Liphatsa tsa lefutso ke likarolo tsa motheo tsa lefutso tse laolang mekhoa.Ntle le liphatsa tsa lefutso tsa livaerase tse ling, tse entsoeng ka RNA, liphatsa tsa lefutso tsa lintho tse ngata li entsoe ka DNA..Bongata ba mafu a lintho tse phelang a bakoa ke tšebelisano pakeng tsa liphatsa tsa lefutso le tikoloho.Kalafo ea liphatsa tsa lefutso ha e le hantle e ka phekola kapa ea fokotsa maloetse a mangata.Phekolo ea liphatsa tsa lefutso e nkuoa e le phetoho lefapheng la bongaka le pharmacy.Lithethefatsi tsa phekolo ea liphatsa tsa lefutso ka kutloisiso e pharaletseng li kenyelletsa litlhare tsa DNA-modified DNA (tse kang litlhare tsa vivo gene therapy tse thehiloeng ho likokoana-hloko tsa likokoana-hloko, meriana ea phekolo ea liphatsa tsa lefutso, lithethefatsi tse hlobotseng tsa plasmid, joalo-joalo) le lithethefatsi tsa RNA (tse kang li-antisense oligonucleotide drugs, siRNA drugs, le mRNA gene therapy, joalo-joalo);Litlhare tsa kalafo ea Gene haholo-holo li kenyelletsa lithethefatsi tsa plasmid DNA, litlhare tsa kalafo tsa lefutso tse thehiloeng ho li-vector tsa vaerase, litlhare tsa kalafo tsa lefutso tse thehiloeng ho li-vector tsa baktheria, litsamaiso tsa ho hlophisa liphatsa tsa lefutso, le litlhare tsa kalafo ea lisele bakeng sa phetoho ea mofuta oa in vitro.Ka mor'a lilemo tsa tsoelo-pele e bohloko, meriana ea phekolo ea lefutso e fihletse liphello tse khothatsang tsa kliniki.(Ho sa baloe liente tsa DNA le liente tsa mRNA) Hajoale, meriana e 45 ea phekolo ea lefutso e amohetsoe hore e bapatsoe lefatšeng ka bophara.Kakaretso ea liphekolo tsa liphatsa tsa lefutso tse 9 li amohetsoe ho bapatsa selemong sena, ho kenyeletsoa le liphekolo tse 7 tsa liphatsa tsa lefutso tse lumelletsoeng ho bapatsa khetlo la pele selemong sena, e leng: CARVYKTI, Amvuttra, Upstaza, Roctavian, Hemgenix, Adstiladrin le Ebvallo, (Hlokomela: Tse ling tse peli li amohetsoe United States selemong sena. FDA bakeng sa ho bapatsa United States ka Phato 2022, 'me e ile ea amoheloa bakeng sa ho bapatsa ke European Union ka 2019; .) Ka ho hlahisoa ha lihlahisoa tse ngata tsa kalafo ea lefutso le nts'etsopele e potlakileng ea thekenoloji ea kalafo ea lefutso, kalafo ea liphatsa tsa lefutso e mothating oa ho tlisa nako ea tsoelo-pele e potlakileng.

Sehlopha sa phekolo ea lefutso (Mohloli oa setšoantšo: Bio-Matrix)

Sengoliloeng sena se thathamisa liphekolo tse 45 tsa lefutso (ho sa kenyeletsoe liente tsa DNA le liente tsa mRNA) tse amohetsoeng hore li bapatsoe.

1. Kalafo ea liphatsa tsa lefutso tsa in vitro

(1) Strimvelis

Khampani: E thehiloe ke GlaxoSmithKline (GSK).

Nako ea ho rekisoa: E amohetsoe ho bapatsa ke European Union ka Mots'eanong 2016.

Lipontšo: Bakeng sa phekolo ea matla a kopantsoeng a immunodeficiency (SCID).

Maikutlo: Mokhoa o akaretsang oa phekolo ena ke ho qala ka ho fumana lisele tsa mokuli tsa hematopoietic stem, ho li atolosa le ho li hlahisa ka har'a vitro, ebe o sebelisa retrovirus ho hlahisa kopi ea liphatsa tsa lefutso tsa ADA (adenosine deaminase) ka har'a lisele tsa stem tsa hematopoietic, 'me qetellong li ente lisele tse fetotsoeng tsa Hematopoietic stem li khutlisetsoa hape' meleng.Liphetho tsa bongaka li bontša hore sekhahla sa ho phela sa lilemo tse 3 sa bakuli ba ADA-SCID ba phekoloang ka Strimvelis ke 100%.

(2) Zalmoxis

Khampani: E hlahisitsoe ke Italy MolMed Company.

Nako ea ho rekisa: E fumane tumello ea ho bapatsa e nang le maemo ho tsoa European Union ka 2016.

Lipontšo: E sebelisoa bakeng sa phekolo ea adjuvant ea tsamaiso ea 'mele ea ho itšireletsa mafung ea bakuli ka mor'a ho fetisetsa lisele tsa stem cell.

Maikutlo: Zalmoxis ke allogeneic T cell suicide gene immunotherapy e fetotsoeng ke li-vector tsa retroviral.Mokhoa ona o sebelisa li-retroviral vectors ho fetola liphatsa tsa lefutso tsa allogeneic T lisele, e le hore lisele tsa T tse fetotsoeng ka liphatsa tsa lefutso li hlalose 1NGFR le HSV-TK Mut2 liphatsa tsa lefutso tsa ho ipolaea li lumella batho ho sebelisa lithethefatsi tsa ganciclovir (ganciclovir) ka nako leha e le efe ho bolaea lisele tsa T tse bakang ts'oaetso e mpe ea 'mele ea ho itšireletsa mafung, ho thibela ho senyeha ho ka 'nang ha etsahala hore ho be le ts'ebetso ea ho itšireletsa mafung ka mor'a ho sebetsa ha GVHD le ho fana ka ts'ebetso ea ho itšireletsa mafung ka mor'a ho sebetsa ha GVHD.

(3) Invossa-K

Khampani: E thehiloe ke TissueGene (KolonTissueGene).

Nako ea ho rekisoa: E amohetsoe ho ngolisoa Korea Boroa ka Phupu 2017.

Lipontšo: Bakeng sa phekolo ea ramatiki ea mangole e senyehang.

Maikutlo: Invossa-K ke allogeneic cell gene therapy e kenyelletsang li-chondrocyte tsa batho.Lisele tsa allogeneic li fetotsoe ka liphatsa tsa lefutso ka in vitro, 'me lisele tse fetotsoeng li ka hlalosa le ho hlahisa phetoho ea kholo ea β1 (TGF-β1) ka mor'a ente ea intra-articular.β1), ka hona ho ntlafatsa matšoao a osteoarthritis.Liphetho tsa bongaka li bontša hore Invossa-K e ka ntlafatsa haholo ramatiki ea lengole.E ile ea hlakoloa ka 2019 ke Tsamaiso ea Lijo le Lithethefatsi ea Korea hobane moetsi o ile a ngola hampe metsoako e sebelisitsoeng.

(4) Zynteglo

Khampani: E batlisisitsoe le ho ntlafatsoa ke bluebird bio.

