Majini ndi mayunitsi oyambira omwe amawongolera mawonekedwe.Kupatulapo majini a mavairasi ena, omwe amapangidwa ndi RNA, majini a zamoyo zambiri amapangidwa ndi DNA..Matenda ambiri a zamoyo amayamba chifukwa cha kugwirizana kwa majini ndi chilengedwe.Thandizo la majini limatha kuchiritsa kapena kuchepetsa matenda ambiri.Gene therapy imatengedwa ngati kusintha pazamankhwala ndi mankhwala.Mankhwala ochizira ma gene m'lingaliro lalikulu amaphatikizapo kutengera mankhwala a DNA osinthidwa a DNA (monga mu vivo gene therapy mankhwala opangidwa ndi ma virus, in vitro gene therapy drugs, maliseche a plasmid, etc.) ndi mankhwala a RNA (monga antisense oligonucleotide drugs, siRNA drugs, ndi mRNA gene therapy, etc.);Mankhwala ochepetsa mphamvu ya Gene makamaka amaphatikiza mankhwala a plasmid DNA, mankhwala ochizira ma jini otengera ma virus, mankhwala opangira ma jini otengera mabakiteriya, makina osintha ma gene, ndi mankhwala ochizira ma cell osinthira jini mu vitro.Pambuyo pazaka zambiri zachitukuko chovutitsa, mankhwala a gene therapy apeza zotsatira zolimbikitsa zachipatala.(Osawerengera katemera wa DNA ndi katemera wa mRNA) Pakali pano, mankhwala 45 ochizira majini avomerezedwa kuti azigulitsidwa padziko lonse lapansi.Ma gene 9 ochiritsira avomerezedwa kuti azigulitsidwa chaka chino, kuphatikiza ma gene 7 ochiritsira omwe adavomerezedwa kuti atsatidwe koyamba chaka chino, omwe ndi: CARVYKTI, Amvuttra, Upstaza, Roctavian, Hemgenix, Adstiladrin ndi Ebvallo, (Zindikirani: Ena awiriwo adavomerezedwa ku United States chaka chino. Gulu loyamba lovomerezedwa ndi ① generapies ① FDA yotsatsa malonda ku United States mu Ogasiti 2022, ndipo idavomerezedwa kuti igulitsidwe ndi European Union mu 2019; .) Ndi kukhazikitsidwa kwazinthu zambiri zopangira ma jini komanso kukula mwachangu kwaukadaulo wa gene therapy, chithandizo cha majini chatsala pang'ono kuyambitsa nyengo yachitukuko chofulumira.

Gulu la gene therapy (Chitsime cha zithunzi: Bio-Matrix)

Nkhaniyi yatchula mitundu 45 yamankhwala ochizira majini (kupatulapo katemera wa DNA ndi katemera wa mRNA) omwe avomerezedwa kuti azigulitsidwa.

1. Chithandizo cha majini mu m'galasi

(1) Strimvelis

Kampani: Yopangidwa ndi GlaxoSmithKline (GSK).

Nthawi yogulitsa: Idavomerezedwa kuti igulidwe ndi European Union mu Meyi 2016.

Zochizira kwambiri kuphatikiza immunodeficiency (SCID).

Ndemanga: Njira yayikulu ya mankhwalawa ndikuyamba kulandira maselo amtundu wa hematopoietic wa wodwalayo, kuwakulitsa ndi kuwakulitsa mu vitro, kenako kugwiritsa ntchito retrovirus kuyambitsa jini yogwira ntchito ya ADA (adenosine deaminase) m'maselo a hematopoietic tsinde, ndipo pomaliza kubaya ma cell osinthidwa a Hematopoietic amabwezeretsedwanso m'thupi.Zotsatira zachipatala zikuwonetsa kuti kupulumuka kwazaka zitatu kwa odwala ADA-SCID omwe amathandizidwa ndi Strimvelis ndi 100%.

(2) Zalmoxis

Kampani: Yopangidwa ndi Italy MolMed Company.

Nthawi Yogulitsa: Adalandira chilolezo chotsatsa kuchokera ku European Union mu 2016.

Iwo ntchito adjuvant mankhwala a chitetezo cha m`thupi odwala pambuyo hematopoietic tsinde cell kupatsidwa zina.

Ndemanga: Zalmoxis ndi allogeneic T cell suicide gene immunotherapy yosinthidwa ndi ma vector a retroviral.Njirayi imagwiritsa ntchito tizilombo toyambitsa matenda kuti tizilombo toyambitsa matenda tisinthe ma cell a allogeneic T, kotero kuti maselo a T osinthidwa kufotokozera 1NGFR ndi HSV-TK Mut2 majini odzipha amalola anthu kugwiritsa ntchito mankhwala a ganciclovir (ganciclovir) nthawi iliyonse kuti aphe maselo a T omwe amachititsa kuti chitetezo chitetezeke, kuteteza kuwonongeka kwina kwa chitetezo cha GVHD, komanso kupereka chitetezo cha GVHD kwa odwala omwe ali ndi GVHD.

(3) Invossa-K

Kampani: Yopangidwa ndi TissueGene (KolonTissueGene).

Nthawi yogulitsa: Zavomerezedwa kuti zilembedwe ku South Korea mu Julayi 2017.

Zochizira osachiritsika bondo nyamakazi.

Ndemanga: Invossa-K ndi allogeneic cell gene therapy yomwe imaphatikizapo ma chondrocyte aumunthu.Maselo a allogeneic amasinthidwa mwachibadwa mu vitro, ndipo maselo osinthidwa amatha kufotokoza ndi kutulutsa kusintha kwa kukula kwa β1 (TGF-β1) pambuyo pa jekeseni wa intra-articular.β1), potero kukonza zizindikiro za osteoarthritis.Zotsatira zachipatala zikuwonetsa kuti Invossa-K imatha kusintha kwambiri nyamakazi ya mawondo.Idathetsedwa mu 2019 ndi Korea Food and Drug Administration chifukwa wopanga adalemba molakwika zosakaniza zomwe zimagwiritsidwa ntchito.

(4) Zynteglo

Kampani: Yofufuzidwa ndikupangidwa ndi bluebird bio.

Nthawi yogulitsa: Yovomerezedwa ndi European Union kuti igulitsidwe mu 2019, ndikuvomerezedwa ndi FDA kuti igulitsidwe ku United States mu Ogasiti 2022.

Zochizira β-thalassemia yodalira magazi.

