Vuba aha, imiti itatu yo kuvura gene yemejwe kugirango yamamaze, aribyo: (1) Ku ya 21 Nyakanga 2022, PTC Therapeutics, Inc..Kwemeza ubuvuzi muri Amerika nta gushidikanya ko ari "ubufasha mu rubura" kuri Bluebird Bio, iri mu bibazo by'amafaranga.. ).Kugeza ubu, imiti 41 yo kuvura gene yemewe yo kwamamaza ku isi hose.

Gene nigice cyibanze kigenga imiterere.Usibye ingirabuzimafatizo za virusi zimwe na zimwe, zigizwe na RNA, ingirabuzima fatizo zigizwe na ADN.Indwara nyinshi z’ibinyabuzima ziterwa n’imikoranire hagati ya gen n’ibidukikije, kandi indwara nyinshi zirashobora gukira cyangwa kugabanuka muri rusange binyuze mu kuvura gene.Ubuvuzi bwa Gene bufatwa nkimpinduramatwara mubuvuzi na farumasi.Imiti yagutse ya gene ikubiyemo imiti ishingiye ku miti ya ADN yahinduwe (nka virusi ya virusi ishingiye ku miti ivura vivo gene, mu miti ivura vitro gene, imiti ya plasmid yambaye ubusa, n'ibindi) n'imiti ya RNA (nk'imiti ya antisense oligonucleotide, imiti ya siRNA, n'ubuvuzi bwa mRNA, n'ibindi);Imiti ivura indwara ya Gene ikubiyemo cyane cyane imiti ya ADN ya plasmide, imiti ivura gene ishingiye kuri virusi, imiti ivura gene ishingiye kuri virusi ya bagiteri, sisitemu yo guhindura gene hamwe n’imiti ivura ingirabuzimafatizo yahinduwe muri vitro.Nyuma yimyaka myinshi yiterambere, imiti ivura gene yageze kubisubizo byubuzima.(ukuyemo inkingo za ADN n'inkingo za mRNA), imiti 41 yo kuvura gene yemejwe yo kwamamaza ku isi.Hamwe nogutangiza ibicuruzwa niterambere ryihuse rya tekinoroji yo kuvura gene, kuvura gene bigiye gutangira mugihe cyiterambere ryihuse.

Ibyiciro byo kuvura gene (Inkomoko yishusho: Biologiya Jingwei)

Iyi ngingo irerekana urutonde 41 rwubuvuzi bwemejwe bwo kwamamaza (ukuyemo inkingo za ADN ninkingo za mRNA).

1. Mu kuvura vitro

(1) Strimvelis

Isosiyete: Yakozwe na GlaxoSmithKline (GSK).

Igihe cyo kwisoko: Byemejwe n’umuryango w’ubumwe bw’ibihugu by’i Burayi muri Gicurasi 2016.

Ibyerekana: Kubuvuzi bukomeye bwo gukingira indwara (SCID).

Icyitonderwa: Inzira rusange yubu buvuzi ni ukubanza kubona ingirangingo z'umurwayi wa hematopoietic stem selile, kwagura no kuzitera umuco muri vitro, hanyuma ugakoresha retrovirus kugirango winjize kopi ya gene ikora ya ADA (adenosine deaminase) mumyanya myibarukiro ya hematopoietic.Ingirabuzimafatizo ya Hematopoietic yinjizwa mu mubiri.Ibisubizo by’amavuriro byerekanaga ko imyaka 3 yo kubaho kwabarwayi ba ADA-SCID bavuwe na Strimvelis yari 100%.

(2) Zalmoxis

Isosiyete: Yakozwe na MolMed, mu Butaliyani.

Igihe cyo kwisoko: Yabonye uburenganzira bwo kwamamaza byemewe na EU muri 2016.

Ibyerekana: Ikoreshwa muburyo bwo kuvura sisitemu yubudahangarwa bw’abarwayi nyuma yo kwimurwa kwa hematopoietic stem selile.

Ijambo: Zalmoxis ni allogeneic T selile yiyahura gene immunotherapy yahinduwe na virusi ya virusi.1NGFR na HSV-TK Mut2 genes yo kwiyahura ituma abantu bakoresha ganciclovir umwanya uwariwo wose kugirango bice selile T itera ubudahangarwa bw'umubiri, bikarinda kwangirika kwa GVHD ishobora kubaho, no kugarura imikorere yubudahangarwa ku barwayi bafite HSCT yanduye nyuma yo kubagwa Escort.

(3) Invossa-K

Isosiyete: Yatejwe imbere na sosiyete ya TissueGene (KolonTissueGene).

Igihe cyo kwisoko: Yemejwe kurutonde muri Koreya yepfo muri Nyakanga 2017.

Ibyerekana: Kubuvuzi bwa rubagimpande.

Ijambo: Invossa-K nubuvuzi bwa allogeneic selile gene irimo chondrocytes zabantu.Ingirabuzimafatizo za Allogeneic zahinduwe muri vitro, kandi selile zahinduwe zirashobora kwerekana no gusohora ibintu bikura bikura β1 (TGF-β1) nyuma yo gutera inshinge.β1), bityo kunoza ibimenyetso bya osteoarthritis.Ibisubizo byubuvuzi byerekana ko Invossa-K ishobora kunoza cyane ivi.Uruhushya rwambuwe n’umuyobozi ushinzwe ibiyobyabwenge muri Koreya yepfo mu 2019 kubera ko uruganda rwanditse nabi ibikoresho byakoreshejwe.

(4) Zynteglo

Isosiyete: Yatejwe imbere na bio yubururu bwabanyamerika bio (bluebird bio).

