Muva nje, izidakamizwa ezintathu zokwelashwa kofuzo zigunyazwe ukumakethwa, okuyilezi: (1) NgoJulayi 21, 2022, i-PTC Therapeutics, Inc. (NASDAQ: PTCT) imemezele ukuthi i-AAV gene therapy Upstaza™ ivunywe yi-European Commission Iwuhlobo lokuqala lokwelapha oludayiswayo olufakwe ngqo ebuchosheni (bona i-athikili eyedlule ku-Generapy ingqondo ivunyelwe ukumaketha).(2) Ngo-Agasti 17, 2022, i-US Food and Drug Administration (FDA) yagunyaza ukwelashwa kofuzo kwe-Bluebird Bio i-Zynteglo (betibeglogene autotemcel, beti-cel) yokwelashwa kwe-beta thalassemia.Ukugunyazwa kokwelashwa e-United States ngokungangabazeki "kuwusizo eqhweni" lweBluebird Bio, esenkingeni yezimali.(3) Ngo-Agasti 24, 2022, i-BioMarin Pharmaceutical (BioMarin) yamemezela ukuthi Ikhomishana YaseYurophu igunyaze ukumaketha okunemibandela kwe-ROCTAVIAN™ (valoctocogene roxaparvovec), ukwelapha ngofuzo lwe-hemophilia A, yokwelapha iziguli ezingenawo umlando we-FVIII factor inhibitors kanye ne-AAV5 i-hemophilia edala i-hemophilia yeziguli ezinama-antibody AAV5 angaphambili Ukwelapha ngofuzo okugunyazwe ukukhangisa).Kuze kube manje, izidakamizwa zokwelashwa kofuzo ezingama-41 sezivunyelwe ukumakethwa emhlabeni jikelele.

I-Gene iyiyunithi yofuzo eyisisekelo elawula izici.Ngaphandle kwezakhi zofuzo zamanye amagciwane, akhiwa i-RNA, izakhi zofuzo zezinto eziphilayo eziningi zakhiwa i-DNA.Izifo eziningi zezinto eziphilayo zibangelwa ukuxhumana phakathi kwezakhi zofuzo kanye nemvelo, futhi izifo eziningi zingelapheka noma zincishiswe empeleni ngokwelashwa kwezakhi zofuzo.Ukwelashwa ngofuzo kuthathwa njengoguquko emkhakheni wezokwelapha kanye nekhemisi.Imithi yokwelapha izakhi zofuzo ebanzi ihlanganisa izidakamizwa ezisekelwe emithini ye-DNA eshintshwe i-DNA (njengemithi yokwelapha i-viral vector-based in vivo gene therapy, 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 echazwe ngokuncane ihlanganisa ikakhulukazi izidakamizwa ze-plasmid DNA, izidakamizwa zokwelashwa kwezakhi zofuzo ezisekelwe kuma-viral vectors, imithi yokwelapha izakhi ezisekelwe kuma-bacterial vectors, izinhlelo zokuhlela izakhi zofuzo kanye nemithi yokwelapha amangqamuzana eguqulwa izakhi zofuzo ku-vitro.Ngemuva kweminyaka yokukhula okuhlukumezayo, izidakamizwa zokwelashwa ngofuzo zithole imiphumela ekhuthazayo yomtholampilo.(ngaphandle kwemijovo ye-DNA kanye nemithi yokugoma ye-mRNA), imithi yokwelapha yofuzo engama-41 igunyazelwe ukumakethwa emhlabeni.Ngokwethulwa kwemikhiqizo kanye nokuthuthuka okusheshayo kobuchwepheshe bokwelapha ngezakhi zofuzo, ukwelashwa ngezakhi zofuzo sekuzongenisa isikhathi sokuthuthuka ngokushesha.

Ukuhlukaniswa kokwelashwa kofuzo (Umthombo wesithombe: Biological Jingwei)

Le ndatshana ibala izindlela zokwelapha zofuzo ezingama-41 ezigunyazwe ukukhangisa (ngaphandle kwemithi yokugomela i-DNA kanye nemithi yokugomela i-mRNA).

1. Ukwelashwa kofuzo lwe-in vitro

(1) Strimvelis

Inkampani: Ithuthukiswe ngabakwa-GlaxoSmithKline (GSK).

Isikhathi sokumaketha: Sigunyazwe 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 olusebenzayo lwe-ADA (adenosine deaminase) emangqamuzaneni awo e-hematopoietic stem, futhi ekugcineni adlulisele ama-stem amaseli e-hematopoietic ashintshiwe.Amaseli we-Hematopoietic stem aphinde afakwe emzimbeni.Imiphumela yomtholampilo ikhombise ukuthi izinga lokusinda leminyaka emi-3 leziguli ze-ADA-SCID elashwe nge-Strimvelis lalingu-100%.

(2) I-Zalmoxis

Inkampani: Ikhiqizwe yi-MolMed, Italy.

Isikhathi sokumaketha: Kutholwe ukugunyazwa kokumaketha okunemibandela kwe-EU 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 i-retroviral vector.Izakhi zofuzo zokuzibulala ze-1NGFR ne-HSV-TK Mut2 zivumela abantu ukuthi basebenzise i-ganciclovir nganoma yisiphi isikhathi ukubulala amaseli e-T abangela ukusabela okungalungile kwamasosha omzimba, avimbele ukuwohloka okwengeziwe kwe-GVHD okungenzeka kwenzeke, futhi abuyisele ukusebenza kwamasosha omzimba ezigulini ezine-haploidentical HSCT ngemva kokuhlinzwa i-Escort.

