tawm tag nrho los ntawm kev phais.

Lus Cim: Imlygic yog ib qho kev hloov kho caj ces (lwm nws cov ICP34.5 thiab ICP47 gene fragments, thiab ntxig rau tib neeg granulocyte-macrophage colony-stimulating factor GM-CSF gene rau hauv tus kab mob) attenuated herpes simplex virus type 1 (HSV-1) oncolytic virus therapy .Txoj kev ntawm kev tswj hwm yog intralesional txhaj.Kev txhaj tshuaj ncaj qha rau hauv cov kab mob melanoma tuaj yeem ua rau tawg ntawm cov qog hlwb thiab tso cov qog-derived antigens thiab GM-CSF los txhawb kev tiv thaiv qog noj ntshav.

(6) Luxturna

Lub tuam txhab: Tsim los ntawm Spark Therapeutics, Roche cov chaw muag khoom.

Sijhawm mus ua lag luam: Pom zoo los ntawm FDA hauv 2017, thiab tom qab ntawd pom zoo rau kev lag luam hauv Tebchaws Europe xyoo 2018.

Cov Lus Qhia: Rau kev kho mob ntawm cov menyuam yaus thiab cov neeg laus uas tsis pom kev vim kev hloov pauv hauv ob daim qauv ntawm RPE65 noob tab sis muaj cov naj npawb txaus ntawm cov hlwb retinal.

Lus Ceeb Toom: Luxturna yog AAV-based gene therapy uas yog tswj hwm los ntawm kev txhaj tshuaj subretinal.Cov noob kev kho mob siv AAV2 los ua tus neeg nqa khoom los qhia txog kev ua haujlwm ntawm cov noob RPE65 ib txwm rau hauv cov hlwb ntawm tus neeg mob, kom cov hlwb sib txuas qhia cov protein RPE65 ib txwm los them rau qhov tsis xws ntawm tus neeg mob cov protein RPE65, yog li txhim kho tus neeg mob lub zeem muag.

(7) Zolgensma

Lub tuam txhab: Tsim los ntawm AveXis, ib lub koom haum ntawm Novartis.

Sijhawm mus ua lag luam: Pom zoo los ntawm FDA thaum lub Tsib Hlis 2019.

Indications: Kev kho mob ntawm tus txha caj qaum atrophy (Spinal Muscular Atrophy, SMA) cov neeg mob hnub nyoog qis dua 2 xyoos.

Lus Cim: Zolgensma yog kev kho noob raws li AAV vector.Cov tshuaj no yog tib txoj kev kho mob ib zaug rau tus txha caj qaum atrophy pom zoo rau kev lag luam hauv ntiaj teb.nplooj ntawv, yog ib qho kev nce qib tseem ceeb.Txoj kev kho noob no siv scAAV9 vector los qhia txog cov noob SMN1 ib txwm rau cov neeg mob los ntawm kev txhaj tshuaj intravenous, tsim cov protein SMN1 li qub, yog li txhim kho cov haujlwm ntawm cov hlwb cuam tshuam xws li lub cev muaj zog neurons.Hauv qhov sib piv, SMA cov tshuaj Spinraza thiab Evrysdi yuav tsum tau txhaj koob tshuaj ntau dua nyob rau lub sijhawm ntev, nrog Spinraza tau tswj hwm tus txha nraub qaum txhua plaub lub hlis, thiab Evrysdi, tshuaj noj qhov ncauj txhua hnub.

(8) Kev txiav txim siab

Lub tuam txhab: Tsim los ntawm Daiichi Sankyo Company Limited (TYO: 4568).

Sijhawm mus ua lag luam: Kev pom zoo los ntawm Nyiv Ministry of Health, Labor thiab Welfare (MHLW) thaum Lub Rau Hli 2021.

Indications: Rau kev kho mob ntawm malignant glioma.