Nako ea ho rekisoa: E amohetsoe ke European Union bakeng sa ho bapatsa ka 2019, mme e amohetsoe ke FDA bakeng sa ho bapatsa United States ka Phato 2022.

Lipontšo: Bakeng sa phekolo ea β-thalassemia e itšetlehileng ka tšelo ea mali.

Maikutlo: Zynteglo ke lentiviral in vitro gene therapy e hlahisang kopi e sebetsang ea mofuta o tloaelehileng oa β-globin (βA-T87Q-globin gene) liseleng tsa stem tsa hematopoietic tse nkiloeng ho mokuli ka vector ea lentiviral, ebe e khutlisetsa liphatsa tsena tsa lefutso tsa autologous liseleng tsa lefutso tsa mokuli.Hang ha mokuli a e-na le mofuta o tloaelehileng oa βA-T87Q-globin, a ka hlahisa protheine e tloaelehileng ea HbAT87Q, e ka fokotsang kapa ea felisa tlhokahalo ea tšelo ea mali ka katleho.Ke phekolo ea nako e le 'ngoe e etselitsoeng ho khutlisa tšelo ea mali ea bophelo bohle le meriana ea bophelo bohle bakeng sa bakuli ba lilemo tse 12 ho ea holimo.

(5) Skysona

Khampani: E batlisisitsoe le ho ntlafatsoa ke bluebird bio.

Nako ea ho mmaraka: E amohetsoe ke European Union bakeng sa ho bapatsa ka Phupu 2021, mme e amohetsoe ke FDA bakeng sa ho bapatsa United States ka Loetse 2022.

Lipontšo: Bakeng sa phekolo ea pele ea cerebral adrenoleukodystrophy (CALD).

Maikutlo: Kalafo ea liphatsa tsa lefutso ea Skysona ke eona feela phekolo ea nako e le 'ngoe ea lefutso e amohetsoeng bakeng sa phekolo ea cerebral adrenoleukodystrophy (CALD) ea mohato oa pele.Skysona (elivaldogene autotemcel, Lenti-D) ke lentiviral in vitro gene therapy ea lenti-D.Mokhoa o akaretsang oa phekolo ke o latelang: lisele tsa stem tsa autologous hematopoietic li ntšoa ho mokuli, li fetisoa le ho fetoloa ke lentivirus e jereng lefutso la ABCD1 in vitro ea motho, ebe e khutlisetsoa ho mokuli.E sebelisoa ho phekola bakuli ba ka tlase ho lilemo tse 18, ba jereng liphetoho tsa liphatsa tsa lefutso tsa ABCD1, le CALD.

(6) Kymria

COMPANY: E thehiloe ke Novartis.

Nako ea ho rekisoa: E amohetsoe ke FDA bakeng sa ho bapatsa ka Phato 2017.

Lipontšo: Kalafo ea precursor B-cell acute lymphoblastic leukemia (ALL) le DLBCL e khutletseng hape le refractory.

Maikutlo: Kymriah ke sethethefatsi sa lentiviral in vitro gene therapy, e leng phekolo ea pele ea CAR-T e amohetsoeng bakeng sa ho bapatsa lefatšeng, e lebisitse ho CD19, le ho sebelisa 4-1BB co-stimulatory factor.E rekoa ka $475,000 US le $313,000 Japane.

(7) Yescarta

Khampani: E Thehiloe ke Kite Pharma, e leng lekala la Gileade (GILD).

Nako ea ho rekisa: E amohetsoe ke FDA bakeng sa ho rekisa ka October 2017;Fosun Kite e hlahisitse theknoloji ea Yescarta ho tsoa ho Kite Pharma mme ea e hlahisa Chaena kamora ho fumana tumello.E amohetsoe ho ngolisoa ka har'a naha.

Lipontšo: Bakeng sa phekolo ea ho khutlela morao kapa ho hanyetsa B-cell lymphoma e kholo.

Maikutlo: Yescarta ke phekolo ea liphatsa tsa lefutso tsa retroviral in vitro, e leng phekolo ea bobeli e amohetsoeng ea CAR-T lefatšeng.E shebane le CD19 mme e amohela li-costimulator tsa CD28.E rekoa ho $373,000 United States.

(8) Tecartus

Khampani: E Thehiloe ke Gileade (GILD).

Nako ea ho rekisoa: E amohetsoe ke FDA bakeng sa ho bapatsa ka Phupu 2020.

Lipontšo: Bakeng sa ho khutlela morao kapa ho hanyetsa mantle cell lymphoma.

Maikutlo: Tecartus ke phekolo ea sele ea CAR-T e ikemetseng e shebileng CD19, 'me ke phekolo ea boraro ea CAR-T e amohelehang bakeng sa ho bapatsa lefatšeng.

(9) Breyanzi

KAMPANI: E Thehiloe ke Bristol-Myers Squibb (BMS).

Nako ea ho rekisoa: E amohetsoe ke FDA bakeng sa ho bapatsa ka Hlakola 2021.

Lipontšo: Ho khutlela morao kapa ho hanyetsa (R / R) e kholo ea B-cell lymphoma (LBCL).

Maikutlo: Breyanzi ke in vitro gene therapy e thehiloeng ho lentivirus, phekolo ea bone ea CAR-T e amohelehang bakeng sa ho bapatsa lefatšeng ka bophara, e lebisitsoe ho CD19.Kamohelo ea Breyanzi ke mohato oa bohlokoa ho Bristol-Myers Squibb lebaleng la immunotherapy ea cellular, eo e e fumaneng ha e fumana Celgene ka $74 bilione ka 2019.

(10) Abecma

Khampani: E thehiloe ke Bristol-Myers Squibb (BMS) le bio bluebird.

Nako ea ho rekisoa: E amohetsoe ke FDA bakeng sa ho bapatsa ka Hlakubele 2021.

Matšoao: ho khutlela morao kapa ho hanyetsa multiple myeloma.

Maikutlo: Abecma ke phekolo ea lefutso ea in vitro e thehiloeng ho lentivirus, phekolo ea pele ea lefats'e ea CAR-T e lebisitseng BCMA, le phekolo ea bohlano ea CAR-T e amohetsoeng ke FDA.Molao-motheo oa moriana ke ho hlahisa li-receptor tsa chimeric BCMA liseleng tsa T tsa mokuli ka ho fetola liphatsa tsa lefutso tsa lentivirus.Kalafo ea ho felisa lisele tsa T tse sa fetoloang ho bakuli, ebe li khutlisetsa lisele tsa T tse fetotsoeng, tse batlang le ho bolaea lisele tsa mofetše tsa BCMA ho bakuli.

(11) Libmeldy

KAMPUNI: E thehiloe ke Orchard Therapeutics.

Nako ea ho rekisoa: E amohetsoe ke European Union bakeng sa ho ngolisoa ka December 2020.

Lipontšo: Bakeng sa phekolo ea metachromatic leukodystrophy (MLD).

