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I kēia mau lā, ua ʻae ʻia nā lāʻau lapaʻau gene therapy ʻekolu no ke kūʻai aku ʻana, ʻo ia hoʻi: (1) Ma Iulai 21, 2022, PTC Therapeutics, Inc. (NASDAQ: PTCT) hoʻolaha i kāna AAV gene therapy Upstaza™ ua ʻae ʻia e ka European Commission ʻO ia ka mua marketed gene therapy i hoʻokomo pololei ʻia i loko o ka lolo (e ʻike i nā ʻatikala mua: ʻO kekahi Milestone in Gene Therapy mua i ʻae ʻia ʻo AV Therapy gene therapy |(2) Ma ʻAukake 17, 2022, ua ʻae ka US Food and Drug Administration (FDA) i ka lāʻau o Bluebird Bio's gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) no ka mālama ʻana i ka beta thalassemia.ʻO ka ʻae ʻana o ka lāʻau lapaʻau ma ʻAmelika Hui Pū ʻIa he mea kānalua ʻole he "kōkua i ka hau" no Bluebird Bio, aia i loko o ka pilikia kālā.(3) Ma ʻAukake 24, 2022, ua hoʻolaha ʻo BioMarin Pharmaceutical (BioMarin) ua ʻae ke Komisina ʻEulopa i ke kūʻai kūʻai kūlana o ROCTAVIAN™ (valoctocogene roxaparvovec), he lāʻau lapaʻau no ka hemophilia A, no ka mālama ʻana i nā poʻe maʻi me ka ʻole o ka moʻolelo FVIII factor inhibitors a me nā antibodies AAV5 maikaʻi ʻole i nā maʻi maʻi me ka hemophilia ma mua o ka maʻi hemophilia.I kēia manawa, ua ʻae ʻia nā lāʻau lapaʻau gene 41 no ke kūʻai ʻana i ka honua.

ʻO Gene ka ʻāpana genetic kumu e hoʻomalu i nā ʻano.Ma waho aʻe o nā genes o kekahi mau maʻi maʻi, i haku ʻia me RNA, ʻo ka DNA ka nui o nā mea ola.ʻO ka hapa nui o nā maʻi o ke kino ma muli o ka pilina ma waena o nā genes a me ke kaiapuni, a he nui nā maʻi e hiki ke ho'ōla a hoʻohaʻahaʻa ʻia paha ma o ka lāʻau lapaʻau.Manaʻo ʻia ʻo Gene therapy he hoʻololi i ke kahua o ka lāʻau lapaʻau a me ka lāʻau lapaʻau.ʻO nā lāʻau lapaʻau ākea ākea e pili ana i nā lāʻau lapaʻau e pili ana i nā lāʻau DNA-modified DNA (e like me ka viral vector-based in vivo gene therapy drugs, in vitro gene therapy drugs, nā plasmid hubad, etc.) a me nā lāʻau RNA (e like me nā lāʻau antisense oligonucleotide, nā lāʻau siRNA, a me ka mRNA gene therapy, etc.);ʻO nā lāʻau lapaʻau Gene therapy ka mea nui e pili ana i nā lāʻau lapaʻau DNA plasmid, nā lāʻau lapaʻau lāʻau e pili ana i nā vectors viral, nā lāʻau lapaʻau gene therapy e pili ana i nā vectors bacterial, nā ʻōnaehana hoʻoponopono gene a me nā lāʻau lapaʻau cell i hoʻololi ʻia i loko o vitro.Ma hope o nā makahiki o ka hoʻomohala ʻana, ua loaʻa i nā lāʻau lapaʻau gene therapy nā hopena lapaʻau hoʻoikaika.(ʻaʻole i komo i nā lāʻau lapaʻau DNA a me nā maʻi mRNA), ua ʻae ʻia nā lāʻau lapaʻau gene 41 no ke kūʻai aku ʻana ma ka honua.Me ka hoʻomaka ʻana o nā huahana a me ka hoʻomohala wikiwiki ʻana o ka ʻenehana hoʻomaʻamaʻa gene, e hoʻomaka ana ka lāʻau lapaʻau i kahi manawa o ka ulu wikiwiki.

honua1

Ka hoʻokaʻawale ʻana o ka lāʻau lapaʻau gene (Ke kumu kiʻi: Biological Jingwei)

Hoʻopaʻa kēia ʻatikala i nā lāʻau lapaʻau gene 41 i ʻae ʻia no ke kūʻai ʻana (koe nā lāʻau lapaʻau DNA a me nā maʻi mRNA).

1. In vitro gene therapy

(1) Strimvelis

Hui: Hoʻomohala ʻia e GlaxoSmithKline (GSK).

Ka manawa i ka mākeke: Apono ʻia e ka European Union i Mei 2016.