Ndemanga: Zynteglo ndi lentiviral in vitro gene therapy yomwe imayambitsa kopi yogwira ntchito ya jini ya β-globin (βA-T87Q-globin gene) m'maselo amtundu wa hematopoietic omwe amatengedwa kuchokera kwa wodwalayo kudzera mu vector ya lentiviral, kenaka amabwezeretsanso ma genetics osinthika a autologous autologous m'maselo amtundu wa odwala.Wodwalayo akakhala ndi jini yachibadwa ya βA-T87Q-globin, amatha kupanga mapuloteni abwinobwino a HbAT87Q, omwe amatha kuchepetsa kapena kuthetsa kufunikira kwa kuikidwa magazi.Ndi chithandizo chanthawi imodzi chomwe chimapangidwa kuti chilowe m'malo mwa kuthiridwa magazi kwa moyo wonse komanso mankhwala amoyo wonse kwa odwala azaka 12 kapena kuposerapo.

(5) Skysona

Kampani: Yofufuzidwa ndikupangidwa ndi bluebird bio.

Nthawi yogulitsa: Adavomerezedwa ndi European Union kuti azitha kutsatsa mu Julayi 2021, ndikuvomerezedwa ndi FDA kuti azitha kutsatsa ku United States mu Seputembala 2022.

Zochizira oyambirira cerebral adrenoleukodystrophy (CALD).

Ndemanga: Chithandizo cha majini cha Skysona ndi njira yokhayo yokhayo yovomerezeka yochizira matenda a cerebral adrenoleukodystrophy (CALD) a nthawi imodzi.Skysona (elivaldogene autotemcel, Lenti-D) ndi hematopoietic stem cell lentiviral in vitro gene therapy Lenti-D.Njira yonse ya mankhwalawa ndi motere: maselo amtundu wa autologous hematopoietic amachotsedwa kwa wodwalayo, kusinthidwa ndi kusinthidwa ndi lentivirus yomwe imanyamula jini ya ABCD1 yaumunthu mu m'galasi, ndikubwezeretsanso kwa wodwalayo.Amagwiritsidwa ntchito pochiza odwala osakwana zaka 18, atanyamula ABCD1 gene mutations, ndi CALD.

(6) Chimiriya

COMPANY: Yopangidwa ndi Novartis.

Nthawi Yogulitsa: Yavomerezedwa ndi FDA kuti igulitse mu Ogasiti 2017.

Chithandizo cha kalambulabwalo B-cell acute lymphoblastic leukemia (ALL) ndi kubwereranso ndi refractory DLBCL.

Ndemanga: Kymriah ndi mankhwala a lentiviral in vitro gene therapy, mankhwala oyamba a CAR-T ovomerezedwa kuti azitha kutsatsa padziko lonse lapansi, akulunjika ku CD19, ndikugwiritsa ntchito 4-1BB co-stimulatory factor.Ndi mtengo wa $475,000 ku US ndi $313,000 ku Japan.

(7) Yescarta

Kampani: Yopangidwa ndi Kite Pharma, nthambi ya Gileadi (GILD).

Nthawi yogulitsa: Yovomerezedwa ndi FDA kuti igulitse mu October 2017;Fosun Kite adayambitsa ukadaulo wa Yescarta kuchokera ku Kite Pharma ndikuupanga ku China atalandira chilolezo.Zavomerezedwa kuti zilembedwe m'dziko.

Zochizira anayambiranso kapena refractory lalikulu B-maselo lymphoma.

Ndemanga: Yescarta ndi retroviral in vitro gene therapy, yomwe ndi yachiwiri yovomerezeka ya CAR-T padziko lonse lapansi.Imayang'ana CD19 ndipo imagwiritsa ntchito makina opangira CD28.Ndi mtengo wa $373,000 ku United States.

(8) Tecartus

Kampani: Yopangidwa ndi Gileadi (GILD).

Nthawi yogulitsa: Yovomerezedwa ndi FDA kuti igulitsidwe mu Julayi 2020.

Kwa kubwereranso kapena refractory mantle cell lymphoma.

Ndemanga: Tecartus ndi autologous CAR-T cell therapy yolunjika ku CD19, ndipo ndi mankhwala achitatu a CAR-T omwe amavomerezedwa kuti azitsatsa padziko lonse lapansi.

(9) Breyanzi

COMPANY: Yopangidwa ndi Bristol-Myers Squibb (BMS).

Nthawi yogulitsa: Yovomerezedwa ndi FDA kuti igulitsidwe mu February 2021.

Zizindikiro: Kubwereranso kapena kukana (R / R) lalikulu B-cell lymphoma (LBCL).

Ndemanga: Breyanzi ndi in vitro gene therapy yotengera lentivirus, chithandizo chachinayi cha CAR-T chovomerezedwa kuti chizigulitsidwa padziko lonse lapansi, cholunjika ku CD19.Kuvomerezedwa kwa Breyanzi ndichinthu chofunikira kwambiri kwa Bristol-Myers Squibb pankhani ya ma cell immunotherapy, omwe adapeza atapeza Celgene kwa $74 biliyoni mu 2019.

(10) Abecma

Kampani: Co-yopangidwa ndi Bristol-Myers Squibb (BMS) ndi bluebird bio.

Nthawi yogulitsa: Yovomerezedwa ndi FDA kuti igulitse mu Marichi 2021.

Zizindikiro: kubwereranso kapena refractory angapo myeloma.

Ndemanga: Abecma ndi mankhwala amtundu wa in vitro otengera lentivirus, chithandizo choyamba chapadziko lonse lapansi cha CAR-T cholunjika ku BCMA, komanso chithandizo chachisanu cha CAR-T chovomerezedwa ndi FDA.Mfundo ya mankhwala ndi kufotokoza chimeric BCMA zolandilira pa wodwala T maselo kudzera lentivirus-mediated jini kusinthidwa m'galasi.Chithandizo chochotsa maselo a T omwe sanasinthidwe mwa odwala, ndikubwezeretsanso maselo a T osinthidwa, omwe amafunafuna ndi kupha maselo a khansa omwe amawonetsa BCMA mwa odwala.

(11) Libmeldy

COMPANY: Yopangidwa ndi Orchard Therapeutics.

Nthawi Yogulitsa: Yovomerezedwa ndi European Union kuti ilembedwe mu Disembala 2020.

Zochizira metachromatic leukodystrophy (MLD).

Ndemanga: Libmeldy ndi chithandizo cha majini chotengera CD34+ cell autologous genetic modified in vitro by lentivirus.Deta yachipatala imasonyeza kuti kulowetsedwa kwa mtsempha wa Libmeldy kungathe kusintha bwino njira yoyambilira ya MLD poyerekeza ndi kuwonongeka kwakukulu kwa galimoto ndi chidziwitso kwa odwala omwe sanalandire chithandizo cha msinkhu womwewo.

