Dhawaan, saddex daawaynta hidda-wadaha ayaa loo oggolaaday suuqgeynta, kuwaas oo kala ah: (1) Bishii Luulyo 21, 2022, PTC Therapeutics, Inc. loo oggolaaday suuqgeyn).(2) Agoosto 17, 2022, Maamulka Cuntada iyo Dawooyinka ee Mareykanka (FDA) ayaa oggolaaday daawaynta hidda-wadaha Bluebird Bio ee Zynteglo (betibeglogene autotemcel, beti-cel) ee daawaynta beta thalassaemia.Oggolaanshaha daawaynta ee Maraykanka ayaa shaki la'aan ah "caawinta barafka" ee Bluebird Bio, oo ku jirta xiisad dhaqaale.(3) Agoosto 24, 2022, BioMarin Pharmaceutical (BioMarin) ayaa ku dhawaaqday in Komishanka Yurub uu ansixiyay suuqgeynta shuruudda ah ee ROCTAVIAN ™ (valoctocogene roxaparvovec), daawaynta hidda-wadaha ee hemophilia A, ee daaweynta bukaannada aan lahayn taariikh FVIII factor inhibitors iyo bukaannada AAV5 ee hore ee antibom philia Daaweynta hidda-wadaha loo oggolaaday suuqgeyn).Ilaa hadda, 41 daawaynta hidda-wadaha ayaa loo oggolaaday suuqgeynta adduunka oo dhan.

Hidde waa unugga hidde-sidaha aasaasiga ah ee xakameeya sifooyinka.Marka laga reebo hiddo-wadaha fayrasyada qaarkood, kuwaas oo ka kooban RNA, hidde-sideyaasha noolaha intooda badan waxay ka kooban yihiin DNA.Cudurada ugu badan ee noolaha waxaa sababa isdhexgalka ka dhexeeya hidde-sideyaasha iyo deegaanka, cudurro badan ayaa lagu daweyn karaa ama lagu yareyn karaa nuxur ahaan iyada oo loo marayo daaweynta hidda-socodka.Daaweynta hidda-wadaha waxaa loo arkaa inay tahay kacaan dhanka daawada iyo farmashiyaha.Daawooyinka daaweynta hidda-wadaha ee ballaadhan waxa ka mid ah dawooyin ku salaysan dawooyinka DNA-ga wax laga beddelay (sida fayraska ku-salaysan dawooyinka hidda-wadaha ee vivo, dawooyinka daawaynta hidda-wadaha ee vitro, dawooyinka plasmid-ka qaawan, iwm.) iyo dawooyinka RNA (sida dawooyinka antisense oligonucleotide, daawooyinka siRNA, iyo daawaynta hidda-wadaha mRNA, iwm.);si cidhiidhi ah loo qeexay dawooyinka daawaynta hidda-wadaha inta badan waxa ka mid ah dawooyinka plasmid DNA-ga, daawooyinka hidda-wadaha ee ku salaysan fayraska, dawooyinka daawaynta hidda-wadaha ee ku salaysan faleebyada bakteeriyada, hababka tafatirka hidda-wadaha iyo dawooyinka daawaynta unugyada kuwaas oo hidde ahaan wax looga beddelay gudaha vitro.Ka dib sannado badan oo horumar ah, dawooyinka daawaynta hidda-wadaha waxay gaadheen natiijooyin caafimaad oo dhiirrigelin leh.(marka laga reebo tallaallada DNA-da iyo tallaallada mRNA), 41 dawooyinka daawaynta hidda-wadaha ayaa loo oggolaaday suuqgeynta adduunka.Iyada oo la bilaabay alaabada iyo horumarinta degdega ah ee tignoolajiyada daaweynta hidda-wadaha, daawaynta hidda-wadaha waxa ay ku dhowdahay in ay keento xilli horumar degdeg ah.

Kala soocida daaweynta hidda-socodka (Isha sawirka: Biological Jingwei)

Maqaalkani waxa uu taxayaa 41 daawaynta hidda-socodka ah oo loo oggolaaday suuq-geyn (marka laga reebo tallaallada DNA-da iyo tallaallada mRNA).

1. Daaweynta hidda-wadaha ee vitro

(1) Strimvelis

Shirkadda: Waxaa soo saartay GlaxoSmithKline (GSK).

Waqtiga suuqa: Waxaa ansixiyay Midowga Yurub bishii Maajo 2016.

Tilmaamaha: Loogu talagalay daawaynta difaac la'aanta isku-darka daran (SCID).

Faallooyinka: Habka guud ee daawayntan waa in marka hore la helo unugyada tarma ee hematopoietic ee bukaanka, la balaadhiyo oo lagu dhaqo gudaha vitro, ka dibna la isticmaalo retrovirus si ay u soo bandhigto nuqul ka mid ah hidda-wadaha ADA (adenosine deaminase) ee shaqaynaya unugyadooda tarma ee hematopoietic, oo ugu dambayntii lagu wareejiyo unugyada tarma ee hematopoietic ee la beddelay.Unugyada tarma ee hematopoietic ayaa dib loogu soo celiyaa jirka.Natiijooyinka caafimaadku waxay muujiyeen in heerka badbaadada 3-sano ee bukaannada ADA-SCID ee lagu daweeyay Strimvelis ay ahayd 100%.

(2) Zalmoxis

Shirkadda: Waxaa soo saaray MolMed, Italy.

Waqtiga suuqa: La helay oggolaanshaha suuqgeynta shuruudda EU ee 2016.

Tilmaamaha: Waxaa loo isticmaalaa daaweynta adjuvant ee habka difaaca jirka ee bukaanada ka dib tallaalka unugyada tarma ee hematopoietic.

Fiiro gaar ah: Zalmoxis waa unug-garabeed allogeneic T-ga is-dilka hidde-socodka difaaca jirka oo ay wax ka beddeleen vector retroviral.1NGFR iyo HSV-TK Mut2 hiddo-wadaha is-dilka ayaa u oggolaanaya dadka inay isticmaalaan ganciclovir wakhti kasta si ay u dilaan unugyada T ee keena jawaabaha difaaca ee xun, ka hortagga sii xumaanshaha GVHD ee laga yaabo inay dhacaan, iyo soo celinta shaqada difaaca ee bukaanada leh HSCT haploidentical ka dib qalliinka Escort.

(3) Invossa-K

Shirkadda: Waxaa soo saartay shirkadda TissueGene (KolonTissueGene).