Nā hōʻailona: No ka mālama ʻana i ka maʻi immunodeficiency hui nui (SCID).

Manaʻo: ʻO ke kaʻina hana maʻamau o kēia lāʻau lapaʻau, ʻo ia ka loaʻa mua ʻana o nā mea maʻi ponoʻī hematopoietic stem cell, hoʻonui a moʻomeheu iā lākou i loko o ka vitro, a laila hoʻohana i ka retrovirus e hoʻokomo i kahi kope o ka gene ADA (adenosine deaminase) hana i loko o kā lākou hematopoietic stem cell, a hope e hoʻololi i nā cell stem hematopoietic i hoʻololi ʻia.Hoʻokomo hou ʻia nā pūnaeʻa hematopoietic i loko o ke kino.Ua hōʻike ʻia nā hopena lapaʻau ʻo 3 mau makahiki ola ola o nā maʻi ADA-SCID i mālama ʻia me Strimvelis he 100%.

(2) Zalmoxis

Hui: Hana ʻia e MolMed, Italia.

Ka manawa i ka mākeke: Loaʻa i ka mana kūʻai kūʻai kūlana EU ma 2016.

Nā hōʻailona: Hoʻohana ʻia ia no ka adjuvant therapy o ka ʻōnaehana pale o nā mea maʻi ma hope o ka hoʻololi ʻana o ka hematopoietic stem cell transplant.

Manaʻo: ʻO Zalmoxis kahi allogeneic T cell suicide gene immunotherapy i hoʻololi ʻia e retroviral vector.ʻO ka 1NGFR a me ka HSV-TK Mut2 genes pepehi kanaka e ʻae i ka poʻe e hoʻohana i ka ganciclovir i kēlā me kēia manawa e pepehi i nā pūnaewele T e hoʻopiʻi i nā pane kūlohelohe ʻino, e pale i ka deterioration hou o ka GVHD e hiki mai ana, a e hoʻihoʻi i ka hana immune i nā maʻi me ka haploidentical HSCT ma hope o ke kaʻina Escort.

(3) Invossa-K

Hui: Hoʻomohala ʻia e ka hui ʻo TissueGene (KolonTissueGene).

Ka manawa i ka mākeke: Apono ʻia no ka papa inoa ʻana ma South Korea i Iulai 2017.

Nā hōʻailona: No ka mālama ʻana i ka maʻi o ka kuli degenerative.

Manaʻo: ʻO Invossa-K kahi allogeneic cell gene therapy e pili ana i nā chondrocytes kanaka.Hoʻololi ʻia nā cell allogeneic i loko o ka vitro, a hiki i nā cell i hoʻololi ʻia ke hōʻike a hūnā i ka hoʻololi ʻana i ka mea ulu β1 (TGF-β1) ma hope o ka hoʻokele intra-articular.β1), ma laila e hoʻomaikaʻi ai i nā hōʻailona o ka osteoarthritis.Hōʻike nā hopena maʻi e hiki ke hoʻomaikaʻi nui ʻo Invossa-K i ka ʻāʻī o ke kuli.Ua hoʻopau ʻia ka laikini e ka mea hoʻoponopono lāʻau lapaʻau o South Korea ma 2019 no ka mea ua kuhi hewa ka mea hana i nā mea i hoʻohana ʻia.

(4) Zynteglo

Hui: Hoʻomohala ʻia e ka hui ʻAmelika bluebird bio (bluebird bio).

Ka manawa i ka mākeke: Ua ʻae ʻia e ka European Union ma 2019, a ua ʻae ʻia e ka FDA i ʻAukake 2022.

Nā hōʻailona: No ka mālama ʻana i ka β-thalassemia e pili ana i ka transfusion.

Manaʻo: ʻO Zynteglo he lentiviral in vitro gene therapy, e hoʻohana ana i ka vector lentiviral e hoʻokomo i kahi kope hana o ka gene β-globin maʻamau (βA-T87Q-globin gene) i loko o nā pūnaewele hematopoietic i wehe ʻia mai nā maʻi., a laila e hoʻokomo hou i kēia mau pūnaeʻa autologous hematopoietic i hoʻololi hou ʻia i loko o ka mea maʻi.I ka manawa e loaʻa ai i ka mea maʻi kahi gene βA-T87Q-globin maʻamau, hiki iā lākou ke hana i kahi protein HbAT87Q maʻamau, hiki ke hōʻemi a hoʻopau paha i ka pono o ke kahe koko.He lāʻau lapaʻau i hoʻokahi manawa i hoʻolālā ʻia e hoʻololi i ke kahe koko o ke ola a me nā lāʻau lapaʻau no ka poʻe maʻi 12 makahiki a ʻoi aku.

