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Munguva pfupi yadarika, matatu emishonga ekurapa majini akabvumidzwa kushambadzira, anoti: (1) Musi waChikunguru 21, 2022, PTC Therapeutics, Inc. (NASDAQ: PTCT) yakazivisa kuti AAV gene therapy Upstaza™ yakabvumidzwa neEuropean Commission Ndiyo yakatanga kutengeswa jena therapy yakanangidzirwa muuropi (ona zvinyorwa zvakapfuura: Chimwe Chikuru muGene Therapy | Mushonga wekutanga weAAV wepasi rose wakapinzwa muuropi unotenderwa kushambadzira).(2) Musi waNyamavhuvhu 17, 2022, iyo US Food and Drug Administration (FDA) yakabvumidza Bluebird Bio's gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) kurapa beta thalassemia.Kubvumirwa kwekurapa muUnited States pasina mubvunzo "rubatsiro muchando" yeBluebird Bio, iyo iri mumatambudziko emari.(3) Musi wa24 Nyamavhuvhu 2022, BioMarin Pharmaceutical (BioMarin) yakazivisa kuti European Commission yakabvumidza kushambadzira kweROCTAVIAN ™ (valoctocogene roxaparvovec), gene therapy yehemophilia A, yekurapa varwere vasina nhoroondo yeFVIII factor inhibitors. neAAV5 masoja ekudzivirira chirwere akanyanya varwere vane hemophilia A yakanyanya (ona chinyorwa chapfuura: Heavy! BioMarin's hemophilia A Gene therapy inobvumidzwa kutengeswa).Parizvino, 41 gene therapy mishonga yakabvumidzwa kushambadzira pasi rese.

Gene ndiyo yakakosha genetic unit inodzora hunhu.Kunze kwemajini emamwe mavhairasi, ayo anoumbwa neRNA, majini ezvipenyu zvakawanda anoumbwa neDNA.Zvirwere zvakawanda zvechipenyu zvinokonzerwa nekudyidzana pakati pemajini uye zvakatipoteredza, uye zvirwere zvakawanda zvinogona kurapwa kana kuderedzwa mukukosha kuburikidza nejene therapy.Gene therapy inoonekwa seshanduko mumunda wemishonga uye pharmacy.Iyo yakafara gene therapy mishonga inosanganisira zvinodhaka zvinoenderana neDNA-modified DNA mishonga (senge viral vector-based in vivo gene therapy drugs, in vitro gene therapy drugs, vasina kupfeka plasmid drugs, etc.) nemishonga yeRNA (senge antisense oligonucleotide zvinodhaka, siRNA mishonga, uye mRNA gene therapy, nezvimwewo);zvakanyatsotsanangurwa Gene therapy mishonga inonyanya kusanganisira plasmid DNA mishonga, gene therapy mishonga yakavakirwa pamavhairasi mavhairasi, gene therapy mishonga yakavakirwa pamabhakitiriya mavheji, masisitimu ekugadzirisa majini uye sero therapy mishonga inogadziriswa in vitro.Mushure memakore ekukura kwakashata, gene therapy mishonga yakawana zvinokurudzira zvekiriniki.(tisingasanganisire majekiseni eDNA uye mRNA vaccines), 41 gene therapy mishonga yakabvumidzwa kutengeswa munyika.Nekutangwa kwezvigadzirwa uye nekukasira kusimukira kweiyo gene therapy tekinoroji, gene therapy yave kuda kuunza nguva yekukurumidza kukura.

pasi rose1

Kukamurwa kwejene kurapwa (Mufananidzo sosi: Biological Jingwei)

Ichi chinyorwa chinonyora 41 gene marapirwo akatenderwa kushambadzira (tisingasanganisire DNA vaccine uye mRNA vaccines).

1. In vitro gene therapy

(1) Strimvelis

Kambani: Yakagadzirwa neGlaxoSmithKline (GSK).

Nguva yemusika: Yakatenderwa neEuropean Union muna Chivabvu 2016.

Zvinoratidza: Pakurapa kwakanyanya kusanganisa immunodeficiency (SCID).

Tsanangudzo: Iyo nzira yekurapa iyi ndeyekutanga kuwana murwere ega masero ehematopoietic stem masero, kuwedzera uye kugadzira iwo mu vitro, uyezve kushandisa retrovirus kuunza kopi inoshanda inoshanda ADA (adenosine deaminase) gene mumasero avo ehematopoietic stem, uye pakupedzisira fambisa iyo yakagadziriswa hematopoietic stem masero.Hematopoietic stem masero anodzoserwa zvakare mumuviri.Mibairo yemakiriniki yakaratidza kuti chiyero chemakore matatu chekurarama kwevarwere veADA-SCID vakarapwa neStrimvelis chaive zana%.

(2) Zalmoxis

Kambani: Yakagadzirwa neMolMed, Italy.

Nguva yemusika: Yakawana EU inomisikidzwa yekushambadzira mvumo muna 2016.

Zvinoratidza: Inoshandiswa kune adjuvant kurapa kwea immune system yevarwere mushure meiyo hematopoietic stem cell transplantation.

Mashoko: Zalmoxis is allogeneic T cell suicide gene immunotherapy yakagadziridzwa neretroviral vector.Iyo 1NGFR uye HSV-TK Mut2 kuzviuraya majini inobvumira vanhu kushandisa ganciclovir chero nguva kuuraya masero eT anokonzera mhinduro dzakaipa dzekudzivirira, kudzivirira kuwedzera kuderera kweGVHD kunogona kuitika, uye kudzorera kushanda kwemuviri kune varwere vane haploidentical HSCT mushure mekuvhiyiwa Escort.