(12) Benoda

Kampani: Yopangidwa ndi WuXi Giant Nuo.

Nthawi yogulitsa: Yavomerezedwa ndi NMPA mu Seputembara 2021.

Zizindikiro: Chithandizo cha odwala akuluakulu omwe akubwereranso kapena kukana B-cell lymphoma (r / r LBCL) pambuyo pa mzere wachiwiri kapena pamwamba pa mankhwala ochiritsira.

Ndemanga: Beinoda ndi anti-CD19 CAR-T gene therapy, komanso ndiye chinthu chachikulu cha WuXi Juro Company.Ndi mankhwala achiwiri a CAR-T omwe amavomerezedwa ku China, kupatulapo B-cell lymphoid yaikulu ya refractory WuXi Giant Nuo akukonzekeranso kupanga jekeseni wa Regiorensai pochiza zizindikiro zina zambiri, kuphatikizapo follicular lymphoma (FL), mantle cell lymphoma (MCL), chronic lymphocytic leukemia (CLL), lymphocytic leukemia (CLL), yachiwiri ya lymphocytic lymphoid lymphoma (DLALL lymphoma lymphoma)

(13) CARVYKTI

Kampani: Chogulitsa choyamba cha Legend Biotech chovomerezeka kutsatsa.

Nthawi yogulitsa: Yovomerezedwa ndi FDA kuti igulitsidwe mu February 2022.

zochizira anayambiranso kapena refractory angapo myeloma (R/R MM).

Ndemanga: CARVYKTI (ciltacabtagene autoleucel, Cilta-cel mwachidule) ndi CAR-T cell immune gene therapy yokhala ndi ma antibodies awiri a domain single omwe akulunjika ku B-cell maturation antigen (BCMA).Deta imasonyeza kuti CARVYKTI Odwala omwe ali ndi myeloma yobwerezabwereza kapena yotsutsa omwe adalandira chithandizo chamankhwala anayi kapena kuposerapo (kuphatikizapo proteasome inhibitors, immunomodulators ndi anti-CD38 monoclonal antibodies), chiwerengero chonse cha 98% chawonetsedwa.

(14)Embala

COMPANY: Yopangidwa ndi Atara Biotherapeutics.

European Commission (EC) yotsatsa mu Disembala 2022, ndi chithandizo choyamba chapadziko lonse lapansi chapadziko lonse lapansi cha T cell chovomerezeka kuti chizigulitsidwa.

Monga monotherapy ya Epstein-Barr virus (EBV) yokhudzana ndi post-transplantation lymphoproliferative disease (EBV+PTLD), odwala omwe akulandira chithandizo ayenera kukhala akuluakulu ndi ana opitirira zaka 2 omwe adalandirapo kale mankhwala amodzi.

Ndemanga: Ebvallo ndi allogeneic EBV-specific universal T-cell gene therapy yomwe imayang'ana ndikuchotsa ma cell omwe ali ndi EBV m'njira yoletsedwa ndi HLA.Kuvomerezeka kwa mankhwalawa kumatengera zotsatira za phunziro lofunika kwambiri la chipatala la gawo 3, ndipo zotsatira zake zinasonyeza kuti ORR ya gulu la HCT ndi gulu la SOT linali 50%.Chiwerengero cha kukhululukidwa kwathunthu (CR) chinali 26.3%, chiwerengero cha kukhululukidwa pang'ono (PR) chinali 23.7%, ndipo nthawi yapakatikati ya kukhululukidwa (TTR) inali miyezi 1.1.Mwa odwala 19 omwe adalandira chikhululukiro, 11 anali ndi nthawi yoyankha (DOR) yopitilira miyezi isanu ndi umodzi.Kuphatikiza apo, pankhani ya chitetezo, palibe zoyipa zomwe zidachitika monga matenda a graft-versus-host (GvHD) kapena matenda okhudzana ndi cytokine okhudzana ndi Ebvallo omwe adachitika.

2. Mu vivo gene therapy yotengera ma vector a virus

(1) Gendicine/Jin Sheng

Kampani: Yopangidwa ndi Shenzhen Saibainuo Company.

Nthawi Yogulitsa: Zavomerezedwa kuti zilembedwe ku China mu 2003.

Zochizira mutu ndi khosi squamous cell carcinoma.

Zindikirani: Recombinant human p53 adenovirus injection Gendicine/Jinyousheng ndi mankhwala a adenovirus vector gene therapy omwe ali ndi ufulu wodziyimira pawokha waumwini wa kampani ya Shenzhen Saibainuo.Anthu amtundu wa 5 adenovirus amapangidwa ndi mtundu wa adenovirus wamtundu wa 5. Wakale ndi dongosolo lalikulu la anti-chotupa zotsatira za mankhwala, ndipo chotsatiracho chimakhala ngati chonyamulira.The adenovirus vekitala amanyamula achire jini p53 mu chandamale selo, kufotokoza chotupa suppressor jini p53 mu chandamale selo, ndi jini mawu The mankhwala akhoza mmwamba-regulating zosiyanasiyana odana ndi khansa majini ndi pansi-kulamulira ntchito zosiyanasiyana oncogenes, potero kupititsa patsogolo cholinga cha thupi ndi kupha cholinga chotupa kuchepetsa chotupa.

(2) Mwadzidzidzi

Kampani: Yopangidwa ndi Latima Company, Latvia.

Nthawi yolembetsa: Adavomerezedwa kuti alembetse ku Latvia mu 2004.

Zochizira melanoma.

Ndemanga: Rigvir ndi chithandizo cha majini chozikidwa pa chibadwa cha ECHO-7 enterovirus vector.Pakalipano, mankhwalawa adatengedwa ku Latvia, Estonia, Poland, Armenia, Belarus, ndi zina zotero, ndipo akulembetsanso EMA m'mayiko a EU.Zachipatala milandu m'zaka khumi zapitazi zatsimikizira kuti Rigvir oncolytic HIV ndi otetezeka ndi ogwira, ndipo akhoza kuonjezera kupulumuka kwa odwala khansa ya pakhungu ndi 4-6 zina.Kuphatikiza apo, mankhwalawa amagwiranso ntchito pamakhansa ena osiyanasiyana, kuphatikiza khansa yapakhungu, khansa ya m'mimba, khansa ya chikhodzodzo, khansa ya impso, khansa ya prostate, khansa ya m'mapapo, khansa ya uterine, lymphosarcoma, ndi zina zambiri.

(3) Okonda

Kampani: Yopangidwa ndi Shanghai Sanwei Biological Company.