Lus Cim: Delytact yog plaub tus kab mob oncolytic gene kho cov khoom tau pom zoo thoob ntiaj teb thiab thawj tus kab mob oncolytic cov khoom pom zoo rau kev kho mob ntawm glioma malignant.Delytact yog ib hom kab mob herpes simplex virus type 1 (HSV-1) oncolytic virus tsim los ntawm Dr. Todo thiab cov npoj yaig.Delytact qhia txog kev hloov pauv ntxiv rau hauv G207 genome ntawm tiam thib ob HSV-1, txhim kho nws cov kev xaiv rov ua dua tshiab hauv cov qog nqaij hlav cancer thiab qhov cuam tshuam ntawm kev tiv thaiv qog lub cev tiv thaiv kab mob, thaum muaj kev ruaj ntseg profile.Delytact yog thawj thib peb-tiam oncolytic HSV-1 tam sim no nyob rau hauv kev soj ntsuam soj ntsuam.Kev pom zoo ntawm Delytact hauv Nyij Pooj tau ua raws li ib leeg-caj npab Phase 2 kev sim tshuaj.Hauv cov neeg mob uas muaj glioblastoma rov tshwm sim, Delytact tau ntsib thawj qhov kawg ntawm kev muaj sia nyob ib xyoos, thiab cov txiaj ntsig tau pom tias Delytact ua tau zoo dua G207.Muaj zog replication thiab ntau dua antitumor kev ua.Qhov no ua tau zoo hauv cov qauv qog nqaij hlav xws li lub mis, prostate, schwannoma, nasopharyngeal, hepatocellular, colorectal, malignant peripheral paj sheath hlav thiab thyroid cancer.

(9) Cov

Lub tuam txhab: Tsim los ntawm PTC Therapeutics, Inc. (NASDAQ: PTCT).

Lub sijhawm ua lag luam: Pom zoo los ntawm EU thaum Lub Xya Hli 2022.

Indication: Rau aromatic L-amino acid decarboxylase (AADC) deficiency, pom zoo rau kev kho mob ntawm cov neeg mob 18 lub hlis thiab laus dua.

Lus Cim: Upstaza™ (eladocagene exuparvovec) yog ib qho kev kho mob hauv vivo siv cov kab mob adeno-associated virus type 2 (AAV2) ua vector.Tus neeg mob tau mob vim muaj kev hloov pauv hauv cov noob encoding AADC enzyme.AAV2 nqa cov noob noj qab haus huv encoding AADC enzyme.Cov nyhuv kho tau tiav nyob rau hauv daim ntawv ntawm kev them nyiaj caj ces.Hauv txoj kev xav, ib koob tshuaj ib zaug siv tau ntev.Nws yog thawj qhov kev lag luam gene therapy ncaj qha txhaj rau hauv lub hlwb.Daim ntawv tso cai ua lag luam muaj feem xyuam rau tag nrho 27 EU cov tswv cuab, nrog rau Iceland, Norway thiab Liechtenstein.

(9) Roctavian

Lub tuam txhab: Tsim los ntawm BioMarin Pharmaceutical (BioMarin).

Sijhawm rau kev ua lag luam: Pom zoo los ntawm EU thaum Lub Yim Hli 2022.

Cov Lus Qhia: Rau kev kho mob ntawm cov neeg laus uas muaj hemophilia A hnyav tsis muaj keeb kwm ntawm FVIII yam inhibition thiab AAV5 antibody tsis zoo.