(3) Invossa-K

Kambani: Yakagadzirwa neTissueGene (KolonTissueGene) kambani.

Nguva yemusika: Yakatenderwa kunyorwa muSouth Korea muna Chikunguru 2017.

Zviratidzo: Kurapa kwekuparara kwemabvi arthritis.

Mashoko: Invossa-K inonzi allogeneic cell gene therapy inosanganisira chondrocytes yevanhu.Masero eAllogeneic ane genetically modified in vitro, uye masero akashandurwa anogona kuratidza uye kuvhara kushandura kushandura kukura chinhu β1 (TGF-β1) mushure mejojo ​​ye intra-articular.β1), nokudaro achivandudza zviratidzo zveosteoarthritis.Migumisiro yemakiriniki inoratidza kuti Invossa-K inogona kuvandudza zvakanyanya arthritis yemabvi.Rezinesi rakabviswa neSouth Korea's drug regulator muna 2019 nekuti mugadziri wacho akanyora zvisizvo izvo zvinoshandiswa.

(4) Zynteglo

Kambani: Yakagadzirwa neAmerican bluebird bio (bluebird bio) kambani.

Nguva yemusika: Yakatenderwa neEuropean Union muna 2019, uye yakatenderwa neFDA muna Nyamavhuvhu 2022.

Zvinoratidza: Zvekurapa kwekuisira ropa-inotsamira β-thalassemia.

Tsanangudzo: Zynteglo inonzi lentiviral in vitro gene therapy, iyo inoshandisa lentiviral vector kuti ibudise kopi inoshanda yegeneti ye β-globin gene (βA-T87Q-globin gene) mumasero ehematopoietic stem akabviswa kubva kuvarwere., uye wozopinza aya masero ane genetically modified autologous hematopoietic stem cells kudzokera kumurwere.Kana murwere angove neakajairika βA-T87Q-globin gene, anogona kugadzira yakajairika HbAT87Q protein, inogona kunyatso kudzikisa kana kubvisa kudiwa kwekuwedzerwa ropa.Inguva imwe chete yekurapa yakagadzirirwa kutsiva kuwedzerwa ropa kwehupenyu hwese uye mishonga yeupenyu hwese kune varwere vane makore gumi nemaviri zvichikwira.

(5) Skysona

Kambani: Yakagadzirwa neAmerican bluebird bio (bluebird bio) kambani.

Nguva yemusika: Yakatenderwa neEU kushambadzira muna Chikunguru 2021.

Zviratidzo: Pakurapa kwekutanga cerebral adrenoleukodystrophy (CALD).

Remarks: Skysona gene therapy ndiyo yega-nguva imwe chete gene therapy inotenderwa kurapwa kwekutanga cerebral adrenoleukodystrophy (CALD).Skysona (elivaldogene autotemcel, Lenti-D) is hematopoietic stem cell lentiviral in vitro gene therapy Lenti-D.Maitiro ekurapa ndeaya anotevera: autologous hematopoietic stem masero anotorwa kubva kumurwere, akagadziridzwa in vitro ne lentivirus inotakura yemunhu ABCD1 gene, yozodzoserwa kumashure kumurwere.Nekurapwa kwevarwere vari pasi pemakore gumi nemasere neABCD1 gene mutation uye CALD.

(6) Kymriah

Kambani: Yakagadzirwa naNovartis.

Nguva yemusika: Yakatenderwa neFDA muna Nyamavhuvhu 2017.

Zviratidzo: Kurapa kwe precursor B-cell acute lymphoblastic leukemia (ALL) uye yakadzoka zvakare uye inoramba DLBCL.

Mashoko: Kymriah mushonga welentiviral in vitro gene therapy, mushonga wekutanga kubvumidzwa pasi rose weCAR-T, wakananga kuCD19 uye uchishandisa co-stimulatory factor 4-1BB.Mitengo i$475,000 muUS uye $313,000 muJapan.

(7) Yescarta

Kambani: Yakagadzirwa naKite Pharma, inotsigira yeGiriyedhi.

Nguva yemusika: Yakatenderwa neFDA muna Gumiguru 2017.

Zviratidzo: Pakurapa kwekudzokazve kana kuti refractory hombe B-cell lymphoma.

Remarks: Yescarta iretroviral in vitro gene therapy.Ndiyo yechipiri CAR-T therapy inobvumidzwa munyika.Inonangana neCD19 uye inoshandisa costimulatory factor yeCD28.Mutengo muUS $373,000.

(8) Tecartus

Kambani: Yakagadzirwa neGiriyedhi (GILD).

Nguva yemusika: Yakatenderwa neFDA muna Chikunguru 2020.

Zviratidzo: Zvekudzokazve kana refractory mantle cell lymphoma.

Mashoko: Tecartus ndeye autologous CAR-T cell therapy yakanangana neCD19, uye ndiyo yechitatu CAR-T kurapwa yakabvumidzwa kushambadzira munyika.

(9) Breyanzi

Kambani: Yakagadzirwa neBristol-Myers Squibb (BMS).

Nguva yemusika: Yakatenderwa neFDA muna Kukadzi 2021.

Zviratidzo: Yakadzoka zvakare kana kuti refractory (R / R) yakakura B-cell lymphoma (LBCL).

Remarks: Breyanzi in vitro gene therapy yakavakirwa pa lentivirus, uye yechina CAR-T therapy inotenderwa kushambadzira munyika, yakanangana neCD19.Kubvumidzwa kwaBreyanzi chiitiko chakakosha cheBristol-Myers squibb mumunda we cellular immunotherapy, iyo Bristol-Myers yakawana payakawana Celgene yemabhiriyoni makumi manomwe nemana emadhora muna 2019.