Maikutlo: Libmeldy ke phekolo ea lefutso e thehiloeng ho autologous CD34+ lisele tse fetotsoeng ka liphatsa tsa lefutso ka lentivirus.Lintlha tsa bongaka li bonts'a hore infusion e le 'ngoe ea methapo ea Libmeldy e ka fetola ka nepo mokhoa oa ho qala MLD ha o bapisoa le kholo e matla ea koloi le kelello ho bakuli ba sa alafshoang ba lilemo tse tšoanang.

(12) Benoda

Khampani: E thehiloe ke WuXi Giant Nuo.

Nako ea ho rekisoa: E amohetsoe ka molao ke NMPA ka Loetse 2021.

Lipontšo: Kalafo ea bakuli ba baholo ba nang le ho khutlela morao kapa ho hanyetsa B-cell lymphoma e kholo (r / r LBCL) ka mor'a mokhoa oa bobeli kapa ka holimo ho phekolo ea tsamaiso.

Maikutlo: Beinoda ke phekolo ea liphatsa tsa lefutso ea anti-CD19 CAR-T, hape ke sehlahisoa sa mantlha sa WuXi Juro Company.Ke sehlahisoa sa bobeli sa CAR-T se amohetsoeng Chaena, ntle le ho khutlela morao / refractory e kholo ea B-cell lymphoid WuXi Giant Nuo e boetse e rera ho hlahisa ente ea Regiorensai bakeng sa phekolo ea matšoao a mang a mangata, ho akarelletsa le follicular lymphoma (FL), mantle cell lymphoma (MCL), chronic lymphocytic leukemia (CLL) , lymphocytic leukemia ea bobeli BLL leukemia e kholo ea DLL lymphoma (DL-cell e kholo ea DL)

(13) CARVYKTI

Khampani: Sehlahisoa sa pele sa Legend Biotech se amohetsoeng hore se bapatsoe.

Nako ea ho rekisoa: E amohetsoe ke FDA bakeng sa ho bapatsa ka Hlakola 2022.

Lipontšo: bakeng sa phekolo ea ho khutlela morao kapa ho hanyetsa myeloma e mengata (R / R MM).

Maikutlo: CARVYKTI (ciltacabtagene autoleucel, Cilta-cel ka bokhutšoanyane) ke CAR-T cell immune gene therapy e nang le li-antibodies tse peli tsa domain tse lebisang B-cell maturation antigen (BCMA).Lintlha li bontša hore CARVYKTI Ho bakuli ba nang le myeloma ea ho khutlela morao kapa ea refractory ba fumaneng mekhoa ea phekolo ea pele e mene kapa ho feta (ho akarelletsa le proteasome inhibitors, immunomodulators le anti-CD38 monoclonal antibodies), tekanyo ea kakaretso ea karabo ea 98% e bontšitsoe.

(14)Evallo

COMPANY: E thehiloe ke Atara Biotherapeutics.

Khomishene ea Europe (EC) bakeng sa ho bapatsa ka Tšitoe 2022, ke kalafo ea pele lefatšeng ka bophara ea T cell e amohetsoeng bakeng sa ho bapatsa.

Lipontšo: Joaloka monotherapy bakeng sa kokoana-hloko ea Epstein-Barr (EBV) e amanang le post-transplantation lymphoproliferative disease (EBV+PTLD), bakuli ba fumanang phekolo e tlameha ho ba batho ba baholo le bana ba ka holimo ho lilemo tse 2 ba kileng ba fumana bonyane phekolo e 'ngoe ea lithethefatsi.

Tlhaloso: Ebvallo ke allogeneic EBV-specific universal T-cell gene therapy e lebisang le ho felisa lisele tse nang le tšoaetso ea EBV ka mokhoa o thibetsoeng ke HLA.Tumello ea kalafo ena e ipapisitse le liphetho tsa thuto ea teko ea bongaka ea karolo ea 3 ea bohlokoa, 'me liphetho li bonts'itse hore ORR ea sehlopha sa HCT le sehlopha sa SOT e ne e le 50%.Sekhahla se feletseng sa tšoarelo (CR) e ne e le 26.3%, tekanyo ea ho fokotsa karolo e itseng (PR) e ne e le 23.7%, 'me nako e bohareng ea ho phomola (TTR) e ne e le likhoeli tse 1.1.Ho bakuli ba 19 ba fumaneng tšoarelo, ba 11 ba bile le nako ea karabelo (DOR) ea likhoeli tse fetang 6.Ho phaella moo, mabapi le ts'ireletso, ha ho na liketso tse mpe tse kang lefu la graft-versus-host (GvHD) kapa lefu la ho lokolloa ha cytokine le amanang le Ebvallo.

2. Kalafo ea liphatsa tsa lefutso ea in vivo e thehiloeng ho likokoana-hloko tsa kokoana-hloko

(1) Gendicine/Jin Sheng

Khampani: E thehiloe ke Khamphani ea Shenzhen Saibainuo.

Nako ea ho rekisoa: E amohetsoe ho ngolisoa Chaena ka 2003.

Lipontšo: Bakeng sa phekolo ea hlooho le molala squamous cell carcinoma.

Ela hloko: Recombinant human p53 adenovirus injection Gendicine/Jinyousheng ke sethethefatsi sa adenovirus vector gene therapy se nang le litokelo tse ikemetseng tsa thepa ea mahlale tseo e leng tsa Company ea Shenzhen Saibainuo.Mofuta oa 5 oa adenovirus oa batho o entsoe ka mofuta oa adenovirus oa motho oa 5. Ea pele ke mohaho o ka sehloohong oa phello e khahlanong le hlahala ea lithethefatsi, 'me ea ho qetela e sebetsa haholo-holo e le mojari.Vector ea adenovirus e jara mofuta oa phekolo p53 ka seleng e reretsoeng, e hlalosa tumor suppressor gene p53 ka seleng e shebiloeng, le polelo ea eona ea liphatsa tsa lefutso Sehlahisoa se ka laola mefuta e fapaneng ea liphatsa tsa lefutso tse khahlanong le mofetše le ho theola-ho laola mesebetsi ea li-oncogenes tse fapaneng, ka tsela eo, ho matlafatsa sepheo sa 'mele oa ho bolaea le ho fokotsa kotsi ea tumor.

(2) Rigvir

Khampani: E thehiloe ke Latima Company, Latvia.

Nako ea ho etsa lethathamo: E amohetsoe ho ngolisoa Latvia ka 2004.

Lipontšo: Bakeng sa phekolo ea melanoma.

Maikutlo: Rigvir ke phekolo ea liphatsa tsa lefutso e thehiloeng ho ECHO-7 enterovirus vector e fetotsoeng ka liphatsa tsa lefutso.Hona joale, lithethefatsi li amohetsoe Latvia, Estonia, Poland, Armenia, Belarus, joalo-joalo, 'me e ntse e ngolisoa ke EMA linaheng tsa EU.Linyeoe tsa bongaka lilemong tse leshome tse fetileng li pakile hore vaerase ea Rigvir oncolytic e bolokehile ebile e sebetsa, 'me e ka eketsa sekhahla sa ho phela ha bakuli ba melanoma ka makhetlo a 4-6.Ntle le moo, kalafo ena e sebetsa ho mefuta e meng ea mofetše, ho kenyelletsa mofets'e oa colorectal, mofets'e oa pancreatic, mofets'e oa senya, mofets'e oa liphio, mofets'e oa senya, mofets'e oa matšoafo, mofets'e oa popelo, lymphosarcoma, jj.