Lus Ceeb Toom: Roctavian (valoctocogene roxaparvovec) siv AAV5 ua tus vector thiab siv tib neeg lub siab tshwj xeeb txhawb HLP los tsav cov kev qhia ntawm tib neeg coagulation yam yim (FVIII) nrog B domain deleted.Qhov kev txiav txim siab ntawm European Commission kom pom zoo rau kev lag luam ntawm valoctocogene roxaparvovec yog raws li cov ntaub ntawv tag nrho ntawm cov tshuaj kho mob kev loj hlob.Ntawm lawv, theem III kev soj ntsuam kuaj GENEr8-1 tau pom tias piv nrog cov ntaub ntawv ntawm lub xyoo ua ntej tso npe kawm, tom qab ib qho kev txhaj tshuaj ntawm valoctocogene roxaparvovec, Cov neeg kawm tau qis dua ib xyoos los ntshav ntau npaum li cas (ABR), tsis tshua muaj kev siv cov tshuaj recombinant factor VIII (F8) protein ntau hauv lub cev, los yog kev ua kom muaj ntshav ntau ntxiv.Tom qab 4 lub lis piam ntawm kev kho mob, cov neeg siv F8 txhua xyoo thiab ABR xav tau kev kho mob raug txo los ntawm 99% thiab 84%, feem, qhov sib txawv tseem ceeb (p<0.001).Cov ntaub ntawv kev nyab xeeb tau txais txiaj ntsig zoo, tsis muaj cov ncauj lus muaj kev cuam tshuam F8 qhov cuam tshuam, kev mob qog noj ntshav, lossis cov kab mob thrombotic, thiab tsis muaj kev kho mob cuam tshuam txog cov xwm txheej tsis zoo (SAEs) tau tshaj tawm.

3. Cov tshuaj me me nucleic acid

(1) Vitravene

Lub tuam txhab: Koom tes tsim los ntawm Ionis Pharma (yav tas los Isis Pharma) thiab Novartis.

Lub sijhawm ua lag luam: Pom zoo los ntawm FDA thiab EU EMA xyoo 1998 thiab 1999.

Cov Lus Qhia: Rau kev kho mob ntawm cytomegalovirus retinitis hauv cov neeg mob HIV.

Lus Cim: Vitravene yog ib qho tshuaj antisense oligonucleotide thiab thawj oligonucleotide tshuaj pom zoo rau kev lag luam hauv ntiaj teb.Thaum pib ntawm kev ua lag luam, kev lag luam xav tau rau cov tshuaj tiv thaiv cytomegalovirus yog ceev heev;Tom qab ntawd vim yog kev txhim kho ntawm kev siv tshuaj tiv thaiv kab mob siab heev, cov neeg mob cytomegalovirus poob qis heev.Vim qhov kev xav tau ntawm kev lag luam qis, cov tshuaj tau tso tawm hauv 2002 thiab 2006 Kev rho tawm hauv EU lub teb chaws thiab Asmeskas.

(2) Macugen

Lub tuam txhab: Co-tsim los ntawm Pfizer thiab Eyetech.

Sijhawm mus ua lag luam: Pom zoo rau npe hauv Tebchaws Meskas hauv xyoo 2004.

Cov Lus Qhia: Rau kev kho mob ntawm neovascular hnub nyoog ntsig txog macular degeneration.

Cov lus ceeb toom: Macugen yog cov tshuaj pegylated hloov kho oligonucleotide uas tuaj yeem tsom thiab khi rau vascular endothelial kev loj hlob zoo (VEGF165 isoform), thiab yog siv los ntawm kev txhaj tshuaj intravitreal.

(3) Defitelio

Lub tuam txhab: Tsim los ntawm Jazz.

Sijhawm mus ua lag luam: Pom zoo los ntawm European Union hauv 2013, thiab pom zoo los ntawm FDA thaum Lub Peb Hlis 2016.

Indications: Rau kev kho mob ntawm hepatic venule occlusive kab mob txuam nrog raum los yog pulmonary dysfunction tom qab hematopoietic qia cell transplantation.

Lus Cim: Defitelio yog ib qho tshuaj oligonucleotide, sib xyaw ntawm oligonucleotides nrog plasmin zog.Nws raug rho tawm hauv 2009 vim kev lag luam.

(4) Cov

Lub tuam txhab: Co-tsim los ntawm Ionis Pharma thiab Kastle.

Sijhawm mus ua lag luam: Pom zoo hauv Tebchaws Meskas raws li cov tshuaj menyuam ntsuag xyoo 2013.

Indications: Rau kev kho mob ntawm homozygous tsev neeg hypercholesterolemia.