(10) Abecma

Kambani: Co-yakagadziridzwa naBristol-Myers Squibb (BMS) uye bluebird bio.

Nguva yemusika: Yakatenderwa neFDA munaKurume 2021.

Zviratidzo: Yakadzokazve kana kuti refractory multiple myeloma.

Mashoko: Abecma ndeye lentivirus-yakavakirwa in vitro gene therapy, yekutanga pasi rose CAR-T cell therapy yakanangana neBCMA, uye yechishanu CAR-T kurapwa yakatenderwa neFDA.Mushonga wemushonga ndeyekutaura chimeric BCMA receptor pane murwere autologous T masero kuburikidza lentivirus-mediated genetic modification in vitro.Pamberi pekuiswa kwesero gene mushonga, murwere akagamuchira maviri emakemikari e cyclophosphamide uye fludarabine kuti atange kurapwa.Kurapa kubvisa masero eT asina kugadziriswa kubva kumurwere, uyezve kupinza masero eT akagadziridzwa zvakare mumuviri wemurwere kutsvaga nekuuraya BCMA-inoratidza cancer maseru.

(11) Libmeldy

Kambani: Yakagadzirwa neOrchard Therapeutics.

Nguva yemusika: Yakatenderwa neEuropean Union kuti inyore muna Zvita 2020.

Zvinoratidza: Pakurapa metachromatic leukodystrophy (MLD).

Remarks: Libmeldy mushonga wegene wakavakirwa palentiviral in vitro gene gadziriso ye autologous CD34 + masero.Data yekiriniki inoratidza kuti imwe intravenous infusion yeLibmeldy inoshanda mukugadzirisa nzira yekutanga-yekutanga MLD uye yakanyanya mota uye kukanganiswa kwekuziva muvarwere vasina kurapwa vezera rimwe chete.

(12) Benoda

Kambani: Yakagadzirwa naWuXi Junuo.

Nguva yemusika: Zvakatenderwa zviri pamutemo neNMPA munaGunyana 2021.

Zviratidzo: Kurapa kwekudzokazve kana kuti refractory yakakura B-cell lymphoma (r / r LBCL) muvarwere vakuru mushure mechipiri-mutsara kana kupfuura systemic therapy.

Remarks: Benoda is anti-CD19 CAR-T gene therapy, uye zvakare iri musimboti chigadzirwa cheWuXi Junuo.Ndiyo yechipiri CAR-T chigadzirwa chakatenderwa muChina, kunze kwekudzokororwa/refractory hombe B-cell lymphoma.Pamusoro pezvo, WuXi Junuo anorongawo kugadzira jekiseni reRuiki Orenza rekurapwa kwezvimwe zviratidzo zvakasiyana, zvinosanganisira follicular lymphoma (FL), mantle cell lymphoma (MCL), chronic lymphocytic leukemia (CLL), mutsara wechipiri diffuse hombe B-cell lymphoma. (DLBCL) uye acute lymphoblastic leukemia (ALL).

(13) CARVYKTI

Kambani: Legend Bio yekutanga yakabvumidzwa chigadzirwa.

Nguva yemusika: Yakatenderwa neFDA muna Kukadzi 2022.

Zviratidzo: Kurapa kwekudzokazve kana kuti refractory multiple myeloma (R / R MM).

Tsanangudzo: CARVYKTI (ciltacabtagene autoleucel, inonzi Cilta-cel) inonzi CAR-T cell immune gene therapy ine masoja maviri e-single-domain anonangana neB cell maturation antigen (BCMA).Iyo data inoratidza kuti CARVYKTI yakaratidza huwandu hwekupindura kusvika ku98% muvarwere vane myeloma yakadzokororwa kana kuti refractory vanga vagamuchira mana kana anopfuura ekurapa kwepamberi, kusanganisira proteasome inhibitors, immunomodulators, uye anti-CD38 monoclonal antibodies.

2. In vivo gene therapy inobva pahutachiona hwehutachiona

(1) Gendicine/kuzvarwa patsva

Kambani: Yakagadzirwa neShenzhen Saibainuo Kambani.

Nguva yemusika: Yakatenderwa kunyorwa muChina muna 2003.

Zviratidzo: Kurapa kwe squamous cell carcinoma yemusoro nemutsipa.

Mashoko: Recombinant human p53 adenovirus jekiseni Gendicine/Jinshengsheng mushonga weadenovirus vector gene therapy une kodzero dzakazvimirira dzepfuma yekambani yeShenzhen Saibainuo.Mushonga unoumbwa neakajairika bundu remunhu suppressor gene p53 uye artificially modified recombinant replication-deficient Human adenovirus type 5 inoumbwa neadenovirus yemunhu rudzi 5. Iyo yekutanga ndiyo huru chimiro chemushonga kuita anti-bundu mhedzisiro, uye yekupedzisira kunyanya. inoshanda semutakuri.Iyo adenovirus vector inotakura therapeutic gene p53 kupinda musero rinonangwa, uye rinoratidza bundu suppressor gene p53 musero rinotarisirwa.Chigadzirwa chacho chinogona kusimudza-kudzora akasiyana-siyana anopesana negomarara majini uye pasi-kudzora zviitiko zveakasiyana oncogenes, nekudaro kuwedzera anti-tumor mhedzisiro yemuviri uye kuzadzisa chinangwa chekuuraya mamota.

(2) Rigvir

Kambani: Yakagadzirwa nekambani yeLatvia Latima.

Nguva yemusika: Yakatenderwa muLatvia muna 2004.