Waqtiga la suuqgeyn karo: Waa la oggolaaday liiska Koonfurta Kuuriya Luulyo 2017.

Tilmaamaha: Loogu talagalay daawaynta arthritis-ka jilibka ee xumaaday.

Xusuusin: Invossa-K waa daaweynta hidde-sideyaasha unugyada allogeneic ee ku lug leh chondrocytes aadanaha.Unugyada Allogeneic waxaa lagu beddelaa hidde ahaan in vitro, unugyada la beddelayna waxay muujin karaan oo ay qarin karaan isbeddelka isbeddelka koritaanka β1 (TGF-β1) ka dib duritaanka intra articular.β1), si loo hagaajiyo calaamadaha osteoarthritis.Natiijooyinka caafimaadku waxay muujinayaan in Invossa-K ay si weyn u wanaajin karto arthritis-ka jilibka.Ruqsadda waxaa kala noqday maandooriyaha Kuuriyada Koonfureed 2019 sababtoo ah soo-saareyaashu waxay si khaldan u calaamadiyeen maaddooyinka la isticmaalay.

(4) Zynteglo

Shirkadda: Waxaa soo saartay shirkadda American bluebird bio (bluebird bio).

Waqtiga suuqa: Waxaa ansixiyay Midowga Yurub 2019, oo ay ansixisay FDA Agoosto 2022.

Tilmaamaha: Daaweynta ku-tiirsanaanta β-thalassaemia.

Xusuusin: Zynteglo waa daawaynta hidda-wadaha lentiviral ee vitro, taas oo adeegsata vector lentiviral si ay u soo bandhigto nuqul shaqeynaya hiddo-wadaha β-globin ee caadiga ah (βA-T87Q-globin gene) oo galay unugyada tarma ee hematopoietic ee laga saaray bukaannada., ka dibna ku geli kuwan hiddo-wadaha la beddelay ee unugyada tarma ee hematopoietic autologous dib ugu geli bukaanka.Marka bukaanku helo hiddo-wadaha βA-T87Q-globin ee caadiga ah, waxa laga yaabaa inay soo saaraan borotiinka HbAT87Q ee caadiga ah, kaas oo si wax ku ool ah u yarayn kara ama meesha ka saari kara baahida dhiig-shubidda.Waa daawayn hal mar ah oo loogu talagalay in lagu beddelo dhiig ku shubista nolosha oo dhan iyo daawaynta cimri-dhererka bukaanka 12 sano iyo ka weyn.

(5) Skysona

Shirkadda: Waxaa soo saartay shirkadda American bluebird bio (bluebird bio).

Waqtiga la suuqgeyn karo: Waxaa ogolaaday EU suuqgeyntiisa Luulyo 2021.

Tilmaamaha: Daawaynta hore ee adrenoleukodystrophy cerebral (CALD).

Xusuusin: Daaweynta hidda-wadaha Skysona waa daawaynta hidda-wadaha hal mar kaliya ee loo oggolaaday daawaynta adrenoleukodystrophy hore ee maskaxda (CALD).Skysona (elivaldogene autotemcel, Lenti-D) waa unug hematopoietic stem lentiviral in vitro therapy therapy Lenti-D.Habka guud ee daawadu waa sida soo socota: Unugyada tarma ee hematopoietic autologous ayaa laga soo saaraa bukaanka, wax ka beddelka in vitro by lentivirus oo sidda hidda-wadaha ABCD1, ka dibna dib loogu celiyo bukaanka.Daawaynta bukaanada da'doodu ka yar tahay 18 jir ee leh ABCD1 beddelka hidda-wadaha iyo CALD.

(6) Kymriah

Shirkadda: Waxaa soo saaray Novartis.

Waqtiga suuqa: Waxaa ogolaatay FDA Agoosto 2017.

Tilmaamaha: Daawaynta hore ee unugyada B-cell acute lymphoblastic leukemia (ALL) iyo soo noqoshada iyo dib u soo noqoshada DLBCL.

Fiiro gaar ah: Kymriah waa daawada lentiviral in vitro hiddo-wadaha therapy, daawaynta CAR-T ee ugu horeysay adduunka ee la ansixiyay, lagu beegsanayo CD19 iyo iyadoo la isticmaalayo factor-stimulatory factor 4-1BB.Qiimuhu waa $475,000 gudaha Maraykanka iyo $313,000 gudaha Japan.

(7)Yescarta

Shirkadda: Waxaa soo saartay Kite Pharma, oo hoos timaada Gilecaad.

Waqtiga suuqa: Waxaa oggolaaday FDA Oktoobar 2017.

Tilmaamaha: Daawaynta soo laabashada ama refractory lymfoma B-cell weyn.

Xusuusin: Yescarta waa daawaynta hidda-sidaha ee fayraska.Waa daawaynta CAR-T ee labaad ee laga ansixiyey aduunka.Waxay beegsataa CD19 waxayna isticmaashaa qodobka qiimaha CD28.Qiimaha Maraykanka waa $373,000.

(8) Tecartus

Shirkadda: Waxaa soo saartay Gilecaad (GILD).

Waqtiga suuqa: Waxaa ogolaatay FDA Luulyo 2020.

Tilmaamaha: Limfoma unugga mantle ee soo noqnoqday ama soo noqnoqda.

Faallooyinka: Tecartus waa daawaynta unug ee CAR-T oo iskeed u gaar ah oo lagu beegsanayo CD19, waana daawaynta CAR-T ee saddexaad ee loo oggolaaday suuqgeynta adduunka.

(9) Breyanzi

Shirkadda: Waxaa soo saartay Bristol-Myers Squibb (BMS).

Waqtiga suuqa: Waxaa ogolaatay FDA Febraayo 2021.

Tilmaamaha: Soo noqoshada ama dib-u-celinta (R/R) lymphoma B-cell (LBCL).

Faallooyinka: Breyanzi waa daawaynta hidda-sidaha in vitro ee ku salaysan lentivirus, iyo daawaynta afraad ee CAR-T ee loo oggolaaday suuqgeynta adduunka, laguna beegsanayo CD19.Oggolaanshaha Breyanzi waa horumar u ah Bristol-Myers Squibb dhanka cilmiga difaaca jirka, taasoo Bristol-Myers ay heshay markii ay ku heshay Celgene $74 bilyan sanadka 2019.

