Cov noob caj noob ces yog cov caj ces tseem ceeb 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.Gene therapy tuaj yeem kho tau lossis txo ntau yam kab mob.Gene therapy yog suav tias yog lub kiv puag ncig hauv kev kho mob thiab chaw muag tshuaj.Gene therapy tshuaj nyob rau hauv ib tug dav dav muaj xws li raws li cov DNA hloov DNA tshuaj (xws li nyob rau hauv vivo gene therapy tshuaj raws li tus kab mob vectors, nyob rau hauv vitro gene therapy tshuaj, liab qab plasmid tshuaj, thiab lwm yam) thiab RNA tshuaj (xws li antisense oligonucleotide tshuaj, siRNA tshuaj, thiab mRNA noob therapy, thiab lwm yam);nqaim siab Gene therapy tshuaj 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 rau hauv vitro gene hloov kho.Tom qab xyoo ntawm kev txhim kho tortuous, cov tshuaj kho noob tau ua tiav kev txhawb nqa kev kho mob.(Tsis suav cov tshuaj tiv thaiv DNA thiab cov tshuaj tiv thaiv mRNA) Tam sim no, 45 cov tshuaj kho noob tau pom zoo rau kev lag luam hauv ntiaj teb.Tag nrho ntawm 9 txoj kev kho noob tau pom zoo rau kev lag luam xyoo no, suav nrog 7 kev kho noob tau pom zoo rau kev lag luam thawj zaug xyoo no, xws li: CARVYKTI, Amvuttra, Upstaza, Roctavian, Hemgenix, Adstiladrin thiab Ebvallo, (Ceeb Toom: Lwm qhov ob tau pom zoo hauv Tebchaws Meskas lub xyoo no. Cov kev ua lag luam tau pom zoo thawj zaug Zenteg batch: los ntawm FDA rau kev lag luam hauv Tebchaws Meskas thaum Lub Yim Hli 2022, thiab tau pom zoo rau kev lag luam los ntawm European Union hauv 2019; .) Nrog rau kev tshaj tawm ntau thiab ntau cov khoom kho noob caj noob ces thiab kev loj hlob sai ntawm kev kho noob 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: Bio-Matrix)

Kab lus no teev 45 cov tshuaj kho noob (tsis suav cov tshuaj tiv thaiv DNA thiab cov tshuaj tiv thaiv mRNA) uas tau pom zoo rau kev lag luam.

1. Kev kho hauv vitro noob

(1) Strimvelis

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

Lub sijhawm ua lag luam: Nws tau pom zoo rau kev lag luam 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, tom qab ntawd siv cov tshuaj retrovirus los qhia txog kev ua haujlwm ADA (adenosine deaminase) cov noob luam rau hauv cov kab mob hematopoietic, thiab thaum kawg txhaj tshuaj hloov kho Hematopoietic qia hlwb rov qab rau hauv lub cev.Cov txiaj ntsig kho mob pom tau 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 Ltalis MolMed Company.

Sijhawm rau kev ua lag luam: Tau txais kev tso cai ua lag luam raws cai los ntawm European Union 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.Txoj kev no siv cov kab mob retroviral los hloov cov noob caj noob ces allogeneic T hlwb, kom cov genetic hloov T hlwb nthuav qhia 1NGFR thiab HSV-TK Mut2 tua tus kab mob tso cai rau tib neeg siv cov tshuaj ganciclovir (ganciclovir) txhua lub sij hawm los tua T hlwb uas ua rau muaj kev phiv tshuaj tiv thaiv kab mob, tiv thaiv kom tsis txhob muaj kev puas tsuaj ntxiv rau cov neeg mob ntshav qab zib hom CT, thiab kev ua haujlwm ntawm HS. cort.

(3) Invossa-K

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

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.Cov hlwb allogeneic yog hloov kho cov noob caj noob ces hauv vitro, thiab cov hlwb hloov tau tuaj yeem nthuav tawm thiab zais qhov hloov pauv 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.Nws raug tshem tawm hauv 2019 los ntawm Kaus Lim Kauslim Food and Drug Administration vim tias cov chaw tsim khoom siv mislabeled cov khoom xyaw siv.

(4) Kev lag luam

Lub tuam txhab: Tshawb nrhiav thiab tsim los ntawm bluebird bio.

Sijhawm rau kev ua lag luam: Pom zoo los ntawm European Union rau kev lag luam hauv 2019, thiab pom zoo los ntawm FDA rau kev lag luam hauv Tebchaws Meskas 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 gene uas qhia txog kev ua haujlwm ntawm β-globin gene (βA-T87Q-globin gene) rau hauv hematopoietic qia hlwb coj los ntawm tus neeg mob los ntawm cov kab mob lentiviral , thiab tom qab ntawd rov ua cov genetic hloov kho rau hauv cov neeg mob hematopoietic.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 kev 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: Tshawb nrhiav thiab tsim los ntawm bluebird bio.

Lub sijhawm ua lag luam: Pom zoo los ntawm European Union rau kev lag luam thaum Lub Xya Hli 2021, thiab tau pom zoo los ntawm FDA rau kev lag luam hauv Tebchaws Meskas thaum lub Cuaj Hli 2022.