Zviratidzo: Pakurapa melanoma.

Remarks: Rigvir mushonga wegene wakavakirwa pane gene-modified ECHO-7 enterovirus vector, iyo yakashandiswa muLatvia, Estonia, Poland, Armenia, Belarus nedzimwe nzvimbo, uye zvakare iri kunyoreswa neEMA yeEuropean Union..Mhosva dzekiriniki mumakore gumi apfuura dzakaratidza kuti hutachiona hweRigvir oncolytic hwakachengeteka uye hunoshanda, uye hunogona kuvandudza huwandu hwekupona kwevarwere vemelanoma ka4-6.Pamusoro pezvo, kurapwa kwacho kwakakodzerawo kune akasiyana mamwe magomarara, kusanganisira colorectal cancer, pancreatic cancer, dundira.kenza, kenza yeitsvo, kenza yeprostate, kenza yemapapu, kenza yechibereko, lymphosarcoma, nezvimwe.

(3) Oncorine/Ankerui

Kambani: Yakagadzirwa neShanghai Sunway Biotechnology Co., Ltd.

Nguva yemusika: Yakatenderwa kunyorwa muChina muna 2005.

Zviratidzo: Kurapa kwemusoro nemutsipa bundu, kenza yechiropa, kenza yepancreatic, kenza yechibereko uye mamwe magomarara.

Manzwiro: Oncorine ndeye oncolytic hutachiona gene therapy chigadzirwa uchishandisa adenovirus sevector.Iyo yakawanikwa oncolytic adenovirus inogona kudzokorora zvakananga mumamota anoshaikwa kana abnormal p53 gene, zvichikonzera tumor cell lysis, nekudaro kuuraya bundu maseru.pasina kukuvadza masero akajairika.Mhedzisiro yekiriniki inoratidza kuti Anke Rui ane chengetedzo yakanaka uye inoshanda kune akasiyana siyana emamota akaipa.

(4) Glybera

Kambani: Yakagadziriswa neUniQure.

Nguva yemusika: Yakatenderwa muEurope muna 2012.

Zviratidzo: Kurapa kwe lipoprotein lipase kushomeka (LPLD) neyakaomarara kana inodzokororwa episodhi yepancreatitis kunyangwe yakanyatso kurambidzwa kudya kwemafuta.

Tsanangudzo: Glybera (alipogene tiparvovec) mushonga wegene therapy wakavakirwa paAAV sevheji.Mushonga uyu unoshandisa AAV sevheji kuendesa jini rekurapa LPL mumasero etsandanyama, kuitira kuti masero anoenderana anogona kuburitsa imwe huwandu hwelipoprotein lipase, Inoita basa mukubvisa chirwere, uye kurapa uku kunoshanda kwenguva yakareba mushure meimwe. kutonga (mhedzisiro inogona kugara kwemakore mazhinji).Mushonga uyu wakabviswa muna 2017, uye zvikonzero zvekubviswa kwayo zvinogona kunge zvine chekuita nezvinhu zviviri: mitengo yakawandisa uye kushomeka kwemusika.Mari yeavhareji yekurapwa kumwe chete kwemushonga yakakwira kusvika miriyoni imwe chete yemadhora ekuAmerica, uye murwere mumwe chete ndiye akatenga nekuishandisa kusvika pari zvino.Kunyange zvazvo kambani yeinishuwarenzi yezvokurapa yakaidzorera 900,000 US madhora, zvakare mutoro mukuru kukambani yeinishuwarenzi.Mukuwedzera, chiratidzo chemushonga hachiwanzoitiki, nehuwandu hwechiitiko che 1 mu 1 mamiriyoni uye nehuwandu hwekusaziva.

(5) Imlygic

Kambani: Yakagadzirwa neAmgen.

Nguva yemusika: Muna 2015, yakatenderwa kunyorwa muUnited States neEuropean Union.

Zvinoratidza: Kurapwa kwemaronda e<em>melanoma asingagoni kubviswa zvachose nokuvhiyiwa.

Remarks: Imlygic is a genetically modified (kubvisa ICP34.5 uye ICP47 gene fragments, nekuisa munhu granulocyte-macrophage colony-stimulating factor GM-CSF gene muhutachiona) attenuated herpes simplex virus type 1 (HSV-1) oncolytic virus. , yekutanga FDA-yakatenderwa oncolytic virus gene therapy.Nzira yehutungamiri ndeye intralesional injection.Jekiseni rakananga mumaronda e-melanoma rinogona kukonzera kuputika kwemasero emudumbu uye kuburitsa maantigen anokonzerwa nebundu uye GM-CSF kukurudzira mhinduro dzekurwisa-tumor immune immune.

(6) Luxturna

Kambani: Yakagadziridzwa neSpark Therapeutics, inotsigira Roche.

Nguva yemusika: Yakatenderwa neFDA muna 2017, uyezve yakabvumidzwa kushambadzira muEurope muna 2018.

Zviratidzo: Kurapa kwevana nevakuru vane kurasikirwa kwekuona nekuda kwekushanduka kwekaviri kopi yeRPE65 gene asi nenhamba yakakwana yemasero eretinal anoshanda.

Matauriro: Luxturna ndeye AAV-yakavakirwa gene therapy iyo inotungamirwa ne subretinal jekiseni.The gene therapy inoshandisa AAV2 semutakuri kuunza kopi inoshanda yeyakajairika RPE65 gene mumasero eretinal emurwere, kuitira kuti masero anowirirana aratidze yakajairika RPE65 protein kubhadharira kuremara kwemurwere RPE65 protein, nekudaro kuvandudza chiono chemurwere.