Nthawi Yogulitsa: Zavomerezedwa kuti zilembedwe ku China mu 2005.

Zizindikiro: mankhwala a mutu ndi khosi zotupa, khansa ya chiwindi, kapamba khansa, khomo pachibelekeropo khansa ndi zina khansa.

Ndemanga: Oncorine (安科瑞) ndi mankhwala a oncolytic virus gene therapy pogwiritsa ntchito adenovirus ngati chonyamulira.Oncolytic adenovirus imapezedwa, yomwe imatha kubwereza mwatsatanetsatane mu zotupa za p53 zoperewera kapena zachilendo, zomwe zimatsogolera ku lysis ya ma cell chotupa, potero kupha ma cell chotupa.popanda kuwononga maselo abwinobwino.Kafukufuku wachipatala awonetsa kuti Ankerui ali ndi chitetezo chabwino komanso chothandiza pamitundu yosiyanasiyana ya zotupa zowopsa.

(4) Glybera

Kampani: Yopangidwa ndi UniQure.

Nthawi Yogulitsa: Zavomerezedwa kuti zilembedwe ku Europe mu 2012.

Zizindikiro: Chithandizo cha kusowa kwa lipoprotein lipase (LPLD) komwe kumakhala kowopsa kapena kobwerezabwereza kwa kapamba ngakhale kudya zakudya zamafuta ochepa.

Ndemanga: Glybera (alipogene tiparvovec) ndi mankhwala a jini opangidwa ndi AAV, omwe amagwiritsa ntchito AAV monga chonyamulira kuti asandutse jini yachirengedwe ya LPL m'maselo a minofu, kotero kuti maselo ogwirizana amatha kupanga kuchuluka kwa lipoprotein lipase, Kuti achepetse matendawa, mankhwalawa ndi othandiza kwa nthawi yaitali (mankhwala osokoneza bongo) amatha kutha kwa zaka zambiri.Mankhwalawa adachotsedwa pamsika mu 2017. Chifukwa chake chikhoza kukhala chokhudzana ndi zinthu ziwiri: mitengo yamtengo wapatali komanso kuchepa kwa msika.Avereji ya mtengo wamankhwala wa mankhwalawa ndi wokwera kwambiri mpaka US$1 miliyoni, ndipo wodwala m'modzi yekha ndiye adagula ndikuigwiritsa ntchito mpaka pano.Ngakhale kampani ya inshuwaransi yachipatala yabwezera US $ 900,000, ndizovuta kwambiri kwa kampani ya inshuwaransi.Kuonjezera apo, zizindikiro zomwe zimagwiritsidwa ntchito ndi mankhwalawa ndizochepa kwambiri, zomwe zimachitika pafupifupi 1 mwa 1 miliyoni komanso kuchuluka kwa matenda olakwika.

(5) Imlygic

Kampani: Yopangidwa ndi Amgen.

Nthawi yogulitsa: Mu 2015, idavomerezedwa kuti ilembedwe ku United States ndi European Union.

Zizindikiro: Chithandizo cha zotupa za melanoma zomwe sizingachotsedwe kwathunthu ndi opaleshoni.

Ndemanga: Imlygic ndi attenuated herpes simplex virus type 1 yomwe yasinthidwa ndi ukadaulo wa majini (kuchotsa zidutswa zake za jini za ICP34.5 ndi ICP47, ndikuyika jini ya granulocyte macrophage colony-stimulating factor GM-CSF mu kachilomboka) (HSV-1) kachilombo ka oncolytic-approve kachirombo ka FDA koyamba pa FDA.Njira yoyendetsera ndi jekeseni wa intralesional, womwe ukhoza kulowetsedwa mwachindunji ku zotupa za melanoma kuti zipangitse kupasuka kwa maselo otupa, kutulutsa ma antigen opangidwa ndi chotupa ndi GM-CSF, ndikulimbikitsanso kuyankha kwa chitetezo cha mthupi.

(6) Luxturna

Kampani: Yopangidwa ndi Spark Therapeutics, wothandizidwa ndi Roche.

Nthawi yogulitsa: Idavomerezedwa kuti igulidwe ndi FDA mu 2017, kenako idavomerezedwa kuti igulidwe ku Europe mu 2018.

Zochizira ana ndi akulu amene ataya masomphenya chifukwa chachiwiri-kope RPE65 jini masinthidwe koma kusunga okwanira chiwerengero cha yotheka retina maselo.

Ndemanga: Luxturna ndi mankhwala amtundu wa AAV omwe amayendetsedwa ndi jakisoni wa subretinal.The gene therapy imagwiritsa ntchito AAV2 monga chonyamulira kuti adziwitse kopi yogwira ntchito ya jini ya RPE65 yachibadwa m'maselo a retina a wodwalayo, kotero kuti maselo ogwirizanawo awonetsere mapuloteni abwino a RPE65, omwe amapanga kusowa kwa mapuloteni a RPE65, motero kusintha masomphenya a wodwalayo.

(7) Zolgensma

Kampani: Yopangidwa ndi AveXis, wothandizira wa Novartis.

Nthawi yogulitsa: Yovomerezedwa ndi FDA kuti igulitse mu Meyi 2019.

Zizindikiro: Chithandizo cha Spinal Muscular Atrophy (SMA) odwala osakwana zaka 2.

Ndemanga: Zolgensma ndi chithandizo cha majini chotengera AAV vector.Mankhwalawa ndi njira yokhayo yothandizira nthawi imodzi ya spinal muscular atrophy yovomerezeka kuti igulitsidwe padziko lonse lapansi.Kukhazikitsidwa kwa mankhwalawa kumatsegula nthawi yatsopano pochiza matenda a msana.tsamba, ndikupita patsogolo.Chithandizo cha jinichi chimagwiritsa ntchito vekitala ya scAAV9 kuti ipangitse jini yabwinobwino ya SMN1 mwa wodwalayo kudzera m'mitsempha kuti ipange mapuloteni abwinobwino a SMN1, potero kumathandizira magwiridwe antchito a maselo okhudzidwa monga ma neuroni agalimoto.Mosiyana ndi izi, mankhwala a SMA Spinraza ndi Evrysdi amafuna kubwereza mobwerezabwereza.Spinraza amaperekedwa ndi jekeseni wa msana miyezi inayi iliyonse, ndipo Evrysdi ndi mankhwala apakamwa tsiku ndi tsiku.

(8) Kusokonezeka

Kampani: Yopangidwa ndi Daiichi Sankyo Company Limited (TYO: 4568).

