Izakhi zofuzo ziyizakhi zofuzo eziyisisekelo ezilawula izici.Ngaphandle kwezakhi zofuzo zamanye amagciwane, akhiwa i-RNA, izakhi zofuzo zezinto eziphilayo eziningi zakhiwa i-DNA..Izifo eziningi zezinto eziphilayo zibangelwa ukuxhumana phakathi kofuzo nendawo ezungezile.Ukwelashwa ngezakhi zofuzo empeleni kungalapha noma kunciphise izifo eziningi.Ukwelashwa ngofuzo kuthathwa njengoguquko emkhakheni wezokwelapha kanye nekhemisi.Imithi yokwelapha izakhi zofuzo ngomqondo obanzi ihlanganisa okusekelwe emithini ye-DNA eshintshwe i-DNA (njengemithi yokwelapha i-vivo gene esekelwe kuma-viral vectors, izidakamizwa ze-in vitro gene therapy, izidakamizwa ze-plasmid enqunu, njll.) kanye nezidakamizwa ze-RNA (njengezidakamizwa ze-antisense oligonucleotide, izidakamizwa ze-siRNA, kanye nokwelashwa kofuzo lwe-mRNA, njll.);Imithi yokwelapha i-Gene yomqondo omncane ikakhulukazi ihlanganisa izidakamizwa ze-plasmid DNA, izidakamizwa zokwelashwa kwezakhi zofuzo ezisekelwe kuma-viral vectors, izidakamizwa zokwelashwa kwezakhi ezisekelwe kuma-bacterial vectors, izinhlelo zokuhlela izakhi zofuzo, kanye nemithi yokwelapha iseli yokuguqulwa kwezakhi zofuzo ze-in vitro.Ngemva kweminyaka yokukhula kanzima, izidakamizwa zokwelashwa ngofuzo zithole imiphumela ekhuthazayo yomtholampilo.(Ingabe singabalwa imigomo ye-DNA kanye nemithi yokugomela i-mRNA) Njengamanje, imithi yokwelapha yofuzo engama-45 igunyazelwe ukumakethwa emhlabeni.Isamba sezindlela zokwelapha zofuzo ezingu-9 zigunyazwe ukuthi zimakethwe kulo nyaka, okuhlanganisa nemithi yokwelapha eyi-7 egunyazwe ukukhangisa okokuqala kulo nyaka, okuyilezi: CARVYKTI, Amvuttra, Upstaza, Roctavian, Hemgenix, Adstiladrin kanye ne-Ebvallo, (Qaphela: Ezinye ezimbili zigunyazwe e-United States kulo nyaka. Iqoqo lokuqala eligunyazwe yi-Z-Glos I-FDA yokumaketha e-United States ngo-Agasti 2022, futhi yagunyazwa ukumakethwa yi-European Union ngo-2019; .) Ngokwethulwa kwemikhiqizo eminingi yokwelapha ngofuzo kanye nokuthuthukiswa okusheshayo kobuchwepheshe bokwelapha ngezakhi zofuzo, ukwelashwa ngezakhi zofuzo sekuzongenisa isikhathi sokuthuthuka okusheshayo.

Ukuhlukaniswa kokwelashwa kofuzo (Umthombo wesithombe: Bio-Matrix)

Le ndatshana ibala izindlela zokwelapha zofuzo ezingama-45 (ngaphandle kwemithi yokugomela i-DNA kanye nemithi yokugomela i-mRNA) egunyazelwe ukumakethwa.

1. Ukwelashwa kofuzo lwe-in vitro

(1) Strimvelis

Inkampani: Ithuthukiswe ngabakwa-GlaxoSmithKline (GSK).

Isikhathi sokumaketha: Sivunyelwe ukumakethwa yi-European Union ngoMeyi 2016.

Izinkomba: Ukuze ekwelapheni ezinzima ezihlangene immunodeficiency (SCID).

Ukuphawula: Inqubo evamile yalokhu kwelashwa iwukuba kuqala kutholwe amangqamuzana esiqu esiguli se-hematopoietic, ukuwakhulisa futhi uwathuthukise ku-vitro, bese usebenzisa i-retrovirus ukwethula ikhophi yofuzo esebenzayo ye-ADA (adenosine deaminase) kumaseli we-hematopoietic stem, futhi ekugcineni ujove amaseli e-Hematopoietic stem abuyiselwe emzimbeni.Imiphumela yomtholampilo ikhombisa ukuthi izinga lokusinda leminyaka emi-3 leziguli ze-ADA-SCID ezilashwa nge-Strimvelis lingu-100%.

(2) I-Zalmoxis

Inkampani: Ikhiqizwe yiNkampani ye-MolMed yase-Italy.

Isikhathi sokumaketha: Kutholwe ukugunyazwa kokumaketha okunemibandela ku-European Union ngo-2016.

Izinkomba: Isetshenziselwa ukwelashwa kwe-adjuvant yamasosha omzimba eziguli ngemuva kokufakelwa kwe-hematopoietic stem cell.

Ukuphawula: I-Zalmoxis iyi-allogeneic T cell suicide gene immunotherapy elungiswe ama-retroviral vectors.Le ndlela isebenzisa ama-retroviral vectors ukuguqula ufuzo ama-allogeneic T cell, ukuze ama-T cell ashintshwe izakhi aveze izakhi zofuzo zokuzibulala ze-HSV-TK Mut2 ze-HSV-TK Mut2 zivumela abantu ukuthi basebenzise izidakamizwa ze-ganciclovir (ganciclovir) nganoma isiphi isikhathi ukuze babulale ama-T cell abangela ukusabela okungalungile kwamasosha omzimba, avimbele ukuwohloka okwengeziwe kwe-immune ye-GVHD kanye nokwakhiwa kabusha kwe-GVHD ye-GVHD ye-GVHD kanye nokwakhiwa kabusha kwe-GVHD kweziguli.

(3) I-Invossa-K

Inkampani: Yathuthukiswa ngabakwa-TissueGene (KolonTissueGene).

Isikhathi sokumaketha: Kugunyazwe ukufakwa kuhlu e-South Korea ngoJulayi 2017.

Izinkomba: Ukwelapha isifo samathambo esiwohlozayo.

Ukuphawula: I-Invossa-K iwukwelapha ngofuzo lweseli ye-allogeneic ehlanganisa ama-chondrocyte abantu.Amaseli e-allogeneic ashintshwa ngokofuzo ku-vitro, futhi amaseli ashintshiwe angaveza futhi akhiphe isici sokukhula esiguqukayo esingu-β1 (TGF-β1) ngemva komjovo we-intra-articular.β1), ngaleyo ndlela kuthuthukiswe izimpawu ze-osteoarthritis.Imiphumela yomtholampilo ibonisa ukuthi i-Invossa-K ingathuthukisa kakhulu isifo samathambo edolweni.Yahoxiswa ngo-2019 yi-Korean Food and Drug Administration ngoba umenzi wazibhala kabi izithako ezisetshenzisiwe.

(4) Zynteglo

Inkampani: Icwaningwe futhi yathuthukiswa yi-bluebird bio.

Isikhathi sokumaketha: Kugunyazwe yi-European Union ukuze kumakethwe ngo-2019, futhi kwagunyazwa i-FDA ukuze kumakethwe e-United States ngo-Agasti 2022.