(10) Abecma

Shirkadda: Waxaa iska kaashaday Bristol-Myers Squibb (BMS) iyo bluebird bio.

Waqtiga suuqa: Waxaa oggolaaday FDA Maarso 2021.

Tilmaamaha: Myeloma badan oo soo noqnoqda ama soo noqnoqda.

Faallooyin: Abecma waa lentivirus-ku-saleysan daawaynta hidda-sidaha vitro, daawaynta unugga CAR-T ee ugu horreysa adduunka ee lagu beegsanayo BCMA, iyo daawaynta CAR-T ee shanaad ee ay ansixisay FDA.Mabda'a daawadu waa in lagu muujiyo reseptor-ka chimeric BCMA ee unugyada T-da ee iswada ee bukaanka iyada oo loo marayo beddelka hidde-wadeyaasha lentivirus-dhexdhexaadinta gudaha vitro.Ka hor inta aan la galin daawada hidda-wadaha unugyada, bukaanku wuxuu helay laba qaybood oo ah cyclophosphamide iyo fludarabine ee daaweynta hore.Daawaynta in laga saaro unugyada T ee aan la beddelin ee bukaanka, ka dibna lagu shubo unugyada T la beddelay ee bukaanka si ay u raadiyaan oo ay u dilaan BCMA-muujinta unugyada kansarka.

(11) Libmeldy

Shirkadda: Waxaa soo saartay Orchard Therapeutics.

Waqtiga suuqa: Waxaa ogolaaday Midowga Yurub liiska Diseembar 2020.

Tilmaamaha: Loogu talagalay daawaynta leukodystrophy metachromatic (MLD).

Faallooyinka: Libmeldy waa daawayn hidde-side ah oo ku salaysan lentiviral in vitro wax ka beddelka unugyada CD34+ ee iswada leh.Xogta caafimaadku waxay muujinaysaa in faleebo hal xidid ah oo Libmeldy ah ay wax ku ool u tahay wax ka beddelka koorsada bilawga hore ee MLD iyo mootada daran iyo daciifnimada garashada ee bukaannada aan la daweyn ee isku da'da ah.

(12) Benoda

Shirkadda: Waxaa soo saaray WuXi Junuo.

Waqtiga suuqa: Si rasmi ah u ansixisay NMPA Sebtembar 2021.

Tilmaamaha: Daaweynta dib-u-soo-noqoshada ama dib-u-celinta weyn ee B-cell lymphoma (r/r LBCL) ee bukaannada qaangaarka ah ka dib daawaynta nidaamka labaad ama ka badan.

Fiiro gaar ah: Benoda waa daawaynta hidda-sidaha CD19 CAR-T, sidoo kale waa sheyga xudunta u ah WuXi Junuo.Waa badeecadii labaad ee CAR-T ee lagu ansixiyey Shiinaha, marka laga reebo dib-u-soo-noqoshada/refractory B-cell lymphoma.Intaa waxaa dheer, WuXi Junuo ayaa sidoo kale qorsheyneysa in ay horumariso cirbadeynta Ruiki Orenza ee daaweynta calaamadaha kale ee kala duwan, oo ay ku jiraan follicular lymphoma (FL), lymphoma cell mantle (MCL), lymphocytic leukemia (CLL) , line-labaad fidin weyn B-cell lymphoma (DLBCL) iyo lymphoblastic leukemia ba'an (ALL).

(13) CARVYKTI

Shirkadda: Legend Bio alaabtii ugu horreysay ee la oggolaaday.

Waqtiga suuqa: Waxaa ogolaatay FDA Febraayo 2022.

Tilmaamaha: Daawaynta soo noqnoqda ama dib u soo noqoshada myeloma badan (R/R MM).

Fiiro gaar ah: CARVYKTI (ciltacabtagene autoleucel, oo loo yaqaan Cilta-cel) waa daawaynta difaaca unugga CAR-T oo leh laba unug ka hortag ah oo hal meel ah oo lagu beegsanayo antigen-ka maturation unug (BCMA).Xogtu waxay muujineysaa in CARVYKTI ay muujisay heerka jawaab celinta guud ee ilaa 98% bukaanada qaba dib-u-soo-kabashada ama dib-u-celinta multiple myeloma kuwaas oo helay afar ama in ka badan oo daaweyn ah oo hore, oo ay ku jiraan proteasome inhibitors, immunomodulators, iyo anti-CD38 monoclonal antibodies.

2. Daaweynta hidda-wadaha ee Vivo oo ku salaysan fayraska

(1) Jinsi/dhalasho mar kale

Shirkadda: Waxaa soo saartay Shirkadda Shenzhen Saibainuo.

Waqtiga suuqa: Waa la oggolaaday liiska Shiinaha 2003.

Tilmaamaha: loogu talagalay daaweynta kansarka unugyada squamous ee madaxa iyo qoorta.

Faallo: Recombinant human p53 cirbadeynta adenovirus Gendicine/Jinshengsheng waa daawaynta hidda-sidaha adenovirus oo leh xuquuq madax-banaan oo hanti aqooneed oo ay leedahay Shirkadda Shenzhen Saibainuo.Daawadu waxay ka kooban tahay hidda-wadaha p53 ee buro-dabiyeedka caadiga ah iyo qaab-dhismeed dib-u-habeyn ah oo dib-u-celin-dhimasho ah Aadanaha adenovirus nooca 5 wuxuu ka kooban yahay adenovirus aadanaha nooca 5. Kan hore waa qaab-dhismeedka ugu weyn ee daawada si loo sameeyo saamaynta anti-buro, iyo kan dambe inta badan u dhaqmo sida side.Fayraska adenovirus wuxuu qaadaa hiddo-wadaha daaweynta p53 unugga bartilmaameedka ah, wuxuuna muujiyaa hidda-wadaha xakameynaya burada p53 unugga bartilmaameedka ah.Badeecadu waxay hagaajin kartaa hiddo-wadaha ka hortagga kansarka ee kala duwan waxayna hoos-u-dhigi kartaa dhaqdhaqaaqyada unugyada kansarka ee kala duwan, taas oo kor u qaadeysa saameynta ka hortagga burooyinka ee jirka iyo gaaritaanka ujeedada dilka burooyinka.

(2) Rigvir

Shirkadda: Waxaa soo saartay shirkadda Latvia ee Latima.

Waqtiga suuqa: Latvia laga ansixiyay 2004.