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 txoj kev kho yog raws li hauv qab no: autologous hematopoietic qia hlwb raug coj tawm ntawm tus neeg mob, hloov pauv thiab hloov kho los ntawm lentivirus nqa tib neeg ABCD1 noob hauv vitro, thiab tom qab ntawd rov qab mus rau tus neeg mob.Nws yog siv los kho cov neeg mob hnub nyoog qis dua 18 xyoo, nqa ABCD1 cov noob hloov pauv, thiab CALD.

(6) Kymriah

COMPANY: Tsim los ntawm Novartis.

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

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

Lus Cim: Kymriah yog ib qho tshuaj tua kab mob hauv vitro gene therapy, thawj CAR-T kho tau pom zoo rau kev lag luam hauv ntiaj teb, tsom rau CD19, thiab siv 4-1BB co-stimulatory factor.Nws yog tus nqi ntawm $ 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 (GILD).

Sijhawm mus ua lag luam: Pom zoo los ntawm FDA rau kev lag luam thaum Lub Kaum Hli 2017;Fosun Kite qhia Yescarta thev naus laus zis los ntawm Kite Pharma thiab tsim tawm hauv Suav teb tom qab tau txais kev tso cai.Pom zoo rau cov npe hauv lub tebchaws.

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, uas yog qhov thib ob pom zoo CAR-T kho hauv ntiaj teb.Nws tsom rau CD19 thiab txais yuav cov costimulator ntawm CD28.Nws yog tus nqi ntawm $ 373,000 hauv Tebchaws Meskas.

(8) Tecartus

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

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

Indications: Rau relapsed los yog refractory mantle cell lymphoma.

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

(9) Breyanzi

COMPANY: Tsim los ntawm Bristol-Myers Squibb (BMS).

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

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

Cov Lus Qhia: Breyanzi yog ib qho kev kho hauv vitro noob raws li lentivirus, 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 nws tau txais thaum nws tau Celgene rau $ 74 nphom hauv 2019.

(10) Abecma

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

Sijhawm rau kev ua lag luam: Pom zoo los ntawm FDA rau kev lag luam hauv lub Peb Hlis 2021.

Indications: relapsed los yog refractory ntau myeloma.

Lus Ceeb Toom: Abecma yog ib qho kev kho hauv vitro noob raws li lentivirus, lub ntiaj teb thawj CAR-T cell therapy targeting BCMA, thiab thib tsib CAR-T kho tau pom zoo los ntawm FDA.Lub hauv paus ntsiab lus ntawm cov tshuaj yog qhia chimeric BCMA receptors ntawm tus neeg mob tus kheej T hlwb los ntawm lentivirus-mediated noob hloov hauv vitro.Kev kho mob txhawm rau tshem tawm cov tsis muaj noob caj noob ces hloov T hlwb hauv cov neeg mob, thiab tom qab ntawd rov kho cov T cells, uas nrhiav thiab tua BCMA-qhia cov qog nqaij hlav cancer hauv cov neeg mob.

(11) Libmeldy

COMPANY: 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 Cim: Libmeldy yog ib txoj kev kho noob raws li autologous CD34+ hlwb hloov kho hauv vitro los ntawm lentivirus.Cov ntaub ntawv kho mob qhia tau hais tias ib qho kev txhaj tshuaj ntawm Libmeldy tuaj yeem hloov kho qhov pib ntawm MLD thaum ntxov piv nrog lub cev muaj zog thiab kev paub tsis meej hauv cov neeg mob uas tsis tau kho ntawm tib lub hnub nyoog.

(12) Benoda

Lub tuam txhab: Tsim los ntawm WuXi Giant Nuo.

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

Indications: Kev kho mob ntawm cov neeg laus uas muaj relapsed los yog refractory loj B-cell lymphoma (r / r LBCL) tom qab ob-kab los yog saum toj no kev kho mob.

Lus Cim: Beinoda yog ib qho tshuaj tiv thaiv CD19 CAR-T noob kho, thiab nws tseem yog cov khoom tseem ceeb ntawm WuXi Juro Company.Nws yog qhov thib ob CAR-T cov khoom tau pom zoo nyob rau hauv Suav teb, tshwj tsis yog rau relapsed / refractory loj B-cell lymphoid WuXi Giant Nuo kuj npaj yuav tsim Regiorensai txhaj rau kev kho mob ntawm ntau lwm yam indications, xws li follicular lymphoma (FL), mantle cell lymphoma (MCL), chronic lymphocytic leukemia (CLL), ob-cell lymphocytic leukemia (CLL) , ob-hlwb (CLL) lymphocytic leukemia (CLL) ).

(13) TXOJ CAI

Lub tuam txhab: Legend Biotech thawj cov khoom pom zoo rau kev lag luam.

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

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

Lus Cim: CARVYKTI (ciltacabtagene autoleucel, Cilta-cel luv luv) yog CAR-T cell immune gene therapy nrog ob hom tshuaj tiv thaiv ib leeg tsom rau B-cell maturation antigen (BCMA).Cov ntaub ntawv qhia tau hais tias CARVYKTI Hauv cov neeg mob uas rov qab los lossis rov qab muaj ntau yam myeloma uas tau txais plaub lossis ntau dua kev kho mob ua ntej (xws li proteasome inhibitors, immunomodulators thiab anti-CD38 monoclonal antibodies), tag nrho cov lus teb ntawm 98% tau pom.