Igihe cyo kwisoko: Byemejwe n’umuryango w’ubumwe bw’ibihugu by’i Burayi muri 2019, kandi byemejwe na FDA muri Kanama 2022.

Ibyerekana: Kubuvuzi bwo guterwa-guterwa β-thalassemia.

Ijambo: Zynteglo ni lentiviral muri vitro gene ivura, ikoresha vector ya lentiviral kugirango itangire kopi yimikorere ya gene isanzwe β-globine (βA-T87Q-globin gene) muri selile stem hematopoietic stem yakuwe mubarwayi., hanyuma ushiremo uturemangingo twa autologique hematopoietic stem selile usubire mumurwayi.Iyo umurwayi amaze kugira gene isanzwe βA-T87Q-globine, barashobora gukora proteine ​​isanzwe ya HbAT87Q, ishobora kugabanya cyangwa gukuraho neza ibikenewe guterwa amaraso.Nubuvuzi bwigihe kimwe bugamije gusimbuza amaraso ubuzima bwawe bwose n'imiti ubuzima bwabo bwose kubarwayi bafite imyaka 12 nayirenga.

(5) Skysona

Isosiyete: Yatejwe imbere na bio yubururu bwabanyamerika bio (bluebird bio).

Igihe cyo kwisoko: Byemejwe na EU yo kwamamaza muri Nyakanga 2021.

Ibyerekana: Kubuvuzi bwubwonko bwambere adrenoleukodystrofiya (CALD).

Ijambo: Skysona ivura gene niyo yonyine ivura gene yemewe rimwe yo kuvura ubwonko bwubwonko bwa adrenoleukodystrophy (CALD).Skysona (elivaldogene autotemcel, Lenti-D) ni hematopoietic stem selile lentiviral muri vitro gene ivura Lenti-D.Inzira rusange yubuvuzi nuburyo bukurikira: autologique hematopoietic stem stem selile ikurwa kumurwayi, igahindurwa muri vitro na lentivirus itwara gene muntu ABCD1, hanyuma igashyirwa mumurwayi.Kuvura abarwayi bari munsi yimyaka 18 hamwe na ABCD1 gene mutation na CALD.

(6) Kymriya

Isosiyete: Yakozwe na Novartis.

Igihe cyo kwisoko: Byemejwe na FDA muri Kanama 2017.

Ibyerekana: Kuvura prursor B-selile acute lymphoblastique leukemia (BYOSE) hamwe na DLBCL yongeye kwisubiraho.

Ijambo: Kymriah ni lentiviral mumiti ya vitro gene therapy, imiti yambere yemewe na CAR-T ku isi, yibasira CD19 kandi ikoresha ibintu bitera imbaraga 4-1BB.Igiciro ni $ 475.000 muri Amerika na 313,000 $ mu Buyapani.

(7) Yescarta

Isosiyete: Yatejwe imbere na Kite Pharma, ishami rya Galeedi.

Igihe cyo kwisoko: Byemejwe na FDA mu Kwakira 2017.

Ibyerekana: Kubuvuzi bwa lymphoma nini ya B-selile.

Ijambo: Yescarta ni virusi itera virusi ya vitro.Nubuvuzi bwa kabiri CAR-T yemewe kwisi.Ireba CD19 kandi ikoresha ibintu byerekana CD28.Igiciro muri Amerika ni $ 373.000.

(8) Tecartus

Isosiyete: Yatejwe imbere na Galeedi (GILD).

Igihe cyo kwisoko: Byemejwe na FDA muri Nyakanga 2020.

Ibyerekana: Kubisubiramo cyangwa byanze bikunze mantle selile lymphoma.

Icyitonderwa: Tecartus nubuvuzi bwa CAR-T bwakorewe CD19, kandi nubuvuzi bwa gatatu CAR-T bwemewe bwo kwamamaza ku isi.

(9) Breyanzi

Isosiyete: Yakozwe na Bristol-Myers Squibb (BMS).

Igihe cyo kwisoko: Byemejwe na FDA muri Gashyantare 2021.

Ibyerekana: Gusubiramo cyangwa kwanga (R / R) lymphoma nini ya B-selile (LBCL).

Ijambo: Breyanzi nubuvuzi bwa vitro gene bushingiye kuri lentivirus, hamwe nubuvuzi bwa kane CAR-T bwemewe bwo kwamamaza ku isi, bwibasira CD19.Iyemezwa rya Breyanzi ni intambwe ikomeye kuri Bristol-Myers Squibb mu rwego rwo gukingira indwara ya selile, Bristol-Myers yaguze igihe yagura Celgene kuri miliyari 74 z'amadolari muri 2019.

(10) Abecma

Isosiyete: Yafatanije na Bristol-Myers Squibb (BMS) na biobird bio.

Igihe cyo kwisoko: Byemejwe na FDA muri Werurwe 2021.

Ibyerekana: Gusubiramo cyangwa kwanga myeloma myinshi.

Ijambo: Abecma ni lentivirus ishingiye kuri vitro gene ivura, ubuvuzi bwa mbere bwa CAR-T ku isi bugamije kurwanya BCMA, hamwe nubuvuzi bwa gatanu CAR-T bwemejwe na FDA.Ihame ry'ibiyobyabwenge ni ukugaragaza imiti ya BCMA ya chimeric kuri selile ya autologique T yumurwayi binyuze muri lentivirus-yunganirwa na genoside muri vitro.Mbere yo kwinjiza imiti ya selile, umurwayi yakiriye ibice bibiri bya cyclophosphamide na fludarabine kugirango abanze avurwe.Umuti kugirango ukureho selile T idahinduwe kumurwayi, hanyuma ushiremo selile T yahinduwe isubira mumubiri wumurwayi kugirango ushake kandi wice selile ya kanseri yerekana BCMA.