Tilmaamaha: Daawaynta melanoma.

Faallooyinka: Rigvir waa daawayn hidde-side ah oo ku salaysan hidda-sidaha la beddelay ee ECHO-7 enterovirus vector, kaas oo laga isticmaalay Latvia, Estonia, Poland, Armenia, Belarus iyo meelo kale, waxaana sidoo kale laga diiwaan gelinayaa EMA ee Midowga Yurub..Kiisaska kiliinikada tobankii sano ee la soo dhaafay waxay caddeeyeen in fayraska Rigvir oncolytic uu yahay badbaado iyo waxtar leh, wuxuuna hagaajin karaa heerka badbaadada bukaanka melanoma 4-6 jeer.Intaa waxaa dheer, daawaynta ayaa sidoo kale ku habboon noocyo kala duwan oo kansar ah, oo ay ku jiraan kansarka mindhicirka, kansarka ganaca, kansarka kaadiheysta.kansarka, kansarka kelyaha, kansarka qanjirka 'prostate', kansarka sanbabada, kansarka ilmo galeenka, lymphosarcoma, iwm.

(3) Oncorine/Ankerui

Shirkadda: Waxaa soo saartay Shanghai Sunway Biotechnology Co., Ltd.

Waqtiga la suuq-geyn karo: Waa la oggolaaday liiska Shiinaha 2005.

Tilmaamaha: Daaweynta burooyinka madaxa iyo qoorta, kansarka beerka, kansarka ganaca, kansarka ilmo-galeenka iyo kansarrada kale.

Fiiro gaar ah: Oncorine waa badeecada daawaynta hidda-wadaha fayraska oncolytic iyadoo la isticmaalayo adenovirus sida fayraska.Adenovirus-ka la helay ee loo yaqaan 'oncolytic adenovirus' wuxuu si gaar ah ugu koobi karaa burooyinka ka maqan ama aan caadiga ahayn ee p53, taasoo keenta lysis unug buro, oo dila unugyada burooyinka.iyada oo aan waxyeello u geysan unugyada caadiga ah.Natiijooyinka caafimaadku waxay muujinayaan in Anke Rui uu leeyahay badbaado wanaagsan iyo waxtarka noocyada kala duwan ee burooyinka halista ah.

(4) Glybera

Shirkadda: Waxaa soo saartay uniQure.

Waqtiga suuqa: Yurub laga ansixiyay 2012.

Tilmaamaha: Daawaynta lipoprotein lipase deficiency (LPLD) oo leh xaalado daran ama soo noqnoqda ee pankreatit inkasta oo ay jirto cunto dufan ah oo si adag loo xaddiday.

Faallooyinka: Glybera (alipogene tiparvovec) waa daawaynta hidda-wadaha oo ku salaysan AAV sida faleebo ahaan.Daaweyntani waxay isticmaashaa AAV sida vector si ay ugu wareejiso hidda-socodka daaweynta LPL unugyada muruqa, si unugyada u dhigma ay soo saari karaan qadar go'an oo ah lipoprotein lipase, Waxay door ka ciyaaraan yareynta cudurrada, iyo daaweyntani waxay waxtar u leedahay waqti dheer ka dib hal maamul (saameyntu waxay socon kartaa sanado badan).Daawada waxaa laga saaray 2017, sababaha meesha looga saarayna waxaa laga yaabaa inay la xiriirto laba arrimood: qiime aad u sarreeya iyo baahida suuqa oo xaddidan.Celcelis ahaan qiimaha hal daawaynta dawadan ayaa gaadhaysa 1 milyan oo doolarka Maraykanka ah, hal bukaan oo kaliya ayaa iibsaday oo isticmaalay ilaa hadda.Inkasta oo shirkadda caymiska caafimaadku ay u soo celisay 900,000 oo dollarka Maraykanka ah, haddana waxay culays weyn ku tahay shirkadda caymiska.Intaa waxaa dheer, calaamadda daawadu waa mid aad dhif u ah, iyada oo heerka dhacdooyinka ee ku saabsan 1 ee 1 milyan iyo heerka sare ee ogaanshaha khaldan.

(5) Macaan

Shirkadda: Waxaa soo saaray Amgen.

Waqtiga suuqa: 2015, waxaa loo ogolaaday liiska Maraykanka iyo Midowga Yurub.

Tilmaamaha: Daawaynta nabarrada melanoma ee aan si buuxda looga saari karin qaliinka.

Fayraska: Imlygic waa hidde ahaan wax laga beddelay (tirtirka jajabkiisa ICP34.5 iyo ICP47, iyo gelinta granulocyte-macrophage colony-stimulating factor GM-CSF ee fayraska) ayaa hoos u dhigay fayraska herpes simplex nooca 1 (HSV-1) fayraska oncolytic, oo ah fayraska ugu horreeya ee FDA-da ah.Habka loo maamulo waa cirbadeynta intralesional.Duritaanka tooska ah ee dhaawacyada melanoma waxay sababi kartaa dillaaca unugyada burooyinka waxayna sii daayaan antigens-ka-soo-baxa buro iyo GM-CSF si kor loogu qaado jawaabaha difaaca-ka-hortagga buro.

(6) Luxturna

Shirkadda: Waxaa soo saartay Spark Therapeutics, oo ah shirkad hoos timaada Roche.

Waqtiga suuqa: Waxaa ogolaatay FDA 2017, ka dibna loo oggolaaday suuqgeynta Yurub 2018.

Tilmaamaha: Daawaynta carruurta iyo dadka waaweyn ee aragga lumay sababtoo ah isbeddellada ku jira nuqulka labanlaabka ah ee hidda-wadaha RPE65 laakiin leh tiro ku filan oo ah unugyo indho-tiran oo macquul ah.

Xusuusin: Luxturna waa daawayn hidde-side ku salaysan AAV kaas oo lagu maamulo duritaanka subretinal.Daaweynta hidda-wadaha waxay isticmaashaa AAV2 sida side si ay u soo bandhigto nuqul shaqeynaya oo ah hiddo-wadaha RPE65 ee caadiga ah ee unugyada retinal ee bukaanka, si unugyada u dhigma ay u muujiyaan borotiinka RPE65 ee caadiga ah si ay u magdhabaan cilladda borotiinka RPE65 ee bukaanka, taas oo hagaajinaysa aragtida bukaanka.