Izinkomba: Okokwelashwa kwe-β-thalassemia encike ekumpontshelweni.

Ukuphawula: I-Zynteglo iwukwelapha ngofuzo lwe-lentviral in vitro eyethula ikhophi esebenzayo yofuzo oluvamile lwe-β-globin (βA-T87Q-globin gene) kumaseli e-hematopoietic stem athathwe esigulini ngevektha ye-lentviral, bese iphinda iphinda ibuyisele lawa maseli e-autologous e-hematopoietic ashintshiwe ngofuzo.Uma isiguli sinofuzo oluvamile lwe-βA-T87Q-globin, singase sikhiqize amaprotheni avamile we-HbAT87Q, anganciphisa noma aqedele isidingo sokumpontshelwa igazi.Ukwelapha okwesikhathi esisodwa okuklanyelwe ukufaka esikhundleni sokumpontshelwa igazi impilo yonke kanye nemithi yempilo yonke ezigulini ezineminyaka engu-12 ubudala nangaphezulu.

(5) Skysona

Inkampani: Icwaningwe futhi yathuthukiswa yi-bluebird bio.

Isikhathi sokumaketha: Kugunyazwe i-European Union ukuze kumakethwe ngoJulayi 2021, futhi kwagunyazwa i-FDA ukuze kumakethwe e-United States ngoSepthemba 2022.

Izinkomba: Ukuze ukwelashwa kwe-cerebral adrenoleukodystrophy (CALD) yokuqala.

Ukuphawula: Ukwelashwa kofuzo kwe-Skysona ukuphela kwendlela yokwelapha yofuzo yesikhathi esisodwa egunyazwe ukwelashwa kwe-cerebral adrenoleukodystrophy (CALD) yesigaba sokuqala.I-Skysona (elivaldogene autotemcel, Lenti-D) iyi-hematopoietic stem cell lentiviral in vitro gene therapy iLenti-D.Inqubo evamile yokwelapha imi kanje: amaseli we-autologous hematopoietic stem akhishwa esigulini, adluliselwe futhi ashintshwe yi-lentivirus ephethe isakhi sofuzo se-ABCD1 in vitro, bese ebuyiselwa esigulini.Isetshenziselwa ukwelapha iziguli ezingaphansi kweminyaka engu-18, ephethe ukuguqulwa kofuzo lwe-ABCD1, kanye ne-CALD.

(6) UKimriya

INKAMPANI: Yathuthukiswa nguNovartis.

Isikhathi sokumaketha: Kugunyazwe yi-FDA ukuze kumakethwe ngo-Agasti 2017.

Izinkomba: Ukwelashwa kwe-precursor B-cell acute lymphoblastic leukemia (ALL) kanye ne-DLBCL ebuyele emuva futhi ephikisayo.

Ukuphawula: I-Kymriah iyisidakamizwa se-lentviral in vitro gene therapy, ukwelashwa kokuqala kwe-CAR-T okugunyazwe ukumakethwa emhlabeni, okuqondiswe ku-CD19, nokusebenzisa i-4-1BB co-stimulatory factor.Ibiza u-$475,000 e-US kanye no-$313,000 e-Japan.

(7) Yescarta

Inkampani: Ithuthukiswe nguKite Pharma, inkampani ephethwe yiGileyadi (GILD).

Isikhathi sokumaketha: Kugunyazwe yi-FDA ukuze kumakethwe ngo-Okthoba 2017;I-Fosun Kite yethule ubuchwepheshe be-Yescarta kwa-Kite Pharma futhi yabukhiqiza e-China ngemuva kokuthola ukugunyazwa.Kugunyazwe ukufakwa kuhlu ezweni.

Izinkomba: Okokwelashwa kwe-B-cell lymphoma enkulu ebuyele emuva noma ephikisayo.

Ukuphawula: I-Yescarta iwukwelapha ngofuzo lwe-retroviral in vitro, okuwukwelashwa kwesibili okuvunyiwe kwe-CAR-T emhlabeni.Iqondise ku-CD19 futhi isebenzisa i-costimulator ye-CD28.Kubiza u-$373,000 e-United States.

(8) I-Tecartus

Inkampani: Ithuthukiswe iGileyadi (GILD).

Isikhathi sokumaketha: Kugunyazwe yi-FDA ukuze kumakethwe ngoJulayi 2020.

Izinkomba: Nge-mantle cell lymphoma ebuyele emuva noma ephikisayo.

Ukuphawula: I-Tecartus iwukwelapha ngokuzenzakalelayo kwe-CAR-T cell eqondiswe ku-CD19, futhi iyindlela yesithathu yokwelapha ye-CAR-T egunyazwe ukumakethwa emhlabeni.

(9) Breyanzi

INKAMPANI: Ithuthukiswe ngabakwaBristol-Myers Squibb (BMS).

Isikhathi sokumaketha: Kugunyazwe yi-FDA ukuze kumakethwe ngoFebhuwari 2021.

Izinkomba: Ibuyele emuva noma i-refractory (R/R) i-B-cell lymphoma enkulu (LBCL).

Ukuphawula: I-Breyanzi iwukwelapha ngofuzo lwe-in vitro okusekelwe ku-lentivirus, ukwelapha kwesine kwe-CAR-T okuvunyelwe ukumakethwa emhlabeni, okuqondiswe ku-CD19.Ukugunyazwa kweBreyanzi kuyingqophamlando yeBristol-Myers Squibb emkhakheni we-cellular immunotherapy, eyakuthola lapho ithola iCelgene ngamabhiliyoni angama-74 ngo-2019.

(10) Abecma

Inkampani: Ihlanganiswe yiBristol-Myers Squibb (BMS) kanye ne-bluebird bio.

Isikhathi sokumaketha: Kugunyazwe yi-FDA ukuze kumakethwe ngoMashi 2021.

Izinkomba: i-myeloma eminingi ebuyele emuva noma ephikisayo.

Ukuphawula: I-Abecma iwukwelapha ngofuzo lwe-in vitro okusekelwe ku-lentivirus, ukwelapha kweseli lokuqala le-CAR-T emhlabeni okuqondiswe ku-BCMA, kanye nokwelapha kwesihlanu kwe-CAR-T okugunyazwe i-FDA.Umgomo womuthi ukuveza ama-chimeric BCMA receptors kuma-T cell esiguli ngokuguqulwa kofuzo kwe-lentivirus-mediated in vitro.Ukwelashwa kokuqeda amaseli e-T angashintshiwe ezigulini, bese kubuyiselwa amaseli e-T ashintshiwe, afuna futhi abulale amaseli omdlavuza aveza i-BCMA ezigulini.

(11) Libmeldy

INKAMPANI: Ithuthukiswe ngabakwa-Orchard Therapeutics.

Isikhathi sokumaketha: Sigunyazwe yi-European Union ukuthi sifakwe ohlwini ngoDisemba 2020.

Izinkomba: Ukuze ukwelashwa kwe-metachromatic leukodystrophy (MLD).

Ukuphawula: I-Libmeldy iwukwelapha ngofuzo okusekelwe kumaseli e-CD34+ e-autologous aguqulwa ngofuzo ku-vitro nge-lentivirus.Idatha yomtholampilo ikhombisa ukuthi ukujova okukodwa kwe-Libmeldy nge-intravenous kungashintsha ngempumelelo inkambo ye-MLD yokuqala uma kuqhathaniswa nokukhubazeka okukhulu kwezimoto nengqondo ezigulini ezingalashwanga zeminyaka efanayo.