(3) I-Invossa-K

Inkampani: Yathuthukiswa yinkampani ye-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.Ilayisensi yahoxiswa umlawuli wezidakamizwa waseNingizimu Korea ngo-2019 ngoba umenzi walebula ngokungalungile izithako ezisetshenzisiwe.

(4) Zynteglo

Inkampani: Ithuthukiswe yinkampani ye-American bluebird bio (bluebird bio) inkampani.

Isikhathi sokumaketha: Sigunyazwe yi-European Union ngo-2019, futhi sagunyazwa yi-FDA ngo-Agasti 2022.

Izinkomba: Okokwelashwa kwe-β-thalassemia encike ekumpontshelweni.

Ukuphawula: I-Zynteglo iwukwelapha nge-lentiviral in vitro gene, esebenzisa i-lentviral vector ukwethula ikhophi esebenzayo yofuzo oluvamile lwe-β-globin (ufuzo lwe-βA-T87Q-globin) kumaseli e-hematopoietic stem akhishwa ezigulini., bese iphinda iphinde ibuyisele esigulini lawa maseli we-autologous hematopoietic stem ashintshwe ngofuzo.Uma isiguli sesinofuzo oluvamile lwe-βA-T87Q-globin, singase sikhiqize iphrotheni evamile ye-HbAT87Q, enganciphisa ngempumelelo noma iqede 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: Ithuthukiswe yinkampani ye-American bluebird bio (bluebird bio) inkampani.

Isikhathi sokumaketha: Kugunyazwe yi-EU ukuze kumakethwe ngoJulayi 2021.

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

Ukuphawula: I-Skysona gene therapy ukuphela kwendlela yokwelapha yofuzo yesikhathi esisodwa egunyazwe ukwelashwa kwe-cerebral adrenoleukodystrophy (CALD) yokuqala.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, ashintshwe ku-vitro yi-lentivirus ephethe isakhi sofuzo se-ABCD1 yomuntu, bese ebuyiselwa esigulini.Ukwelashwa kweziguli ezingaphansi kweminyaka engu-18 nge-ABCD1 gene mutation kanye ne-CALD.

(6) UKimriya

Inkampani: Yathuthukiswa ngabakwaNovartis.

Isikhathi sokumaketha: Sigunyazwe yi-FDA 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 okuvunyelwe emhlabeni kwe-CAR-T, okuqondiswe ku-CD19 nokusebenzisa i-co-stimulatory factor 4-1BB.Intengo ingu-$475,000 e-US kanye no-$313,000 e-Japan.

(7) Yescarta

Inkampani: Yathuthukiswa nguKite Pharma, inkampani engaphansi kweGileyadi.

Isikhathi sokumaketha: Sigunyazwe yi-FDA ngo-Okthoba 2017.

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

Ukuphawula: I-Yescarta iwukwelapha nge-retroviral in vitro gene.Ukwelashwa kwe-CAR-T kwesibili okugunyazwe emhlabeni.Iqondise ku-CD19 futhi isebenzisa i-costimulatory factor ye-CD28.Intengo e-US ingu-$373,000.

(8) I-Tecartus

Inkampani: Ithuthukiswe iGileyadi (GILD).

Isikhathi sokumaketha: Kugunyazwe yi-FDA 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 yokwelapha yesithathu ye-CAR-T egunyazwe ukumakethwa emhlabeni.

(9) Breyanzi

Inkampani: Yathuthukiswa ngabakwaBristol-Myers Squibb (BMS).

Isikhathi sokumaketha: Kugunyazwe yi-FDA 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, kanye nokwelapha kwesine kwe-CAR-T okugunyazwe ukumakethwa emhlabeni, okuqondiswe ku-CD19.Ukugunyazwa kweBreyanzi kuyingqophamlando yeBristol-Myers Squibb emkhakheni we-cellular immunotherapy, iBristol-Myers eyawuthola ngenkathi ithola iCelgene ngamaRandi ayizigidi eziyizinkulungwane ezingama-74 ngo-2019.

(10) Abecma

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

Isikhathi sokumaketha: Kugunyazwe yi-FDA ngoMashi 2021.

Izinkomba: I-myeloma eminingi ebuyele emuva noma ephikisayo.

Ukuphawula: I-Abecma iwukwelashwa kwe-lentivirus okusekelwe ku-in vitro gene, ukwelashwa kwamaseli okuqala e-CAR-T emhlabeni okuqondise ku-BCMA, kanye nokwelapha kwesihlanu kwe-CAR-T okugunyazwe yi-FDA.Umgomo womuthi ukuveza isamukeli se-chimeric BCMA kumaseli e-T azenzakalelayo esiguli ngokusebenzisa ukuguqulwa kofuzo kwe-lentivirus-mediated in vitro.Ngaphambi kokufakwa kwesidakamizwa sofuzo lweseli, isiguli sathola izinhlanganisela ezimbili ze-cyclophosphamide ne-fludarabine ukuze zelashwe ngaphambilini.Ukwelashwa kokukhipha ama-T cell angashintshiwe esigulini, bese kuphinda kufakwe ama-T cell ashintshiwe abuyele emzimbeni wesiguli ukuze afune futhi abulale amaseli omdlavuza aveza i-BCMA.