(7) Zolgensma

Shirkadda: Waxaa soo saartay AveXis, oo hoos timaada Novartis.

Waqtiga suuqa: Waxaa ogolaatay FDA May 2019.

Tilmaamaha: Daaweynta atrophy muruqa laf-dhabarta (Spinal Muscular Atrophy, SMA) bukaanada ka yar 2 sano.

Xusuusin: Zolgensma waa daawayn hidde-side ku salaysan AAV vector.Daawadani waa qorshaha kaliya ee daawaynta hal mar ah ee atrophy muruqa laf dhabarta ee loo oggolaaday suuqgeynta adduunka.bogga, waa horumar taariikhi ah.Daaweynta hidda-wadaha Tani waxay isticmaashaa vector-ka scAAV9 si ay u soo bandhigto hidda-wadaha SMN1 ee caadiga ah bukaanka iyada oo loo marayo faleebo xididka, soo saarida borotiinka SMN1 caadiga ah, taas oo kor u qaadeysa shaqada unugyada ay saameeyeen sida dareemayaasha dhaqdhaqaaqa.Taas bedelkeeda, dawooyinka SMA Spinraza iyo Evrysdi waxay u baahan yihiin qiyaaso soo noqnoqda muddo dheer, iyadoo Spinraza loo maamulo cirbad laf dhabarta afartii biloodba mar, iyo Evrysdi, oo ah daroogo afka laga qaato maalin kasta.

(8) Daalimiin

Shirkadda: Waxaa soo saaray Daiichi Sankyo Company Limited (TYO: 4568).

Waqtiga la suuqgeynayo: Oggolaanshaha shuruuda ah ee ka yimid Wasaaradda Caafimaadka, Shaqada iyo Daryeelka ee Japan (MHLW) ee Juun 2021.

Tilmaamaha: Daawaynta glioma malignant.

Faallooyin: Delytact waa badeecada afraad ee daawaynta hidda-wadaha fayraska oncolytic ee la ansixiyey adduunka oo dhan iyo badeecadii ugu horreysay ee fayraska oncolytic ee loo oggolaaday daawaynta glioma malignant.Delytact waa fayraska loo yaqaan 'herpes simplex virus type 1' (HSV-1) oo ay soo saareen Dr. Todo iyo asxaabtiisa.Delytact waxay soo bandhigaysaa isbeddelka tirtirka dheeraadka ah ee G207 genome ee jiilka labaad ee HSV-1, iyada oo kor u qaadaysa ku-noqoshadeeda xulashada ee unugyada kansarka iyo soo-saarka jawaabaha difaaca-ka-hortagga buro, iyada oo la ilaalinayo astaanta badbaadada sare.Delytact waa jiilka saddexaad ee oncolytic HSV-1 ee hadda ku jira qiimeynta bukaan-socodka.Oggolaanshaha Delytact ee Japan waxay ku salaysnayd hal gacan oo waji 2 tijaabo caafimaad ah.Bukaannada qaba glioblastoma oo soo noqnoqda, Delytact waxay la kulantay barta ugu dambeysa ee badbaadada hal sano, natiijaduna waxay muujisay in Delytact ay ka fiican tahay G207.Dib u soo celin xoog leh iyo dhaqdhaqaaqa antitumor sare.Tani waxay wax ku ool u tahay moodooyinka burooyinka adag oo ay ku jiraan naaska, prostate, schwannoma, nasopharyngeal, hepatocellular, colorectal, malignant burooyinka galka dareemaha ee dareemayaasha iyo kansarka tayroodhka.

(9) Upstaza

Shirkadda: Waxaa soo saartay PTC Therapeutics, Inc. (NASDAQ: PTCT).

Waqtiga suuqa: Waxaa ansixiyay Midowga Yurub Luulyo 2022.

Tilmaam: La'aanta L-amino acid decarboxylase (AADC), oo loo oggolaaday daaweynta bukaannada 18 bilood jir iyo ka weyn.

Xusuusin: Upstaza™ (eladocagene exuparvovec) waa daawaynta hidda-sidaha ee vivo iyadoo la isticmaalayo adeno-la-xidhiidha fayraska nooca 2 (AAV2) sida fayraska.Bukaanku wuu bukooday sababta oo ah isbeddel ku yimi hidda-socodka oo dhigaya enzyme AADC.AAV2 waxa uu sido hiddo-wade caafimaad qaba oo codaynaya ensaymka AADC.Saamaynta daaweynta waxaa lagu gaaraa qaabka magdhowga hidda-socodka.Aragti ahaan, hal dose ayaa waxtar leh muddo dheer.Waa daawaynta hiddo-wadaha ee ugu horreeya ee suuqa la galiyo oo si toos ah maskaxda loogu durayo.Oggolaanshaha suuqgeyntu waxay khusaysaa dhammaan 27ka waddan ee xubnaha ka ah EU, iyo sidoo kale Iceland, Norway iyo Liechtenstein.

(9) Roctavian

Shirkadda: Waxaa soo saartay BioMarin Pharmaceutical (BioMarin).

Waqtiga suuqa: Waxaa ansixiyay Midowga Yurub Agoosto 2022.

Tilmaamaha: Daawaynta bukaanada qaangaarka ah ee qaba hemophilia daran oo aan lahayn taariikh ka hortagga FVIII factor iyo AAV5 antibody negative.

Faallooyinka: Roctavian (valoctocogene roxaparvovec) waxay u isticmaashaa AAV5 sida vector oo waxay isticmaashaa dhiirigeliyaha beerka-gaarka ah ee HLP si uu u wado muujinta xinjirowga aadanaha sideed (FVIII) iyadoo domainka B la tirtiray.Go'aanka Komishanka Yurub ee lagu ansixiyay suuq-geynta valoctocogene roxaparvovec wuxuu ku salaysan yahay xogta guud ee barnaamijka horumarinta kiliinikada ee daawada.Waxaa ka mid ah, wajiga III tijaabada bukaan-socodka GENEr8-1 waxay muujisay in marka la barbardhigo xogta sanadka ka hor intaan la qorin, ka dib hal faleebo oo ah valoctocogene roxaparvovec, Mawduucyada ayaa si aad ah hoos ugu dhacay heerka dhiig-baxa sanadlaha ah (ABR), isticmaalka joogtada ah ee dib-u-soo-celinta VIII (F8) diyaarinta borotiinka, ama korodhka weyn ee dhaqdhaqaaqa F8 ee dhiigga jirka.Kadib 4 todobaad oo daaweyn ah, maaddooyinka isticmaalkooda F8 sanadlaha ah iyo ABR ee u baahan daawaynta ayaa la dhimay 99% iyo 84%, siday u kala horreeyaan, farqi weyn oo tirakoobka ah (p<0.001).Astaanta badbaadada ayaa ahayd mid wanaagsan, iyada oo aanay jirin maaddooyin la kulma ka-hortagga F8, malignant, ama waxyeelooyinka xinjirowga, iyo dhacdooyinka xun xun ee daawaynta ee la xidhiidha (SAEs) lama soo sheegin.