(7) Zolgensma

Kambani: Yakagadziridzwa neAveXis, inotsigira yeNovartis.

Nguva yemusika: Yakatenderwa neFDA muna Chivabvu 2019.

Zviratidzo: Kurapa kwepinal muscular atrophy (Spinal Muscular Atrophy, SMA) varwere vari pasi pemakore maviri.

Mazwi: Zolgensma ijena rekurapa rakavakirwa paAAV vector.Mushonga uyu ndiwo wega-nguva imwe chete chirongwa chekurapa chespinal muscular atrophy chakabvumidzwa kushambadzira munyika.peji, kufambira mberi kukuru.Iyi gene therapy inoshandisa scAAV9 vector kuunza yakajairika SMN1 gene muvarwere kuburikidza ne intravenous infusion, ichigadzira yakajairika SMN1 protein, nekudaro inovandudza basa reakakanganisika maseru akadai semotor neurons.Kusiyana neizvi, mishonga yeSMA Spinraza neEvrysdi inoda kudzokororwa kudzokororwa kwenguva yakareba, neSpinraza inoshandiswa sejekiseni remusana mwedzi mina yega yega, uye Evrysdi, mushonga wezuva nezuva wemukanwa.

(8) Delytact

Kambani: Yakagadzirwa neDaiichi Sankyo Company Limited (TYO: 4568).

Nguva yemusika: Mvumo ine mamiriro kubva kuJapan Ministry of Health, Labor and Welfare (MHLW) muna Chikumi 2021.

Zvinoratidza: Zvekurapa malignant glioma.

Matauriro: Delytact ndiyo yechina oncolytic hutachiona gene therapy chigadzirwa chakabvumidzwa pasi rose uye yekutanga oncolytic hutachiona chigadzirwa chakabvumidzwa kurapwa kwemalignant glioma.Delytact is a genetically engineered herpes simplex virus type 1 (HSV-1) oncolytic virus yakagadzirwa naDr. Todo nevamwe vaaishanda navo.Delytact inounza imwezve yekudzima mutation muG207 genome yechipiri-chizvarwa HSV-1, ichiwedzera yakasarudzika kudzokororwa mumaseru egomarara uye kuiswa kweanopesana netumor immune mhinduro, uku ichichengetedza yakakwirira chengetedzo chimiro.Delytact ndiyo yekutanga chizvarwa chechitatu oncolytic HSV-1 parizvino mukuongororwa kwekiriniki.Mvumo yeDelytact muJapan yaive yakavakirwa pane imwe-ruoko Phase 2 yekiriniki yekuyedza.Mune varwere vane inodzokororwa glioblastoma, Delytact yakasangana nemagumo ekupedzisira ekupona kwegore rimwe, uye mhedzisiro yakaratidza kuti Delytact yakaita zvirinani pane G207.Kudzokorora kwakasimba uye yakakwirira antitumor chiitiko.Izvi zvinoshanda mumhando dzakasimba dzebundu dzinosanganisira zamu, prostate, schwannoma, nasopharyngeal, hepatocellular, colorectal, malignant peripheral nerve sheath tumors uye gomarara rethyroid.

(9) Upstaza

Kambani: Yakagadzirwa nePTC Therapeutics, Inc. (NASDAQ: PTCT).

Nguva yemusika: Yakatenderwa neEU muna Chikunguru 2022.

Chiratidzo: Kushomeka kweL-amino acid decarboxylase (AADC) inonhuhwirira, yakatenderwa kurapwa kwevarwere vane mwedzi gumi nemisere zvichikwira.

Remarks: Upstaza™ (eladocagene exuparvovec) iri in vivo gene therapy uchishandisa adeno-associated virus type 2 (AAV2) sevheta.Murwere ari kurwara nekuda kwekushanduka kwejini encoding iyo AADC enzyme.AAV2 inotakura gene ine hutano inokodha iyo AADC enzyme.Therapeutic effect inowanikwa nenzira ye genetic compensation.Mukutaura, imwe dhesi inoshanda kwenguva yakareba.Ndiyo yekutanga kutengeswa kwejene therapy yakananga muuropi.Mvumo yekushambadzira inoshanda kune ese makumi maviri nenomwe enhengo dzeEU, pamwe neIceland, Norway neLiechtenstein.

(9) Roctavian

Kambani: Yakagadzirwa neBioMarin Pharmaceutical (BioMarin).

Nguva yemusika: Yakatenderwa neEU muna Nyamavhuvhu 2022.

Zviratidzo: Kurapa kwevarwere vakuru vane hemophilia yakaoma A vasina nhoroondo yeFVIII factor inhibition uye AAV5 antibody negative.

Mashoko: Roctavian (valoctocogene roxaparvovec) inoshandisa AAV5 sevheta uye inoshandisa chiropa chemunhu chakananga HLP kufambisa kutaura kwemunhu coagulation factor sere (FVIII) neB domain yakabviswa.Sarudzo yeEuropean Commission yekubvumidza kutengeswa kwevaloctocogene roxaparvovec yakavakirwa pane yakazara data yechirongwa chekiriniki yekuvandudza mushonga.Pakati pavo, chikamu chechitatu chekliniki yekuedza GENEr8-1 yakaratidza kuti kana ichienzaniswa nedheta regore risati ranyoreswa, mushure mekuiswa kamwe chete kwevaloctocogene roxaparvovec, Zvidzidzo zvaive nehuwandu hwekubuda ropa kwegore negore (ABR), kushandiswa kusingawanzoitiki kwechimwe chinhu chechipiri VIII. (F8) mapuroteni ekugadzirira, kana kuwedzera kukuru kwekuita kweF8 muropa remuviri.Mushure memavhiki e4 ekurapwa, zvidzidzo zvegore negore zveF8 kushandiswa uye ABR inoda kurapwa yakaderedzwa ne99% uye 84%, maererano, mutsauko wakakosha (p <0.001).Nhoroondo yekuchengetedza yakanga yakanaka, pasina zvidzidzo zvinosangana neF8 factor inhibition, kuipa, kana thrombotic side effects, uye hapana zviitiko zvakakomba zvine chokuita nekurapa (SAEs) zvakataurwa.