(11) Libmeldy

Isosiyete: Yateguwe na Orchard Therapeutics.

Igihe cyo kwisoko: Byemejwe n’umuryango w’ubumwe bw’ibihugu by’i Burayi gushyira ku rutonde mu Kuboza 2020.

Ibyerekana: Kubuvura metachromatic leukodystrophy (MLD).

Ijambo: Libmeldy nubuvuzi bwa gene bushingiye kuri lentiviral muri vitro gene ihindura ingirabuzimafatizo za CD34 +.Amavuriro yerekana ko kwinjiza imitsi imwe ya Libmeldy bigira akamaro muguhindura inzira ya MLD hakiri kare ndetse nubumuga bukomeye bwa moteri nubwenge kubarwayi batavuwe bafite imyaka imwe.

(12) Benoda

Isosiyete: Yakozwe na WuXi Junuo.

Igihe cyo kwisoko: Byemejwe na NMPA muri Nzeri 2021.

Ibyerekana: Kuvura lymphoma nini ya B-selile yisubiramo cyangwa yangiritse (r / r LBCL) kubarwayi bakuze nyuma yumurongo wa kabiri cyangwa se uburyo bwinshi bwo kuvura.

Ijambo: Benoda ni imiti igabanya ubukana bwa CD19 CAR-T, kandi nigicuruzwa cyibanze cya WuXi Junuo.Nibicuruzwa bya kabiri CAR-T byemejwe mubushinwa, usibye gusubiramo lymphoma nini ya B-selile.Byongeye kandi, WuXi Junuo arateganya kandi gutera urushinge rwa Ruiki Orenza mu kuvura ibindi bimenyetso bitandukanye, birimo lymphoma follicular (FL), lymphoma selile mantle (MCL), lymphocytique leukemia idakira (CLL), umurongo wa kabiri ukwirakwiza lymphoma nini ya B-selile (DLBCL) na lymphoblastique ikaze (BYOSE).

(13) CARVYKTI

Isosiyete: Igicuruzwa cya mbere cyemewe cya Legio Bio.

Igihe cyo kwisoko: Byemejwe na FDA muri Gashyantare 2022.

Ibyerekana: Kuvura myeloma isubirwamo cyangwa yangiritse (R / R MM).

Ijambo: CARVYKTI (ciltacabtagene autoleucel, bita Cilta-cel) nubuvuzi bwa CAR-T bwakorewe ubudahangarwa bw'umubiri hamwe na antibodi ebyiri zo mu bwoko bwa antibodiyite zibasira antigen yo gukura kwa B (BCMA).Aya makuru yerekana ko CARVYKTI yerekanye igipimo rusange cy’ibisubizo bigera kuri 98% ku barwayi barwaye myeloma yongeye kwisubiraho cyangwa yangiritse, bakaba barahawe imiti ine cyangwa irenga mbere, harimo na proteasome inhibitor, immunomodulator, na antibodiyite zirwanya CD38.

2. Muri vivo gene ivura ishingiye kuri virusi

(1) Gendicine / yavutse ubwa kabiri

Isosiyete: Yatejwe imbere na Shenzhen Saibainuo.

Igihe cyo kwisoko: Yemejwe kurutonde mubushinwa muri 2003.

Ibyerekana: Kubuvura kanseri y'udukoko twa kanseri y'umutwe n'ijosi.

Ijambo: Recombinant muntu p53 adenovirus inshinge Gendicine / Jinshengsheng numuti wa adenovirus vector gene therapy hamwe nuburenganzira bwumutungo wubwenge bwigenga bufitwe na Shenzhen Saibainuo.Uyu muti ugizwe na gene p53 isanzwe yibibyimba byabantu hamwe na artificiel artificiel recombinant replication-yabuze Ubwoko bwa adenovirus bwubwoko bwa 5 bugizwe nubwoko bwa adenovirus bwabantu 5. Iyambere niyo miterere nyamukuru yibiyobyabwenge kugirango igire ingaruka zo kurwanya ibibyimba, kandi ibyanyuma bikora nkibitwara.Indwara ya adenovirus itwara gene ivura p53 mu ngirabuzimafatizo, kandi ikagaragaza gene p53 ikuramo kanseri.Igicuruzwa kirashobora kugenzura ingirabuzimafatizo zitandukanye zo kurwanya kanseri no kugabanya ibikorwa bya oncogène zitandukanye, bityo bikazamura ingaruka zo kurwanya ibibyimba umubiri kandi bikagera ku ntego yo kwica ibibyimba.

(2) Rigvir

Isosiyete: Yatejwe imbere na Lativiya isosiyete Latima.

Igihe cyo kwisoko: Byemejwe muri Lativiya muri 2004.

Ibyerekana: Kubuvuzi bwa melanoma.

Ijambo: Rigvir nubuvuzi bwa gene bushingiye kuri gene ya ECHO-7 enterovirus yahinduwe, yakoreshejwe muri Lativiya, Esitoniya, Polonye, ​​Arumeniya, Biyelorusiya n'ahandi, kandi yandikwa muri EMA y’ubumwe bw’ibihugu by’Uburayi..Indwara z’amavuriro mu myaka icumi ishize zerekanye ko virusi ya Rigvir oncolytic ifite umutekano kandi ikora neza, kandi ishobora kuzamura ubuzima bw’abarwayi ba melanoma inshuro 4-6.Byongeye kandi, ubuvuzi bukwiriye kandi izindi kanseri zitandukanye, zirimo kanseri yibara, kanseri yandura, kanseri y'uruhago.kanseri, kanseri y'impyiko, kanseri ya prostate, kanseri y'ibihaha, kanseri y'inda, lymphosarcoma, n'ibindi.