(14)Ebvallo

COMPANY: Tsim los ntawm Atara Biotherapeutics.

European Commission (EC) rau kev lag luam nyob rau lub Kaum Ob Hlis 2022, nws yog lub ntiaj teb thawj universal T cell kho tau pom zoo rau kev lag luam.

Cov Lus Qhia: Raws li kev kho mob monotherapy rau Epstein-Barr tus kab mob (EBV) cuam tshuam txog tom qab hloov pauv cov kab mob lymphoproliferative (EBV + PTLD), cov neeg mob tau txais kev kho mob yuav tsum yog cov neeg laus thiab cov menyuam yaus hnub nyoog qis dua 2 xyoos uas yav tas los tau txais tsawg kawg ib qho kev kho tshuaj.

Lus Ceeb Toom: Ebvallo yog ib qho allogeneic EBV-specific universal T-cell noob kho uas lub hom phiaj thiab tshem tawm cov kab mob EBV nyob rau hauv HLA-txheej txheem.Kev pom zoo ntawm txoj kev kho no yog ua raws li cov txiaj ntsig ntawm qhov tseem ceeb ntawm theem 3 kev sim tshuaj ntsuam xyuas, thiab cov txiaj ntsig tau pom tias ORR ntawm pawg HCT thiab pawg SOT yog 50%.Qhov ua tiav kev zam txim (CR) tus nqi yog 26.3%, qhov kev tshem tawm ib nrab (PR) tus nqi yog 23.7%, thiab lub sijhawm nruab nrab ntawm kev zam txim (TTR) yog 1.1 lub hlis.Ntawm 19 tus neeg mob uas tau ua tiav kev zam txim, 11 muaj lub sijhawm teb (DOR) ntau dua 6 lub hlis.Tsis tas li ntawd, hais txog kev nyab xeeb, tsis muaj kev cuam tshuam tsis zoo xws li kab mob graft-tiv thaiv tus tswv tsev (GvHD) lossis Ebvallo-related cytokine release syndrome tshwm sim.

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

(1) Gendicine/Jin Sheng

Lub tuam txhab: Tsim los ntawm Shenzhen Saibainuo Company.

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

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

Nco tseg: Recombinant human p53 adenovirus txhaj Gendicine/Jinyouheng yog ib qho tshuaj adenovirus vector gene therapy nrog cov tswv cuab ywj pheej ywj pheej uas yog los ntawm Shenzhen Saibainuo Company.Tib neeg hom 5 adenovirus yog tsim los ntawm tib neeg adenovirus hom 5. Tus qub yog cov qauv tseem ceeb rau kev tiv thaiv qog noj ntshav ntawm cov tshuaj, thiab tom kawg yog ua raws li tus neeg nqa khoom.Lub adenovirus vector nqa cov tshuaj gene p53 mus rau hauv lub hom phiaj cell, qhia cov qog suppressor noob p53 nyob rau hauv lub hom phiaj cell, thiab nws cov gene qhia Cov khoom muaj peev xwm up-regulate ntau yam anti-cancer genes thiab down-regulate cov dej num ntawm ntau yam oncogenes, li no pab txhawb lub cev lub qog suppressor lub hom phiaj ntawm tua.

(2) Rigvir

Lub tuam txhab: Tsim los ntawm Latima Company, Latvia.

Lub Sijhawm Teev Npe: Pom zoo rau npe hauv Latvia xyoo 2004.

Indications: Rau kev kho mob ntawm melanoma.

Lus Cim: Rigvir yog ib txoj kev kho noob raws li kev hloov kho caj ces ECHO-7 enterovirus vector.Tam sim no, cov tshuaj tau txais kev pom zoo hauv Latvia, Estonia, Poland, Armenia, Belarus, thiab lwm yam, thiab tseem tab tom ua EMA rau npe hauv EU lub teb chaws.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 ua rau kom muaj sia nyob ntawm cov neeg mob melanoma los ntawm 4-6 zaug.Tsis tas li ntawd, txoj kev kho no kuj tseem siv tau 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 qog noj ntshav, mob raum, mob qog noj ntshav, mob ntsws cancer, mob qog noj ntshav hauv tsev menyuam, lymphosarcoma, thiab lwm yam.

(3) Oncorine

Lub tuam txhab: Tsim los ntawm Shanghai Sanwei Biological Company.

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

Cov lus qhia: kho mob taub hau thiab caj dab, mob qog noj ntshav, mob qog noj ntshav, mob qog noj ntshav thiab lwm yam qog noj ntshav.

Lus Cim: Oncorine (安科瑞) yog ib qho kev kho kab mob oncolytic gene kho cov khoom siv adenovirus ua tus cab.Tus kab mob oncolytic adenovirus tau txais, uas tuaj yeem rov ua dua tshiab hauv p53 gene deficiency lossis qog nqaij hlav txawv txav, ua rau cov lysis ntawm cov qog hlwb, yog li tua cov qog hlwb.tsis ua kom puas cov hlwb.Cov kev tshawb fawb soj ntsuam tau pom tias Ankerui muaj kev nyab xeeb zoo thiab ua haujlwm tau zoo rau ntau hom qog nqaij hlav.