(3) Oncorine

Khampani: E thehiloe ke Shanghai Sanwei Biological Company.

Nako ea ho rekisoa: E amohetsoe ho ngolisoa Chaena ka 2005.

Lipontšo: phekolo ea lihlahala tsa hlooho le molala, kankere ea sebete, kankere ea pancreatic, kankere ea popelo le mefuta e meng ea kankere.

Maikutlo: Oncorine (安科瑞) ke sehlahisoa sa phekolo ea liphatsa tsa lefutso tsa oncolytic se sebelisang adenovirus joalo ka sejari.Ho fumanoe oncolytic adenovirus, e ka ikatisang ka ho khetheha ho lihlahala tse haelloang ke lefutso tsa p53 kapa tse sa tloaelehang, tse lebisang ho lysis ea lisele tsa tumor, ka hona, li bolaea lisele tsa hlahala.ntle le ho senya lisele tse tloaelehileng.Liphuputso tsa bongaka li bontšitse hore Ankerui e na le tšireletso e ntle le katleho bakeng sa mefuta e sa tšoaneng ea lihlahala tse kotsi.

(4) Glybera

Khampani: E thehiloe ke UniQure.

Nako ea ho rekisoa: E amohetsoe ho ngolisoa Europe ka 2012.

Lipontšo: Phekolo ea khaello ea lipoprotein lipase (LPLD) e nang le liketsahalo tse matla kapa tse iphetang tsa pancreatitis ho sa tsotellehe lijo tse nang le mafura a mangata.

Litlhaloso: Glybera (alipogene tiparvovec) ke lithethefatsi tsa phekolo ea liphatsa tsa lefutso tse thehiloeng ho AAV, e sebelisang AAV e le sesebelisoa sa ho fetisetsa liphatsa tsa lefutso tsa LPL ka lisele tsa mesifa, e le hore lisele tse tsamaisanang li khone ho hlahisa palo e itseng ea lipoprotein lipase, Ho fokotsa lefu lena, phekolo ena e sebetsa ka nako e telele bakeng sa nako e telele ea tsamaiso ea lithethefatsi hang.Lithethefatsi li ile tsa tlosoa 'marakeng ka 2017. Lebaka la ho tlosoa ha eona le ka amana le lintlha tse peli: theko e phahameng le tlhokahalo e fokolang ea' maraka.Kakaretso ea litšenyehelo tsa phekolo ea moriana ona e ka holimo ho US$1 million, 'me ke mokuli a le mong feela ea e rekileng le ho e sebelisa ho fihlela joale.Leha k'hamphani ea inshorense ea bongaka e buselitse US $ 900,000 bakeng sa eona, hape ke moroalo o batlang o le moholo bakeng sa k'hamphani ea inshorense.Ho phaella moo, lipontšo tse lebisitsoeng ke lithethefatsi ha li fumanehe haholo, ka sekhahla sa liketsahalo tse ka bang 1 ho limilione tse 1 le tekanyo e phahameng ea ho se tsebe hantle.

(5) Imlygic

Khampani: E thehiloe ke Amgen.

Nako ea ho rekisoa: Ka 2015, e ile ea amoheloa hore e ngolisoe United States le European Union.

Lipontšo: Kalafo ea melanoma e ke keng ea tlosoa ka ho feletseng ka ho buuoa.

Maikutlo: Imlygic ke attenuated herpes simplex kokoana-hloko ea mofuta oa 1 e fetotsoeng ke theknoloji ea liphatsa tsa lefutso (ho hlakola likaroloana tsa eona tsa ICP34.5 le ICP47, le ho kenya motho granulocyte macrophage colony-stimulating factor GM-CSF gene ka har'a kokoana-hloko) (HSV-1) oncolytic-approve virus ea FDA ke eona ea pele ea FDA.Mokhoa oa tsamaiso ke ente ea intralesional, e ka kenngoa ka ho toba ka lisosa tsa melanoma ho baka ho phatloha ha lisele tsa hlahala, ho lokolla li-antigen tse nkiloeng ke tumor le GM-CSF, le ho khothalletsa likarabo tsa ho itšireletsa mafung.

(6) Luxturna

Khampani: E thehiloe ke Spark Therapeutics, e leng setsi sa Roche.

Nako ea ho rekisa: E ile ea amoheloa bakeng sa ho bapatsa ke FDA ka 2017, 'me ea lumelloa ho bapatsa Europe ka 2018.

Lipontšo: Bakeng sa phekolo ea bana le batho ba baholo ba lahlehetsoeng ke pono ka lebaka la liphetoho tse peli tsa liphatsa tsa lefutso tsa RPE65 empa ba boloka palo e lekaneng ea lisele tse sebetsang tsa retinal.

Maikutlo: Luxturna ke phekolo ea lefutso e thehiloeng ho AAV e tsamaisoang ke ente ea subretinal.Phekolo ea liphatsa tsa lefutso e sebelisa AAV2 e le sejari ho hlahisa kopi e sebetsang ea liphatsa tsa lefutso tse tloaelehileng tsa RPE65 liseleng tsa retinal tsa mokuli, e le hore lisele tse tsamaisanang le tsona li hlahise protheine e tloaelehileng ea RPE65, e etsang hore mokuli a be le khaello ea protheine ea RPE65, kahoo e ntlafatsa pono ea mokuli.

(7) Zolgensma

Khamphani: E thehiloe ke AveXis, e leng setsi sa Novartis.

Nako ea ho rekisoa: E amohetsoe ke FDA bakeng sa ho bapatsa ka Mots'eanong 2019.

Matšoao: Phekolo ea bakuli ba Spinal Muscular Atrophy (SMA) ba ka tlase ho lilemo tse 2.

Maikutlo: Zolgensma ke phekolo ea lefutso e thehiloeng ho vector ea AAV.Setlhare sena ke morero oa nako e le 'ngoe feela oa kalafo bakeng sa mokokotlo oa mokokotlo oa mokokotlo o lumelletsoeng ho rekisoa lefatšeng.Ho qala ha lithethefatsi ho bula nako e ncha ea phekolo ea mokokotlo oa mokokotlo oa mesifa.leqephe, ke tsoelo-pele ea bohlokoahali.Pheko ena ea liphatsa tsa lefutso e sebelisa scAAV9 vector ho hlahisa mofuta o tloaelehileng oa SMN1 ho mokuli ka ho kenngoa ka methapo ho hlahisa protheine e tloaelehileng ea SMN1, kahoo e ntlafatsa mosebetsi oa lisele tse amehileng tse kang li-motor neurons.Ka lehlakoreng le leng, lithethefatsi tsa SMA Spinraza le Evrysdi li hloka ho pheta-pheta nako e telele.Spinraza e fanoa ka ente ea mokokotlo ka mor'a likhoeli tse ling le tse ling tse 'nè,' me Evrysdi ke lithethefatsi tse nooang letsatsi le letsatsi.

(8) Delytact

Khampani: E Thehiloe ke Daiichi Sankyo Company Limited (TYO: 4568).