3. Small nucleic acid mishonga

(1) Vitravene

Kambani: Yakabatanidzwa neIonis Pharma (yaimbova Isis Pharma) uye Novartis.

Nguva yemusika: Yakatenderwa neFDA neEU EMA muna 1998 na1999.

Zviratidzo: Kurapa kwe cytomegalovirus retinitis muvarwere vane HIV.

Tsanangudzo: Vitravene is an antisense oligonucleotide drug uye yekutanga oligonucleotide mushonga unobvumirwa kushambadzira munyika.Pakutanga kwemusika, kudiwa kwemusika kwemishonga inorwisa-cytomegalovirus yakanga yakakurumidza zvikuru;zvino nekuda kwekuvandudzwa kweanoshanda zvakanyanya antiretroviral therapy, nhamba ye cytomegalovirus kesi yakaderera zvakanyanya.Nekuda kwekushomeka kwemusika, mushonga wakabudiswa muna 2002 uye 2006 Kubviswa munyika dzeEU uye US.

(2) Macugen

Kambani: Co-yakagadziridzwa naPfizer uye Eyetech.

Nguva yemusika: Yakatenderwa kunyorwa muUnited States muna 2004.

Zviratidzo: Pakurapa neovascular age-related macular degeneration.

Mashoko: Macugen is pegylated modified oligonucleotide drug inogona kutarisa uye kusunga kune vascular endothelial growth factor (VEGF165 isoform), uye inotungamirirwa nejekiseni ye intravitreal.

(3) Defitelio

Kambani: Yakagadzirwa neJazz.

Nguva yemusika: Yakatenderwa neEuropean Union muna 2013, uye yakabvumidzwa neFDA munaKurume 2016.

Zviratidzo: Kurapa kwehepatic venule occlusive chirwere chakabatana nerenal kana pulmonary dysfunction mushure mekuita hematopoietic stem cell transplantation.

Tsanangudzo: Defitelio is oligonucleotide drug, musanganiswa we oligonucleotides ane plasmin properties.Yakabviswa muna 2009 nekuda kwezvikonzero zvekutengesa.

(4) Kynamro

Kambani: Co-yakagadziridzwa naIonis Pharma naKastle.

Nguva yemusika: Yakatenderwa muUnited States semushonga wenherera muna 2013.

Zvinoratidza: Zvekurapa adjuvant ye homozygous mhuri hypercholesterolemia.

Tsanangudzo: Kynamro is an antisense oligonucleotide drug, an antisense oligonucleotide inotarisa munhu apo B-100 mRNA.Kynamro inoshandiswa se 200 mg subcutaneously kamwe pavhiki.

(5) Spinraza

Kambani: Yakagadziriswa neIonis Pharmaceuticals.

Nguva yemusika: Yakatenderwa neFDA muna Zvita 2016.

Zviratidzo: Kurapa kwespinal muscular atrophy (SMA).

Tsanangudzo: Spinraza (nusinersen) is antisense oligonucleotide drug.Spinraza inogona kushandura iyo RNA splicing yeSMN2 gene nekusunga kune splicing saiti yeSMN2 exon 7, nekudaro ichiwedzera kugadzirwa kweanoshanda zvizere SMN protein.Muna Nyamavhuvhu 2016, BIOGEN Corporation yakashandisa sarudzo yayo kuwana kodzero dzepasi rose dzeSpinraza.Spinraza yakatanga kuyerwa kwekiriniki yekutanga muvanhu muna 2011. Mumakore mashanu chete, yakabvumidzwa neFDA muna 2016, zvichiratidza kucherechedzwa kweFDA kuzere kwekuita kwayo.Mushonga uyu wakatenderwa kushambadzira muChina muna Kubvumbi 2019. Kutenderera kwese kwemvumo kweSpinraza muChina kuri pasi pemwedzi mitanhatu.Pave nemakore maviri nemwedzi miviri kubva Spinraza yakatanga kubvumidzwa muUnited States.Chirwere chakadaro che blockbuster chekune dzimwe nyika chisingawanzo mushonga chiri muKumhanya kwekunyora muChina kwatove nekukurumidza.Izvi zvinokonzerwawo ne "Chiziviso Chekuburitswa Kwekutanga Rondedzero YeOverseas Mishonga Itsva Inodiwa Nekukurumidza Kutsvagisa Kliniki" yakapihwa neCentre for Drug Evaluation munaNovember 1, 2018, iyo yakaverengerwa muchikamu chekutanga chemakumi mana akakosha ekunze matsva. mishonga yekukurumidza kuongorora, uye Spinraza yakaiswa mukati.

(6) Eksodho 51

Kambani: Yakagadzirwa neAVI BioPharma (yakazonzi Sarepta Therapeutics).

Nguva yemusika: Yakatenderwa neFDA munaGunyana 2016.

Zviratidzo: Kurapa kweDuchenne muscular dystrophy (DMD) neDMD gene mutation mu exon 51 skipping gene.