(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 ekuguqulweni kofuzo lwe-lentiviral in vitro yamaseli e-CD34+ e-autologous.Idatha yomtholampilo ibonisa ukuthi ukujova okukodwa kwe-Libmeldy nge-intravenous kuphumelela ekuguquleni inkambo ye-MLD yokuqala kanye nokukhubazeka okukhulu kwezimoto nengqondo ezigulini ezingalashwanga ezineminyaka efanayo.

(12) Benoda

Inkampani: Yathuthukiswa yi-WuXi Junuo.

Isikhathi sokumaketha: Sigunyazwe ngokusemthethweni yi-NMPA ngoSepthemba 2021.

Izinkomba: Ukwelashwa kwe-B-cell lymphoma enkulu ebuyele emuva noma ephikisayo (r/r LBCL) ezigulini ezikhulile ngemuva komugqa wesibili noma ukwelashwa okuhlelekile okwengeziwe.

Ukuphawula: I-Benoda iwukwelapha ngofuzo olwa ne-CD19 CAR-T, futhi iphinde ibe umkhiqizo oyinhloko we-WuXi Junuo.Ingumkhiqizo wesibili we-CAR-T ogunyazwe e-China, ngaphandle kwe-B-cell lymphoma enkulu ephindekile/esabelayo.Ngaphezu kwalokho, i-WuXi Junuo ihlela nokwenza umjovo we-Ruiki Orenza wokwelapha ezinye izinkomba ezihlukahlukene, okuhlanganisa i-follicular lymphoma (FL), i-mantle cell lymphoma (MCL), i-chronic lymphocytic leukemia (CLL), yomugqa wesibili isakaza i-B-cell lymphoma enkulu (DLBCL) kanye ne-acute lymphoblastic leukemia (YONKE).

(13) I-CARYKTI

Inkampani: Umkhiqizo wokuqala ovunyelwe we-Legend Bio.

Isikhathi sokumaketha: Kugunyazwe yi-FDA ngoFebhuwari 2022.

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

Ukuphawula: I-CARVYKTI (i-ciltacabtagene autoleucel, ebizwa ngokuthi i-Cilta-cel) iwukwelapha ngofuzo lweseli ye-CAR-T enamasosha omzimba amabili esizinda esisodwa aqondise i-B cell maturation antigen (BCMA).Idatha ibonisa ukuthi i-CARVYKTI ibonise izinga lokuphendula eliphelele elifika ku-98% ezigulini ezine-myeloma eminingi ebuyele emuva noma ephikisayo eyayithole izindlela zokwelapha ezine noma ngaphezulu zangaphambili, okuhlanganisa ama-proteasome inhibitors, ama-immunomodulators, nama-anti-CD38 monoclonal antibodies.

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

(1) I-Gendicine/ukuzalwa kabusha

Inkampani: Ithuthukiswe yiNkampani ye-Shenzhen Saibainuo.

Isikhathi sokumaketha: Sigunyazwe ukufakwa ohlwini e-China ngo-2003.

Izinkomba: Ukwelashwa kwe-squamous cell carcinoma yekhanda nentamo.

Ukuphawula: Umjovo we-adenovirus we-Recombinant human p53 I-Gendicine/Jinshengsheng iwumuthi we-adenovirus vector gene therapy onamalungelo azimele empahla yengqondo ephethwe yi-Shenzhen Saibainuo Company.Umuthi wakhiwe uhlobo oluvamile lwe-tumor suppressor gene p53 kanye ne-artificially modified recombinant replication-deficint Human adenovirus type 5 yakhiwe uhlobo lwe-adenovirus yomuntu 5. Olokuqala luyisakhiwo esiyinhloko somuthi ukuze sibe nomphumela wokulwa nesimila, futhi lesi sakamuva sisebenza ikakhulukazi njengesithwali.I-adenovirus vector ithwala isakhi sokwelapha esingu-p53 iyise kuseli eliqondiwe, futhi iveze isakhi sokucindezela isimila p53 kuseli eliqondiwe.Umkhiqizo ungaphakamisa-ulawula izakhi zofuzo ezilwa nomdlavuza futhi wehlisele phansi imisebenzi yama-oncogenes ahlukahlukene, ngaleyo ndlela uthuthukise umphumela wokulwa nesimila womzimba futhi ufeze inhloso yokubulala izimila.

(2) I-Rigvir

Inkampani: Ithuthukiswe yinkampani yaseLatvia iLatima.

Isikhathi sokumaketha: Kuvunywe e-Latvia ngo-2004.

Izinkomba: Ukuze ekwelapheni melanoma.