Nako ea ho mmaraka: Tumello e nang le maemo ho tsoa Lefapheng la Bophelo bo Botle, Basebetsi le Boiketlo ba Japane (MHLW) ka Phuptjane 2021.

Lipontšo: Bakeng sa phekolo ea malignant glioma.

Maikutlo: Delytact ke sehlahisoa sa bone sa oncolytic virus gene therapy se amohetsoeng lefatšeng ka bophara, 'me ke sehlahisoa sa pele sa vaerase sa oncolytic se amohetsoeng bakeng sa kalafo ea malignant glioma.Delytact ke kokoana-hloko e entsoeng ka liphatsa tsa lefutso ea herpes simplex virus mofuta oa 1 (HSV-1) oncolytic virus e entsoeng ke Dr. Todo le basebetsi-'moho.Delytact e hlahisa liphetoho tse ling tsa ho hlakoloa ho genome ea G207 ea HSV-1 ea moloko oa bobeli, e ntlafatsa ho ikatisa ha eona ka har'a lisele tsa mofetše le ho kenngoa ha likarabo tsa 'mele oa ho itšireletsa mafung ha li ntse li boloka tšireletso e phahameng.Delytact ke moloko oa boraro oa oncolytic HSV-1 hajoale o ntseng o hlahlojoa ke lingaka.Tumello ea Delytact naheng ea Japane e thehiloe haholo-holo tekong ea bongaka ea karolo ea bobeli ea letsoho le le leng.Ho bakuli ba nang le glioblastoma e iphetang, Delytact e fihletse ntlha ea mantlha ea sekhahla sa ho phela sa selemo se le seng, mme liphetho li bonts'itse hore Delytact e bonts'itse katleho e ntle ha e bapisoa le G207.Matla a matla a ho ikatisa le ts'ebetso e phahameng ea antitumor.Sena se ne se sebetsa mefuteng e tiileng ea hlahala ea matsoele, prostate, schwannomas, nasopharyngeal, hepatocellular, colorectal, malignant peripheral nerve sheath tumors le mofets'e oa qoqotho.

(9) Upstaza

COMPANY: E Thehiloe ke PTC Therapeutics, Inc. (NASDAQ: PTCT).

Nako ea ho mmaraka: E amohetsoe ke European Union bakeng sa ho bapatsa ka Phupu 2022.

Matšoao: Bakeng sa khaello e nkhang hamonate ea L-amino acid decarboxylase (AADC), e amohetsoe bakeng sa kalafo ea bakuli ba likhoeli tse 18 ho ea holimo.

Maikutlo: Upstaza™ (eladocagene exuparvovec) ke in vivo gene therapy e nang le adeno-associated virus type 2 (AAV2) e le eona e tsamaisang.Bakuli baa kula ka lebaka la liphetoho tsa lefutso tse kenyang enzyme ea AADC.AAV2 e na le mofuta o phetseng hantle o kenyang enzyme ea AADC.Mofuta oa matšeliso a liphatsa tsa lefutso o finyella phello ea phekolo.Ka khopolo, tsamaiso e le 'ngoe e sebetsa ka nako e telele.Ke phekolo ea pele ea liphatsa tsa lefutso e rekisoang e kenngoa ka ho toba bokong.Tumello ea ho bapatsa e sebetsa ho linaha tsohle tse 27 tseo e leng litho tsa EU, hammoho le Iceland, Norway le Liechtenstein.

(10) Roctavian

Khampani: E thehiloe ke BioMarin Pharmaceutical (BioMarin).

Nako ea ho rekisoa: E amohetsoe ho bapatsa ke European Union ka Phato 2022;tumello ea ho bapatsa ke UK Medicines and Healthcare Products Administration (MHRA) ka Pulungoana 2022.

Lipontšo: Bakeng sa kalafo ea bakuli ba baholo ba nang le hemophilia e matla ea A ba se nang nalane ea FVIII factor inhibition mme ba le mpe bakeng sa li-antibodies tsa AAV5.

Maikutlo: Roctavian (valoctocogene roxaparvovec) e sebelisa AAV5 joalo ka vector 'me e sebelisa HLP e ikhethileng ka sebete sa motho ho tsamaisa polelo ea human coagulation factor VIII (FVIII) mme sebaka sa B se hlakotsoe.Qeto ea Khomishene ea Europe ea ho fana ka tumello ea ho bapatsa valoctocogene roxaparvovec e ipapisitse le lintlha tse akaretsang tsa projeke ea ntlafatso ea bongaka ea moriana.Har'a tsona, liphetho tsa teko ea kliniki ea GENER8-1 e bontšitse hore ha ho bapisoa le lintlha tsa selemo pele ho ngolisoa, ka mor'a ho kenngoa ha valoctocogene roxaparvovec, tekanyo ea selemo le selemo ea ho tsoa mali (ABR) e fokotsehile haholo, ho sebelisoa ha protheine ea recombinant coagulation factor VIII (F8) ho fokotsehile, kapa ts'ebetso ea mali ea F8 e eketseha haholo 'meleng.Ka mor'a libeke tse 4 tsa phekolo, tekanyo ea tšebeliso ea selemo le selemo ea F8 le ABR e hlokang phekolo e fokotsoe ka 99% le 84%, ka ho latellana, 'me phapang e ne e le ea bohlokoa ka lipalo (p <0.001).Boemo ba ts'ireletso bo ne bo le molemo, 'me ha ho motho ea kileng a ba le bothata ba F8 factor inhibition, malignancy kapa thrombosis side effects,' me ha ho liketsahalo tse mpe tse amanang le phekolo (SAEs) tse tlalehiloeng.

(11) Hemgenix

Khampani: E thehiloe ke UniQure Corporation.

Nako ea ho rekisa: E amohetsoe ke FDA bakeng sa ho bapatsa ka Pulungoana 2022.

Lipontšo: Bakeng sa kalafo ea bakuli ba baholo ba nang le hemophilia B.

Maikutlo: Hemgenix ke phekolo ea liphatsa tsa lefutso e thehiloeng ho vector ea AAV5.Setlhare sena se na le mofuta oa coagulation factor IX (FIX) oa mofuta oa FIX-Padua, o tsamaisoang ka methapo.Ka mor'a tsamaiso, liphatsa tsa lefutso li ka hlalosa FIX coagulation factor sebeteng le secrete Ka mor'a ho kena maling ho etsa mosebetsi oa coagulation, e le ho finyella morero oa phekolo, ka khopolo, tsamaiso e le 'ngoe e sebetsa ka nako e telele.

(12) Adstiladrin

Khampani: E Thehiloe ke Ferring Pharmaceuticals .

Nako ea ho rekisa: E amohetsoe ke FDA bakeng sa ho bapatsa ka December 2022.

Lipontšo: Bakeng sa phekolo ea kankere ea senya e se nang mesifa e kotsi haholo (NMIBC) e sa arabeleng ho Bacillus Calmette-Guerin (BCG) .