(3) Oncorine / Ankerui

Isosiyete: Yatejwe imbere na Shanghai Sunway Biotechnology Co., Ltd.

Igihe cyo kwisoko: Yemejwe kurutonde mubushinwa muri 2005.

Ibyerekana: Kuvura ibibyimba byo mu mutwe no mu ijosi, kanseri y'umwijima, kanseri y'urwagashya, kanseri y'inkondo y'umura n'izindi kanseri.

Icyitonderwa: Oncorine nigicuruzwa cya virusi ya oncolytike ikoresheje adenovirus nka vector.Indwara ya oncolytic adenovirus irashobora kwigana cyane cyane mubibyimba bidafite cyangwa p53 idasanzwe, bigatera lysis selile yibibyimba, bityo bikica selile.utangiza selile zisanzwe.Ibisubizo by’amavuriro byerekana ko Anke Rui afite umutekano n’ingirakamaro ku bibyimba bibi bitandukanye.

(4) Glybera

Isosiyete: Yatejwe imbere na uniQure.

Igihe cyo kwisoko: Byemejwe mu Burayi muri 2012.

Ibyerekana: Kuvura ibura rya lipoprotein (LPLD) hamwe nibice bikabije cyangwa bigaruka kenshi bya pancreatite nubwo indyo yuzuye ibinure.

Ijambo: Glybera (alipogene tiparvovec) numuti wo kuvura gene ushingiye kuri AAV nka vector.Ubu buvuzi bukoresha AAV nk'ubuvuzi bwo kwimura gene LPL ivura mu ngirangingo z'imitsi, kugira ngo ingirabuzimafatizo zijyanye nazo zishobora gutanga urugero rwa lipoprotein lipase, Ifite uruhare mu kugabanya indwara, kandi ubu buvuzi bukora igihe kirekire nyuma y'ubuyobozi bumwe (ingaruka zishobora kumara imyaka myinshi).Uyu muti washyizwe ku rutonde muri 2017, kandi impamvu zo kuwushyira ku rutonde zishobora kuba zifitanye isano n’ibintu bibiri: ibiciro biri hejuru cyane n’isoko ridakenewe ku isoko.Ikigereranyo cyo kuvura imiti imwe igera kuri miliyoni imwe y'amadolari y'Amerika, kandi umurwayi umwe ni we waguze kandi arakoresha kugeza ubu.Nubwo isosiyete y'ubwishingizi bw'ubuvuzi yishyuye amadorari 900.000 y'Abanyamerika, ni n'umutwaro munini ku kigo cy'ubwishingizi.Byongeye kandi, kwerekana imiti ni gake cyane, aho usanga abantu bagera kuri 1 kuri miliyoni imwe n’igipimo kinini cyo kwisuzumisha nabi.

(5) Imlygic

Isosiyete: Yakozwe na Amgen.

Igihe cyo kwisoko: Muri 2015, hemejwe gutondekanya muri Amerika no mu muryango w’ubumwe bw’ibihugu by’Uburayi.

Ibyerekana: Kuvura ibikomere bya melanoma bidashobora gukurwaho burundu no kubagwa.

Ijambo: Imlygic ni ihindurwa rya genetike (gusiba ibice byayo bya ICP34.5 na ICP47, no kwinjiza granulocyte-macrophage colony-itera ibintu GM-CSF muri virusi) byanduye virusi ya herpes simplex virusi ya 1 (HSV-1) virusi ya oncolytic.Uburyo bwo kuyobora ni inshinge zo mu nda.Gutera mu buryo butaziguye ibikomere bya melanoma birashobora gutera guturika kw'ibibyimba no kurekura antigene zikomoka ku bibyimba na GM-CSF kugira ngo bitume umubiri urwanya indwara.

(6) Luxturna

Isosiyete: Yatejwe imbere na Spark Therapeutics, ishami rya Roche.

Igihe cyo kwisoko: Byemejwe na FDA muri 2017, hanyuma byemezwa kwamamaza muburayi muri 2018.

Ibyerekana: Kubuvuzi bwabana nabakuze bafite ikibazo cyo kutabona bitewe na mutation muri kopi ebyiri za gene RPE65 ariko hamwe numubare uhagije wingirabuzimafatizo zifatika.

Icyitonderwa: Luxturna nubuvuzi bwa AAV bushingiye ku gutera inshinge.Ubuvuzi bwa gene bukoresha AAV2 nk'umwikorezi kugira ngo bumenye kopi ikora ya gene isanzwe ya RPE65 mu ngirabuzimafatizo z'umurwayi, ku buryo ingirabuzimafatizo zijyanye na zo zigaragaza poroteyine isanzwe ya RPE65 kugira ngo yishyure inenge ya poroteyine ya RPE65 y'umurwayi, bityo imitekerereze ye irusheho kuba myiza.

(7) Zolgensma

Isosiyete: Yatejwe imbere na AveXis, ishami rya Novartis.

Igihe cyo kwisoko: Byemejwe na FDA muri Gicurasi 2019.

Ibyerekana: Kuvura abarwayi b'umugongo atrophyi (Spinal Muscular Atrophy, SMA) abarwayi bari munsi yimyaka 2.