Lus Cim: Kynamro yog ib qho tshuaj tiv thaiv oligonucleotide, ib qho tshuaj tiv thaiv oligonucleotide tsom rau tib neeg apo B-100 mRNA.Kynamro yog muab raws li 200 mg subcutaneously ib zaug ib lub lim tiam.

(5) Spinraza

Lub tuam txhab: Tsim los ntawm Ionis Pharmaceuticals.

Sijhawm mus ua lag luam: Pom zoo los ntawm FDA thaum Lub Kaum Ob Hlis 2016.

Indications: Rau kev kho mob ntawm tus txha caj qaum atrophy (SMA).

Lus Cim: Spinraza (nusinersen) yog tshuaj tua kab mob oligonucleotide.Spinraza tuaj yeem hloov pauv RNA splicing ntawm SMN2 noob los ntawm kev khi rau qhov chaw splicing ntawm SMN2 exon 7, yog li ua kom cov khoom ua tau zoo ntawm SMN protein ntau.Thaum Lub Yim Hli 2016, BIOGEN Corporation tau siv nws txoj kev xaiv kom tau Spinraza txoj cai thoob ntiaj teb.Spinraza tau pib nws thawj qhov kev sim tshuaj rau tib neeg hauv xyoo 2011. Tsuas yog 5 xyoos xwb, nws tau pom zoo los ntawm FDA hauv 2016, qhia txog FDA qhov kev lees paub tag nrho ntawm nws cov txiaj ntsig.Cov tshuaj tau pom zoo rau kev lag luam hauv Suav teb thaum lub Plaub Hlis 2019. Tag nrho cov kev pom zoo ntawm Spinraza hauv Suav teb yog tsawg dua 6 lub hlis.Nws tau 2 xyoos thiab 2 lub hlis txij li Spinraza tau pom zoo thawj zaug hauv Tebchaws Meskas.Xws li ib tug blockbuster txawv teb chaws tsis tshua muaj kab mob tshiab tshuaj yog nyob rau hauv Qhov ceev ntawm cov npe nyob rau hauv Tuam Tshoj yog twb ceev heev.Qhov no kuj yog vim "Daim Ntawv Ceeb Toom Txog Kev Tshaj Tawm ntawm Thawj Daim Ntawv Teev Npe ntawm Cov Tshuaj Kho Mob Tshiab Tseem Ceeb Yuav Tsum Tau Siv rau Kev Tshawb Fawb" muab los ntawm Lub Chaw Saib Xyuas Tshuaj rau Lub Kaum Ib Hlis 1, 2018, uas tau suav nrog thawj pawg ntawm 40 tseem ceeb txawv teb chaws cov tshuaj tshiab rau kev tshuaj xyuas nrawm, thiab Spinraza ranked nyob rau hauv.

(6) Exondys 51

Lub tuam txhab: Tsim los ntawm AVI BioPharma (tom qab hloov npe hu ua Sarepta Therapeutics).

Sijhawm mus ua lag luam: Pom zoo los ntawm FDA thaum lub Cuaj Hlis 2016.

Indications: Rau kev kho mob ntawm Duchenne nqaij dystrophy (DMD) nrog DMD noob hloov nyob rau hauv exon 51 hla gene.

Lus Cim: Exondys 51 yog ib qho tshuaj antisense oligonucleotide.Lub antisense oligonucleotide tuaj yeem khi rau exon 51 txoj haujlwm ntawm pre-mRNA ntawm DMD gene, uas ua rau tsim cov mRNA paub tab.Kev txiav tawm, yog li kho ib feem ntawm mRNA nyeem ntawv, pab tus neeg mob los ua ke qee yam kev ua haujlwm ntawm dystrophin uas luv dua li cov protein ib txwm, yog li txhim kho tus neeg mob cov tsos mob.

(7) Cov

Lub tuam txhab: Tsim los ntawm Ionis Pharmaceuticals.

Sijhawm rau kev ua lag luam: Pom zoo los ntawm European Union rau kev lag luam thaum Lub Xya Hli 2018.

Cov Lus Qhia: Rau kev kho mob ntawm cov kab mob transthyretin amyloidosis (hATTR).