Nthawi yopita kumsika: Chivomerezo chokhazikika kuchokera ku Unduna wa Zaumoyo, Ntchito ndi Zaumoyo ku Japan (MHLW) mu Juni 2021.

Zochizira malignant glioma.

Ndemanga: Delytact ndi chinthu chachinayi cha oncolytic virus gene therapy chovomerezeka padziko lonse lapansi, komanso chida choyamba cha virus cha oncolytic chovomerezeka kuchiza glioma yoyipa.Delytact ndi kachilombo ka herpes simplex virus mtundu 1 (HSV-1) oncolytic yopangidwa ndi Dr. Todo ndi anzake.Delytact imayambitsa zosintha zina zochotsa mu G207 genome ya m'badwo wachiwiri wa HSV-1, kupititsa patsogolo kasankhidwe kake m'maselo a khansa komanso kulowetsedwa kwa mayankho a anti-tumor immune immune ndikusunga chitetezo chokwanira.Delytact ndiye m'badwo woyamba wachitatu wa oncolytic HSV-1 womwe ukuwunikiridwa pano.Kuvomerezedwa kwa Delytact ku Japan makamaka kumachokera ku kuyesa kwachipatala kwa gawo limodzi la mkono umodzi.Odwala omwe ali ndi glioblastoma yobwereza, Delytact adakwanitsa kupulumuka kwa chaka chimodzi, ndipo zotsatira zake zidawonetsa kuti Delytact idawonetsa kuchita bwino poyerekeza ndi G207.Mphamvu yamphamvu yobwerezabwereza komanso ntchito yayikulu ya antitumor.Izi zinali zogwira mtima m'mitundu yolimba ya zotupa za bere, prostate, schwannomas, nasopharyngeal, hepatocellular, colorectal, zotupa zotumphukira zamitsempha zam'mitsempha, ndi khansa ya chithokomiro.

(9) Zosangalatsa

COMPANY: Yopangidwa ndi PTC Therapeutics, Inc. (NASDAQ: PTCT).

Nthawi yopita kumsika: Yovomerezedwa ndi European Union kuti igulitsidwe mu Julayi 2022.

Zizindikiro: Pakuchepa kwa L-amino acid decarboxylase (AADC) wonunkhira, amavomerezedwa kuti azichiza odwala omwe ali ndi miyezi 18 kapena kuposerapo.

Ndemanga: Upstaza™ (eladocagene exuparvovec) ndi mu vivo gene therapy yokhala ndi adeno-associated virus type 2 (AAV2) monga chonyamulira.Odwala amadwala chifukwa cha masinthidwe a jini omwe amasunga enzyme ya AADC.AAV2 imakhala ndi jini yathanzi yolemba enzyme ya AADC.Mawonekedwe a jini chipukuta misozi amakwaniritsa achire zotsatira.Mwachidziwitso, utsogoleri umodzi umagwira ntchito kwa nthawi yayitali.Ndilo chithandizo choyamba cha jini chomwe chimaperekedwa mwachindunji muubongo.Chilolezo cha malonda chimagwira ntchito ku mayiko onse 27 a EU, komanso Iceland, Norway ndi Liechtenstein.

(10) Roctavian

Kampani: Yopangidwa ndi BioMarin Pharmaceutical (BioMarin).

Nthawi yopita kumsika: Kuvomerezedwa kutsatsa ndi European Union mu Ogasiti 2022;chilolezo chotsatsa ndi UK Medicines and Healthcare products Administration (MHRA) mu Novembala 2022.

Zochizira odwala wamkulu hemophilia A amene alibe mbiri FVIII factor chopinga ndi zoipa AAV5 chitetezo.

Ndemanga: Roctavian (valoctocogene roxaparvovec) amagwiritsa ntchito AAV5 ngati vekitala ndipo amagwiritsa ntchito cholimbikitsa chiwindi chamunthu cha HLP kuyendetsa mawu a human coagulation factor VIII (FVIII) pomwe domain B itachotsedwa.Lingaliro la European Commission lovomereza kugulitsidwa kwa valoctocogene roxaparvovec zatengera zonse zomwe zachitika pakukula kwachipatala kwa mankhwalawa.Pakati pawo, zotsatira za gawo lachitatu la mayeso azachipatala GENER8-1 adawonetsa kuti poyerekeza ndi zomwe zidachitika chaka chisanalembetsedwe, pambuyo pa kulowetsedwa kamodzi kwa valoctocogene roxaparvovec, The mutu wapachaka wamagazi (ABR) wachepa kwambiri, pafupipafupi kugwiritsa ntchito recombinant coagulation factor VIII (F8) kukonzekera kwa protein kumachepetsedwa, kapena kuchuluka kwa magazi m'thupi la F8 kumawonjezeka kwambiri.Pambuyo pa masabata a 4 a chithandizo, mlingo wa F8 wogwiritsidwa ntchito pachaka ndi ABR wofuna chithandizo unachepetsedwa ndi 99% ndi 84%, motero, ndipo kusiyana kunali kofunikira kwambiri (p <0.001).Mbiri yachitetezo inali yabwino, ndipo palibe phunziro lomwe linakumana ndi F8 factor inhibition, malignancy kapena thrombosis side effects, ndipo palibe zochitika zazikulu zokhudzana ndi chithandizo (SAEs) zomwe zinanenedwa.

(11) Hemgenix

Kampani: Yopangidwa ndi UniQure Corporation.

Nthawi yogulitsa: Yovomerezedwa ndi FDA kuti igulitsidwe mu Novembala 2022.

Zochizira odwala wamkulu hemophilia B.

Ndemanga: Hemgenix ndi chithandizo cha majini chotengera AAV5 vekitala.Mankhwalawa amakhala ndi coagulation factor IX (FIX) mtundu wa FIX-Padua, womwe umaperekedwa kudzera m'mitsempha.Pambuyo poyang'anira, jini imatha kufotokoza FIX coagulation factor mu chiwindi ndi kubisala Pambuyo polowa m'magazi kuti agwiritse ntchito coagulation, kuti akwaniritse cholinga cha chithandizo, mwachidziwitso, utsogoleri umodzi umagwira ntchito kwa nthawi yaitali.

(12) Adstiladrin

Kampani: Yopangidwa ndi Ferring Pharmaceuticals.

Nthawi yogulitsa: Yovomerezedwa ndi FDA kuti igulitsidwe mu Disembala 2022.

Zizindikiro: Zochizira khansa ya chikhodzodzo yomwe ili pachiwopsezo chachikulu (NMIBC) yosalabadira Bacillus Calmette-Guerin (BCG) .