(12) Benoda

Inkampani: Ithuthukiswe yi-WuXi Giant Nuo.

Isikhathi sokumaketha: Sigunyazwe ngokusemthethweni yi-NMPA ngoSepthemba 2021.

Izinkomba: Ukwelashwa kweziguli ezikhulile ezine-B-cell lymphoma enkulu ebuyele emuva noma ephikisayo (r/r LBCL) ngemva komugqa wesibili noma ngaphezulu kokwelashwa kwe-systemic.

Ukuphawula: I-Beinoda iwukwelapha ngofuzo olwa ne-CD19 CAR-T, futhi iphinde ibe umkhiqizo oyinhloko we-WuXi Juro Company.Ingumkhiqizo wesibili we-CAR-T ogunyazwe eShayina, ngaphandle kwe-B-cell lymphoid enkulu e-refractory enkulu i-WuXi Giant Nuo futhi ihlela ukwenza umjovo we-Regiorensai ukuze ulaphe ezinye izinkomba eziningi, okuhlanganisa i-follicular lymphoma (FL), i-mantle cell lymphoma (MCL), i-lymphocytic leukemia (CLL), i-lymphocytic leukemia (CLL) yomugqa wesibili we-BBC lympho diffuse (DLL lymphoma enkulu) i-lymphoma enkulu ye-DLL.

(13) I-CARYKTI

Inkampani: Umkhiqizo wokuqala weLegend Biotech ogunyazwe ukumakethwa.

Isikhathi sokumaketha: Kugunyazwe yi-FDA ukuze kumakethwe ngoFebhuwari 2022.

Izinkomba: zokwelashwa kwe-myeloma eminingi ebuyele emuva noma ephikisayo (R/R MM).

Ukuphawula: I-CARVYKTI (i-ciltacabtagene autoleucel, i-Cilta-cel ngamafuphi) iwukwelapha ngofuzo lweseli ye-CAR-T enamasosha omzimba amabili esizinda esisodwa aqondise i-B-cell maturation antigen (BCMA).Idatha ibonisa ukuthi i-CARVYKTI Ezigulini ezine-myeloma eminingi ebuyele emuva noma ephikisayo ezithole izindlela zokwelapha ezine noma ngaphezulu zangaphambili (kuhlanganise nama-proteasome inhibitors, ama-immunomodulators nama-anti-CD38 monoclonal antibodies), isilinganiso sokuphendula sisonke se-98% sibonisiwe.

(14)Evela

INKAMPANI: Ithuthukiswe ngabakwa-Atara Biotherapeutics.

i-European Commission (EC) yokumaketha ngoDisemba 2022, iwukwelapha kokuqala emhlabeni wonke kwe-T cell okugunyazwe ukumakethwa.

Izinkomba: Njenge-monotherapy ye-Epstein-Barr virus (EBV)-related post-transplantation lymphoproliferative disease (EBV+PTLD), iziguli ezithola ukwelashwa kufanele kube abantu abadala kanye nezingane ezineminyaka engaphezu kwengu-2 ubudala ezike zithole okungenani enye imithi yokwelapha eyodwa.

Ukuphawula: I-Ebvallo iwukwelashwa kwe-allogeneic EBV-specific universal T-cell gene eqondisa futhi iqede amaseli anegciwane le-EBV ngendlela ekhawulelwe ye-HLA.Ukugunyazwa kwalokhu kwelashwa kusekelwe emiphumeleni yocwaningo lomtholampilo lwesigaba sesi-3 esibalulekile, futhi imiphumela yabonisa ukuthi i-ORR yeqembu le-HCT neqembu le-SOT lalingama-50%.Izinga lokuxolelwa okuphelele (CR) lalingu-26.3%, izinga lokuxolelwa okuyingxenye (PR) lalingu-23.7%, kanti isikhathi esimaphakathi sokuxolelwa (TTR) kwakuyizinyanga ezingu-1.1.Ezigulini eziyi-19 ezithole ukuxolelwa, eziyi-11 zinesikhathi sokuphendula (DOR) esingaphezu kwezinyanga eziyi-6.Ukwengeza, mayelana nokuphepha, akukho ukusabela okubi okufana ne-graft-versus-host disease (GvHD) noma i-Ebvallo-related cytokine release syndrome eyenzekayo.

2. Ukwelashwa kwe-vivo gene okusekelwe kuma-viral vectors

(1) Gendicine/Jin Sheng

Inkampani: Ithuthukiswe yiNkampani ye-Shenzhen Saibainuo.

Isikhathi sokumaketha: Kugunyazwe ukuthi kufakwe ohlwini e-China ngo-2003.

Izinkomba: Okokwelashwa kwekhanda nentamo i-squamous cell carcinoma.

Qaphela: Umjovo we-recombinant human p53 adenovirus injection i-Gendicine/Jinyousheng iwumuthi we-adenovirus vector gene therapy onamalungelo azimele empahla yengqondo ephethwe yi-Shenzhen Saibainuo Company.I-adenovirus yomuntu ye-5 yakhiwe uhlobo lwe-adenovirus yomuntu 5. Olokuqala luyisakhiwo esiyinhloko somphumela wokulwa ne-tumor wesidakamizwa, futhi lesi sakamuva sisebenza ngokuyinhloko njengesithwali.I-adenovirus vector ithwala isakhi sokwelapha i-p53 kuseli eliqondiwe, iveza isakhi sofuzo sokucindezela isimila p53 kuseli eliqondiwe, kanye nokubonakaliswa kofuzo lwalo Umkhiqizo ungalawula izakhi zofuzo ezilwa nomdlavuza futhi wehlise ukulawulwa kwemisebenzi yama-oncogenes ahlukahlukene, ngaleyo ndlela uthuthukise inhloso yomzimba yokucindezela isimila noma ucindezele isimila.

(2) I-Rigvir

Inkampani: Yathuthukiswa yiLatima Company, Latvia.

Isikhathi sokufakwa kuhlu: Kugunyazwe ukufakwa kuhlu e-Latvia ngo-2004.

Izinkomba: Ukuze ekwelapheni melanoma.

Ukuphawula: I-Rigvir iwukwelapha ngofuzo okusekelwe ku-ECHO-7 i-enterovirus vector eguqulwe ngofuzo.Njengamanje, umuthi wamukelwe e-Latvia, Estonia, Poland, Armenia, Belarus, njll., futhi ubhalisela i-EMA emazweni e-EU .Amacala omtholampilo eminyakeni eyishumi edlule afakazele ukuthi igciwane le-Rigvir oncolytic liphephile futhi liyasebenza, futhi linganyusa izinga lokusinda kweziguli ze-melanoma izikhathi ezi-4-6.Ngaphezu kwalokho, lokhu kwelashwa kuyasebenza nakwezinye izinhlobo zomdlavuza, okuhlanganisa umdlavuza we-colorectal, umdlavuza we-pancreatic, umdlavuza wesinye, umdlavuza wezinso, umdlavuza wendlala yesinye, umdlavuza wamaphaphu, umdlavuza wesibeletho, i-lymphosarcoma, njll.

(3) I-Oncorine

Inkampani: Ithuthukiswe yi-Shanghai Sanwei Biological Company.