(4) Glybera

Lub tuam txhab: Tsim los ntawm uniQure.

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

Cov lus ceeb toom: Glybera (alipogene tiparvovec) yog cov tshuaj kho noob raws li AAV, uas siv AAV ua tus cab kuj los hloov cov tshuaj kho cov noob LPL rau hauv cov leeg hlwb, kom cov hlwb sib txuas tuaj yeem tsim cov lipoprotein lipase ntau npaum li cas, txhawm rau txo tus kab mob, qhov kev kho no siv tau ntau xyoo.Cov tshuaj raug rho tawm ntawm kev ua lag luam hauv 2017. Qhov laj thawj rau nws qhov kev rho tawm tuaj yeem cuam tshuam rau ob yam: tus nqi siab thiab kev xav tau ntawm kev lag luam tsawg.Tus nqi kho mob nruab nrab ntawm cov tshuaj yog siab li US $ 1 lab, 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 US $ 900,000 rau nws, nws tseem yog lub nra hnyav rau lub tuam txhab pov hwm.Tsis tas li ntawd, cov lus qhia tsom los ntawm 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 xyoo 2015, nws tau pom zoo los teev 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.

Cov lus ceeb toom: Imlygic yog tus kab mob herpes simplex virus hom 1 uas tau hloov kho los ntawm cov cuab yeej cuab tam (tso tseg 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 no) (HSV-1tic gene) oncoly virus.Cov txheej txheem ntawm kev tswj hwm yog kev txhaj tshuaj intralesional, uas tuaj yeem raug txhaj ncaj qha rau hauv cov kab mob melanoma kom ua rau cov qog hlwb tawg, tso cov qog-derived antigens thiab GM-CSF, thiab txhawb kev tiv thaiv qog noj ntshav.

(6) Luxturna

Lub tuam txhab: Tsim los ntawm Spark Therapeutics, ib lub koom haum ntawm Roche.

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

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

Lus Cim: Luxturna yog AAV-raws li kev kho noob caj noob ces tswj 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 tus neeg mob cov hlwb retinal, kom cov hlwb sib txuas qhia cov protein RPE65 ib txwm, ua rau tus neeg mob tsis muaj protein ntau 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 rau kev ua lag luam: Pom zoo los ntawm FDA rau kev lag luam hauv lub Tsib Hlis 2019.

Indications: Kev kho mob ntawm 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.Kev tso tawm ntawm cov tshuaj qhib lub sijhawm tshiab hauv kev kho tus txha caj qaum atrophy.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 hauv tus neeg mob los ntawm kev txhaj tshuaj intravenous los tsim cov protein ntau SMN1, yog li txhim kho kev ua haujlwm ntawm cov hlwb cuam tshuam xws li lub cev muaj zog neurons.Hauv qhov sib piv, SMA tshuaj Spinraza thiab Evrysdi yuav tsum tau siv sijhawm ntev dua.Spinraza yog muab los ntawm kev txhaj tshuaj txha caj qaum txhua plaub lub hlis, thiab Evrysdi yog tshuaj noj txhua hnub.

(8) Kev txiav txim siab

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

Sijhawm rau kev 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.

Cov lus ceeb toom: Delytact yog plaub tus kab mob oncolytic gene kho cov khoom tau pom zoo thoob ntiaj teb, thiab thawj tus kab mob oncolytic khoom tau 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 thib ob tiam 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 noj ntshav thaum tswj kev nyab xeeb siab.Delytact yog thawj thib peb-tiam oncolytic HSV-1 tam sim no tab tom soj ntsuam kev soj ntsuam.Kev pom zoo ntawm Delytact hauv Nyij Pooj feem ntau yog ua raws li ib leeg-caj npab theem 2 kev sim tshuaj.Hauv cov neeg mob uas muaj glioblastoma rov tshwm sim, Delytact tau ua tiav thawj qhov kawg ntawm kev muaj sia nyob ib xyoos, thiab cov txiaj ntsig tau pom tias Delytact tau pom tias muaj txiaj ntsig zoo dua piv nrog G207.Muaj zog replicate quab yuam thiab ntau dua kev ua haujlwm antitumor.Qhov no tau zoo nyob rau hauv cov qauv qog nqaij hlav ntawm lub mis, prostate, schwannomas, nasopharyngeal, hepatocellular, colorectal, malignant peripheral paj sheath hlav, thiab thyroid cancer.

(9) Cov

COMPANY: Tsim los ntawm PTC Therapeutics, Inc. (NASDAQ: PTCT).

Sijhawm rau kev ua lag luam: Pom zoo los ntawm European Union rau kev lag luam thaum Lub Xya Hli 2022.

Cov Lus Qhia: Rau cov tshuaj tsw qab L-amino acid decarboxylase (AADC) tsis txaus, nws raug pom zoo rau kev kho mob ntawm cov neeg mob hnub nyoog 18 hli thiab laus dua.