Ukuphawula: I-Rigvir iwukwelapha ngofuzo okusekelwe ku-gene-modified enterovirus vector ye-ECHO-7, esetshenziswe e-Latvia, Estonia, Poland, Armenia, Belarus nakwezinye izindawo, futhi isabhaliswa ne-EMA ye-European Union..Amacala omtholampilo eminyakeni eyishumi edlule afakazele ukuthi igciwane le-Rigvir oncolytic liphephile futhi liyasebenza, futhi lingathuthukisa izinga lokusinda leziguli ze-melanoma izikhathi ezi-4-6.Ngaphezu kwalokho, ukwelashwa kulungele futhi ezinhlobonhlobo zeminye imidlavuza, okuhlanganisa umdlavuza we-colorectal, umdlavuza we-pancreatic, umdlavuza wesinye.umdlavuza, umdlavuza wezinso, umdlavuza wendlala yesinye, umdlavuza wamaphaphu, umdlavuza wesibeletho, i-lymphosarcoma, njll.

(3) I-Oncorine/Ankerui

Inkampani: Yathuthukiswa yi-Shanghai Sunway Biotechnology Co., Ltd.

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

Izinkomba: Ukwelashwa kwamathumba ekhanda nentamo, umdlavuza wesibindi, umdlavuza wepancreatic, umdlavuza wesibeletho kanye neminye imidlavuza.

Ukuphawula: I-Oncorine ingumkhiqizo we-oncolytic virus gene therapy usebenzisa i-adenovirus njenge-vector.I-adenovirus ye-oncolytic etholakalayo ingaphindaphinda ikakhulukazi kumathumba antula noma angajwayelekile ofuzo lwe-p53, okubangela i-tumor cell lysis, ngaleyo ndlela ibulale amaseli wesimila.ngaphandle kokulimaza amaseli avamile.Imiphumela yomtholampilo ibonisa ukuthi u-Anke Rui unokuphepha okuhle nokusebenza ngempumelelo kwezinhlobonhlobo zamathumba ayingozi.

(4) Glybera

Inkampani: Ithuthukiswe yi-uniQure.

Isikhathi sokumaketha: Kuvunywe eYurophu 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) umuthi wokwelapha ngofuzo osuselwe ku-AAV njengevekhtha.Lokhu kwelashwa kusebenzisa i-AAV njenge-vector ukudlulisa isakhi sofuzo sokwelapha i-LPL kumaseli emisipha, ukuze amangqamuzana ahambisanayo akwazi ukukhiqiza inani elithile le-lipoprotein lipase, Idlala indima ekudambiseni izifo, futhi lokhu kwelashwa kusebenza kahle isikhathi eside ngemva kokuphatha okukodwa (umphumela ungahlala iminyaka eminingi).Umuthi wasuswa ohlwini ngo-2017, futhi izizathu zokukhishwa kwawo ohlwini zingase zihlobane nezinto ezimbili: amanani entengo aphezulu kanye nesidingo esilinganiselwe semakethe.Izindleko ezimaphakathi zokwelashwa okukodwa komuthi ziphezulu njengesigidi esingu-1 samadola aseMelika, futhi sinye kuphela isiguli esisithengile futhi sasisebenzisa kuze kube manje.Yize inkampani yomshwalense wezokwelashwa iwubuyisele imali engu-900,000 US dollars, futhi kuwumthwalo omkhulu enkampanini yomshwalense.Ngaphezu kwalokho, inkomba yomuthi ayivamile kakhulu, ngezinga lezigameko cishe eli-1 esigidini esi-1 kanye nezinga eliphezulu lokungaxilongwa kahle.

(5) Imlygic

Inkampani: Yathuthukiswa ngabakwa-Amgen.

Isikhathi sokumaketha: Ngo-2015, sagunyazwa ukufakwa kuhlu e-United States nase-European Union.

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

Ukuphawula: I-Imlygic iwufuzo olushintshiwe (isusa izingcezu zayo zofuzo ze-ICP34.5 kanye ne-ICP47, futhi ifaka isakhi sofuzo se-granulocyte-macrophage colony-stimulating factor GM-CSF kugciwane) encishisiwe i-herpes simplex virus type 1 (HSV-1) oncolytic virus, i-FDA-approve virus yokuqala eyakhiwe ku-FDA-approve.Indlela yokuphatha umjovo we-intralesional.Umjovo oqondile ezilonda ze-melanoma ungabangela ukugqashuka kwamaseli wesimila futhi ukhulule ama-antigen atholakala ngesimila kanye ne-GM-CSF ukuze kuthuthukiswe ukusabela kwamasosha omzimba okulwa nesimila.

(6) Luxturna

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

Isikhathi sokumaketha: Kugunyazwe yi-FDA ngo-2017, kwase kugunyazwa ukumaketha eYurophu ngo-2018.

Izinkomba: Ezokwelashwa kwezingane kanye nabantu abadala abalahlekelwe umbono ngenxa yokuguqulwa kwekhophi ephindwe kabili yofuzo lwe-RPE65 kodwa enezinombolo ezanele zamaseli 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 ukuze anxephezele ukukhubazeka kweprotheni ye-RPE65 yesiguli, ngaleyo ndlela athuthukise umbono wesiguli.

(7) Zolgensma

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

Isikhathi sokumaketha: Kugunyazwe yi-FDA ngoMeyi 2019.