Litlhaloso: Adstiladrin ke mofuta oa kalafo o thehiloeng ho adenoviral vector e sa ikatisang, e ka fetisang protheine ea alfa-2b ka har'a lisele tse shebiloeng, 'me e tsamaisoa ka catheter ea moroto ka har'a senya (e fanoang hang ka mor'a likhoeli tse ling le tse ling tse tharo), vector ea kokoana-hloko e ka tšoaetsa lisele tsa "bladder" ka "bladder" ea "bladder" le "bladder". phello ea utic.Ka hona, mokhoa ona o mocha oa phekolo ea liphatsa tsa lefutso o fetola lisele tsa mokuli tsa lebota la senya hore e be “fektheri” e nyenyane e hlahisang interferon, kahoo e matlafatsa matla a mokuli a ho loantša kankere.

Tšireletseho le katleho ea Adstiladrin li ile tsa hlahlojoa thutong ea meriana e mengata e kenyeletsang bakuli ba 157 ba nang le kotsi e kholo ea BCG-e sa arabeleng NMIBC.Bakuli ba ile ba amohela Adstiladrin likhoeli tse ling le tse ling tse tharo ho fihlela likhoeling tsa 12, kapa ho fihlela chefo e sa amoheleheng ea phekolo kapa ho khutla ha NMIBC ea boemo bo phahameng.Ka kakaretso, karolo ea 51 lekholong ea bakuli ba ngolisitsoeng ba phekoloang ka Adstiladrin ba fihletse karabelo e feletseng (ho nyamela ha matšoao 'ohle a kankere a bonoang ka cystoscopy, biopsy tissue, le moroto).

3. Lithethefatsi tse nyenyane tsa nucleic acid

(1) Vitravene

Khampani: E entsoe ka kopanelo ke Ionis Pharma (eo pele e neng e le Isis Pharma) le Novartis.

Nako ea ho rekisa: Ka 1998 le 1999, e ile ea lumelloa ho bapatsa ke FDA le EU EMA.

Lipontšo: Bakeng sa phekolo ea cytomegalovirus retinitis ho bakuli ba nang le HIV.

Litlhaloso: Vitravene ke lithethefatsi tsa antisense oligonucleotide, e leng lithethefatsi tsa pele tsa oligonucleotide tse amohelehang bakeng sa ho rekisoa lefatšeng.Boemong ba pele ba lethathamo, tlhoko ea 'maraka ea lithethefatsi tse khahlanong le CMV e ne e potlakile haholo;hamorao, ka lebaka la nts'etsopele ea phekolo e sebetsang haholo ea li-antiretroviral, palo ea linyeoe tsa CMV e ile ea theoha haholo.Ka lebaka la tlhokeho e botsoa ea 'maraka, sethethefatsi se ile sa qalisoa ka 2002 le 2006 Ho tlohela linaheng tsa EU le United States.

(2) Macugen

Khampani: E thehiloe ke Pfizer le Eyetech.

Nako ea ho rekisoa: E amohetsoe ho ngolisoa United States ka 2004.

Lipontšo: Bakeng sa phekolo ea ho senyeha ha macular ho amanang le lilemo tsa neovascular.

Tlhaloso: Macugen ke pegylated modified oligonucleotide drug, e ka lebisang le ho tlama vascular endothelial growth factor (VEGF165 subtype), 'me mokhoa oa tsamaiso ke ente ea intravitreal.

(3) Defitelio

Khampani: E Thehiloe ke Jazz Pharmaceuticals.

Nako ea ho rekisoa: E amohetsoe ho bapatsa ke European Union ka 2013 mme ea amoheloa ke FDA bakeng sa ho bapatsa ka Hlakubele 2016.

Lipontšo: Bakeng sa phekolo ea lefu la sebete la veno-occlusive le amanang le ho se sebetse ha renal kapa pulmonary ka mor'a ho fetisoa ha lisele tsa mali tsa hematopoietic.

Maikutlo: Defitelio ke lithethefatsi tsa oligonucleotide, e leng motsoako oa oligonucleotides le thepa ea plasmin.E tlositsoe 'marakeng ka 2009 ka mabaka a khoebo.

(4) Kynamro

Khampani: E thehiloe ke Ionis Pharma le Kastle.

Nako ea ho rekisa: Ka 2013, e ile ea lumelloa ho bapatsa United States e le sethethefatsi sa likhutsana.

Lipontšo: Bakeng sa phekolo ea adjuvant ea homozygous family hypercholesterolemia.

Maikutlo: Kynamro ke sethethefatsi sa oligonucleotide sa antisense, e leng oligonucleotide ea antisense e shebileng motho apo B-100 mRNA.Kynamro e tsamaisoa e le 200 mg subcutaneously hang ka beke.

(5) Spinraza

Khampani: E thehiloe ke Ionis Pharmaceuticals.

Nako ea ho rekisoa: E amohetsoe ke FDA bakeng sa ho bapatsa ka December 2016.

Lipontšo: Bakeng sa phekolo ea spinal muscular atrophy (SMA).

Maikutlo: Spinraza (nusinersen) ke sethethefatsi sa oligonucleotide sa antisense.Ka ho tlama sebaka sa cleavage sa SMN2 exon 7, Spinraza e ka fetola RNA cleavage ea SMN2 gene, kahoo e eketsa tlhahiso ea protheine ea SMN e sebetsang ka botlalo.Ka Phato 2016, BIOGEN e sebelisitse khetho ea eona ea ho fumana litokelo tsa lefats'e tsa Spinraza.Spinraza e qalile feela teko ea eona ea pele ea kliniki ho batho ka 2011. Ka lilemo tse 5 feela, e ile ea amoheloa ke FDA bakeng sa ho bapatsa ka 2016, e bontšang hore FDA e ananela ka botlalo ho sebetsa ha eona.Sethethefatsi se ile sa amoheloa bakeng sa ho rekisoa Chaena ka April 2019. Nako eohle ea tumello ea Spinraza Chaena e ne e le ka tlase ho likhoeli tsa 6, 'me e ne e le lilemo tse 2 le likhoeli tsa 2 ho tloha ha Spinraza e qala ho amoheloa United States.Lebelo la ho thathamisa China le se le ntse le potlakile haholo.Sena se boetse se bakoa ke taba ea hore Setsi sa Tlhahlobo ea Lithethefatsi se fane ka "Tsebiso mabapi le Phatlalatso ea Lethathamo la Sehlopha sa Pele sa Lithethefatsi tse Ncha tsa Overseas tse Hlokehang ka Potlako Ts'ebetsong ea Tleliniki" ka la 1 Pulungoana 2018, mme e kenyellelitsoe sehlopheng sa pele sa lithethefatsi tse ncha tse 40 bakeng sa tlhahlobo e potlakileng, eo Spinraza e neng e behiloe ho eona.

(6) Exoda 51

Khampani: E thehiloe ke AVI BioPharma (hamorao e ile ea rehoa Sarepta Therapeutics).

Nako ea ho rekisa: Ka Loetse 2016, e ile ea amoheloa bakeng sa ho bapatsa ke FDA.

Lipontšo: Bakeng sa phekolo ea Duchenne muscular dystrophy (DMD) e nang le exon 51 skipping phetoho ea liphatsa tsa lefutso mofuteng oa DMD.