3. Daawooyinka yar yar ee nucleic acid

(1) Vitravene

Shirkadda: Waxaa si wadajir ah u sameeyay Ionis Pharma (oo hore u ahaan jiray Isis Pharma) iyo Novartis.

Waqtiga suuqa: Waxaa ansixiyay FDA iyo EU EMA 1998 iyo 1999.

Tilmaamaha: Daawaynta cytomegalovirus retinitis ee bukaanka HIV-ga qaba.

Faallooyinka: Vitravene waa daawada oligonucleotide antisense iyo daawada oligonucleotide ee ugu horreysa ee loo oggolaaday suuqgeynta adduunka.Bilowgii suuqa, baahida suuqa ee dawooyinka anti-cytomegalovirus waxay ahayd mid degdeg ah;ka dibna ay sabab u tahay horumarinta daaweynta antiretroviral oo aad u firfircoon, tirada kiisaska cytomegalovirus ayaa aad hoos ugu dhacay.Baahida suuqa oo yar awgeed, dawada waxaa la sii daayay 2002 iyo 2006 ka bixitaan wadamada EU iyo Mareykanka.

(2) Macugen

Shirkadda: Waxaa iska kaashaday Pfizer iyo Eyetech.

Waqtiga la suuq-geyn karo: Waa la oggolaaday liiska Maraykanka 2004tii.

Tilmaamaha: loogu talagalay daaweynta neovascular da'da la xiriirta macular degeneration.

Faallooyinka: Macugen waa daawada oligonucleotide pegylated oo wax laga beddelay kaas oo bartilmaameedsan kara kuna xidhi kara factor koritaanka endothelial vascular (VEGF165 isoform), waxaana lagu maamulaa duritaanka intravitreal.

(3) Defitelio

Shirkadda: Waxaa soo saaray Jazz.

Waqtiga suuqa: Waxaa ansixiyay Midowga Yurub 2013, oo ay ansixisay FDA Maarso 2016.

Tilmaamaha: Daawaynta cudurka occlusive venule hepatic venule ee la xidhiidha kelyaha ama sambabada cillad la'aanta ka dib markii unugyada tarma ee hematopoietic ay ku tallaalaan.

Faallooyinka: Defitelio waa daawada oligonucleotide, isku dar ah oligonucleotides oo leh sifooyinka plasmin.Waxaa laga noqday 2009 sababo ganacsi awgeed.

(4) Kynamro

Shirkadda: Waxaa iska kaashaday Ionis Pharma iyo Kastle.

Waqtiga la suuqgeyn karo: Waxaa laga oggolaaday Mareykanka sida agoonta daroogada 2013.

Tilmaamaha: Daawaynta adjuvant ee homozygous familial hypercholesterolemia.

Xusuusin: Kynamro waa daawada oligonucleotide-ka-hortagga ah, oligonucleotide-ka-hortagga ah oo bartilmaameedsanaya apo B-100 mRNA.Kynamro waxaa loo maamulaa sida 200 mg oo hoostiisa ah hal mar todobaadkii.

(5) Spinraza

Shirkadda: Waxaa soo saartay Ionis Pharmaceuticals.

Waqtiga suuqa: Waxaa ogolaatay FDA bishii Disembar 2016.

Tilmaamaha: Daawaynta atrophy muruqa laf dhabarta (SMA).

Xusuusin: Spinraza (nusinersen) waa daawada oligonucleotide lidka ku ah.Spinraza waxay bedeli kartaa kala-baxa RNA ee hiddo-wadaha SMN2 iyadoo lagu xirayo goobta kala-baxa ee SMN2 exon 7, taasoo kordhinaysa soosaarka borotiinka SMN oo si buuxda u shaqeeya.Bishii Agoosto 2016, Shirkadda BIOGEN waxay adeegsatay ikhtiyaarkeeda si ay u hesho xuquuqda caalamiga ah ee Spinraza.Spinraza waxay bilawday tijaabadii ugu horeysay ee kiliinikada ee bini'aadamka 2011. Kaliya 5 sano, waxaa ansixiyay FDA 2016, taasoo ka tarjumaysa aqoonsiga buuxa ee FDA ee waxtarkeeda.Daawada waxaa loo oggolaaday suuqgeynta Shiinaha Abriil 2019. Dhammaan wareegga oggolaanshaha ee Spinraza ee Shiinaha waa wax ka yar 6 bilood.Waxaa laga joogaa 2 sano iyo 2 bilood tan iyo markii Spinraza markii ugu horreysay lagu ansixiyay Mareykanka.Noocan oo kale ah xannibaadda shisheeye ee dhifka ah ee dawooyinka cusub ayaa ku jira Xawaaraha liiska Shiinaha ayaa durba aad u dheereeya.Tani waxay sidoo kale sabab u tahay "Ogaysiis ku saabsan Siideynta Liisaska Koowaad ee Daawooyinka Cusub ee Dibadda Si Degdeg ah Loogu Baahan Yahay Cilmi-baarista Caafimaadka" oo ay soo saartay Xarunta Qiimaynta Maandooriyaha ee Noofambar 1, 2018, kaas oo lagu daray dufcaddii ugu horreysay ee 40 dawooyin cusub oo ajnabi ah oo loogu talagalay dib-u-eegis degdeg ah, iyo Spinraza ayaa ku jira.

(6) Fannaaniinta 51

Shirkadda: Waxaa soo saartay AVI BioPharma (oo markii dambe loo beddelay Sarepta Therapeutics).

Waqtiga suuqa: Waxaa ogolaatay FDA Sebtembar 2016.

Tilmaamaha: Daawaynta Duchenne muscular dystrophy (DMD) oo leh isbeddelka hidda-wadaha DMD ee exon 51 skipping gene.