Izinkomba: Ukwelashwa kwe-spinal muscular atrophy (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.ikhasi, inqubekelaphambili eyingqophamlando.Lokhu kwelashwa kwezakhi zofuzo kusebenzisa i-scAAV9 vector ukwethula isakhi sofuzo esivamile se-SMN1 ezigulini ngokufakwa komthambo, okukhiqiza amaprotheni avamile e-SMN1, ngaleyo ndlela kuthuthukiswe ukusebenza kwamangqamuzana athintekile njengama-motor neurons.Ngokuphambene, izidakamizwa ze-SMA i-Spinraza ne-Evrysdi zidinga ukulinganisa okuphindaphindiwe isikhathi eside, kanti i-Spinraza ilawulwa njengomjovo womgogodla njalo ngemva kwezinyanga ezine, kanye ne-Evrysdi, isidakamizwa somlomo nsuku zonke.

(8) I-Delytact

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

Isikhathi sokumaketha: Ukugunyazwa okunemibandela okuvela eMnyangweni 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 ingumkhiqizo 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 ukuguqulwa okwengeziwe kokususwa ku-G207 genome ye-HSV-1 yesizukulwane sesibili, ithuthukisa ukuphindaphinda kwayo okukhethayo kumaseli omdlavuza kanye nokungeniswa kwezimpendulo zokuzivikela ezilwa nesimila, kuyilapho kugcinwa iphrofayili ephezulu yokuphepha.I-Delytact iyisizukulwane sokuqala sesithathu se-oncolytic HSV-1 okwamanje ekuhlolweni komtholampilo.Ukugunyazwa kwe-Delytact e-Japan bekusekelwe ocwaningweni lomtholampilo lweSigaba sesi-2 sengalo eyodwa.Ezigulini ezine-glioblastoma ephindaphindiwe, i-Delytact ihlangabezane nesiphetho sokuqala sokusinda konyaka owodwa, futhi imiphumela yabonisa ukuthi i-Delytact yenze kangcono kune-G207.Ukuphindaphinda okuqinile nomsebenzi ophezulu we-antitumor.Lokhu kusebenza kahle kumamodeli aqinile wesimila okuhlanganisa ibele, i-prostate, schwannoma, 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-EU ngoJulayi 2022.

Inkomba: Ngokushoda kwe-L-amino acid decarboxylase (AADC) enephunga elimnandi, egunyazwe ukwelashwa kweziguli ezinezinyanga eziyi-18 nangaphezulu.

Ukuphawula: I-Upstaza™ (eladocagene exuparvovec) iwukwelapha ngofuzo lwe-vivo kusetshenziswa i-adeno-associated virus type 2 (AAV2) njengevektha.Isiguli sinesifo ngenxa yokuguquka kofuzo olufaka ikhodi ye-enzyme ye-AADC.I-AAV2 iphethe isakhi sofuzo esinempilo esifaka i-enzyme ye-AADC.Umphumela wokwelapha utholakala ngendlela yesinxephezelo sofuzo.Ngokombono, umthamo owodwa usebenza isikhathi eside.Iwukwelashwa kofuzo kokuqala okudayiswayo okujovelwe ngqo ebuchosheni.Isigunyazo sokumaketha sisebenza kuwo wonke amazwe angamalungu e-EU angama-27, kanye ne-Iceland, i-Norway ne-Liechtenstein.

(9) Roctavian

Inkampani: Yathuthukiswa ngabakwa-BioMarin Pharmaceutical (BioMarin).

Isikhathi sokumaketha: Kugunyazwe yi-EU ngo-Agasti 2022.

Izinkomba: Ekwelapheni iziguli ezikhulile ezine-hemophilia A enzima ngaphandle komlando we-FVIII factor inhibition kanye ne-AAV5 antibody negative.

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 eight (FVIII) nesizinda esingu-B sisusiwe.Isinqumo se-European Commission sokugunyaza ukumakethwa kwe-valoctocogene roxaparvovec sisekelwe kuyo yonke idatha yohlelo lokuthuthukiswa komtholampilo lomuthi.Phakathi kwazo, isigaba sesi-III sesilingo somtholampilo i-GENEr8-1 sibonise ukuthi uma kuqhathaniswa nedatha yonyaka ngaphambi kokubhaliswa, ngemva kokujova okukodwa kwe-valoctocogene roxaparvovec, Izihloko zazinezinga lokuphuma kwegazi eliphansi kakhulu lonyaka (ABR), ukusetshenziswa kancane kancane kwe-recombinant factor VIII (F8) amalungiselelo amaprotheni, noma ukwanda okuphawulekayo komsebenzi we-F8 egazini lomzimba.Ngemva kwamaviki angu-4 okwelashwa, ukusetshenziswa konyaka kwe-F8 kwezifundo kanye ne-ABR edinga ukwelashwa kwehliswe ngo-99% no-84%, ngokulandelana, umehluko ophawulekayo wezibalo (p<0.001).Iphrofayili yokuphepha ibithandeka, zingekho izifundo ezibona i-F8 factor inhibition, ubuthi, noma imiphumela engemihle ye-thrombotic, futhi azikho izehlakalo ezimbi kakhulu (ama-SAE) ezihlobene nokwelashwa ezibikiwe.

3. Izidakamizwa ezincane ze-nucleic acid

(1) I-Vitravene

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

Isikhathi sokumaketha: Sigunyazwe yi-FDA kanye ne-EU EMA ngo-1998 nango-1999.