Lus Cim: Tegsedi yog ib qho tshuaj tua kab mob oligonucleotide tsom rau transthyretin mRNA.Nws yog lub ntiaj teb thawj cov tshuaj pom zoo rau kev kho mob hATTR.Cov txheej txheem ntawm kev tswj hwm yog txhaj tshuaj subcutaneous.Cov tshuaj txo qis kev tsim cov ATTR protein los ntawm kev tsom mus rau mRNA ntawm transthyretin (ATTR), thiab muaj txiaj ntsig zoo-kev pheej hmoo ntawm kev kho ATTR.Tsis yog theem kab mob lossis qhov muaj ntawm cardiomyopathy tsis cuam tshuam.

(8) Cov

Lub tuam txhab: Co-tsim los ntawm Alnylam thiab Sanofi.

Sijhawm rau kev ua lag luam: Pom zoo rau npe hauv Tebchaws Meskas hauv 2018.

Cov Lus Qhia: Rau kev kho mob ntawm cov kab mob transthyretin amyloidosis (hATTR).

Cov Lus Qhia: Onpattro yog ib qho tshuaj siRNA tsom rau transthyretin mRNA, uas txo qis kev tsim cov ATTR protein nyob rau hauv daim siab thiab tsub zuj zuj ntawm amyloid deposits nyob rau hauv peripheral qab haus huv los ntawm lub hom phiaj ntawm mRNA ntawm transthyretin (ATTR)., yog li txhim kho thiab txo cov tsos mob ntawm tus kab mob.

(9) Givlaari

Lub tuam txhab: Tsim los ntawm Alnylam Corporation.

Sijhawm mus ua lag luam: Pom zoo los ntawm FDA thaum lub Kaum Ib Hlis 2019.

Cov Lus Qhia: Rau kev kho mob ntawm tus mob hepatic porphyria (AHP) hauv cov neeg laus.

Lus Cim: Givlaari yog ib qho tshuaj siRNA, cov tshuaj siRNA thib ob tau pom zoo rau kev lag luam tom qab Onpattro.Cov tshuaj yog siv rau subcutaneously thiab tsom rau mRNA rau degradation ntawm ALAS1 protein.Kev kho mob Givlaari txhua hli tuaj yeem txo qis qib ntawm ALAS1 hauv daim siab, yog li txo qis cov qib neurotoxic ALA thiab PBG mus rau qhov qub, yog li txo tus neeg mob cov tsos mob.Cov ntaub ntawv tau qhia tias cov neeg mob kho nrog Givlaari tau txo qis 74% ntawm cov kab mob flares piv rau cov placebo pawg.

(10) Vyondys53

Lub tuam txhab: Tsim los ntawm Sarepta Therapeutics.

Sijhawm mus ua lag luam: Pom zoo los ntawm FDA thaum Lub Kaum Ob Hlis 2019.

Indication: Rau kev kho mob ntawm DMD cov neeg mob nrog dystrophin gene exon 53 splice mutation.

Lus Cim: Vyondys 53 yog ib qho tshuaj antisense oligonucleotide.Cov tshuaj oligonucleotide tsom rau cov txheej txheem splicing ntawm dystrophin mRNA precursor.Nyob rau hauv cov txheej txheem indirect ntawm dystrophin mRNA precursor, sab nraud Exon 53 yog ib feem spliced ​​tawm, piv txwv li tsis muaj nyob rau mRNA mRNA paub tab, thiab tau tsim los tsim ib tug truncated tab sis tseem functional dystrophin protein, yog li txhim kho kev tawm dag zog lub cev rau cov neeg mob.

(11) Kev

Lub tuam txhab: Tsim los ntawm Ionis Pharmaceuticals thiab nws cov koom haum Akcea Therapeutics.

Sijhawm mus ua lag luam: Pom zoo los ntawm European Medicines Agency (EMA) thaum lub Tsib Hlis 2019.

Kev taw qhia: Raws li kev kho mob ntxiv rau kev tswj hwm kev noj zaub mov hauv cov neeg laus nrog tsev neeg chylomicronemia syndrome (FCS).