Ndemanga: Adstiladrin ndi mankhwala a jini otengera adenoviral vector osabwerezabwereza, omwe amatha kutulutsa mapuloteni ambiri a interferon alfa-2b m'maselo omwe mukufuna, ndipo amaperekedwa kudzera mu catheter ya mkodzo kulowa m'chikhodzodzo (yomwe imaperekedwa kamodzi miyezi itatu iliyonse) utic zotsatira.Motero njira yatsopano yochizira majini imeneyi imasintha maselo a m'chikhodzodzo cha wodwalayo kukhala “fakitale” yaing’ono imene imatulutsa interferon, motero zimathandiza kuti wodwalayo athe kulimbana ndi khansa.

Chitetezo ndi mphamvu ya Adstiladrin inayesedwa mu kafukufuku wachipatala wambiri kuphatikizapo odwala 157 omwe ali ndi chiopsezo chachikulu cha BCG-osayankha NMIBC.Odwala adalandira Adstiladrin miyezi itatu iliyonse kwa miyezi 12, kapena mpaka poizoni wosavomerezeka ku chithandizo kapena kubwereranso kwa NMIBC yapamwamba kwambiri.Ponseponse, 51 peresenti ya odwala omwe adalandira chithandizo ndi Adstiladrin adapeza yankho lathunthu (kutayika kwa zizindikiro zonse za khansa zomwe zimawonedwa pa cystoscopy, biopsy tishu, ndi mkodzo).

3. Mankhwala ang'onoang'ono a nucleic acid

(1) Vitravene

Kampani: Yopangidwa ndi Ionis Pharma (yemwe kale anali Isis Pharma) ndi Novartis.

Nthawi yogulitsa: Mu 1998 ndi 1999, idavomerezedwa kutsatsa ndi FDA ndi EU EMA.

Zochizira cytomegalovirus retinitis odwala HIV.

Ndemanga: Vitravene ndi antisense oligonucleotide mankhwala, omwe ndi mankhwala oyamba a oligonucleotide ovomerezeka kuti agulitse malonda padziko lapansi.Pa gawo loyambirira la ndandanda, kufunikira kwa msika kwa mankhwala odana ndi CMV kunali kofulumira kwambiri;kenako, chifukwa cha chitukuko cha kwambiri yogwira ma ARV, chiwerengero cha CMV milandu anatsika kwambiri.Chifukwa cha kuchepa kwa msika, mankhwalawa adayambitsidwa mu 2002 ndi 2006 Kuchotsa m'maiko a EU ndi United States.

(2) Magulu

Kampani: Co-yopangidwa ndi Pfizer ndi Eyetech.

Nthawi yopita kumsika: Adavomerezedwa kuti alembetse ku United States mu 2004.

Zochizira neovascular zaka zokhudzana macular alibe.

Ndemanga: Macugen ndi pegylated modified oligonucleotide mankhwala, omwe angathe kulunjika ndi kumangirira vascular endothelial growth factor (VEGF165 subtype), ndipo njira yoyendetsera ntchito ndi jekeseni wa intravitreal.

(3) Defitelio

Kampani: Yopangidwa ndi Jazz Pharmaceuticals.

Nthawi yogulitsa: Idavomerezedwa kuti igulidwe ndi European Union mu 2013 ndikuvomerezedwa ndi FDA kuti igulitsidwe mu Marichi 2016.

Zochizira kwa chiwindi veno-occlusive matenda kugwirizana ndi aimpso kapena m`mapapo mwanga kukanika pambuyo hematopoietic tsinde cell kupatsidwa zina.

Ndemanga: Defitelio ndi oligonucleotide mankhwala, amene ndi osakaniza oligonucleotides ndi plasmin katundu.Anachotsedwa pamsika mu 2009 pazifukwa zamalonda.

(4) Chinamro

Kampani: Yopangidwa ndi Ionis Pharma ndi Kastle.

Nthawi Yogulitsa: Mu 2013, idavomerezedwa kuti igulitsidwe ku United States ngati mankhwala amasiye.

Pakuti adjuvant mankhwala a homozygous banja hypercholesterolemia.

Ndemanga: Kynamro ndi antisense oligonucleotide mankhwala, amene ndi antisense oligonucleotide kulunjika anthu apo B-100 mRNA.Kynamro amaperekedwa ngati 200 mg subcutaneously kamodzi pa sabata.

(5) Spinraza

Kampani: Yopangidwa ndi Ionis Pharmaceuticals.

Nthawi yogulitsa: Yovomerezedwa ndi FDA kuti igulitse mu Disembala 2016.

Zochizira msana minofu atrophy (SMA).

Ndemanga: Spinraza (nusinersen) ndi antisense oligonucleotide mankhwala.Pomangiriza ku malo ophatikizika a SMN2 exon 7, Spinraza imatha kusintha RNA cleavage ya SMN2 jini, potero kukulitsa kupanga kwa protein ya SMN yogwira ntchito mokwanira.Mu Ogasiti 2016, BIOGEN idagwiritsa ntchito mwayi wake kupeza ufulu wapadziko lonse wa Spinraza.Spinraza idangoyamba kuyesa koyamba kwachipatala mwa anthu ku 2011. M'zaka 5 zokha, idavomerezedwa ndi FDA kuti igulitse mu 2016, zomwe zikuwonetsa kuzindikira kwathunthu kwa FDA kwa mphamvu yake.Mankhwalawa adavomerezedwa kuti agulitse ku China mu April 2019. Njira yonse yovomerezeka ya Spinraza ku China inali yosakwana miyezi 6, ndipo panali zaka 2 ndi miyezi 2 kuchokera pamene Spinraza inavomerezedwa koyamba ku United States.Kuthamanga kwa mindandanda ku China kuli kale mwachangu kwambiri.Izi zilinso chifukwa chakuti Center for Drug Evaluation idapereka "Chidziwitso Pakusindikiza Mndandanda wa Gulu Loyamba la Mankhwala Osokoneza Bongo Omwe Akufunika Mwamsanga mu Clinical Practice" pa Novembara 1, 2018, ndipo adaphatikizidwa mugulu loyamba lamankhwala 40 akunja kuti awunikenso mwachangu, pomwe Spinraza adayikidwamo.

(6) Eksodo 51

Kampani: Yopangidwa ndi AVI BioPharma (yemwe inadzatchedwanso Sarepta Therapeutics).

Nthawi yogulitsa: Mu Seputembala 2016, idavomerezedwa kuti igulidwe ndi FDA.

Zochizira Duchenne muscular dystrophy (DMD) ndi exon 51 kulumpha masinthidwe a jini mu jini ya DMD.