Litlhaloso: Exondys 51 ke lithethefatsi tsa oligonucleotide tsa antisense, oligonucleotide ea antisense e ka tlama sebakeng sa exon 51 ea pre-mRNA ea liphatsa tsa lefutso tsa DMD, e leng se etsang hore ho thehoe mRNA e hōlileng tsebong, karolo ea exon 51 e thibetsoe Excision, ka tsela eo, ka mokhoa o itseng o lokisa liforomo tse khutšoanyane tsa protheine, ho thusa bakuli ba bang ho lokisa li-protein tse tloaelehileng tsa mRNA. , kahoo ho ntlafatsa matšoao a mokuli.

(7) Tegsedi

Khampani: E thehiloe ke Ionis Pharmaceuticals.

Nako ea ho rekisoa: E amohetsoe ho bapatsa ke European Union ka Phupu 2018.

Lipontšo: Bakeng sa phekolo ea hereditary transthyretin amyloidosis (hATTR).

Maikutlo: Tegsedi ke sethethefatsi sa oligonucleotide sa antisense se lebisang ho transthyretin mRNA.Ke moriana oa pele o amohetsoeng lefatšeng ka bophara bakeng sa kalafo ea hATTR.E tsamaisoa ka ente ea subcutaneous.Sethethefatsi se fokotsa tlhahiso ea protheine ea ATTR ka ho lebisa mRNA ea transthyretin (ATTR), 'me e na le karolelano e ntle ea kotsi ea phekolo ea ATTR,' me methapo ea kutlo le boleng ba bophelo ba mokuli li ntlafetse haholo, 'me e lumellana le mefuta ea phetoho ea TTR, Leha ho le joalo boemo ba lefu kapa boteng ba lefu la pelo bo ne bo le bohlokoa.

(8) Onpattro

Khampani: E entsoe ka kopanelo ke Alnylam Corporation le Sanofi Corporation.

Nako ea ho rekisoa: E amohetsoe ho ngolisoa United States ka 2018.

Lipontšo: Bakeng sa phekolo ea hereditary transthyretin amyloidosis (hATTR).

Litlhaloso: Onpattro ke lithethefatsi tsa siRNA tse lebisang transthyretin mRNA, e fokotsang tlhahiso ea protheine ea ATTR sebeteng le ho fokotsa ho bokellana ha li-amyloid ho methapo ea peripheral ka ho lebisa tlhokomelo ho mRNA ea transthyretin (ATTR) , kahoo e ntlafatsa le ho fokotsa matšoao a lefu lena.

(9) Givlaari

Khampani: E Thehiloe ke Alnylam Corporation.

Nako ea ho rekisoa: E amohetsoe ke FDA bakeng sa ho bapatsa ka Pulungoana 2019.

Lipontšo: Bakeng sa phekolo ea acute hepatic porphyria (AHP) ho batho ba baholo.

Maikutlo: Givlaari ke sethethefatsi sa siRNA, e leng moriana oa bobeli oa siRNA o amohetsoeng bakeng sa ho rekisoa ka mor'a Onpattro.Mokhoa oa tsamaiso ke subcutaneous injection.Sethethefatsi se lebisa mRNA ea protheine ea ALAS1, 'me phekolo ea khoeli le khoeli le Givlaari e ka fokotsa haholo le ka ho sa feleng boemo ba ALAS1 sebeteng, ka tsela eo ea fokotsa maemo a neurotoxic ALA le PBG ho ea boemong bo tloaelehileng, kahoo e Fokotsa matšoao a lefu la mokuli.Lintlha li bontšitse hore bakuli ba tšoaroang ka Givlaari ba ne ba fokotsehile ka 74% palo ea ho tsieleha ha ho bapisoa le sehlopha sa placebo.

(10) Vyondys53

KAMPUNI: E thehiloe ke Sarepta Therapeutics.

Nako ea ho rekisoa: E amohetsoe ke FDA bakeng sa ho bapatsa ka December 2019.

Lipontšo: Bakeng sa phekolo ea bakuli ba DMD ba nang le lefutso la dystrophin exon 53 splicing mutation.

Maikutlo: Vyondys 53 ke sethethefatsi sa oligonucleotide sa antisense, se shebaneng le ts'ebetso ea splicing ea dystrophin pre-mRNA.Exon 53 e fokotsehile ka mokhoa o itseng, ke hore ha e teng ho mRNA e hōlileng, 'me e etselitsoe ho hlahisa dystrophin e fokolang empa e ntse e sebetsa, kahoo e ntlafatsa bokhoni ba boikoetliso ho bakuli.

(11) Waylivra

Khampani: E thehiloe ke Ionis Pharmaceuticals le setsi sa eona sa Akcea Therapeutics.

Nako ea ho rekisoa: E amohetsoe ho bapatsa ke European Medicines Agency (EMA) ka Mots'eanong 2019.

Lipontšo: E le phekolo ea adjuvant ho phaella taolong ea lijo ho bakuli ba baholo ba nang le lefu la malapa la chylomicronemia (FCS).

Maikutlo: Waylivra ke sethethefatsi sa oligonucleotide sa antisense, e leng setlhare sa pele se lumelletsoeng ho rekisoa lefatšeng ka bophara bakeng sa kalafo ea FCS.

(12) Leqvio

Khampani: E thehiloe ke Novartis.

Nako ea ho rekisoa: E amohetsoe ke European Union bakeng sa ho bapatsa ka December 2020.

Lipontšo: Bakeng sa kalafo ea batho ba baholo ba nang le hypercholesterolemia ea mantlha (heterozygous family and non-family) kapa dyslipidemia e tsoakiloeng.

Maikutlo: Leqvio ke sethethefatsi sa siRNA se shebileng PCSK9 mRNA.Ke phekolo ea pele lefatšeng ea siRNA bakeng sa ho theola k'holeseterole (LDL-C).E tsamaisoa ka ente ea subcutaneous.Setlhare se fokotsa boemo ba protheine ea PCSK9 ka ho kena-kenana le RNA, ka hona ho fokotsa boemo ba LDL-C.Lintlha tsa bongaka li bonts'a hore ho bakuli ba sa khoneng ho fokotsa maemo a LDL-C ho fihlela moo ho lebelletsoeng kamora kalafo ka tekanyetso e phahameng e mamelloang ea li-statins, Leqvio e ka fokotsa LDL-C ka hoo e ka bang 50%.

(13) Ntlafatso

Khampani: E Thehiloe ke Alnylam Pharmaceuticals.

Nako ea ho rekisoa: E amohetsoe ke European Union bakeng sa ho bapatsa ka Pulungoana 2020.

Lipontšo: Bakeng sa phekolo ea mofuta oa mantlha oa hyperoxaluria 1 (PH1).

Maikutlo: Oxlumo ke moriana oa siRNA o lebisitseng hydroxyacid oxidase 1 (HAO1) ​​mRNA, 'me mokhoa oa tsamaiso ke ente e ka tlas'a letlalo.Setlhare sena se ile sa ntlafatsoa ho sebelisoa k'hemistri ea morao-rao e ntlafalitsoeng ea Alnylam, theknoloji ea conjugation ea ESC-GalNAc, e nolofalletsang siRNA e tsamaisoang ka mokhoa o sa tsitsang ka phehello le matla a maholo.Sethethefatsi sena se theola kapa se thibela hydroxyacid oxidase 1 (HAO1) ​​mRNA, se fokotsa boemo ba glycolate oxidase sebeteng, ebe se sebelisa substrate e hlokahalang bakeng sa tlhahiso ea oxalate, e fokotsa tlhahiso ea oxalate ho laola tsoelo-pele ea lefu lena ho bakuli le ho ntlafatsa matšoao a lefu lena.