Lus Cim: Waylivra yog ib qho tshuaj antisense oligonucleotide, uas yog thawj cov tshuaj pom zoo rau kev kho mob ntawm FCS hauv ntiaj teb.

(12) Cov

Lub tuam txhab: Tsim los ntawm Novartis.

Sijhawm rau kev ua lag luam: Pom zoo los ntawm EU thaum Lub Kaum Ob Hlis 2020.

Indications: Rau kev kho mob ntawm cov neeg laus thawj hypercholesterolemia (heterozygous tsev neeg thiab tsis yog tsev neeg) los yog mixed dyslipidemia.

Lus Cim: Leqvio yog cov tshuaj siRNA tsom rau PCSK9 mRNA.Nws yog lub ntiaj teb thawj zaug txo cov roj cholesterol (LDL-C) siRNA therapy.Cov txheej txheem ntawm kev tswj hwm yog txhaj tshuaj subcutaneous.Cov tshuaj ua haujlwm los ntawm RNA cuam tshuam kom txo qis PCSK9 protein ntau, uas ua rau txo qis LDL-C qib.Cov ntaub ntawv kho mob qhia tau tias Leqvio tuaj yeem txo LDL-C los ntawm kwv yees li 50% hauv cov neeg mob uas nws qib LDL-C tsis tuaj yeem txo mus rau qib theem txawm tias qhov siab tshaj plaws ntawm cov tshuaj statins.

(13) Cov

Lub tuam txhab: Tsim los ntawm Alnylam Pharmaceuticals.

Sijhawm rau kev ua lag luam: Pom zoo los ntawm EU thaum Lub Kaum Ib Hlis 2020.

Indications: Rau kev kho mob ntawm thawj hyperoxaluria hom 1 (PH1).

Lus Cim: Oxlumo yog cov tshuaj siRNA tsom rau hydroxy acid oxidase 1 (HAO1) ​​mRNA, uas yog siv rau subcutaneously.Cov tshuaj tau tsim los siv Alnylam qhov tseeb txhim kho stabilization tshuaj ESC-GalNAc conjugation technology, uas ua rau subcutaneously tswj siRNAs nrog ntau dua persistence thiab ua tau zoo.Cov tshuaj tsom mus rau kev degradation lossis inhibition ntawm hydroxy acid oxidase 1 (HAO1) ​​mRNA, txo cov qib ntawm glycolate oxidase hauv daim siab, thiab tom qab ntawd noj cov substrate uas xav tau rau kev tsim cov oxalate thiab txo cov oxalate ntau lawm los tswj cov kab mob thiab txhim kho cov tsos mob ntawm tus mob.

(14) Cov

Lub tuam txhab: Tsim los ntawm NS Pharma, ib lub koom haum ntawm Nippon Shinyaku.

Sijhawm mus ua lag luam: Pom zoo los ntawm FDA thaum Lub Yim Hli 2020.

Cov Lus Qhia: Rau kev kho mob ntawm Duchenne cov leeg nqaij dystrophy (DMD) nrog DMD noob hloov hauv exon 53 hla cov noob.

Lus Cim: Viltepso yog phosphorodiamide morpholino oligonucleotide tshuaj.Cov tshuaj oligonucleotide no tuaj yeem khi rau exon 53 txoj haujlwm ntawm pre-mRNA ntawm DMD gene, uas ua rau tsim cov mRNA paub tab.Cov exon raug tshem tawm ib nrab, yog li ib feem kho cov mRNA nyeem ntawv, pab tus neeg mob los ua ke qee cov haujlwm ntawm dystrophin uas luv dua li cov protein ib txwm, yog li txhim kho tus neeg mob cov tsos mob.

(15) Lub cev muaj zog

Lub tuam txhab: Tsim los ntawm Alnylam Pharmaceuticals.

Sijhawm mus ua lag luam: Pom zoo los ntawm FDA thaum Lub Rau Hli 2022.