Ndemanga: Exondys 51 ndi antisense oligonucleotide mankhwala, antisense oligonucleotide akhoza kumangirira ku malo a exon 51 pre-mRNA wa DMD jini, kuchititsa mapangidwe okhwima mRNA, mbali ya exon 51 oletsedwa Excision, potero kuwongolera pang'onopang'ono mawonekedwe a puloteni yafupipafupi ya mRNA, kuthandiza odwala omwe ali ndi mRNA. , potero kusintha zizindikiro za wodwalayo.

(7) Tegsedi

Kampani: Yopangidwa ndi Ionis Pharmaceuticals.

Nthawi yogulitsa: Idavomerezedwa kuti igulidwe ndi European Union mu Julayi 2018.

Zochizira hereditary transthyretin amyloidosis (hATTR).

Ndemanga: Tegsedi ndi antisense oligonucleotide mankhwala olunjika transthyretin mRNA.Ndiwo mankhwala oyamba omwe amavomerezedwa padziko lapansi kuti azichiza hATTR.Imayendetsedwa ndi subcutaneous jekeseni.Mankhwalawa amachepetsa kupanga kwa mapuloteni a ATTR poyang'ana mRNA ya transthyretin (ATTR), ndipo ali ndi chiŵerengero chabwino cha chiopsezo pochiza ATTR, ndipo matenda a ubongo ndi umoyo wa wodwalayo zakhala zikuyenda bwino, ndipo zimagwirizana ndi mitundu yosinthika ya TTR, Palibe gawo la matenda kapena kukhalapo kwa cardiomyopathy kunali koyenera.

(8) Pamwamba

Kampani: Yopangidwa pamodzi ndi Alnylam Corporation ndi Sanofi Corporation.

Nthawi Yogulitsa: Zavomerezedwa kuti zilembedwe ku United States mu 2018.

Zochizira hereditary transthyretin amyloidosis (hATTR).

Ndemanga: Onpattro ndi mankhwala a siRNA omwe amayang'ana transthyretin mRNA, omwe amachepetsa kupanga mapuloteni a ATTR m'chiwindi komanso amachepetsa kuchuluka kwa ma amyloid deposits mu mitsempha yozungulira poyang'ana mRNA ya transthyretin (ATTR) , potero amawongolera ndi kuchepetsa zizindikiro za matenda.

(9) Givlaari

Kampani: Yopangidwa ndi Alnylam Corporation.

Nthawi yogulitsa: Yovomerezedwa ndi FDA kuti igulitse mu Novembala 2019.

Zochizira pachimake kwa chiwindi porphyria (AHP) akuluakulu.

Ndemanga: Givlaari ndi mankhwala a siRNA, omwe ndi mankhwala achiwiri a siRNA ovomerezeka kuti agulitsidwe pambuyo pa Onpattro.Njira ya makonzedwe ndi subcutaneous jekeseni.Mankhwalawa amayang'ana mRNA ya mapuloteni a ALAS1, ndipo chithandizo cha mwezi ndi mwezi ndi Givlaari chingathe kuchepetsa kwambiri komanso kwamuyaya mlingo wa ALAS1 m'chiwindi, potero kuchepetsa milingo ya neurotoxic ALA ndi PBG kumagulu abwinobwino, potero Kuchepetsa zizindikiro za matenda a wodwalayo.Detayo inasonyeza kuti odwala omwe amathandizidwa ndi Givlaari anali ndi 74% kuchepetsa chiwerengero cha kugwidwa poyerekeza ndi gulu la placebo.

(10) Vyondys53

COMPANY: Yopangidwa ndi Sarepta Therapeutics.

Nthawi yogulitsa: Yovomerezedwa ndi FDA kuti igulitsidwe mu Disembala 2019.

Zochizira odwala DMD ndi dystrophin jini exon 53 splicing masinthidwe.

Ndemanga: Vyondys 53 ndi antisense oligonucleotide mankhwala, amene amalimbana splicing ndondomeko dystrophin pre-mRNA.Exon 53 imachepetsedwa pang'ono, mwachitsanzo, palibe pa mRNA yokhwima, ndipo idapangidwa kuti ipangitse dystrophin yocheperako koma yogwira ntchito, potero imakulitsa mphamvu zolimbitsa thupi mwa odwala.

(11) Waylivra

Kampani: Yopangidwa ndi Ionis Pharmaceuticals ndi othandizira ake Akcea Therapeutics.

Nthawi yogulitsa: Idavomerezedwa kuti igulidwe ndi European Medicines Agency (EMA) mu Meyi 2019.

Zizindikiro: Monga chithandizo chothandizira kuwonjezera pa kuwongolera zakudya mwa odwala akuluakulu omwe ali ndi matenda a chylomicronemia (FCS).

Ndemanga: Waylivra ndi mankhwala a antisense oligonucleotide, omwe ndi mankhwala oyamba omwe amavomerezedwa kuti agulitsidwe padziko lonse lapansi pochiza FCS.

(12) Levi

Kampani: Yopangidwa ndi Novartis.

Nthawi Yogulitsa: Yovomerezedwa ndi European Union kuti igulidwe mu Disembala 2020.

Zochizira akuluakulu omwe ali ndi hypercholesterolemia yayikulu (heterozygous family and non-familia) kapena dyslipidemia wosakanikirana.

Ndemanga: Leqvio ndi mankhwala a siRNA omwe akulunjika ku PCSK9 mRNA.Ndi chithandizo choyamba cha siRNA padziko lonse lapansi chotsitsa cholesterol (LDL-C).Imayendetsedwa ndi subcutaneous jekeseni.Mankhwalawa amachepetsa mlingo wa mapuloteni a PCSK9 kupyolera mu kusokoneza kwa RNA, motero amachepetsa mlingo wa LDL-C.Zambiri zachipatala zikuwonetsa kuti kwa odwala omwe sangathe kutsitsa LDL-C kufika pamlingo womwe akufuna atalandira chithandizo ndi mlingo waukulu wololedwa wa ma statins, Leqvio amatha kuchepetsa LDL-C pafupifupi 50%.

(13) Chikhalidwe

Kampani: Yopangidwa ndi Alnylam Pharmaceuticals.

Nthawi Yogulitsa: Yovomerezedwa ndi European Union kuti igulitse mu Novembala 2020.

Zochizira chachikulu hyperoxaluria mtundu 1 (PH1).