Ijambo: Zolgensma nubuvuzi bwa gene bushingiye kuri vector ya AAV.Uyu muti niyo gahunda yonyine yo kuvura inshuro imwe yo kuvura imitsi yumugongo yemewe yo kwamamaza ku isi.urupapuro, ni intambwe yiterambere.Ubu buvuzi bwa gene bukoresha virusi ya scAAV9 kugirango yinjize abarwayi basanzwe ba SMN1 binyuze mu kwinjiza imitsi, itanga poroteyine isanzwe ya SMN1, bityo bitezimbere imikorere ya selile zanduye nka neuron ya moteri.Ibinyuranye n'ibyo, imiti ya SMA Spinraza na Evrysdi isaba kunywa inshuro nyinshi mugihe kirekire, Spinraza ikaba yaratewe inshinge zumugongo buri mezi ane, na Evrysdi, ibiyobyabwenge kumanwa.

(8) Kureka

Isosiyete: Yatejwe imbere na Daiichi Sankyo Company Limited (TYO: 4568).

Igihe cyo kwisoko: Icyemezo cya minisiteri yubuzima, umurimo n’imibereho myiza y’Ubuyapani (MHLW) muri Kamena 2021.

Ibyerekana: Kubuvuzi bwa glioma mbi.

Icyitonderwa: Delytact nigicuruzwa cya kane cya virusi ya oncolytike ivura imiti yemewe ku isi yose kandi nigicuruzwa cya mbere cya virusi ya oncolytike cyemewe kuvura glioma mbi.Delytact ni virusi ya herpes simplex virusi yo mu bwoko bwa 1 (HSV-1) virusi ya oncolytike yakozwe na Dr. Todo na bagenzi be.Delytact yinjiza ihinduka ryimiterere ya genoside ya G207 yo mu gisekuru cya kabiri HSV-1, ikongerera uburyo bwo guhitamo ingirabuzimafatizo za kanseri no kwinjiza imiti irwanya ibibyimba, mu gihe ikomeza umutekano w’umutekano.Delytact niyambere yambere ya gatatu oncolytike HSV-1 kurubu mugusuzuma amavuriro.Iyemezwa rya Delytact mu Buyapani ryashingiye ku kigeragezo kimwe cy’icyiciro cya 2 cy’amavuriro.Ku barwayi barwaye glioblastoma isubirwamo, Delytact yujuje iherezo ryibanze ryo kubaho umwaka umwe, kandi ibisubizo byagaragaje ko Delytact yitwaye neza kurusha G207.Kwigana gukomeye nibikorwa birwanya antitumor.Ibi bigira akamaro mubyitegererezo byibibyimba birimo amabere, prostate, schwannoma, nasofaryngeal, hepatocellular, colorectal, malignant peripheral nerv sheath tumers na kanseri ya tiroyide.

(9) Upstaza

Isosiyete: Yakozwe na PTC Therapeutics, Inc. (NASDAQ: PTCT).

Igihe cyo kwisoko: Byemejwe na EU muri Nyakanga 2022.

Icyerekana: Kubura Aromatic L-amino acide decarboxylase (AADC), yemerewe kuvura abarwayi amezi 18 nayirenga.

Ijambo: Upstaza ™ (eladocagene exuparvovec) nubuvuzi bwa vivo gene ukoresheje virusi ya adeno ifitanye isano na 2 (AAV2) nka vector.Umurwayi ararwaye kubera ihinduka ryimiterere ya gene irimo enzyme ya AADC.AAV2 itwara gene nzima irimo enzyme ya AADC.Ingaruka zo kuvura zigerwaho muburyo bwo kwishyurwa.Mubyigisho, ikinini kimwe gifite akamaro mugihe kirekire.Nibintu byambere byashyizwe ahagaragara imiti ivura ubwonko.Uruhushya rwo kwamamaza rurakoreshwa mu bihugu 27 bigize Umuryango w’ubumwe bw’ibihugu by’Uburayi, ndetse na Islande, Noruveje na Liechtenstein.

(9) Roctavian

Isosiyete: Yakozwe na BioMarin Pharmaceutical (BioMarin).

Igihe cyo kwisoko: Byemejwe na EU muri Kanama 2022.

Ibyerekana: Kubuvuzi bw'abarwayi bakuze bafite hemofilia ikabije A idafite amateka yo kubuza FVIII na antibody mbi ya AAV5.

Ijambo: Roctavian (valoctocogene roxaparvovec) ikoresha AAV5 nka vector kandi ikoresha umwirondoro wihariye wumwijima wumuntu HLP kugirango igaragaze imvugo yibintu byabantu umunani (FVIII) hamwe na B yasibwe.Icyemezo cya komisiyo y’ibihugu by’i Burayi cyo kwemeza ibicuruzwa bya valoctocogene roxaparvovec bishingiye ku mibare rusange ya gahunda yo guteza imbere imiti ivura imiti.Muri byo, icyiciro cya gatatu cy’amavuriro GENEr8-1 cyerekanye ko ugereranije n’amakuru y’umwaka mbere yo kwiyandikisha, nyuma yo kwinjiza inshuro imwe ya valoctocogene roxaparvovec, Ibintu byari bifite umuvuduko muke w’amaraso buri mwaka (ABR), bidakunze gukoreshwa cyane na recombinant factor VIII (F8) mu maraso y’umubiri.Nyuma yibyumweru 4 byo kuvurwa, amasomo ya F8 yumwaka hamwe na ABR bisaba kuvurwa yagabanutseho 99% na 84%, itandukaniro rikomeye mubarurishamibare (p <0.001).Umwirondoro w’umutekano wari mwiza, nta ngingo zifite ikibazo cya F8 kibuza, indwara mbi, cyangwa ingaruka ziterwa na trombotique, kandi nta kibazo kijyanye no kuvura (SAEs) cyigeze kivugwa.