(14) Viltepso

Khampani: E thehiloe ke NS Pharma, e leng setsi sa Nippon Shinyaku.

Nako ea ho rekisoa: E amohetsoe ke FDA bakeng sa ho bapatsa ka Phato 2020.

Lipontšo: Bakeng sa phekolo ea Duchenne muscular dystrophy (DMD) e nang le exon 53 skipping phetoho ea liphatsa tsa lefutso mofuteng oa DMD.

Litlhaloso: Viltepso ke lithethefatsi tsa antisense oligonucleotide tse ka tlamang boemong ba exon 53 ea pre-mRNA ea liphatsa tsa lefutso tsa DMD, e leng se etsang hore karolo ea exon 53 e hlahisoe ka mor'a hore ho thehoe mRNA e hōlileng, ka tsela e itseng e lokisoe foreimi ea ho bala ea mRNA Lebokose le thusa bakuli ho kopanya mefuta e meng e tloaelehileng ea protheine ho feta bakuli ba tloaelehileng ba liprotheine tse nang le mefuta e sa tšoaneng ea liprotheine.

(15) Li-Amondys 45

Khampani: E thehiloe ke Sarepta Therapeutics.

Nako ea ho rekisoa: E amohetsoe ke FDA bakeng sa ho bapatsa ka Hlakola 2021.

Lipontšo: Bakeng sa phekolo ea Duchenne muscular dystrophy (DMD) e nang le exon 45 skipping phetoho ea liphatsa tsa lefutso mofuteng oa DMD.

Litlhaloso: Amondys 45 ke lithethefatsi tsa oligonucleotide tsa antisense, oligonucleotide ea antisense e ka tlama sebakeng sa exon 45 ea pre-mRNA ea liphatsa tsa lefutso tsa DMD, e leng se etsang hore karolo ea exon 45 e thibeloe ka mor'a hore ho thehoe mRNA e hōlileng tsebong Excision, ka tsela eo, ka tsela e itseng e lokisa liforomo tse khutšoanyane tsa ho bala ha mRNA, ho thusa bakuli ba bang ka mokhoa o fokolang oa ho bala, ho thusa ho lokisa liforomo tse fokolang tsa mRNA. protheine, ka hona ho ntlafatsa matšoao a mokuli.

(16) Amvuttra (vutrisiran)

Khampani: E Thehiloe ke Alnylam Pharmaceuticals.

Nako ea ho rekisoa: E amohetsoe ke FDA bakeng sa ho bapatsa ka Phuptjane 2022.

Lipontšo: Bakeng sa phekolo ea hereditary transthyretin amyloidosis le polyneuropathy (hATTR-PN) ho batho ba baholo.

Maikutlo: Amvuttra (Vutrisiran) ke sethethefatsi sa siRNA se lebisitseng transthyretin (ATTR) mRNA, se tsamaisoang ka ente ea subcutaneous.Vutrisiran e thehiloe ho Alnylam's Enhanced Stability Chemistry (ESC)-GalNAc conjugate delivery platform design ka matla a eketsehileng le botsitso ba metabolism.Tumello ea phekolo e itšetlehile ka lintlha tsa likhoeli tse 9 tsa thuto ea eona ea bongaka ea Phase III (HELIOS-A), 'me liphello tse akaretsang li bontša hore phekolo e ntlafalitse matšoao a hATTR-PN,' me ho feta 50% ea boemo ba bakuli bo ile ba khutlisetsoa morao kapa ba emisoa ho mpefala.

4. Lithethefatsi tse ling tsa phekolo ea lefutso

(1) Rexin-G

Khampani: E thehiloe ke Epeius Biotech.

Nako ea ho rekisa: Ka 2005, e ile ea lumelloa ho bapatsa ke Tsamaiso ea Lijo le Lithethefatsi ea Philippines (BFAD).

Lipontšo: Bakeng sa phekolo ea kankere e tsoetseng pele e hanyetsanang le chemotherapy.

Maikutlo: Rexin-G ke ente ea nanoparticle e tletseng liphatsa tsa lefutso.E hlahisa mofuta oa cyclin G1 o fetohang liseleng tse lebisitsoeng ka har'a vector ea retroviral ho bolaea ka ho khetheha lihlahala tse tiileng.Mokhoa oa tsamaiso ke intravenous infusion.Joalo ka setlhare se lebisitsoeng ho hlahala se batlang le ho senya lisele tsa mofetše oa metastatic, se na le phello e itseng ea pheko ho bakuli ba hlolehileng ho sebelisa lithethefatsi tse ling tsa mofetše, ho kenyeletsoa le biologics e lebisitsoeng ho tsona.

(2) Neovasculgen

Khampani: E thehiloe ke Setsi sa Li-Stem cell.

Nako ea ho thathamisoa: E ile ea amoheloa hore e ngolisoe Russia ka la 7 Tšitoe, 2011, eaba e thakholoa Ukraine ka 2013.

Lipontšo: Bakeng sa phekolo ea lefu la peripheral vascular arterial, ho akarelletsa le ischemia e matla ea maoto.

Maikutlo: Neovasculgen ke phekolo ea liphatsa tsa lefutso e thehiloeng ho li-plasmids tsa DNA.The vascular endothelial growth factor (VEGF) 165 gene e hahiloe mokokotlong oa plasmid mme e kenngoa ho bakuli.

(3) Collategene

Khamphani: E kopantsoe ka kopanelo ke Univesithi ea Osaka le lik'hamphani tsa capital capital.

Nako ea ho rekisoa: E amohetsoe ke Lekala la Bophelo bo Botle, Basebetsi le Boiketlo ba Japane ka Phato 2019.

Lipontšo: Kalafo ea ischemia e tebileng e tlaase.

Maikutlo: Collategene ke phekolo ea liphatsa tsa lefutso e thehiloeng ho plasmid, setlhare sa pele sa lehae sa phekolo ea lefutso se hlahisoang ke AnGes, k'hamphani ea phekolo ea lefutso Japane.Karolo e ka sehloohong ea lithethefatsi ena ke plasmid e hlobotseng e nang le tatellano ea liphatsa tsa lefutso tsa hepatocyte growth factor (HGF).Haeba moriana o kenngoa ka har'a mesifa ea maoto a ka tlaase, HGF e hlalositsoeng e tla khothalletsa ho thehoa ha methapo e mecha ea mali ho pota-potile methapo ea mali e koetsoeng.Liteko tsa bongaka li netefalitse phello ea eona ho ntlafatsa liso.

Foregene e ka thusa joang nts'etsopele ea kalafo ea lefutso?

Re thusa ho boloka nako ea tlhahlobo tlhahlobong e kholo, qalong ea nts'etsopele ea lithethefatsi tsa siRNA.

Lintlha tse ling etela:

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