Ndemanga: Oxlumo ndi mankhwala a siRNA omwe akulunjika ku hydroxyacid oxidase 1 (HAO1) ​​mRNA, ndipo njira yoyendetsera ndi jekeseni wa subcutaneous.The mankhwala anayamba ntchito Alnylam atsopano kumatheka kukhazikika zimapangidwira, ESC-GalNAc conjugation luso, amene zimathandiza subcutaneously kutumikiridwa siRNA ndi kulimbikira kwambiri ndi potency.Mankhwalawa amawononga kapena kulepheretsa hydroxyacid oxidase 1 (HAO1) ​​mRNA, amachepetsa mlingo wa glycolate oxidase m'chiwindi, ndiyeno amadya gawo lapansi lofunika kuti apange oxalate, kuchepetsa kupanga oxalate kuti athe kuchepetsa kufalikira kwa matendawa kwa odwala komanso kusintha zizindikiro za matenda.

(14) Viltepso

Company: Yopangidwa ndi NS Pharma, subsidiary ya Nippon Shinyaku.

Nthawi yogulitsa: Yovomerezedwa ndi FDA kuti igulitsidwe mu Ogasiti 2020.

Zochizira Duchenne muscular dystrophy (DMD) ndi exon 53 kulumpha masinthidwe a jini mu jini ya DMD.

Ndemanga: Viltepso ndi antisense oligonucleotide mankhwala omwe amatha kumangirira ku malo a exon 53 a pre-mRNA a DMD jini, kuchititsa kuti gawo la exon 53 lichotsedwe pambuyo pa mapangidwe a mRNA okhwima, potero kuwongolera pang'ono mawonekedwe a mRNA owerengera Bokosi limathandiza odwala kuti azitha kuphatikizira odwala omwe ali ndi mawonekedwe afupikitsa a protein, omwe amawongolera ma protein afupipafupi.

(15) Amondi 45

Kampani: Yopangidwa ndi Sarepta Therapeutics.

Nthawi yogulitsa: Yovomerezedwa ndi FDA kuti igulitsidwe mu February 2021.

Zochizira Duchenne muscular dystrophy (DMD) ndi exon 45 kulumpha masinthidwe a jini mu jini ya DMD.

Ndemanga: Amondys 45 ndi antisense oligonucleotide mankhwala, antisense oligonucleotide akhoza kumangirira ku malo a exon 45 a pre-mRNA a DMD jini, kuchititsa kuti mbali ya exon 45 kutsekedwa pambuyo mapangidwe okhwima mRNA Excision, potero mwapang`onopang`ono kuwerenga ma dystrophin mawonekedwe afupikitsa ma syntrophin kuthandizira odwala kuti asinthe mawonekedwe a mRNA. mapuloteni, potero kusintha zizindikiro za wodwalayo.

(16) Amvuttra (vutrisiran)

Kampani: Yopangidwa ndi Alnylam Pharmaceuticals.

Nthawi yogulitsa: Yovomerezedwa ndi FDA kuti igulitse mu June 2022.

Zochizira hereditary transthyretin amyloidosis ndi polyneuropathy (hATTR-PN) mwa akulu.

Ndemanga: Amvuttra (Vutrisiran) ndi mankhwala a siRNA omwe akulunjika ku transthyretin (ATTR) mRNA, yoyendetsedwa ndi jakisoni wa subcutaneous.Vutrisiran imachokera ku Alnylam's Enhanced Stability Chemistry (ESC)-GalNAc conjugate delivery platform design ndi kuwonjezereka kwa potency ndi kukhazikika kwa metabolic.Chivomerezo cha mankhwalawa chimachokera ku deta ya miyezi ya 9 ya kafukufuku wake wachipatala wa Phase III (HELIOS-A), ndipo zotsatira zake zonse zimasonyeza kuti mankhwalawa adasintha zizindikiro za hATTR-PN, ndipo oposa 50% a odwalawo adasinthidwa kapena anasiya kuwonjezereka.

4. Mankhwala ena ochizira majini

(1) Rexin-G

Kampani: Yopangidwa ndi Epeius Biotech.

Nthawi yogulitsa: Mu 2005, idavomerezedwa kuti igulidwe ndi a Philippine Food and Drug Administration (BFAD).

Zizindikiro: Zochizira khansa yapamwamba yosamva mankhwala amphamvu.

Ndemanga: Rexin-G ndi jakisoni wodzaza jini wa nanoparticle.Imayambitsa jini yosinthika ya cyclin G1 m'maselo omwe akuwafunira kudzera mu vector yobwezeretsanso kuti aphe makamaka zotupa zolimba.Ulamuliro njira ndi mtsempha wa magazi kulowetsedwa.Monga mankhwala olimbana ndi chotupa omwe amafufuza mwachangu ndikuwononga maselo a khansa ya metastatic, amakhala ndi zotsatira zochiritsa odwala omwe alephera mankhwala ena a khansa, kuphatikiza biologics.

(2) Neovasculgen

Kampani: Yopangidwa ndi Human stem cell Institute.

Nthawi yolembedwa: Anavomerezedwa kuti alembetsedwe ku Russia pa Disembala 7, 2011, kenako adakhazikitsidwa ku Ukraine mu 2013.

Zochizira zotumphukira mitsempha yamitsempha matenda, kuphatikizapo kwambiri nthambi ischemia.

Ndemanga: Neovasculgen ndi mankhwala a jini otengera ma plasmids a DNA.The vascular endothelial growth factor (VEGF) 165 jini imapangidwa pamsana wa plasmid ndikulowetsedwa mwa odwala.

(3) Collategene

Kampani: Yopangidwa pamodzi ndi Osaka University ndi makampani opangira ndalama.

Nthawi Yogulitsa: Kuvomerezedwa ndi Unduna wa Zaumoyo, Ntchito ndi Ubwino waku Japan mu Ogasiti 2019.

Chithandizo cha kwambiri m`munsi malekezero ischemia.

Ndemanga: Collategene ndi plasmid-based gene therapy, mankhwala oyamba amtundu wapakhomo opangidwa ndi AnGes, kampani yopanga ma gene ku Japan.Chigawo chachikulu cha mankhwalawa ndi plasmid wamaliseche yomwe ili ndi ndondomeko ya jini ya hepatocyte growth factor (HGF).Ngati mankhwala jekeseni mu minofu ya m`munsi miyendo, anasonyeza HGF kulimbikitsa mapangidwe atsopano mitsempha ya magazi kuzungulira occluded mitsempha.Mayesero azachipatala atsimikizira zotsatira zake pakuwongolera zilonda.

Kodi Foregene angathandize bwanji kukula kwa gene therapy?

Timathandizira kupulumutsa nthawi yowunikira pakuwunika kwakukulu, kumayambiriro kwa chitukuko cha mankhwala a siRNA.

Zambiri pitani:

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