3. Imiti mito ya nucleic aside

(1) Vitravene

Isosiyete: Yatejwe imbere na Ionis Pharma (ahahoze ari Isis Pharma) na Novartis.

Igihe cyo kwisoko: Byemejwe na FDA na EU EMA muri 1998 na 1999.

Ibyerekana: Kuvura cytomegalovirus retinitis ku barwayi banduye virusi itera SIDA.

Ijambo: Vitravene numuti wa antisense oligonucleotide numuti wa mbere wa oligonucleotide wemejwe kwamamariza kwisi.Mu gutangira isoko, isoko ryasabye imiti irwanya cytomegalovirus yari yihutirwa cyane;noneho kubera iterambere ryimiti igabanya ubukana bwa virusi, umubare wa virusi ya cytomegalovirus wagabanutse cyane.Kubera isoko rike, ibiyobyabwenge byasohotse mu 2002 na 2006 Kuvana mu bihugu by’Uburayi no muri Amerika.

(2) Macugen

Isosiyete: Yafatanije na Pfizer na Eyetech.

Igihe cyo kwisoko: Yemejwe kurutonde muri Amerika muri 2004.

Ibyerekana: Kubuvuzi bwimitsi yimitsi ijyanye na macula degeneration.

Icyitonderwa: Macugen numuti wahinduwe na oligonucleotide wa pegylated ushobora kwibasira no guhuza ibintu bikura byimitsi (VEGF165 isoform), kandi bigakorwa no gutera inshinge.

(3) Defitelio

Isosiyete: Yakozwe na Jazz.

Igihe cyo kwisoko: Byemejwe n’umuryango w’ubumwe bw’ibihugu by’Uburayi muri 2013, kandi byemejwe na FDA muri Werurwe 2016.

Ibyerekana: Kugirango bavure indwara ya hepatike venule idasanzwe ifitanye isano no gukora impyiko cyangwa ibihaha nyuma yo guterwa ingirabuzimafatizo ya hematopoietic.

Ijambo: Defitelio numuti wa oligonucleotide, uruvange rwa oligonucleotide hamwe na plasmin.Yakuweho muri 2009 kubera impamvu z'ubucuruzi.

(4) Kynamro

Isosiyete: Yafatanije na Ionis Pharma na Kastle.

Igihe cyo kwisoko: Byemejwe muri Amerika nkumuti wimfubyi muri 2013.

Ibyerekana: Kubuvuzi bujyanye na homosexous familial hypercholesterolemia.

Ijambo: Kynamro numuti wa antisense oligonucleotide, antisense oligonucleotide yibasira abantu apo B-100 mRNA.Kynamro itangwa nka 200 mg munsi yubutaka rimwe mu cyumweru.

(5) Spinraza

Isosiyete: Yakozwe na Pharmaceuticals ya Ionis.

Igihe cyo kwisoko: Byemejwe na FDA mu Kuboza 2016.

Ibyerekana: Kubuvuzi bwimitsi yumugongo (SMA).

Ijambo: Spinraza (nusinersen) numuti wa antisense oligonucleotide.Spinraza irashobora guhindura RNA itera gene ya SMN2 muguhuza urubuga rwa SMN2 exon 7, bityo bikongera umusaruro wa proteine ​​ikora neza.Muri Kanama 2016, BIOGEN Corporation yakoresheje uburyo bwo kubona uburenganzira bwa Spinraza ku isi.Spinraza yatangiye igeragezwa ryayo rya mbere ry’amavuriro mu bantu mu mwaka wa 2011. Mu myaka 5 gusa, yemejwe na FDA mu 2016, byerekana ko FDA yemeye neza imikorere yayo.Uyu muti wemejwe gucuruzwa mu Bushinwa muri Mata 2019. Inzira zose zemewe za Spinraza mu Bushinwa ntizirenza amezi 6.Hari hashize imyaka 2 n'amezi 2 Spinraza yemerewe bwa mbere muri Amerika.Indwara nkiyi yo mu mahanga indwara idasanzwe imiti mishya iri mu muvuduko wo gutondekanya mu Bushinwa umaze kwihuta cyane.Ibi kandi biterwa n "" Itangazo ryerekeye Gusohora Urutonde rwa mbere rw’ibiyobyabwenge byo mu mahanga bikenewe byihutirwa mu bushakashatsi bw’ubuvuzi "rwatanzwe n’ikigo gishinzwe gusuzuma ibiyobyabwenge ku ya 1 Ugushyingo 2018, rwashyizwe mu cyiciro cya mbere cy’imiti 40 y’amahanga y’ibanze kugira ngo isuzumwe byihuse, kandi Spinraza iri ku mwanya wa mbere.

(6) Exondys 51

Isosiyete: Yakozwe na AVI BioPharma (nyuma yiswe Sarepta Therapeutics).

Igihe cyo kwisoko: Byemejwe na FDA muri Nzeri 2016.

Ibyerekana: Kubuvuzi bwa Duchenne muscular dystrofiya (DMD) hamwe na DMD ihindagurika muri exon 51 gusimbuka gene.

Ijambo: Exondys 51 numuti wa antisense oligonucleotide.Antisense oligonucleotide irashobora guhuza na exon 51 ya pre-mRNA ya gene DMD, bikavamo gushiraho mRNA ikuze.Gusuzumwa, bityo bikosora igice cyo gusoma mRNA cyo gusoma, bifasha umurwayi guhuza uburyo bumwebumwe bukora bwa dystrofine bugufi kuruta poroteyine isanzwe, bityo bikazamura ibimenyetso byumurwayi.