Isikhathi sokumaketha: Kuvunyelwe ukufakwa ohlwini e-China ngo-2005.

Izinkomba: ukwelashwa kwamathumba ekhanda nentamo, umdlavuza wesibindi, umdlavuza we-pancreatic, umdlavuza womlomo wesibeletho kanye neminye imidlavuza.

Ukuphawula: I-Oncorine (安科瑞) iwumkhiqizo we-oncolytic virus gene therapy usebenzisa i-adenovirus njengesithwali.Kutholwa i-oncolytic adenovirus, engaphindaphinda ngokuqondile ku-p53 yezakhi zofuzo ezintula noma ezingavamile, okuholela ekuhlaziyweni kwamaseli wesimila, ngaleyo ndlela kubulale amaseli wesimila.ngaphandle kokulimaza amaseli avamile.Ucwaningo lwezokwelapha lubonise ukuthi i-Ankerui inokuphepha okuhle nokusebenza ngempumelelo kwezinhlobonhlobo zamathumba ayingozi.

(4) Glybera

Inkampani: Ithuthukiswe yi-uniQure.

Isikhathi sokumaketha: Sigunyazwe ukufakwa kuhlu e-Europe ngo-2012.

Izinkomba: Ukwelashwa kokuntuleka kwe-lipoprotein lipase (LPLD) ngeziqephu ezinzima noma eziphindaphindayo ze-pancreatitis naphezu kokudla okunomkhawulo oqinile wamafutha.

Ukuphawula: I-Glybera (i-alipogene tiparvovec) iyisidakamizwa sokwelapha izakhi zofuzo esekelwe ku-AAV, esebenzisa i-AAV njengesithwali sokuguqula isakhi sofuzo sokwelapha i-LPL sibe amangqamuzana emisipha, ukuze amangqamuzana ahambisanayo akwazi ukukhiqiza inani elithile le-lipoprotein lipase, Ukuze unciphise lesi sifo, lokhu kwelashwa kusebenza kahle isikhathi eside iminyaka eminingi yokuphatha izidakamizwa kanye nomphumela wokuphatha izidakamizwa ungahlala isikhathi eside.Umuthi uhoxiswe emakethe ngo-2017. Isizathu sokuhoxiswa kwawo singase sihlobane nezici ezimbili: amanani entengo aphezulu kanye nesidingo esilinganiselwe semakethe.Izindleko zokwelashwa ezimaphakathi zomuthi ziphezulu njenge-US$1 million, futhi yisiguli esisodwa kuphela esisithengile futhi sasisebenzisa kuze kube manje.Yize inkampani yomshwalense wezokwelapha isibuyisele u-US$900,000 ngayo, futhi kuwumthwalo omkhulu enkampanini yomshwalense.Ukwengeza, izinkomba eziqondiswe yisidakamizwa azivamile kakhulu, ngezinga lezigameko cishe eli-1 ku-1 million kanye nezinga eliphezulu lokungaxilongwa kahle.

(5) Imlygic

Inkampani: Yathuthukiswa ngabakwa-Amgen.

Isikhathi sokumaketha: Ngo-2015, savunyelwa ukuthi sifakwe ohlwini e-United States nase-European Union.

Izinkomba: Ukwelashwa kwezilonda ze-melanoma ezingakwazi ukususwa ngokuphelele ngokuhlinzwa.

Ukuphawula: I-Imlygic iwuhlobo 1 lwegciwane le-herpes simplex elincishisiwe eliye lashintshwa ubuchwepheshe bofuzo (ukusula izingcezu zalo zofuzo ze-ICP34.5 kanye ne-ICP47, nokufaka isakhi sofuzo se-granulocyte macrophage colony-stimulating factor GM-CSF kugciwane) (HSV-1) i-oncolytic-virus ye-FDA ekhiqizwa okokuqala ku-FDA.Indlela yokuphatha umjovo we-intralesional, ongajovwa ngokuqondile ezilonda ze-melanoma ukuze ubangele ukuphuka kwamangqamuzana e-tumor, ukhulule ama-antigen atholakala ngesimila kanye ne-GM-CSF, futhi ukhuthaze ukusabela kwe-anti-tumor immune.

(6) Luxturna

Inkampani: Ithuthukiswe ngabakwa-Spark Therapeutics, inkampani ephethwe yi-Roche.

Isikhathi sokumaketha: Sagunyazwa ukukhangisa yi-FDA ngo-2017, sase sigunyazwa ukumakethwa eYurophu ngo-2018.

Izinkomba: Ezokwelashwa kwezingane kanye nabantu abadala abalahlekelwe ukubona ngenxa yokuguqulwa kofuzo okuphindwe kabili kwe-RPE65 kodwa bagcine inani elanele lamaseli e-retina asebenzayo.

Ukuphawula: I-Luxturna iwukwelapha ngofuzo olususelwa ku-AAV olulawulwa ngomjovo we-subretinal.Ukwelashwa kwezakhi zofuzo kusebenzisa i-AAV2 njengesithwali ukwethula ikhophi esebenzayo yofuzo oluvamile lwe-RPE65 kumangqamuzana e-retinal esiguli, ukuze amangqamuzana ahambisanayo aveze iphrotheni evamile ye-RPE65, okwenza ukuntuleka kwamaprotheni esiguli e-RPE65, ngaleyo ndlela athuthukise umbono wesiguli.

(7) Zolgensma

Inkampani: Ithuthukiswe ngabakwa-AveXis, inkampani ephethwe yi-Novartis.

Isikhathi sokumaketha: Kugunyazwe yi-FDA ukuze kumakethwe ngoMeyi 2019.

Izinkomba: Ukwelashwa kwe-Spinal Muscular Atrophy (SMA) iziguli ezingaphansi kweminyaka engu-2 ubudala.

Ukuphawula: I-Zolgensma iwukwelapha ngofuzo okusekelwe ku-AAV vector.Lesi sidakamizwa wuhlelo lokwelapha lwesikhathi esisodwa kuphela lwe-spinal muscular atrophy oluvunyelwe ukumakethwa emhlabeni.Ukwethulwa komuthi kuvula inkathi entsha ekwelapheni i-spinal muscular atrophy.ikhasi, inqubekelaphambili eyingqophamlando.Lokhu kwelashwa kwezakhi zofuzo kusebenzisa i-scAAV9 vector ukwethula isakhi sofuzo esivamile se-SMN1 esigulini ngokufakwa emthanjeni ukuze kukhiqizwe amaprotheni avamile e-SMN1, ngaleyo ndlela kuthuthukiswe ukusebenza kwamangqamuzana athintekile njengama-motor neurons.Ngokuphambene, izidakamizwa ze-SMA i-Spinraza ne-Evrysdi zidinga umthamo ophindaphindiwe wesikhathi eside.I-Spinraza inikezwa ngomjovo womgogodla njalo ezinyangeni ezine, kanti i-Evrysdi iyisidakamizwa esithathwa ngomlomo nsuku zonke.

(8) I-Delytact

Inkampani: Ithuthukiswe yi-Daiichi Sankyo Company Limited (TYO: 4568).

Isikhathi sokumaketha: Ukugunyazwa okunemibandela okuvela kuMnyango Wezempilo, Wezabasebenzi Nenhlalakahle Yase-Japan (MHLW) ngoJuni 2021.

Izinkomba: Ukwelashwa kwe-glioma eyingozi.