Lus Cim: Upstaza™ (eladocagene exuparvovec) yog ib qho kev kho hauv vivo gene nrog adeno-associated virus type 2 (AAV2) ua tus nqa khoom.Cov neeg mob tau mob vim kev hloov pauv hauv cov noob encoding AADC enzyme.AAV2 nqa cov noob noj qab haus huv encoding AADC enzyme.Daim ntawv ntawm cov nyiaj them rau cov noob tau txais kev kho mob.Hauv txoj kev xav, ib qho kev tswj hwm tau zoo rau lub sijhawm ntev.Nws yog thawj qhov kev lag luam gene therapy uas 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.

(10) Roctavian

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

Lub sijhawm ua lag luam: Pom zoo rau kev lag luam los ntawm European Union thaum Lub Yim Hli 2022;kev tso cai ua lag luam los ntawm UK Tshuaj thiab Cov Khoom Siv Khomob Kev Noj Qab Haus Huv (MHRA) thaum Lub Kaum Ib Hlis 2022.

Cov lus qhia: Rau kev kho mob ntawm cov neeg laus uas muaj hemophilia A hnyav uas tsis muaj keeb kwm ntawm FVIII yam inhibition thiab tsis zoo rau AAV5 cov tshuaj tiv thaiv.

Lus Cim: Roctavian (valoctocogene roxaparvovec) siv AAV5 ua vector thiab siv tib neeg lub siab tshwj xeeb txhawb HLP los tsav cov kev qhia ntawm tib neeg coagulation factor VIII (FVIII) nrog B domain deleted.Lub European Commission qhov kev txiav txim siab pom zoo rau kev lag luam ntawm valoctocogene roxaparvovec yog raws li cov ntaub ntawv tag nrho ntawm cov tshuaj kho mob txoj haujlwm.Ntawm lawv, cov txiaj ntsig ntawm theem III kev soj ntsuam kuaj GENER8-1 tau pom tias piv nrog cov ntaub ntawv ntawm lub xyoo ua ntej tso npe, tom qab ib qho kev txhaj tshuaj ntawm valoctocogene roxaparvovec, Cov kev kawm txhua xyoo los ntshav tus nqi (ABR) txo qis, qhov zaus ntawm kev siv cov recombinant coagulation factor VIII ntawm lub cev yog qhov tseem ceeb hauv kev ua haujlwm ntawm F8.Tom qab 4 lub lis piam ntawm kev kho mob, qhov kev kawm txhua xyoo F8 kev siv tus nqi thiab ABR xav tau kev kho mob raug txo los ntawm 99% thiab 84%, feem, thiab qhov sib txawv yog qhov tseem ceeb (p<0.001).Cov ntaub ntawv kev nyab xeeb tau zoo, thiab tsis muaj kev cuam tshuam txog F8 yam inhibition, malignancy lossis thrombosis phiv, thiab tsis muaj kev kho mob ntsig txog cov xwm txheej tsis zoo (SAEs) tau tshaj tawm.

(11) Hemgenix

Lub tuam txhab: Tsim los ntawm UniQure Corporation.

Sijhawm mus ua lag luam: Pom zoo los ntawm FDA rau kev lag luam thaum lub Kaum Ib Hlis 2022.

Cov Lus Qhia: Rau kev kho mob ntawm cov neeg laus uas muaj hemophilia B.

Lus Cim: Hemgenix yog kev kho noob raws li AAV5 vector.Cov tshuaj yog nruab nrog lub coagulation factor IX (FIX) gene variant FIX-Padua, uas yog muab tso rau hauv cov hlab ntsha.Tom qab kev tswj hwm, cov noob tuaj yeem qhia txog FIX coagulation yam hauv lub siab thiab zais cia Tom qab nkag mus rau hauv cov ntshav kom ua tiav cov haujlwm coagulation, kom ua tiav lub hom phiaj ntawm kev kho mob, theoretically, ib qho kev tswj hwm tau siv sijhawm ntev.

(12) Adstiladrin

Lub tuam txhab: Tsim los ntawm Ferring Pharmaceuticals.

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

Cov Lus Qhia: Rau kev kho mob ntawm cov qog nqaij hlav qog nqaij hlav tsis zoo (NMIBC) tsis teb rau Bacillus Calmette-Guerin (BCG).

Cov lus ceeb toom: Adstiladrin yog ib txoj kev kho noob raws li cov kab mob uas tsis yog replicating adenoviral vector, uas muaj peev xwm overexpress interferon alfa-2b protein nyob rau hauv lub hom phiaj hlwb, thiab yog muab los ntawm ib tug urinary catheter rau hauv lub zais zis (tso tshuaj ib zaug txhua txhua peb lub hlis), tus kab mob vector tuaj yeem kis tau mus rau hauv cov hlwb ntawm lub zais zis thiab cov protein ntau dhau los ntawm lub zais zis. tic nyhuv.Txoj kev kho cov noob tshiab no yog li hloov tus neeg mob lub zais zis phab ntsa rau hauv ib qho me me "lub Hoobkas" uas tsim cov interferon, yog li txhim kho tus neeg mob lub peev xwm tiv thaiv qog noj ntshav.