Mashoko: Exondys 51 is an antisense oligonucleotide drug.Iyo antisense oligonucleotide inogona kusunga kune exon 51 nzvimbo yepre-mRNA yeDMD gene, zvichiita kuti pave nekuumbwa kwemRNA yakakura.Kubvisa, nokudaro kururamisa chikamu chekuverenga mRNA, kunobatsira murwere kugadzira mamwe maitiro anoshanda edystrophin ari mapfupi pane akajairika mapuroteni, nekudaro achivandudza zviratidzo zvemurwere.

(7) Tegsedi

Kambani: Yakagadziriswa neIonis Pharmaceuticals.

Nguva yemusika: Yakatenderwa neEuropean Union yekushambadzira muna Chikunguru 2018.

Zviratidzo: Pakurapa kwenhaka transthyretin amyloidosis (hATTR).

Tsanangudzo: Tegsedi inonzi antisense oligonucleotide drug inotarisa transthyretin mRNA.Ndiwo mushonga wekutanga kubvumidzwa pasi rose wekurapa hATTR.Nzira yehutungamiri ndeye subcutaneous injection.Mushonga unoderedza kugadzirwa kweATTR protein nekutarisa mRNA ye transthyretin (ATTR), uye ine mukana wakanaka wekubatsira-ngozi mukurapa kweATTR.Hapana danho rechirwere kana kuvepo kwecardiomyopathy yaive yakakosha.

(8) Onpattro

Kambani: Co-yakagadziridzwa neAlnylam naSanofi.

Nguva yemusika: Yakatenderwa kunyorwa muUnited States muna 2018.

Zviratidzo: Pakurapa kwenhaka transthyretin amyloidosis (hATTR).

Tsanangudzo: Onpattro is a siRNA drug inotarisa transthyretin mRNA, iyo inoderedza kugadzirwa kweATTR protein muchiropa uye kuunganidzwa kweamyloid deposits mu peripheral nerve nekutarisa mRNA ye transthyretin (ATTR)., nokudaro kuvandudza uye kubvisa zviratidzo zvechirwere.

(9) Givlaari

Kambani: Yakagadzirwa neAlnylam Corporation.

Nguva yemusika: Yakatenderwa neFDA munaNovember 2019.

Zviratidzo: Kurapa kweacute hepatic porphyria (AHP) muvakuru.

Mashoko: Givlaari mushonga we siRNA, wechipiri siRNA mushonga unobvumidzwa kushambadzira mushure meOnpattro.Mushonga unopihwa pasi uye unonangana nemRNA yekushatisa ALAS1 protein.Mwedzi wega wega Givlaari kurapwa kunogona zvakanyanya uye nekuramba kudzikisa mwero weALAS1 muchiropa, nekudaro kuderedza mazinga eurotoxic ALA nePBG kusvika kune yakajairika, nekudaro Rerutsira zviratidzo zvechirwere chemurwere.Iyo data yakaratidza kuti varwere vanobatwa neGivlaari vaive ne74% kuderedzwa kwenhamba yezvirwere zvinopisa zvichienzaniswa neboka re placebo.

(10) Vyondys53

Kambani: Yakagadzirwa neSarepta Therapeutics.

Nguva yemusika: Yakatenderwa neFDA muna Zvita 2019.

Chiratidzo: Kurapwa kwevarwere veDMD vane dystrophin gene exon 53 splice mutation.

Mashoko: Vyondys 53 is an antisense oligonucleotide drug.Mushonga we oligonucleotide unonangana neiyo splicing process ye dystrophin mRNA precursor.Mune nzira isina kunanga yedystrophin mRNA precursor, iyo yekunze Exon 53 yakanga yakamurwa zvishoma, kureva kuti haipo pane yakakura mRNA, uye yakagadzirirwa kuburitsa truncated asi ichiri inoshanda dystrophin protein, nekudaro ichivandudza kurovedza muviri muvarwere.

(11) Waylivra

Kambani: Yakagadziridzwa neIonis Pharmaceuticals uye inotsigira Akcea Therapeutics.

Nguva yemusika: Yakatenderwa neEuropean Medicines Agency (EMA) muna Chivabvu 2019.

Chiratidzo: Sechirongwa chekuwedzera kune chikafu chinodzorwa muvarwere vakuru vane mhuri chylomicronemia syndrome (FCS).

Tsanangudzo: Waylivra is an antisense oligonucleotide drug, inova mushonga wekutanga unobvumidzwa kurapwa kweFCS munyika.

(12) Leqvio

Kambani: Yakagadzirwa naNovartis.

Nguva yemusika: Yakabvumidzwa neEU muna Zvita 2020.

Zviratidzo: Pakurapa kwevakuru vekutanga hypercholesterolemia (heterozygous yemhuri uye isiri yemhuri) kana yakasanganiswa dyslipidemia.

Mashoko: Leqvio mushonga we siRNA wakanangana nePCSK9 mRNA.Ndiyo yekutanga pasi rose cholesterol-kudzikisa (LDL-C) siRNA therapy.Nzira yehutungamiri ndeye subcutaneous injection.Mushonga unoshanda neRNA kupindira kudzikisa PCSK9 mapuroteni mazinga, ayo anozodzikisa mazinga eLDL-C.Data yekiriniki inoratidza kuti Leqvio inogona kuderedza LDL-C neinosvika makumi mashanu muzana muvarwere vane mazinga eLDL-C asingagone kudzikiswa kusvika panhanho dzakanangwa kunyangwe akaremerwa madosi emastatins.

(13) Oslumo

Kambani: Yakagadzirwa neAlnylam Pharmaceuticals.

Nguva yemusika: Yakabvumidzwa neEU munaNovember 2020.