Ukuphawula: I-Delytact ingumkhiqizo wesine we-oncolytic virus gene therapy ovunyelwe emhlabeni jikelele, futhi umkhiqizo wokuqala wegciwane le-oncolytic ogunyazwe ukwelapha i-glioma eyingozi.I-Delytact igciwane le-herpes simplex elifakwe ngofuzo uhlobo 1 (HSV-1) oncolytic virus elakhiwe uDkt. Todo kanye nozakwabo.I-Delytact yethula izinguquko ezengeziwe zokususa ku-G207 genome ye-HSV-1 yesizukulwane sesibili, ithuthukisa ukuphindaphinda kwayo okukhethayo kumaseli omdlavuza kanye nokungeniswa kwezimpendulo zokuzivikela ezilwa nesimila kuyilapho kugcinwa ukuphepha okuphezulu.I-Delytact iyisizukulwane sokuqala sesithathu se-oncolytic HSV-1 okwamanje esihlolwa ngomtholampilo.Ukugunyazwa kwe-Delytact e-Japan kusekelwe ngokuyinhloko ocwaningweni lomtholampilo lwesigaba sesi-2 sengalo eyodwa.Ezigulini ezine-glioblastoma ephindaphindayo, i-Delytact ifinyelele isiphetho esiyinhloko sezinga lokusinda lonyaka owodwa, futhi imiphumela yabonisa ukuthi i-Delytact ibonise ukusebenza kahle kangcono uma kuqhathaniswa ne-G207.Amandla anamandla okuphindaphinda kanye nomsebenzi ophezulu we-antitumor.Lokhu kwasebenza kahle kumamodeli aqinile wesimila webele, i-prostate, schwannomas, nasopharyngeal, hepatocellular, colorectal, malignant peripheral nerve sheath tumors, kanye nomdlavuza wegilo.

(9) Ukuphila

INKAMPANI: Ithuthukiswe yi-PTC Therapeutics, Inc. (NASDAQ: PTCT).

Isikhathi sokumaketha: Kugunyazwe yi-European Union ukuze kumakethwe ngoJulayi 2022.

Izinkomba: Ngokushoda kwe-L-amino acid decarboxylase (AADC) enephunga elimnandi, igunyazwe ukwelapha iziguli ezinezinyanga eziyi-18 nangaphezulu.

Ukuphawula: I-Upstaza™ (eladocagene exuparvovec) iwukwelapha ngofuzo lwe-vivo ene-adeno-associated virus type 2 (AAV2) njengesithwali.Iziguli ziyagula ngenxa yokuguquka kofuzo olufaka ikhodi ye-enzyme ye-AADC.I-AAV2 iphethe isakhi sofuzo esinempilo esifaka i-enzyme ye-AADC.Indlela yesinxephezelo sofuzo izuza umphumela wokwelapha.Ngokombono, ukuphatha okukodwa kusebenza isikhathi eside.Iwukwelashwa kwezakhi zofuzo zokuqala ezithengiswayo ezijovwa ngqo ebuchosheni.Isigunyazo sokumaketha sisebenza kuwo wonke amazwe angamalungu e-EU angama-27, kanye ne-Iceland, i-Norway ne-Liechtenstein.

(10) Roctavian

Inkampani: Yathuthukiswa ngabakwa-BioMarin Pharmaceutical (BioMarin).

Isikhathi sokumaketha: Kugunyazwe ukumakethwa yi-European Union ngo-Agasti 2022;ukugunyazwa kokumaketha yi-UK Medicines and Healthcare products Administration (MHRA) ngoNovemba 2022.

Izinkomba: Ekwelapheni iziguli zabantu abadala ezine-hemophilia enzima A ezingenawo umlando we-FVIII factor inhibition futhi ezingenayo amasosha omzimba we-AAV5.

Amazwana: I-Roctavian (valoctocogene roxaparvovec) isebenzisa i-AAV5 njengevekhtha futhi isebenzisa umgqugquzeli oqondene nesibindi somuntu i-HLP ukuze iqhubekisele phambili ukubonakaliswa kwe-human coagulation factor VIII (FVIII) nesizinda esingu-B sisusiwe.Isinqumo se-European Commission sokugunyaza ukumakethwa kwe-valoctocogene roxaparvovec sisekelwe kuyo yonke idatha yephrojekthi yokuthuthukiswa komtholampilo yomuthi.Phakathi kwazo, imiphumela yesigaba sesi-III sesilingo somtholampilo i-GENER8-1 ibonise ukuthi uma kuqhathaniswa nedatha yonyaka ngaphambi kokubhaliswa, ngemva kokujova okukodwa kwe-valoctocogene roxaparvovec, izinga lokuphuma kwegazi lonyaka lesihloko (ABR) lincishiswe kakhulu, imvamisa yokusetshenziswa kwamalungiselelo amaprotheni we-recombinant coagulation factor VIII (F8) ancishisiwe, noma umsebenzi wegazi we-F8 ukhuphuka kakhulu emzimbeni.Ngemva kwamaviki angu-4 okwelashwa, izinga lokusebenzisa i-F8 lonyaka lesifundo kanye ne-ABR edinga ukwelashwa kwehliswe ngo-99% no-84%, ngokulandelana, futhi umehluko wawubalulekile ngokwezibalo (p<0.001).Iphrofayili yokuphepha yayiyinhle, futhi asikho isihloko esihlangabezane ne-F8 factor inhibition, ukonakala noma imiphumela engemihle ye-thrombosis, futhi azikho izehlakalo ezimbi kakhulu ezihlobene nokwelashwa (ama-SAE) ezibikiwe.

(11) Hemgenix

Inkampani: Ithuthukiswe yi-UniQure Corporation.

Isikhathi sokumaketha: Kugunyazwe yi-FDA ukuze kumakethwe ngoNovemba 2022.

Izinkomba: Ezokwelapha iziguli ezikhulile ezine-hemophilia B.

Ukuphawula: I-Hemgenix iwukwelapha ngofuzo olususelwe kuvektha ye-AAV5.Umuthi ufakwe i-coagulation factor IX (FIX) ehlukile yofuzo FIX-Padua, elawulwa ngomthambo.Ngemuva kokuphathwa, isakhi sofuzo singaveza isici se-FIX coagulation esibindi futhi sifihle Ngemva kokungena egazini ukuze senze umsebenzi we-coagulation, ukuze kuzuzwe inhloso yokwelashwa, ngokwengqondo, ukuphathwa okukodwa kusebenza isikhathi eside.

(12) I-Adstiladrin

Inkampani: Ithuthukiswe ngabakwaFerring Pharmaceuticals.

Isikhathi sokumaketha: Kugunyazwe yi-FDA ukuze kumakethwe ngoDisemba 2022.

Izinkomba: Ezokwelashwa komdlavuza wesinye ongeyona imisipha ohlaselayo (i-NMIBC) oyingozi kakhulu ongaphenduli ku-Bacillus Calmette-Guerin (BCG) .

Ukuphawula: I-Adstiladrin iwukwelapha ngofuzo okusekelwe ku-adenoviral vector engaphindaphindeki, engacindezela ngokweqile i-interferon alfa-2b protein kumaseli aqondiwe, futhi inikezwa nge-catheter yomchamo ingene esinyeni (inikezwa kanye njalo ezinyangeni ezintathu) , i-virus vector ingangena ngempumelelo kumaseli we-bladder and aprotein-over-2 umphumela we-utic.Le ndlela entsha yokwelapha isakhi sofuzo ishintsha amangqamuzana odonga lwesinye sesiguli abe “imboni” encane ekhiqiza i-interferon, ngaleyo ndlela ithuthukise ikhono lesiguli lokulwa nomdlavuza.