Faallooyinka: Exondys 51 waa daawada oligonucleotide-ka-hortagga ah.Antisense oligonucleotide waxay ku xidhmi kartaa booska exon 51 ee pre-mRNA ee hidda-wadaha DMD, taasoo keentay samaynta mRNA baaluq.Jeexitaanka, oo qayb ahaan saxaya qaabka akhriska mRNA, waxay ka caawisaa bukaanka inuu isku daro noocyada shaqada qaarkood ee dystrophin oo ka gaaban borotiinka caadiga ah, taas oo hagaajinaysa calaamadaha bukaanka.

(7) Tegsedi

Shirkadda: Waxaa soo saartay Ionis Pharmaceuticals.

Waqtiga la suuqgeyn karo: Waxaa ansixiyay Midowga Yurub suuqgeyntiisa Luulyo 2018.

Tilmaamaha: Daawaynta amyloidosis transthyretin ee la iska dhaxlo (hATTR).

Faallooyinka: Tegsedi waa daawada oligonucleotide-ka-hortagga ah ee lagu beegsanayo transthyretin mRNA.Waa dawadii ugu horaysay ee la ansixiyay aduunka ee daawaynta hatTR.Habka loo maamulo waa cirbadeynta subcutaneous.Daawadu waxay yaraynaysaa soo saarista borotiinka ATTR iyadoo la beegsanayo mRNA of transthyretin (ATTR), waxayna leedahay faa'iido-khatarta wanaagsan ee daaweynta ATTR.Marxaladda cudurka iyo joogitaanka wadnaha wadnaha midna ma khusayn.

(8) Onpattro

Shirkad: Waxaa iska kaashaday Alnylam iyo Sanofi.

Waqtiga la suuqgeyn karo: Waa la oggolaaday liiska Maraykanka 2018.

Tilmaamaha: Daawaynta amyloidosis transthyretin ee la iska dhaxlo (hATTR).

Faallooyinka: Onpattro waa daawada siRNA ee lagu beegsanayo transthyretin mRNA, taas oo yaraynaysa soo saarista borotiinka ATTR ee beerka iyo ururinta kaydka amyloid ee neerfayaasha durugsan iyada oo la beegsanayo mRNA of transthyretin (ATTR)., si ay u wanaajiso oo ay yareyso calaamadaha cudurka.

(9) Givlaari

Shirkadda: Waxaa soo saartay Alnylam Corporation.

Waqtiga suuqa: Waxaa ogolaatay FDA Noofambar 2019.

Tilmaamaha: Loogu talagalay daawaynta cagaarshow ba'an (AHP) ee dadka waaweyn.

Faallooyinka: Givlaari waa daawada siRNA, dawada labaad ee siRNA ee loo ogolaaday suuqgeyn kadib Onpattro.Daawada waxaa loo maamulaa si hoose oo ka hooseysa waxayna bartilmaameed ka dhigtaa mRNA si loo dhimo borotiinka ALAS1.Daawaynta bilaha ah ee Givlaari waxay si weyn oo joogto ah u yarayn kartaa heerka ALAS1 ee beerka, taasoo hoos u dhigaysa heerarka neurotoxic ALA iyo PBG ilaa heerka caadiga ah, si ay u yareeyaan calaamadaha cudurka bukaanka.Xogta ayaa muujisay in bukaannada lagu daweeyay Givlaari ay 74% hoos u dhigeen tirada cudurrada dillaaca marka loo eego kooxda placebo.

(10) Vyondys53

Shirkadda: Waxaa soo saartay Sarepta Therapeutics.

Waqtiga suuqa: Waxaa ogolaatay FDA Disembar 2019.

Tilmaan: Daaweynta bukaanka DMD ee leh hidda-wadaha dystrophin exon 53 mutation.

Fiiro gaar ah: Vyondys 53 waa daawada oligonucleotide antisense.Daawada oligonucleotide waxay bartilmaameed ka dhigtaa habka kala qaybinta ee dystrophin mRNA horudhac.Habka aan tooska ahayn ee dystrophin mRNA precursor, dibedda Exon 53 ayaa qayb ahaan la kala saaray, ie kuma jiraan mRNA baaluq, waxaana loogu talagalay in lagu soo saaro borotiinka dystrophin oo go'ay laakiin wali shaqeynaya, taas oo kor u qaadaysa awoodda jimicsiga bukaanka.

(11) Waylivra

Shirkad: Waxaa soo saartay Ionis Pharmaceuticals iyo qaybteeda Akcea Therapeutics.

Waqtiga suuqa: Waxaa oggolaaday Wakaaladda Daawooyinka Yurub (EMA) Maajo 2019.

Tilmaan: Sida daawayn dheeri ah oo loogu talagalay cuntada la kontoroolo ee bukaanada qaangaarka ah ee qaba qoyska chylomicronemia syndrome (FCS).

Faallooyinka: Waylivra waa daawada oligonucleotide antisense, taas oo ah daawadii ugu horreysay ee loo oggolaaday daawaynta FCS adduunka.

(12) Leqvio

Shirkadda: Waxaa soo saaray Novartis.

Waqtiga suuqa: Waxaa ansixiyay Midowga Yurub Disembar 2020.

Tilmaamaha: Daawaynta hypercholesterolemia aasaasiga ah ee dadka waaweyn (heterozygous familial iyo non-familial) ama dyslipidemia isku dhafan.

Fiiro gaar ah: Leqvio waa daawada siRNA ee lagu beegsanayo PCSK9 mRNA.Waa daawaynta siRNA ee ugu horeysay ee hoos u dhigista kolestaroolka (LDL-C).Habka loo maamulo waa cirbadeynta subcutaneous.Daroogadu waxay ku shaqeysaa faragelinta RNA si ay hoos ugu dhigto heerarka borotiinka PCSK9, taas oo markaa hoos u dhigaysa heerarka LDL-C.Xogta caafimaadku waxay tusinaysaa in Leqvio ay hoos u dhigi karto LDL-C ku dhawaad ​​50% bukaanada aan heerarkooda LDL-C la dhimi karin ilaa heerar la beegsanayo inkasta oo qiyaasta ugu badan ee statins loo dulqaadan karo.

(13) Oxlumo

Shirkadda: Waxaa soo saartay Alnylam Pharmaceuticals.

Waqtiga suuqa: Waxaa ansixiyay Midowga Yurub Noofambar 2020.