(7) Tegsedi

Isosiyete: Yakozwe na Pharmaceuticals ya Ionis.

Igihe cyo kwisoko: Byemejwe n’ubumwe bw’ibihugu by’i Burayi byo kwamamaza muri Nyakanga 2018.

Ibyerekana: Kubuvuzi bwa hereditori transthyretin amyloidose (HATTR).

Ijambo: Tegsedi numuti wa antisense oligonucleotide yibasira transthyretin mRNA.Nibiyobyabwenge byambere byemewe kwisi kuvura HATTR.Uburyo bwo kuyobora ni inshinge zidasanzwe.Uyu muti ugabanya umusaruro wa poroteyine ya ATTR yibasira mRNA ya transthyretine (ATTR), kandi ifite igipimo cyiza-cyiza cyo kuvura ATTR.Ntabwo icyiciro cyindwara cyangwa kubaho kwa cardiomyopathie byari ngombwa.

(8) Onpattro

Isosiyete: Yafatanije na Alnylam na Sanofi.

Igihe cyo kwisoko: Yemejwe kurutonde muri Amerika muri 2018.

Ibyerekana: Kubuvuzi bwa hereditori transthyretin amyloidose (HATTR).

Ijambo: Onpattro ni imiti ya siRNA yibasira transthyretine mRNA, igabanya umusaruro wa poroteyine ya ATTR mu mwijima no kwegeranya amiloide yabitswe mu mitsi ya periferique yibasira mRNA ya transthyretine (ATTR)., bityo kunoza no kugabanya ibimenyetso byindwara.

(9) Givlaari

Isosiyete: Yatejwe imbere na Alnylam Corporation.

Igihe cyo kwisoko: Byemejwe na FDA mu Gushyingo 2019.

Ibyerekana: Kubuvuzi bukabije bwa hepatike porphyria (AHP) kubantu bakuru.

Ijambo: Givlaari numuti wa siRNA, ikiyobyabwenge cya kabiri cya siRNA cyemewe kwamamaza nyuma ya Onpattro.Umuti utangwa mu buryo butagaragara kandi ugamije mRNA yo kwangirika kwa poroteyine ALAS1.Buri kwezi kuvura Givlaari birashobora kugabanya cyane kandi bikomeje kugabanya urugero rwa ALAS1 mu mwijima, bityo bikagabanya urugero rwa neurotoxic ALA na PBG kurwego rusanzwe, bityo bikagabanya ibimenyetso byindwara zumurwayi.Amakuru yerekanaga ko abarwayi bavuwe na Givlaari bagabanutseho 74% umubare w’umuriro ugereranije n’itsinda rya placebo.

(10) Vyondys53

Isosiyete: Yateguwe na Sarepta Therapeutics.

Igihe cyo kwisoko: Byemejwe na FDA mu Kuboza 2019.

Icyerekana: Kubuvuzi bw'abarwayi ba DMD bafite dystrofine gene exon 53 gucamo ibice.

Ijambo: Vyondys 53 numuti wa antisense oligonucleotide.Imiti ya oligonucleotide yibasira uburyo bwo gutera dystrofine mRNA ibanziriza.Muburyo butaziguye bwa dystrofine mRNA ibanziriza, Exon 53 yo hanze yasohotse igice, ni ukuvuga ko itagaragara kuri mRNA ikuze, kandi yari igamije gukora poroteyine ya dystrofine yaciwe ariko iracyakora, bityo ubushobozi bwimyitozo ngororamubiri ku barwayi.

(11) Waylivra

Isosiyete: Yatejwe imbere na Pharmaceuticals ya Ionis hamwe n’ishami ryayo Akcea Therapeutics.

Igihe cyo kwisoko: Byemejwe n’ikigo cy’ubuvuzi cy’uburayi (EMA) muri Gicurasi 2019.

Kwerekana: Nubuvuzi bujyanye nimirire igenzurwa kubarwayi bakuze barwaye syndrome ya familial chylomicronemia (FCS).

Ijambo: Waylivra ni imiti igabanya ubukana bwa oligonucleotide, ikaba ariwo muti wa mbere wemejwe kuvura FCS ku isi.

(12) Leqvio

Isosiyete: Yakozwe na Novartis.

Igihe cyo kwisoko: Byemejwe na EU mu Kuboza 2020.

Ibyerekana: Kubuvuzi bwa hypercholesterolemia yabantu bakuru (heterozygous familial and non familial) cyangwa dyslipidemiya ivanze.

Ijambo: Leqvio numuti wa siRNA wibasira PCSK9 mRNA.Nibwo bwa mbere ku isi bugabanya cholesterol (LDL-C) siRNA ivura.Uburyo bwo kuyobora ni inshinge zidasanzwe.Ibiyobyabwenge bikorana na RNA kugirango igabanye urugero rwa poroteyine PCSK9, nayo igabanya urugero rwa LDL-C.Amakuru y’amavuriro yerekana ko Leqvio ishobora kugabanya LDL-C hafi 50% ku barwayi bafite LDL-C idashobora kugabanuka kugera ku ntego nubwo umubare wa statine wihanganirwa.

(13) Oxlumo

Isosiyete: Yakozwe na Pharmaceuticals ya Alnylam.

Igihe cyo kwisoko: Byemejwe na EU mu Gushyingo 2020.