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

Ukuphawula: I-Vitravene iyisidakamizwa se-oligonucleotide ye-antisense kanye nesidakamizwa sokuqala se-oligonucleotide esivunyelwe ukumaketha emhlabeni.Ekuqaleni kwemakethe, isidingo semakethe sezidakamizwa ze-anti-cytomegalovirus sasiphuthuma kakhulu;khona-ke ngenxa yokuthuthukiswa kokwelashwa kwe-antiretroviral esebenzayo kakhulu, inani lamacala e-cytomegalovirus lehla kakhulu.Ngenxa yesidingo esiphansi semakethe, umuthi wakhululwa ngo-2002 nango-2006 Ukuhoxiswa emazweni e-EU nase-US.

(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 esishintshiwe se-pegylated esingakhomba futhi sibophe ku-vascular endothelial growth factor (VEGF165 isoform), futhi ilawulwa umjovo we-intravitreal.

(3) Defitelio

Inkampani: Ithuthukiswe yi-Jazz.

Isikhathi sokumaketha: Sigunyazwe yi-European Union ngo-2013, futhi sagunyazwa yi-FDA ngoMashi 2016.

Izinkomba: Ukwelashwa kwe-hepatic venule occlusive disease ehambisana nokungasebenzi kahle kwezinso noma i-pulmonary ngemva kokufakelwa kwe-hematopoietic stem cell.

Ukuphawula: I-Defitelio iyisidakamizwa se-oligonucleotide, ingxube ye-oligonucleotides enezakhiwo ze-plasmin.Yahoxiswa ngo-2009 ngenxa yezizathu zezohwebo.

(4) Kynamro

Inkampani: Ihlanganiswe ngu-Ionis Pharma noKastle.

Isikhathi sokumaketha: Sagunyazwa e-United States njengesidakamizwa sezintandane ngo-2013.

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

Ukuphawula: I-Kynamro iyisidakamizwa se-oligonucleotide esiphikisayo, i-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: Sigunyazwe yi-FDA ngoDisemba 2016.

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

Ukuphawula: I-Spinraza (nusinersen) iyisidakamizwa esilwa ne-oligonucleotide.I-Spinraza ingashintsha ukuhlanganisa kwe-RNA kofuzo lwe-SMN2 ngokubophezela kusayithi lokuhlanganisa le-SMN2 exon 7, ngaleyo ndlela ikhulise ukukhiqizwa kwephrotheni ye-SMN esebenza ngokugcwele.Ngo-Agasti 2016, i-BIOGEN Corporation yasebenzisa inketho yayo ukuze ithole amalungelo omhlaba we-Spinraza.I-Spinraza yaqala ukuhlolwa kwayo kokuqala komtholampilo kubantu ngo-2011. Eminyakeni engu-5 nje, yagunyazwa yi-FDA ngo-2016, okubonisa ukuqaphela okuphelele kwe-FDA ukusebenza kwayo.Umuthi ugunyazwe ukumakethwa e-China ngo-Ephreli 2019. Wonke umjikelezo wokugunyaza we-Spinraza e-China ungaphansi kwezinyanga ezingu-6.Sekuyiminyaka emi-2 nezinyanga ezi-2 kusukela i-Spinraza yagunyazwa okokuqala e-United States.Isifo esinjalo esingavamile se-blockbuster sangaphandle umuthi omusha ungaphakathi Ijubane lokufakwa kuhlu e-China selivele lishesha kakhulu.Lokhu futhi kungenxa yokuthi "Isaziso Sokukhishwa Kohlu Lokuqala Lwezidakamizwa Ezintsha Zaphesheya Ezidingeka Ngokuphuthumayo Ukuze Ucwaninge Ngomtholampilo" esikhishwe yiCentre for Drug Evaluation ngoNovemba 1, 2018, esifakwe kuqeqebana lokuqala lemithi emisha engu-40 ebalulekile yangaphandle ukuze ibuyekezwe ngokushesha, futhi i-Spinraza ikleliswe kuyo.

(6) Exondys 51

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

Isikhathi sokumaketha: Sigunyazwe yi-FDA ngoSepthemba 2016.

Izinkomba: Okokwelashwa kwe-Duchenne muscular dystrophy (DMD) ne-DMD gene mutation in exon 51 skipping gene.

Ukuphawula: I-Exondys 51 iyisidakamizwa se-oligonucleotide esiphikisayo.I-oligonucleotide ye-antisense ingabophezela ku-exon 51 isikhundla se-pre-mRNA yofuzo lwe-DMD, okuholela ekwakhekeni kwe-mRNA evuthiwe.I-Excision, ngaleyo ndlela ilungisa ingxenye yohlaka lokufunda lwe-mRNA, isiza isiguli ukuthi sihlanganise izinhlobo ezithile ezisebenzayo ze-dystrophin ezimfishane kuneprotheni evamile, ngaleyo ndlela kuthuthukiswe izimpawu zesiguli.

(7) Tegsedi

Inkampani: Ithuthukiswe ngabakwa-Ionis Pharmaceuticals.

Isikhathi sokumaketha: Kugunyazwe yi-European Union ukuze kumakethwe ngoJulayi 2018.