Tilmaamaha: Daaweynta hyperoxaluria aasaasiga ah nooca 1 (PH1).

Fiiro gaar ah: Oxlumo waa dawada siRNA oo bar-tilmaameedsanaysa hydroxy acid oxidase 1 (HAO1) ​​mRNA, kaas oo si hoose loo maamulo.Daawada waxaa la soo saaray iyadoo la adeegsanayo tignoolajiyada isku xidhka kiimikada ESC-GalNAc ee xasilinta ee Alnylam ee ugu dambeysay, taasoo u sahlaysa siRNA-yada hoosta laga maamulo oo leh adkaysi iyo waxtar weyn.Daroogadu waxay bartilmaameedsanaysaa hoos u dhigista ama joojinta hydroxy acid oxidase 1 (HAO1) ​​mRNA, waxay yaraynaysaa heerka glycolate oxidase ee beerka, ka dibna waxay isticmaashaa substrate-ka looga baahan yahay soo saarista oxalate waxayna yaraynaysaa wax soo saarka oxalate si loo xakameeyo horumarka cudurka iyo hagaajinta calaamadaha cudurka bukaanka.

(14) Viltepso

Shirkadda: Waxaa soo saartay NS Pharma, oo hoos timaada Nippon Shinyaku.

Waqtiga suuqa: Waxaa ogolaatay FDA Agoosto 2020.

Tilmaamaha: Daawaynta Duchenne muscular dystrophy (DMD) oo leh isbeddelka hidda-wadaha DMD ee exon 53 skipping gene.

Fiiro gaar ah: Viltepso waa daawada phosphorodiamide morpholino oligonucleotide.Daawada oligonucleotide waxay ku xidhmi kartaa booska exon 53 ee pre-mRNA ee hidda-wadaha DMD, taasoo keentay samaynta mRNA baaluq.Exon ayaa qayb ahaan meesha laga saaray, iyada oo qayb ahaan la saxayo qaabka akhriska mRNA, ka caawinta bukaanka in uu sameeyo qaar ka mid ah noocyada shaqada ee dystrophin oo ka gaaban borotiinka caadiga ah, taas oo hagaajinaysa calaamadaha bukaanka.

(15) Amvuttra (vutrisiran)

Shirkadda: Waxaa soo saartay Alnylam Pharmaceuticals.

Waqtiga suuqa: Waxaa ogolaatay FDA Juun 2022.

Tilmaamaha: Daawaynta dadka waaweyn ee dhaxalka ah ee transthyretin amyloidosis oo leh polyneuropathy (hATTR-PN).

Faallooyinka: Amvuttra (Vutrisiran) waa daawada siRNA ee bar-tilmaameedsanaysa transthyretin (ATTR) mRNA, kaas oo lagu maamulo duritaanka maqaarka hoostiisa.Vutrisiran waxaa loo nashqadeeyay iyadoo lagu salaynayo Alnylam's Enhanced Stabilization Chemistry (ESC) -GalNAc madal gaarsiinta oo leh awood korodhay iyo xasiloonida dheef-shiid kiimikaadka.Oggolaanshaha daawaynta waxay ku saleysan tahay xogta 9-bilood ee daraasaddeeda caafimaad ee Wajiga III (HELIOS-A), iyadoo natiijooyinka guud ay muujinayaan in daawaynta ay hagaajisay calaamadaha hATTR-PN, iyada oo in ka badan 50% bukaannada ay dib u noqdaan ama joojiyaan horumarka.

4. Daawooyinka kale ee daaweynta hidda-wadaha

(1) Rexin-G

Shirkadda: Waxaa soo saartay Epeius Biotech.

Waqtiga suuqa: Waxaa ansixiyay Maamulka Cuntada iyo Dawooyinka ee Filibiin (BFAD) 2005.

Tilmaamaha: Loogu talagalay daawaynta kansarrada sare u kacay ee u adkaysta daaweynta kiimikaad.

Xusuusin: Rexin-G waa cirbad nanoparticle ah oo hiddo-wadaha ku raran yahay.Waxay soo bandhigaysaa hiddasidaha mutant G1 cyclin ee unugyada bartilmaameedka ah iyada oo loo marayo vector retroviral si si gaar ah loogu dilo burooyinka adag.Habka maamulka waa faleebo xididka.Sida daroogo buro-bartilmaameed ah oo si firfircoon u raadisa oo burburisa unugyada kansarka dheef-shiid kiimikaadka, waxay saameyn gaar ah ku leedahay bukaanada aan waxtarka u lahayn daawooyinka kale ee kansarka, oo ay ku jiraan bayooloji la beegsaday.

(2) Neovasculgen

Shirkad: Waxaa soo saaray Machadka Unugyada Asalka ee Human.

Waqtiga liiska: Waxaa la oggolaaday liiska Ruushka Diseembar 7, 2011, ka dibna lagu qoray Ukraine 2013.

Tilmaamaha: Loogu talagalay daawaynta cudurrada xididdada durugsan, oo ay ku jiraan ischemia addimada daran.

Faallooyinka: Neovasculgen waa daawaynta hidda-wadaha plasmid-ku-salaysan ee DNA-da kaas oo xuubka kobaca xididdada xididdada dhiigga (VEGF) 165 uu ku dhisan yahay laf-dhabarta plasmid oo lagu geliyo bukaannada.

(3) Collategene

Shirkad: Waxaa iska kaashaday Jaamacadda Osaka iyo shirkadaha raasumaalka ah.

Waqtiga liiska: Waxaa oggolaaday Wasaaradda Caafimaadka, Shaqada iyo Samafalka Japan ee liiska Agoosto 2019.

Tilmaamaha: Daaweynta ischemia hoose ee daran.

Faallooyinka: Collategene waa daawaynta hidda-wadaha plasmid-ku-salaysan, daawadii ugu horreysay ee daawaynta hidde-sidaha Japan ee ay soo saarto AnGes.Qaybta ugu muhiimsan ee daawadani waa plasmid qaawan oo ka kooban taxanaha hidda-wadaha ee factor koritaanka hepatocytes (HGF).Haddii daawada lagu duro murqaha lugaha hoose, HGF ee la muujiyay ayaa kor u qaadi doonta samaynta xididdada cusub ee dhiigga ee agagaarka xididdada dhiigga ee xiran.Tijaabooyin caafimaad ayaa xaqiijiyay waxtarkeeda hagaajinta boogaha.

DHAMAAD