Ibyerekana: Kubuvuzi bwa hyperoxaluria yibanze ubwoko bwa 1 (PH1).

Ijambo: Oxlumo numuti wa siRNA yibasira hydroxy aside oxydease 1 (HAO1) ​​mRNA, itangwa muburyo bwihuse.Uyu muti wakozwe hifashishijwe uburyo bwa Alnylam bwa kijyambere bwongerewe imbaraga bwo gukoresha imiti ya ESC-GalNAc ikorana buhanga, ifasha siRNAs mu buryo bwihuse hamwe no gukomera no gukora neza.Uyu muti ugamije kwangirika cyangwa kubuza aside aside hydroxy 1 (HAO1) ​​mRNA, igabanya urugero rwa okiside ya glycolate mu mwijima, hanyuma ikarya insimburangingo isabwa kugira ngo ikore oxalate kandi igabanye umusaruro wa oxalate kugira ngo igenzure iterambere ry’indwara no kunoza ibimenyetso by’indwara ku barwayi.

(14) Viltepso

Isosiyete: Yatejwe imbere na NS Pharma, ishami rya Nippon Shinyaku.

Igihe cyo kwisoko: Byemejwe na FDA muri Kanama 2020.

Ibyerekana: Kubuvuzi bwa Duchenne muscular dystrofiya (DMD) hamwe na mutation ya DMD muri exon 53 gusimbuka gene.

Ijambo: Viltepso numuti wa fosiforiodiamide morpholino oligonucleotide.Uyu muti wa oligonucleotide urashobora guhuza na exon 53 ya pre-mRNA ya gene DMD, bigatuma habaho mRNA ikuze.Exon yakuweho igice, bityo ikosora igice cyo gusoma mRNA, ifasha umurwayi guhuza uburyo bumwe na bumwe bwa dystrofine bugufi kuruta poroteyine isanzwe, bityo bikazamura ibimenyetso byumurwayi.

(15) Amvuttra (vutrisiran)

Isosiyete: Yakozwe na Pharmaceuticals ya Alnylam.

Igihe cyo kwisoko: Byemejwe na FDA muri kamena 2022.

Ibyerekana: Kubuvura abakuze barazwe transthyretin amyloidose hamwe na polyneuropathie (HATTR-PN).

Ijambo: Amvuttra (Vutrisiran) numuti wa siRNA wibasira transthyretine (ATTR) mRNA, itangwa no gutera inshinge.Vutrisiran yateguwe hashingiwe kuri Alnylam's Enhanced Stabilization Chemistry (ESC) -GalNAc ihujwe no gutanga imbaraga hamwe no kongera imbaraga no guhindagurika.Kwemeza ubwo buvuzi bishingiye ku mibare y’amezi 9 yavuye mu bushakashatsi bw’ubuvuzi bwa Phase III (HELIOS-A), ibisubizo muri rusange byerekana ko ubuvuzi bwateje imbere ibimenyetso bya HATTR-PN, abarwayi barenga 50% basubira inyuma cyangwa bahagarika iterambere.

4. Indi miti ivura gene

(1) Rexin-G

Isosiyete: Yakozwe na Epeius Biotech.

Igihe cyo kwisoko: Byemejwe n’ubuyobozi bushinzwe ibiryo n’ibiyobyabwenge bya Philippine (BFAD) muri 2005.

Ibyerekana: Kubuvuzi bwa kanseri yateye imbere irwanya chimiotherapie.

Ijambo: Rexin-G ni inshinge ya gene yuzuye inshinge.Yinjiza cyclin G1 mutant gene mungirangingo ikoresheje virusi itera virusi kugirango yice ibibyimba bikomeye.Uburyo bwo kuyobora ni kwinjiza imitsi.Nkumuti wibasiwe nibibyimba ushakisha kandi ukangiza ingirabuzimafatizo za kanseri metastatike, bigira ingaruka runaka kubarwayi badashobora kurwanya indi miti ya kanseri, harimo na biologiya yibasiwe.

(2) Neovasculgen

Isosiyete: Yatejwe imbere na stem stem selile.

Igihe cyo gutondeka: Byemejwe gutondekanya mu Burusiya ku ya 7 Ukuboza 2011, hanyuma bikandikwa muri Ukraine muri 2013.

Ibyerekana: Kubuvuzi bwindwara ya arterial periferique, harimo ischemia ikomeye.

Icyitonderwa: Neovasculgen nubuvuzi bwa ADN bushingiye ku mikorere ya ADN aho imiterere yo gukura kw'imitsi y'amaraso (VEGF) 165 yubatswe ku mugongo wa plasmid hanyuma ikinjizwa mu barwayi.

(3) Collategene

Isosiyete: Yafatanije na kaminuza ya Osaka hamwe n’ibigo bishora imari.

Igihe cyo gutondekanya: Byemejwe na minisiteri yubuzima, umurimo n’imibereho myiza y’Ubuyapani kurutonde muri Kanama 2019.

Ibyerekana: Kuvura ischemia yo hepfo cyane.

Ijambo: Collategene nubuvuzi bushingiye kuri plasmide, imiti yambere yubuvuzi bwa kiyapani kavukire yakozwe na AnGes.Ibyingenzi bigize uyu muti ni plasmid yambaye ubusa irimo gene ikurikirana ya hepatocyte yumuntu ikura (HGF).Niba imiti yatewe mumitsi yingingo zo hepfo, HGF yerekanwe izateza imbere imitsi mishya y'amaraso ikikije imiyoboro y'amaraso ifunze.Igeragezwa rya Clinical ryemeje akamaro karyo mugutezimbere ibisebe.

IHEREZO