Kev nyab xeeb thiab kev ua tau zoo ntawm Adstiladrin tau raug soj ntsuam hauv ntau qhov chaw kho mob nrog rau 157 tus neeg mob uas muaj kev pheej hmoo siab BCG-tsis teb NMIBC.Cov neeg mob tau txais Adstiladrin txhua peb lub hlis mus txog rau 12 lub hlis, lossis txog thaum tsis muaj tshuaj lom rau kev kho lossis rov ua dua ntawm qib siab NMIBC.Zuag qhia tag nrho, 51 feem pua ​​​​ntawm cov neeg mob cuv npe kho nrog Adstiladrin tau ua tiav cov lus teb (qhov ploj ntawm tag nrho cov tsos mob ntawm tus mob qog noj ntshav pom ntawm cystoscopy, cov ntaub so ntswg biopsy, thiab zis).

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: Hauv xyoo 1998 thiab 1999, nws tau pom zoo rau kev lag luam los ntawm FDA thiab EU EMA.

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

Lus Cim: Vitravene yog ib qho tshuaj antisense oligonucleotide, uas yog thawj oligonucleotide tshuaj pom zoo rau kev lag luam hauv ntiaj teb.Nyob rau theem pib ntawm kev teev npe, kev lag luam thov rau cov tshuaj tiv thaiv CMV yog qhov ceev heev;Tom qab ntawd, vim yog kev txhim kho ntawm kev siv tshuaj tiv thaiv kab mob siab heev, cov neeg mob CMV poob qis heev.Vim tias kev lag luam qeeb qeeb, cov tshuaj tau tsim tawm hauv xyoo 2002 thiab 2006 Kev rho tawm hauv EU lub tebchaws thiab Tebchaws Meskas.

(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 Qhia: Macugen yog cov tshuaj pegylated hloov kho oligonucleotide, uas tuaj yeem tsom thiab khi vascular endothelial kev loj hlob zoo (VEGF165 subtype), thiab txoj kev tswj hwm yog txhaj tshuaj intravitreal.

(3) Defitelio

Lub tuam txhab: Tsim los ntawm Jazz Pharmaceuticals.

Lub sijhawm ua lag luam: Nws tau pom zoo rau kev lag luam los ntawm European Union hauv 2013 thiab pom zoo los ntawm FDA rau kev lag luam hauv lub Peb Hlis 2016.

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

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

(4) Cov

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

Sijhawm rau kev ua lag luam: Xyoo 2013, nws tau pom zoo rau kev lag luam hauv Tebchaws Meskas raws li cov tshuaj menyuam ntsuag.

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

Lus Cim: Kynamro yog ib qho tshuaj tiv thaiv oligonucleotide, uas yog 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 rau kev lag luam 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.Los ntawm kev khi rau qhov chaw cleavage ntawm SMN2 exon 7, Spinraza tuaj yeem hloov RNA cleavage ntawm SMN2 noob, yog li ua kom cov khoom ua haujlwm ntawm SMN protein ntau.Thaum Lub Yim Hli 2016, BIOGEN tau siv nws txoj kev xaiv kom tau txais txoj cai thoob ntiaj teb rau Spinraza.Spinraza tsuas yog pib nws thawj qhov kev sim tshuaj rau tib neeg hauv 2011. Tsuas yog 5 xyoo, nws tau pom zoo los ntawm FDA rau kev lag luam hauv xyoo 2016, uas 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 rau Spinraza hauv Suav teb yog tsawg dua 6 lub hlis, thiab nws yog 2 xyoo thiab 2 lub hlis txij li Spinraza tau pom zoo thawj zaug hauv Tebchaws Meskas.Qhov ceev ntawm kev teev npe nyob rau hauv Suav teb yog twb ceev heev.Qhov no kuj yog vim qhov tseeb tias Lub Tsev Haujlwm Saib Xyuas Tshuaj Tshaj Tawm tau tshaj tawm "Daim Ntawv Ceeb Toom Txog Kev Tshaj Tawm Cov Ntawv Tshaj Tawm ntawm Thawj Pawg Tshuaj Kho Mob Tshiab Tseem Ceeb Yuav Tsum Tau Ua Hauv Kev Kho Mob" thaum Lub Kaum Ib Hlis 1, 2018, thiab tau suav nrog thawj pawg ntawm 40 cov tshuaj tshiab txawv teb chaws rau kev tshuaj xyuas nrawm, cov uas Spinraza tau nyob rau hauv.

(6) Exondys 51

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

Lub sijhawm ua lag luam: Lub Cuaj Hli 2016, nws tau pom zoo rau kev lag luam los ntawm FDA.

Cov Lus Qhia: Rau kev kho mob ntawm Duchenne leeg nqaij dystrophy (DMD) nrog exon 51 hla kev hloov pauv hauv cov noob DMD.

Lus Cim: Exondys 51 yog ib qho tshuaj antisense oligonucleotide, antisense oligonucleotide tuaj yeem khi rau txoj hauj lwm ntawm exon 51 ntawm pre-mRNA ntawm DMD gene, uas ua rau tsim cov mRNA mRNA, ib feem ntawm exon 51 yog thaiv Excision, yog li ib feem ntawm kev nyeem ntawv luv luv ntawm cov neeg mob, ua rau muaj kev cuam tshuam ntawm mRNA. er 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.