Ukuphepha nokusebenza kahle kwe-Adstiladrin kwahlolwa ocwaningweni lwemitholampilo olunezikhungo eziningi ezihlanganisa iziguli eziyi-157 ezinengozi ephezulu ye-BCG-engaphenduli i-NMIBC.Iziguli zithole i-Adstiladrin njalo ezinyangeni ezintathu kuze kufike ezinyangeni eziyi-12, noma kuze kube yilapho ubuthi obungamukelekile bokwelashwa noma ukuphindeka kwe-NMIBC yezinga eliphezulu.Sekukonke, amaphesenti angama-51 eziguli ezibhalisiwe ezelashwe nge-Adstiladrin athole impendulo ephelele (ukunyamalala kwazo zonke izimpawu zomdlavuza ezibonwa ku-cystoscopy, izicubu ze-biopsy, nomchamo).

3. Izidakamizwa ezincane ze-nucleic acid

(1) I-Vitravene

Inkampani: Ithuthukiswe ngokuhlanganyela ngu-Ionis Pharma (owayekade eyi-Isis Pharma) kanye noNovartis.

Isikhathi sokumaketha: Ngo-1998 nango-1999, sagunyazwa ukukhangisa yi-FDA kanye ne-EU EMA.

Izinkomba: Ukwelashwa kwe-cytomegalovirus retinitis ezigulini ezine-HIV.

Ukuphawula: I-Vitravene iyisidakamizwa se-oligonucleotide ye-antisense, okuyisidakamizwa sokuqala se-oligonucleotide esivunyelwe ukumakethwa emhlabeni.Esigabeni sokuqala sokufakwa kuhlu, isidingo semakethe semithi elwa ne-CMV sasiphuthuma kakhulu;kamuva, ngenxa yokuthuthukiswa kokwelashwa ngezidambisigciwane ezisebenza kakhulu, inani lamacala e-CMV lehla kakhulu.Ngenxa yesidingo semakethe esintengayo, umuthi wethulwa ngo-2002 kanye no-2006 Ukuhoxiswa emazweni e-EU nase-United States.

(2) I-Macugen

Inkampani: Ihlanganiswe yi-Pfizer ne-Eyetech.

Isikhathi sokumaketha: Sigunyazwe ukufakwa kuhlu e-United States ngo-2004.

Izinkomba: Ukuze ukwelashwa kwe-neovascular age-related macular degeneration.

Amazwana: I-Macugen iyisidakamizwa se-oligonucleotide esilungisiwe se-pegylated, esingakhomba futhi sibophe i-vascular endothelial growth factor (VEGF165 subtype), futhi indlela yokuphatha i-intravitreal injection.

(3) Defitelio

Inkampani: Ithuthukiswe ngabakwaJazz Pharmaceuticals.

Isikhathi sokumaketha: Sagunyazwa ukumakethwa yi-European Union ngo-2013 futhi sagunyazwa yi-FDA ukuze simakethwe ngoMashi 2016.

Izinkomba: Ukwelashwa kwesifo se-hepatic veno-occlusive esihambisana nokungasebenzi kahle kwezinso noma amaphaphu ngemva kokufakelwa kwe-hematopoietic stem cell.

Ukuphawula: I-Defitelio iyisidakamizwa se-oligonucleotide, esiyingxube ye-oligonucleotides enezakhiwo ze-plasmin.Ihoxiswe emakethe ngo-2009 ngenxa yezizathu zokuthengisa.

(4) Kynamro

Inkampani: Ihlanganiswe ngu-Ionis Pharma noKastle.

Isikhathi sokumaketha: Ngo-2013, sagunyazwa ukumakethwa e-United States njengesidakamizwa esiyintandane.

Izinkomba: Ukwelashwa kwe-adjuvant ye-homozygous yomndeni hypercholesterolemia.

Ukuphawula: I-Kynamro iyisidakamizwa se-oligonucleotide esine-antisense, okuyi-oligonucleotide ye-antisense eqondise i-apo yomuntu ye-B-100 mRNA.I-Kynamro iphathwa njenge-200 mg ngaphansi kwesikhumba kanye ngesonto.

(5) Spinraza

Inkampani: Ithuthukiswe ngabakwa-Ionis Pharmaceuticals.

Isikhathi sokumaketha: Kugunyazwe yi-FDA ukuze kumakethwe ngoDisemba 2016.

Izinkomba: Ukwelashwa kwe-spinal muscular atrophy (SMA).

Ukuphawula: I-Spinraza (nusinersen) iyisidakamizwa esilwa ne-oligonucleotide.Ngokubophezela kusayithi lokuqhekeka kwe-SMN2 exon 7, i-Spinraza ingashintsha i-RNA cleavage yofuzo lwe-SMN2, ngaleyo ndlela ikhulise ukukhiqizwa kwephrotheni ye-SMN esebenza ngokugcwele.Ngo-Agasti 2016, i-BIOGEN yasebenzisa inketho yayo ukuze ithole amalungelo omhlaba e-Spinraza.I-Spinraza iqale kuphela uhlolo lwayo lokuqala lomtholampilo kubantu ngo-2011. Eminyakeni engu-5 nje, yagunyazwa yi-FDA ukuze imakethwe ngo-2016, okubonisa ukuqashelwa okugcwele kwe-FDA kokusebenza kwayo.Umuthi wagunyazwa ukuthi ukhangiswe e-China ngo-Ephreli 2019. Wonke umjikelezo wokugunyazwa kwe-Spinraza e-China wawungaphansi kwezinyanga ezingu-6, futhi kwase kuyiminyaka emi-2 nezinyanga ezingu-2 kusukela i-Spinraza yagunyazwa okokuqala e-United States.Isivinini sokufakwa kuhlu e-China sesivele sishesha kakhulu.Lokhu futhi kungenxa yokuthi Isikhungo Sokuhlolwa Kwezidakamizwa sikhiphe “Isaziso Sokushicilela Uhlu Lweqoqo Lokuqala Lezidakamizwa Ezintsha Zaphesheya Ezidingeka Ngokuphuthumayo Emsebenzini Womtholampilo” ngoNovemba 1, 2018, futhi lafakwa eqeqebeni lokuqala lemithi emisha engama-40 ukuze ibuyekezwe ngokushesha, phakathi kwayo i-Spinraza ifakwe phakathi kwayo.

(6) Exondys 51

Inkampani: Yathuthukiswa yi-AVI BioPharma (kamuva eyaqanjwa kabusha ngokuthi i-Sarepta Therapeutics).

Isikhathi sokumaketha: NgoSepthemba 2016, sagunyazwa ukukhangisa yi-FDA.

Izinkomba: Ekwelapheni i-Duchenne muscular dystrophy (DMD) ene-exon 51 ukweqa ukuguqulwa kofuzo kufuzo lwe-DMD.