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

Ukuphawula: I-Tegsedi iyisidakamizwa se-oligonucleotide esiphikisayo esiqondise i-transthyretin mRNA.Umuthi wokuqala emhlabeni wonke ogunyazwe ukwelapha i-hATTR.Indlela yokuphatha umjovo we-subcutaneous.Umuthi wehlisa ukukhiqizwa kwephrotheni ye-ATTR ngokukhomba i-mRNA ye-transthyretin (ATTR), futhi unenani elihle lenzuzo engozini ekwelapheni i-ATTR.Asikho isigaba sesifo noma ubukhona be-cardiomyopathy obubalulekile.

(8) Onpattro

Inkampani: Ihlanganiswe yi-Alnylam kanye ne-Sanofi.

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

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

Ukuphawula: I-Onpattro iyisidakamizwa se-siRNA esiqondise ku-transthyretin mRNA, esinciphisa ukukhiqizwa kweprotheyini ye-ATTR esibindini kanye nokunqwabelana kwama-amyloid deposits ku-peripheral nerves ngokukhomba i-mRNA ye-transthyretin (ATTR)., ngaleyo ndlela kube ngcono futhi kudambise izimpawu zesifo.

(9) Givlaari

Inkampani: Ithuthukiswe yi-Alnylam Corporation.

Isikhathi sokumaketha: Kugunyazwe yi-FDA ngoNovemba 2019.

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

Ukuphawula: I-Givlaari umuthi we-siRNA, umuthi wesibili we-siRNA ogunyazwe ukumakethwa ngemuva kwe-Onpattro.Umuthi ulawulwa ngaphansi kwesikhumba futhi uqondise ku-mRNA ukuze kucekelwe phansi amaprotheni e-ALAS1.Ukwelashwa kwanyanga zonke kwe-Givlaari kunganciphisa ngokuphawulekayo nangokuqhubekayo izinga le-ALAS1 esibindini, ngaleyo ndlela kwehlise amazinga e-neurotoxic ALA ne-PBG aye ebangeni elivamile, ngaleyo ndlela Kwehlise izimpawu zesifo sesiguli.Idatha ibonise ukuthi iziguli eziphathwe nge-Givlaari ziye zancipha ngo-74% inani lezifo ezivuthayo uma kuqhathaniswa neqembu le-placebo.

(10) I-Vyondys53

Inkampani: Ithuthukiswe ngabakwaSarepta Therapeutics.

Isikhathi sokumaketha: Kugunyazwe yi-FDA ngoDisemba 2019.

Inkomba: Ukwelashwa kweziguli ze-DMD ezine-dystrophin gene exon 53 splice mutation.

Ukuphawula: I-Vyondys 53 iyisidakamizwa se-oligonucleotide esiphikisayo.Umuthi we-oligonucleotide uhlose inqubo yokuhlanganisa ye-dystrophin mRNA precursor.Enqubweni engaqondile ye-dystrophin mRNA precursor, i-Exon 53 yangaphandle yahlukaniswa ngokwengxenye, okusho ukuthi ayikho ku-mRNA ekhulile, futhi yayiklanyelwe ukukhiqiza iphrotheni ye-dystrophin encishisiwe kodwa esasebenzayo, ngaleyo ndlela ithuthukise amandla okuzivocavoca ezigulini.

(11) Waylivra

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

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

Inkomba: Njengokwelashwa okungeziwe ekudleni okulawulwayo ezigulini ezikhulile ezine-familia chylomicronemia syndrome (FCS).

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

(12) Leqvio

Inkampani: Yathuthukiswa ngabakwaNovartis.

Isikhathi sokumaketha: Kugunyazwe yi-EU ngoDisemba 2020.

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

Ukuphawula: I-Leqvio iyisidakamizwa se-siRNA esiqondise ku-PCSK9 mRNA.Iyindlela yokuqala emhlabeni yokwelapha yokwehlisa i-cholesterol (LDL-C) ye-siRNA.Indlela yokuphatha umjovo we-subcutaneous.Umuthi usebenza ngokuphazanyiswa kwe-RNA ukwehlisa amazinga e-PCSK9 amaprotheni, nawo ehlise amazinga e-LDL-C.Idatha yomtholampilo ikhombisa ukuthi i-Leqvio ingakwazi ukwehlisa i-LDL-C cishe ngama-50% ezigulini ezinamazinga e-LDL-C angeke ehliselwe kumazinga aqondiwe naphezu kwemithamo ephezulu ebekezelelwe yama-statins.

(13) Uxolo

Inkampani: Ithuthukiswe yi-Alnylam Pharmaceuticals.

Isikhathi sokumaketha: Kugunyazwe yi-EU ngoNovemba 2020.

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

Ukuphawula: I-Oxlumo umuthi we-siRNA oqondise i-hydroxy acid oxidase 1 (HAO1) ​​mRNA, elawulwa ngaphansi kwesikhumba.Umuthi wasungulwa kusetshenziswa ikhemikhali ye-Alnylam yokuzinzisa yakamuva ethuthukisiwe ye-ESC-GalNAc, eyenza ama-siRNA alawulwa ngaphansi kwesikhumba akwazi ukuphikelela okukhulu nokusebenza ngempumelelo.Umuthi uhlose ukucekelwa phansi noma ukuvinjwa kwe-hydroxy acid oxidase 1 (HAO1) ​​mRNA, wehlisa izinga le-glycolate oxidase esibindini, bese usebenzisa i-substrate edingekayo ukuze kukhiqizwe i-oxalate futhi wehlise ukukhiqizwa kwe-oxalate ukulawula ukuqhubekela phambili kwesifo nokuthuthukisa izimpawu zesifo ezigulini.