Lub sijhawm ua lag luam: Nws tau pom zoo rau kev lag luam los ntawm European Union 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 thawj cov tshuaj pom zoo hauv ntiaj teb rau kev kho mob hATTR.Nws yog siv los ntawm kev txhaj tshuaj subcutaneous.Cov tshuaj txo cov kev tsim tawm ntawm ATTR protein los ntawm kev tsom mus rau mRNA ntawm transthyretin (ATTR), thiab muaj txiaj ntsig zoo-kev pheej hmoo ntawm kev kho mob ntawm ATTR, thiab tus neeg mob neuropathy thiab lub neej zoo tau zoo dua qub, thiab nws yog sib xws nrog TTR kev hloov pauv hom, tsis muaj kab mob theem lossis muaj cov cardiomyopathy cuam tshuam.

(8) Cov

Lub tuam txhab: Koom tes tsim los ntawm Alnylam Corporation thiab Sanofi Corporation.

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 ceeb toom: Onpattro yog ib qho tshuaj siRNA tsom rau transthyretin mRNA, uas txo qhov tsim ntawm ATTR protein nyob rau hauv daim siab thiab txo cov 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 alleviating cov tsos mob.

(9) Givlaari

Lub tuam txhab: Tsim los ntawm Alnylam Corporation.

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

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

Cov lus ceeb toom: Givlaari yog cov tshuaj siRNA, uas yog thib ob siRNA tshuaj pom zoo rau kev lag luam tom qab Onpattro.Txoj kev ntawm kev tswj hwm yog txhaj tshuaj subcutaneous.Cov tshuaj tsom mus rau mRNA ntawm ALAS1 cov protein, thiab kev kho mob txhua hli nrog Givlaari tuaj yeem txo qis ntawm ALAS1 hauv daim siab mus tas li, yog li txo qis cov qib neurotoxic ALA thiab PBG mus rau qhov qub, yog li txo cov tsos mob ntawm tus neeg mob.Cov ntaub ntawv qhia tau hais tias cov neeg mob kho nrog Givlaari muaj 74% txo qis ntawm cov qaug dab peg piv rau pawg placebo.

(10) Vyondys53

COMPANY: Tsim los ntawm Sarepta Therapeutics.

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

Indications: Rau kev kho mob ntawm DMD cov neeg mob nrog dystrophin noob exon 53 splicing kev hloov.

Lus Cim: Vyondys 53 yog ib qho tshuaj tiv thaiv oligonucleotide, uas yog lub hom phiaj ntawm kev sib txuas ntawm dystrophin pre-mRNA.Exon 53 yog ib feem truncated, piv txwv li tsis muaj nyob rau ntawm mRNA paub tab, thiab yog tsim los tsim ib tug truncated tab sis tseem ua hauj lwm dystrophin, 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 rau kev ua lag luam: Nws tau pom zoo rau kev lag luam los ntawm European Medicines Agency (EMA) thaum lub Tsib Hlis 2019.

Indications: Raws li kev kho mob ntxiv nrog rau kev tswj 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 lag luam hauv ntiaj teb rau kev kho mob ntawm FCS.

(12) Cov

Lub tuam txhab: Tsim los ntawm Novartis.

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

Cov Lus Qhia: Rau kev kho mob ntawm cov neeg laus nrog thawj hypercholesterolemia (heterozygous tsev neeg thiab tsis yog tsev neeg) lossis sib xyaw dyslipidemia.

Lus Cim: Leqvio yog ib qho tshuaj siRNA tsom rau PCSK9 mRNA.Nws yog lub ntiaj teb thawj txoj kev kho siRNA rau txo cov roj (LDL-C).Nws yog siv los ntawm kev txhaj tshuaj subcutaneous.Cov tshuaj txo cov qib ntawm PCSK9 protein los ntawm RNA cuam tshuam, yog li txo qis LDL-C.Cov ntaub ntawv kho mob qhia tau hais tias rau cov neeg mob uas tsis tuaj yeem txo LDL-C qib mus rau theem hom phiaj tom qab kev kho mob nrog qhov siab tshaj plaws ntawm cov tshuaj statins, Leqvio tuaj yeem txo LDL-C li ntawm 50%.

(13) Cov

Lub tuam txhab: Tsim los ntawm Alnylam Pharmaceuticals.

Sijhawm rau kev ua lag luam: Pom zoo los ntawm European Union rau kev lag luam thaum lub Kaum Ib Hlis 2020.

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

Lus Cim: Oxlumo yog ib qho tshuaj siRNA tsom rau hydroxyacid oxidase 1 (HAO1) ​​mRNA, thiab txoj kev tswj hwm yog txhaj tshuaj subcutaneous.Cov tshuaj tau tsim los siv Alnylam qhov tseeb txhim kho stabilization chemistry, ESC-GalNAc conjugation technology, uas ua rau subcutaneously tswj siRNA nrog ntau dua persistence thiab potency.Cov tshuaj degrades los yog inhibits hydroxyacid oxidase 1 (HAO1) ​​mRNA, txo cov qib ntawm glycolate oxidase nyob rau hauv daim siab, thiab ces noj cov substrate yuav tsum tau rau zus tau tej cov oxalate, txo oxalate ntau lawm los tswj kev loj hlob ntawm tus kab mob nyob rau hauv cov neeg mob thiab txhim kho cov tsos mob ntawm tus kab 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 rau kev lag luam thaum Lub Yim Hli 2020.