Amazwana: I-Exondys 51 iyisidakamizwa se-oligonucleotide e-antisense, i-oligonucleotide ye-antisense ingabophezela endaweni ye-exon 51 ye-pre-mRNA yofuzo lwe-DMD, okuholela ekwakhekeni kwe-mRNA evuthiwe, ingxenye ye-exon 51 ivinjiwe i-Excision, ngaleyo ndlela ilungise ingxenye ethile ye-protein esebenzayo i-dystrophin i-syndrome evamile, ukusiza iziguli ziqondise amafomu amafushane we-mRNA. , ngaleyo ndlela kube ngcono izimpawu zesiguli.

(7) Tegsedi

Inkampani: Ithuthukiswe ngabakwa-Ionis Pharmaceuticals.

Isikhathi sokumaketha: Sivunyelwe ukumakethwa yi-European Union ngoJulayi 2018.

Izinkomba: Okokwelashwa kwe-hereditary transthyretin amyloidosis (hATTR).

Ukuphawula: I-Tegsedi iyisidakamizwa se-oligonucleotide esiphikisayo esiqondise i-transthyretin mRNA.Uwumuthi wokuqala ogunyazwe emhlabeni wonke ukwelapha i-hATTR.Ilawulwa ngomjovo we-subcutaneous.Umuthi wehlisa ukukhiqizwa kwephrotheni ye-ATTR ngokukhomba i-mRNA ye-transthyretin (ATTR), futhi unenani elihle lenzuzo-lengozi ekwelapheni i-ATTR, futhi i-neuropathy yesiguli kanye nezinga lempilo kuye kwathuthukiswa kakhulu, futhi kuyahambisana nezinhlobo zokuguquka kwe-TTR, Isigaba sesifo noma ubukhona be-cardiomyopathy obubalulekile.

(8) Onpattro

Inkampani: Ithuthukiswe ngokuhlanganyela yi-Alnylam Corporation kanye ne-Sanofi Corporation.

Isikhathi sokumaketha: Sigunyazwe ukufakwa kuhlu e-United States ngo-2018.

Izinkomba: Okokwelashwa kwe-hereditary transthyretin amyloidosis (hATTR).

Ukuphawula: I-Onpattro iyisidakamizwa se-siRNA esiqondise i-transthyretin mRNA, esinciphisa ukukhiqizwa kwephrotheni ye-ATTR esibindini futhi sinciphise ukunqwabelana kwama-amyloid emithanjeni ye-peripheral ngokukhomba i-mRNA ye-transthyretin (ATTR) , ngaleyo ndlela ithuthukise futhi inciphise izimpawu zesifo.

(9) Givlaari

Inkampani: Ithuthukiswe yi-Alnylam Corporation.

Isikhathi sokumaketha: Kugunyazwe yi-FDA ukuze kumakethwe ngoNovemba 2019.

Izinkomba: Ukwelashwa kwe-acute hepatic porphyria (AHP) kubantu abadala.

Ukuphawula: I-Givlaari umuthi we-siRNA, okuwumuthi we-siRNA wesibili ogunyazwe ukumakethwa ngemuva kwe-Onpattro.Indlela yokuphatha umjovo we-subcutaneous.Umuthi uqondise ku-mRNA yephrotheni ye-ALAS1, futhi ukwelashwa kwanyanga zonke nge-Givlaari kunganciphisa ngokuphawulekayo nangokuphelele izinga le-ALAS1 esibindini, ngaleyo ndlela kwehlise amazinga e-neurotoxic ALA ne-PBG ebangeni elivamile, ngaleyo ndlela Kwehlise izimpawu zesifo sesiguli.Idatha ibonise ukuthi iziguli eziphathwe nge-Givlaari ziye zancipha ngo-74% inani lokubanjwa uma kuqhathaniswa neqembu le-placebo.

(10) I-Vyondys53

INKAMPANI: Ithuthukiswe ngabakwaSarepta Therapeutics.

Isikhathi sokumaketha: Kugunyazwe yi-FDA ukuze kumakethwe ngoDisemba 2019.

Izinkomba: Ukwelashwa kweziguli ze-DMD ezine-dystrophin gene exon 53 splicing mutation.

Ukuphawula: I-Vyondys 53 iyisidakamizwa se-oligonucleotide esiphikisayo, esiqondise inqubo yokuhlanganisa i-dystrophin pre-mRNA.I-Exon 53 incishisiwe kancane, okusho ukuthi ayikho ku-mRNA evuthiwe, futhi iklanyelwe ukukhiqiza i-dystrophin encishisiwe kodwa esasebenzayo, ngaleyo ndlela ithuthukise umthamo wokuzivocavoca ezigulini.

(11) Waylivra

Inkampani: Ithuthukiswe ngabakwa-Ionis Pharmaceuticals kanye nenkampani engaphansi kwayo i-Akcea Therapeutics.

Isikhathi sokumaketha: Kuvunyelwe ukumakethwa yi-European Medicines Agency (EMA) ngoMeyi 2019.

Izinkomba: Njengokwelashwa kwe-adjuvant ngaphezu kokulawulwa kokudla ezigulini ezikhulile ezine-familia chylomicronemia syndrome (FCS).

Ukuphawula: I-Waylivra iyisidakamizwa se-oligonucleotide esiphikisayo, okuwumuthi wokuqala ogunyazwe ukumakethwa emhlabeni ukuze welaphe i-FCS.

(12) Leqvio

Inkampani: Yathuthukiswa ngabakwaNovartis.

Isikhathi sokumaketha: Kugunyazwe yi-European Union ukuze kumakethwe ngoDisemba 2020.

Izinkomba: Ekwelapheni abantu abadala abane-hypercholesterolemia eyinhloko (i-heterozygous yomndeni nokungeyona yomndeni) noma i-dyslipidemia exubile.

Ukuphawula: I-Leqvio iyisidakamizwa se-siRNA esiqondise ku-PCSK9 mRNA.Ukwelashwa kwe-siRNA kokuqala emhlabeni wonke kokwehlisa i-cholesterol (LDL-C).Ilawulwa ngomjovo we-subcutaneous.Umuthi wehlisa izinga lephrotheni ye-PCSK9 ngokuphazanyiswa kwe-RNA, ngaleyo ndlela wehlise izinga le-LDL-C.Idatha yomtholampilo ikhombisa ukuthi ezigulini ezingakwazi ukwehlisa amazinga e-LDL-C afinyelele ezingeni okuhlosiwe ngemuva kokwelashwa ngomthamo omkhulu obekezelelwe wama-statins, i-Leqvio inganciphisa i-LDL-C cishe ngama-50%.

(13)Uxolo

Inkampani: Ithuthukiswe yi-Alnylam Pharmaceuticals.

Isikhathi sokumaketha: Kugunyazwe yi-European Union ukuze kumakethwe ngoNovemba 2020.

Izinkomba: Ukuze ukwelashwa kwe-hyperoxaluria eyinhloko yohlobo 1 (PH1).

Ukuphawula: I-Oxlumo iyisidakamizwa se-siRNA esiqondise ku-hydroxyacid oxidase 1 (HAO1) ​​mRNA, futhi indlela yokuphatha iwumjovo ongaphansi kwesikhumba.Umuthi wathuthukiswa kusetshenziswa ikhemikhali yakamuva yokuqina ye-Alnylam, ubuchwepheshe bokuhlanganiswa kwe-ESC-GalNAc, obuvumela i-siRNA elawulwa ngaphansi kwesikhumba ukuphikelela okukhulu namandla.Umuthi wehlisa noma uvimbela i-hydroxyacid oxidase 1 (HAO1) ​​mRNA, wehlisa izinga le-glycolate oxidase esibindini, bese usebenzisa i-substrate edingekayo ukuze kukhiqizwe i-oxalate, wehlise ukukhiqizwa kwe-oxalate ukulawula ukuqhubeka kwesifo ezigulini futhi kuthuthukiswe izimpawu zesifo.