Zviratidzo: Pakurapa kwekutanga hyperoxaluria mhando 1 (PH1).

Tsanangudzo: Oxlumo is a siRNA drug inotarisa hydroxy acid oxidase 1 (HAO1) ​​mRNA, iyo inotungamirirwa pasi pecutaneously.Mushonga uyu wakagadzirwa uchishandisa Alnylam's ichangoburwa yakagadziridzwa kemikari ESC-GalNAc conjugation tekinoroji, iyo inogonesa inodzorwa pasiRNAs nekushingirira kukuru uye kushanda nesimba.Mushonga wacho unotarisa kuora kana kuvharidzirwa kwehydroxy acid oxidase 1 (HAO1) ​​mRNA, inoderedza chiyero che glycolate oxidase muchiropa, uye inopedza substrate inodiwa pakugadzirwa kweoxalate uye inoderedza kugadzirwa kweoxalate kudzora kufambira mberi kwechirwere nekuvandudza. zviratidzo zvechirwere muvarwere.

(14) Viltepso

Kambani: Yakagadzirwa neNS Pharma, inotsigira yeNippon Shinyaku.

Nguva yemusika: Yakatenderwa neFDA muna Nyamavhuvhu 2020.

Zviratidzo: Kurapa kweDuchenne muscular dystrophy (DMD) neDMD gene mutation mu exon 53 skipping gene.

Mashoko: Viltepso is phosphorodiamide morpholino oligonucleotide mushonga.Uyu oligonucleotide mushonga unogona kusunga kune exon 53 nzvimbo yepre-mRNA yeDMD gene, zvichiita kuti pave nekuumbwa kwemRNA yakakura.Iyo exon inobviswa zvishoma, nekudaro ichigadzirisa chikamu chemRNA yekuverenga furemu, ichibatsira murwere kugadzira mamwe maitiro anoshanda edystrophin ari mapfupi pane akajairika mapuroteni, nekudaro achivandudza zviratidzo zvemurwere.

(15) Amvuttra (vutrisiran)

Kambani: Yakagadzirwa neAlnylam Pharmaceuticals.

Nguva yemusika: Yakatenderwa neFDA muna Chikumi 2022.

Zviratidzo: Kurapa kwevakuru venhaka transthyretin amyloidosis ine polyneuropathy (hATTR-PN).

Tsanangudzo: Amvuttra (Vutrisiran) mushonga we siRNA wakanangana ne transthyretin (ATTR) mRNA, inotungamirwa neiyo subcutaneous jekiseni.Vutrisiran yakagadzirwa zvichibva paAlnylam's Enhanced Stabilization Chemistry (ESC) -GalNAc conjugated delivery platform ine kuwedzera potency uye metabolic kugadzikana.Kubvumidzwa kwekurapa kunobva padatha yemwedzi we9 kubva kuChikamu chechitatu chekiriniki yekudzidza (HELIOS-A), ine mibairo yese inoratidza kuti kurapwa kwakavandudza zviratidzo zvehATTR-PN, nevanopfuura 50% yevarwere vanodzosera kana kumisa kufambira mberi.

4. Mamwe magene therapy mishonga

(1) Rexin-G

Kambani: Yakagadzirwa neEpeius Biotech.

Nguva yemusika: Yakatenderwa nePhilippine Food and Drug Administration (BFAD) muna 2005.

Zviratidzo: Kurapwa kwekenza yepamusoro inopesana nechemotherapy.

Mazwi: Rexin-G ijene-rakatakura nanoparticle jekiseni.Inosuma iyo cyclin G1 mutant gene mumaseru anotangwa kuburikidza neretroviral vector kuti iuraye chaizvo mabundu akasimba.Nzira yekutonga ndeye intravenous infusion.Semushonga wakanangana nebundu uyo ​​unoshingairira kutsvaga nekuparadza maseru egomarara remetastatic, zvine zvimwe zvinokonzeresa kune varwere vasingashande kurwisa mamwe mishonga yegomarara, kusanganisira yakanangwa biologics.

(2) Neovasculgen

Kambani: Yakagadzirwa neHuman stem cell Institute.

Nguva yekunyora: Yakatenderwa kuti inyore muRussia musi waZvita 7, 2011, uyezve yakanyorwa muUkraine muna 2013.

Zvinoratidza: Kurapa kweperipheral arterial disease, kusanganisira yakaoma nhengo ischemia.

Tsanangudzo: Neovasculgen iDNA plasmid-based gene therapy umo vascular endothelial growth factor (VEGF) 165 gene inovakwa pamusana weplasmid uye inopinzwa muvarwere.

(3) Collategene

Kambani: Co-yakagadziridzwa neOsaka University uye venture capital mafemu.

Nguva yekunyora: Yakatenderwa neJapan Ministry of Health, Labor and Welfare kuti inyore muna Nyamavhuvhu 2019.

Zviratidzo: Kurapa kweyakanyanya kuderera kwekupedzisira ischemia.

Mashoko: Collategene iplasmid-based gene therapy, yekutanga yeJapan gene therapy mushonga unogadzirwa neAnGes.Chinhu chikuru chemushonga uyu iplasmid isina kupfeka ine gene sequence ye human hepatocyte growth factor (HGF).Kana mushonga wacho wakaiswa mumisumbu yemakumbo ezasi, iyo HGF inoratidzwa ichakurudzira kuumbwa kwetsinga itsva dzeropa dzakatenderedza tsinga dzeropa dzakavharika.Miedzo yemakiriniki yakasimbisa kushanda kwayo mukuvandudza maronda.

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Nguva yekutumira: Nov-10-2022