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

Lus Cim: Viltepso yog cov tshuaj antisense oligonucleotide uas tuaj yeem khi rau txoj hauj lwm ntawm exon 53 ntawm pre-mRNA ntawm DMD gene, ua rau ib feem ntawm exon 53 yuav tsum tau excised tom qab tsim mRNA mRNA paub tab, yog li ib feem ntawm kev kho cov mRNA nyeem ntawv lub thawv pab cov neeg mob ua kom cov txheej txheem luv luv ntawm cov protein ntau dua, yog li kev kho ib feem ntawm mRNA nyeem cov ncej cov tsos mob ntawm cov neeg mob.

(15) Amondys 45

Lub tuam txhab: Tsim los ntawm Sarepta Therapeutics.

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

Cov Lus Qhia: Rau kev kho mob ntawm Duchenne leeg nqaij dystrophy (DMD) nrog exon 45 hla kev hloov pauv hauv cov noob DMD.

Lus Cim: Amondys 45 yog ib qho tshuaj antisense oligonucleotide, antisense oligonucleotide tuaj yeem khi rau txoj hauj lwm ntawm exon 45 ntawm pre-mRNA ntawm DMD gene, uas ua rau ib feem ntawm exon 45 raug thaiv tom qab tsim mphim mRNA Excision, yog li kev nyeem ntawv ib nrab ntawm cov kev ua haujlwm ntawm mRNA synthesis ntawm cov neeg mob. luv dua li cov protein ib txwm, yog li txhim kho tus neeg mob cov tsos mob.

(16) Kev hlub

Lub tuam txhab: Tsim los ntawm Alnylam Pharmaceuticals.

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

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

Lus Cim: Amvuttra (Vutrisiran) yog ib qho tshuaj siRNA tsom rau transthyretin (ATTR) mRNA, muab los ntawm kev txhaj tshuaj subcutaneous.Vutrisiran yog ua raws li Alnylam's Enhanced Stability Chemistry (ESC)-GalNAc conjugate xa platform tsim nrog nce zog thiab metabolic stability.Qhov kev pom zoo ntawm txoj kev kho yog raws li 9-hli cov ntaub ntawv ntawm nws qhov kev tshawb fawb Phase III (HELIOS-A), thiab tag nrho cov txiaj ntsig tau pom tias qhov kev kho no tau txhim kho cov tsos mob ntawm hATTR-PN, thiab ntau dua 50% ntawm cov neeg mob tus mob tau thim rov qab los yog nres los ntawm kev mob hnyav dua.

4. Lwm yam tshuaj kho noob caj noob ces

(1) Rexin-G

Lub tuam txhab: Tsim los ntawm Epeius Biotech.

Lub sijhawm ua lag luam: Hauv xyoo 2005, nws tau pom zoo rau kev lag luam los ntawm Philippine Food and Drug Administration (BFAD).

Indications: Rau kev kho mob cancer siab heev resistant rau chemotherapy.

Lus Cim: Rexin-G yog ib qho tshuaj gene-loaded nanoparticle txhaj.Nws qhia txog cyclin G1 mutant gene rau hauv lub hom phiaj hlwb los ntawm ib tug retroviral vector los tua cov qog nqaij hlav.Txoj kev tswj hwm yog intravenous infusion.Raws li cov tshuaj qog nqaij hlav uas nquag nrhiav thiab rhuav tshem cov qog nqaij hlav cancer metastatic, nws muaj qee yam kev kho mob rau cov neeg mob uas ua tsis tau lwm yam 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: Nws tau pom zoo rau kev sau npe hauv tebchaws Russia thaum Lub Kaum Ob Hlis 7, 2011, thiab tom qab ntawd pib hauv Ukraine hauv 2013.

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

Lus Cim: Neovasculgen yog kev kho noob raws li DNA plasmids.Lub vascular endothelial kev loj hlob zoo tshaj plaws (VEGF) 165 noob yog tsim los ntawm plasmid qaum qaum thiab infused rau cov neeg mob.

(3) Collategene

Lub tuam txhab: Koom tes tsim los ntawm Osaka University thiab venture peev tuam txhab.

Sijhawm mus ua lag luam: Pom zoo los ntawm Ministry of Health, Labor thiab Welfare ntawm Nyiv thaum Lub Yim Hli 2019.

Cov Lus Qhia: Kev kho mob ntawm qhov tseem ceeb hauv qis kawg ischemia.

Lus Cim: Collategene yog plasmid-based gene therapy, thawj cov tshuaj kho noob hauv tsev uas tsim los ntawm AnGes, lub tuam txhab kho noob hauv Nyij Pooj.Lub ntsiab tseem ceeb ntawm cov tshuaj no yog liab qab plasmid uas muaj tib neeg hepatocyte growth factor (HGF) gene sequence.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.Cov kev sim tshuaj ntsuam xyuas tau lees paub nws cov txiaj ntsig ntawm kev txhim kho rwj.

Foregene tuaj yeem pab txoj kev kho noob li cas?

Peb pab txuag lub sijhawm tshuaj ntsuam hauv kev tshuaj ntsuam loj, nyob rau theem pib ntawm kev txhim kho tshuaj siRNA.

Xav paub ntxiv mus saib:

https://www.foreivd.com/cell-direct-rt-qpcr-kit-direct-rt-qpcr-series/