(14) I-Viltepso

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

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

Izinkomba: Okokwelashwa kwe-Duchenne muscular dystrophy (DMD) ne-DMD gene mutation in exon 53 skipping gene.

Ukuphawula: I-Viltepso iyisidakamizwa se-phosphorodiamide morpholino oligonucleotide.Lesi sidakamizwa se-oligonucleotide singabophezela ku-exon 53 isikhundla se-pre-mRNA yofuzo lwe-DMD, okuholela ekwakhekeni kwe-mRNA evuthiwe.I-exon isuswa kancane, ngaleyo ndlela ilungise ingxenye yohlaka lokufunda lwe-mRNA, kusize isiguli ukuthi sihlanganise izinhlobo ezithile ezisebenzayo ze-dystrophin ezimfushane kuneprotheni evamile, ngaleyo ndlela kuthuthukiswe izimpawu zesiguli.

(15) Amvuttra (vutrisiran)

Inkampani: Ithuthukiswe yi-Alnylam Pharmaceuticals.

Isikhathi sokumaketha: Kugunyazwe yi-FDA ngoJuni 2022.

Izinkomba: Ezokwelapha i-transthyretin amyloidosis yofuzo yabantu abadala ene-polyneuropathy (hATTR-PN).

Ukuphawula: I-Amvuttra (Vutrisiran) umuthi we-siRNA oqondise i-transthyretin (ATTR) mRNA, elawulwa ngomjovo ongaphansi kwesikhumba.I-Vutrisiran yakhelwe ngokusekelwe ku-Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc conjugated delivery platform with potency and metabolic stability.Ukugunyazwa kokwelashwa kusekelwe emininingwaneni yezinyanga eziyi-9 yocwaningo lwayo lomtholampilo lweSigaba sesi-III (HELIOS-A), okunemiphumela isiyonke ebonisa ukuthi ukwelapha kwathuthukisa izimpawu ze-hATTR-PN, ngaphezu kuka-50% weziguli ezihlehlisa noma ezimisa ukuqhubeka.

4. Eminye imithi yokwelapha ngofuzo

(1) I-Rexin-G

Inkampani: Yathuthukiswa ngabakwa-Epeius Biotech.

Isikhathi sokumaketha: Sivunywe yiPhilippine Food and Drug Administration (BFAD) ngo-2005.

Izinkomba: Ezokwelashwa komdlavuza oseqophelweni eliphezulu ongazweli ku-chemotherapy.

Ukuphawula: I-Rexin-G iwumjovo we-nanoparticle ogcwele isakhi sofuzo.Yethula isakhi sofuzo esiguquguqukayo se-cyclin G1 kumaseli okuqondiwe kusetshenziswa i-retroviral vector ukuze kubulawe izimila eziqinile.Indlela yokuphatha ukumnika nge-intravenous.Njengomuthi ohloselwe isimila ofuna futhi ucekele phansi amangqamuzana omdlavuza we-metastatic, unomphumela othile ezigulini ezingasebenzi ngokumelene neminye imithi yomdlavuza, kuhlanganise ne-biologics ehlosiwe.

(2) Neovasculgen

Inkampani: Ithuthukiswe yi-Human stem cell Institute.

Isikhathi sokufakwa kuhlu: Kugunyazwe ukufakwa kuhlu e-Russia ngo-December 7, 2011, kwase kufakwa ohlwini e-Ukraine ngo-2013.

Izinkomba: Ukuze ukwelashwa kwe-peripheral arterial disease, kuhlanganise ne-ischemia enzima yemilenze.

Ukuphawula: I-Neovasculgen iyi-DNA plasmid-based gene therapy lapho isakhi sofuzo se-vascular endothelial growth factor (VEGF) 165 sakhiwe ngomgogodla we-plasmid futhi sifakwe ezigulini.

(3) I-Collategene

Inkampani: Ithuthukiswe i-Osaka University kanye namafemu emali eyibhizinisi.

Isikhathi sokufakwa kuhlu: Sigunyazwe Umnyango Wezempilo, Wezabasebenzi Nenhlalakahle Yase-Japan ukuze sifakwe kuhlu ngo-Agasti 2019.

Izinkomba: Ukwelashwa kwe-ischemia ephansi kakhulu yomkhawulo ophansi.

Ukuphawula: I-Collategene iwukwelapha ngofuzo olususelwa ku-plasmid, umuthi wokuqala womdabu waseJapan wokwelapha ngofuzo okhiqizwe yi-AnGes.Ingxenye eyinhloko yalesi sidakamizwa i-plasmid enqunu equkethe ukulandelana kofuzo lwe-human hepatocyte growth factor (HGF).Uma umuthi ujovwa emisipha yemilenze engezansi, i-HGF eveziwe izokhuthaza ukwakheka kwemithambo yegazi emisha ezungeze imithambo yegazi evalekile.Izivivinyo zemitholampilo zikuqinisekisile ukusebenza kahle kwayo ekwenzeni ngcono izilonda.

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