Cov Lus Qhia: Rau kev kho mob ntawm cov neeg laus cov kab mob transthyretin amyloidosis nrog polyneuropathy (hATTR-PN).

Lus Cim: Amvuttra (Vutrisiran) yog ib qho tshuaj siRNA tsom rau transthyretin (ATTR) mRNA, uas yog siv los ntawm kev txhaj tshuaj subcutaneous.Vutrisiran yog tsim los ntawm Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc conjugated tus me nyuam platform nrog nce potency thiab metabolic stability.Qhov kev pom zoo ntawm txoj kev kho yog raws li 9-hli cov ntaub ntawv los ntawm nws qhov kev tshawb fawb Phase III (HELIOS-A), nrog rau tag nrho cov txiaj ntsig qhia tau hais tias kev kho kom zoo dua cov tsos mob ntawm hATTR-PN, nrog ntau dua 50% ntawm cov neeg mob thim rov qab lossis nres kev loj hlob.

4. Lwm yam tshuaj kho noob caj noob ces

(1) Rexin-G

Lub tuam txhab: Tsim los ntawm Epeius Biotech.

Sijhawm mus ua lag luam: Pom zoo los ntawm Philippine Food and Drug Administration (BFAD) hauv 2005.

Cov Lus Qhia: Rau kev kho mob qog noj ntshav siab heev uas tiv taus cov kws khomob.

Lus Cim: Rexin-G yog ib qho tshuaj gene-loaded nanoparticle txhaj.Nws qhia txog cyclin G1 mutant gene mus rau hauv lub hom phiaj hlwb los ntawm retroviral vector los tua cov qog nqaij hlav.Txoj kev ntawm kev tswj hwm yog intravenous infusion.Raws li cov tshuaj qog nqaij hlav uas nquag nrhiav thiab rhuav tshem cov qog nqaij hlav qog noj ntshav, nws muaj qee yam cuam tshuam rau cov neeg mob uas tsis muaj txiaj ntsig tiv thaiv lwm cov tshuaj mob qog noj ntshav, suav nrog cov tshuaj biologics.

(2) Neovasculgen

Lub tuam txhab: Tsim los ntawm Human stem cells institute.

Lub Sijhawm Teev Npe: Pom zoo rau npe hauv tebchaws Russia thaum Lub Kaum Ob Hlis 7, 2011, thiab tom qab ntawd sau npe hauv Ukraine hauv 2013.

Indications: Rau kev kho mob ntawm peripheral arterial kab mob, nrog rau mob heev limb ischemia.

Lus Cim: Neovasculgen yog DNA plasmid-based gene therapy nyob rau hauv uas vascular endothelial kev loj hlob yam (VEGF) 165 noob yog tsim los ntawm ib tug plasmid qaum thiab infused rau cov neeg mob.

(3) Collategene

Lub tuam txhab: Co-tsim los ntawm Osaka University thiab venture peev tuam txhab.

Lub Sijhawm Teev Npe: Pom zoo los ntawm Nyiv Lub Tsev Haujlwm Saib Xyuas Kev Noj Qab Haus Huv, Kev Ua Haujlwm thiab Kev Noj Qab Haus Huv rau kev teev npe thaum Lub Yim Hli 2019.

Cov Lus Qhia: Kev kho mob ntawm qhov qis qis qis kawg nkaus ischemia.

Lus Cim: Collategene yog plasmid-based gene therapy, thawj haiv neeg Japanese tshuaj kho cov tshuaj uas tsim los ntawm AnGes.Lub ntsiab tseem ceeb ntawm cov tshuaj no yog liab qab plasmid uas muaj cov noob sib lawv liag ntawm human hepatocyte growth factor (HGF).Yog tias cov tshuaj tau txhaj rau hauv cov leeg ntawm cov ceg qis, qhov qhia HGF yuav txhawb kev tsim cov hlab ntsha tshiab nyob ib ncig ntawm cov hlab ntsha.Kev sim tshuaj kho mob tau lees paub tias nws cov txiaj ntsig zoo hauv kev txhim kho rwj.

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