(14) I-Viltepso

Inkampani: Ithuthukiswe yi-NS Pharma, inkampani ephethwe yi-Nippon Shinyaku.

Isikhathi sokumaketha: Kugunyazwe yi-FDA ukuze kumakethwe ngo-Agasti 2020.

Izinkomba: Ekwelapheni i-Duchenne muscular dystrophy (DMD) ene-exon 53 ukweqa ukuguqulwa kofuzo kufuzo lwe-DMD.

Amazwana: I-Viltepso iyisidakamizwa se-oligonucleotide e-antisense engakwazi ukubopha endaweni ye-exon 53 ye-pre-mRNA yofuzo lwe-DMD, okubangela ukuthi ingxenye ye-exon 53 ikhishwe ngemva kokwakhiwa kwe-mRNA evuthiwe, ngaleyo ndlela ilungise ingxenye yohlaka lokufunda lwe-mRNA Ibhokisi lisiza iziguli ukuba zihlanganise ezinye izinhlobo ze-dystrophin ezisebenzayo ze-dystrophin ezivamile lapho amafomu asebenzayo asebenza kahle.

(15) Amamondi 45

Inkampani: Ithuthukiswe ngabakwaSarepta Therapeutics.

Isikhathi sokumaketha: Kugunyazwe yi-FDA ukuze kumakethwe ngoFebhuwari 2021.

Izinkomba: Okokwelashwa kwe-Duchenne muscular dystrophy (DMD) nge-exon 45 ukweqa ukuguqulwa kofuzo kufuzo lwe-DMD.

Amazwana: I-Amondys 45 iyisidakamizwa se-oligonucleotide e-antisense, i-oligonucleotide ye-antisense ingabophezela endaweni ye-exon 45 ye-pre-mRNA yofuzo lwe-DMD, okuholela ekutheni ingxenye ye-exon 45 ivinjwe ngemva kokwakhiwa kwe-mRNA evuthiwe Excision, ngaleyo ndlela ukufundwa kwe-dystrophin evamile ku-mRNA okuvamile ukulungisa uhlaka oluvamile lwe-mRNA okusiza ukuguqula i-mRNA isikhashana. amaprotheni, ngaleyo ndlela athuthukise izimpawu zesiguli.

(16) Amvuttra (vutrisiran)

Inkampani: Ithuthukiswe yi-Alnylam Pharmaceuticals.

Isikhathi sokumaketha: Kugunyazwe yi-FDA ukuze kumakethwe ngoJuni 2022.

Izinkomba: Okokwelashwa kwe-hereditary transthyretin amyloidosis ene-polyneuropathy (hATTR-PN) kubantu abadala.

Ukuphawula: I-Amvuttra (Vutrisiran) iyisidakamizwa se-siRNA esiqondise i-transthyretin (ATTR) mRNA, elawulwa ngomjovo ongaphansi kwesikhumba.I-Vutrisiran isuselwe kumklamo weplathifomu yokulethwa kwe-Alnylam's Enhanced Stability Chemistry (ESC)-GalNAc enamandla akhulayo nokuzinza kwe-metabolic.Ukugunyazwa kokwelashwa kusekelwe emininingwaneni yezinyanga eziyi-9 yocwaningo lwayo lomtholampilo lweSigaba sesi-III (HELIOS-A), futhi imiphumela iyonke ibonisa ukuthi ukwelapha kwathuthukisa izimpawu ze-hATTR-PN, futhi ngaphezu kuka-50% wesimo seziguli sahlehliswa noma samiswa ekubeni sibi kakhulu.

4. Eminye imithi yokwelapha ngofuzo

(1) I-Rexin-G

Inkampani: Yathuthukiswa ngabakwa-Epeius Biotech.

Isikhathi sokumaketha: Ngo-2005, sagunyazwa ukukhangisa yi-Philippine Food and Drug Administration (BFAD).

Izinkomba: Ezokwelashwa komdlavuza oseqophelweni eliphezulu ukumelana ne-chemotherapy.

Ukuphawula: I-Rexin-G iwumjovo we-nanoparticle ogcwele isakhi sofuzo.Yethula isakhi sofuzo esiguquguqukayo se-cyclin G1 kumaseli okuqondiwe ngokusebenzisa i-retroviral vector ukuze ibulale ngokukhethekile izimila eziqinile.Indlela yokuphatha ukumnika nge-intravenous.Njengomuthi oqondiswe kwisimila ofuna ngenkuthalo futhi ucekele phansi amaseli omdlavuza we-metastatic, unomphumela othile wokwelapha ezigulini ezihlulekile kwezinye izidakamizwa zomdlavuza, okuhlanganisa ne-biologics ehlosiwe.

(2) Neovasculgen

Inkampani: Ithuthukiswe yi-Human stem cell Institute.

Isikhathi sokufakwa kuhlu: Yagunyazwa ukuthi ifakwe ohlwini e-Russia ngo-December 7, 2011, yase yethulwa e-Ukraine ngo-2013.

Izinkomba: Ukwelashwa kwe-peripheral vascular arterial disease, kuhlanganise ne-ischemia enzima yesitho.

Ukuphawula: I-Neovasculgen iwukwelapha ngofuzo okusekelwe kuma-plasmids e-DNA.I-vascular endothelial growth factor (VEGF) 165 gene yakhiwe kumgogodla we-plasmid futhi ifakwe ezigulini.

(3) I-Collategene

Inkampani: Ithuthukiswe ngokuhlanganyela yiNyuvesi yase-Osaka kanye nezinkampani ezinkulu zebhizinisi.

Isikhathi sokumaketha: Sigunyazwe Umnyango Wezempilo, Wezabasebenzi Nenhlalakahle Yase-Japan ngo-August 2019.

Izinkomba: Ukwelashwa kwe-ischemia ebucayi ephansi.

Ukuphawula: I-Collategene iwukwelapha ngofuzo okusekelwe ku-plasmid, umuthi wokuqala wasekhaya wokwelapha ngezakhi zofuzo okhiqizwe yi-AnGes, inkampani yokwelapha ngezakhi zofuzo e-Japan.Ingxenye eyinhloko yalesi sidakamizwa i-plasmid enqunu equkethe ukulandelana kofuzo lwe-hepatocyte growth factor (HGF).Uma umuthi ujovwa emisipha yemilenze engezansi, i-HGF eveziwe izokhuthaza ukwakheka kwemithambo yegazi emisha ezungeze imithambo yegazi evalekile.Izivivinyo zemitholampilo ziwuqinisekisile umphumela wawo ekwenzeni ngcono izilonda.

I-Foregene ingasiza kanjani ekuthuthukisweni kokwelashwa kofuzo?

Sisiza ukulondoloza isikhathi sokuhlolwa ekuhlolweni okukhulu, esigabeni sokuqala sokuthuthukiswa kwezidakamizwa ze-siRNA.

Imininingwane eyengeziwe vakashela:

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