(5) Skysona

Hui: Hoʻomohala ʻia e ka hui ʻAmelika bluebird bio (bluebird bio).

Ka manawa i ka mākeke: Apono ʻia e ka EU no ke kūʻai aku ma Iulai 2021.

Nā hōʻailona: No ka mālama ʻana i ka cerebral adrenoleukodystrophy (CALD).

Manaʻo: ʻO Skysona gene therapy ʻo ia wale nō ka lāʻau lapaʻau i ʻae ʻia no ka mālama ʻana i ka cerebral adrenoleukodystrophy mua (CALD).ʻO Skysona (elivaldogene autotemcel, Lenti-D) he hematopoietic stem cell lentiviral in vitro gene therapy Lenti-D.Penei ke kaʻina hana maʻamau o ka lāʻau: autologous hematopoietic stem cell i waho o ka mea maʻi, hoʻololi ʻia i loko o ka vitro e ka lentivirus e lawe ana i ka gene ABCD1 kanaka, a laila hoʻihoʻi ʻia i loko o ka mea maʻi.No ka mālama ʻana i nā maʻi ma lalo o 18 makahiki me ka ABCD1 gene mutation a me CALD.

(6) Kymriah

Hui: Kūkulu ʻia e Novartis.

Ka manawa i ka mākeke: ʻApono ʻia e ka FDA i ʻAukake 2017.

Nā hōʻailona: Hoʻomaʻamaʻa i ka maʻi leukemia lymphoblastic acute B-cell acute a me DLBCL i hoʻihoʻi ʻia a refractory.

Manaʻo: ʻO Kymriah kahi lāʻau lapaʻau lentiviral in vitro gene therapy, ka lāʻau lapaʻau CAR-T mua i ʻae ʻia ma ka honua, e kuhikuhi ana i ka CD19 a me ka hoʻohana ʻana i ka co-stimulatory factor 4-1BB.ʻO ke kumu kūʻai he $475,000 ma US a me $313,000 ma Iapana.

(7) Yescarta

Hui: Hoʻomohala ʻia e Kite Pharma, he lālā o Gileada.

Ka manawa i ka mākeke: ʻApono ʻia e ka FDA i ʻOkakopa 2017.

Nā hōʻailona: No ka mālama ʻana i ka lymphoma B-cell nui.

Manaʻo: Yescarta he retroviral in vitro gene therapy.ʻO ia ka lua o ka lāʻau lapaʻau CAR-T i ʻae ʻia ma ka honua.Hoʻohana ia i ka CD19 a hoʻohana i ke kumu costimulatory o CD28.ʻO ke kumu kūʻai ma US he $373,000.

(8) Tecartus

Hui: Hoʻomohala ʻia e Gileada (GILD).

Ka manawa i ka mākeke: ʻApono ʻia e ka FDA i Iulai 2020.

Nā hōʻailona: No ka lymphoma cell mantle i hoʻihoʻi hou ʻia a i ʻole refractory.

Manaʻo: ʻO Tecartus kahi autologous CAR-T cell therapy e kuhikuhi ana i ka CD19, a ʻo ia ke kolu o ka lāʻau lapaʻau CAR-T i ʻae ʻia no ke kūʻai aku ma ka honua.

(9) Breyanzi

Hui: Hoʻomohala ʻia e Bristol-Myers Squibb (BMS).

Ka manawa i ka mākeke: ʻApono ʻia e ka FDA i Pepeluali 2021.

Nā hōʻailona: Relapsed a refractory (R/R) nui B-cell lymphoma (LBCL).

Manaʻo: Breyanzi he in vitro gene therapy e pili ana i ka lentivirus, a ʻo ka hā o ka lāʻau lapaʻau CAR-T i ʻae ʻia no ke kūʻai aku ʻana ma ka honua, e kuhikuhi ana i ka CD19.ʻO ka ʻae ʻana o Breyanzi he mea nui ia no Bristol-Myers Squibb i ke kahua o ka immunotherapy cellular, i loaʻa iā Bristol-Myers i ka wā i loaʻa ai iā Celgene no $ 74 biliona ma 2019.

(10) Abecma

Hui: Hoʻokumu pū ʻia e Bristol-Myers Squibb (BMS) a me bluebird bio.

Ka manawa i ka mākeke: ʻApono ʻia e ka FDA i Malaki 2021.

Nā ʻōlelo hōʻike: ka hoʻihoʻi ʻana a i ʻole refractory multiple myeloma.

Manaʻo: ʻO Abecma kahi lentivirus-based in vitro gene therapy, ka mua o ka honua CAR-T cell therapy e kuhikuhi ana iā BCMA, a me ka lima o ka CAR-T therapy i ʻae ʻia e ka FDA.ʻO ke kumu o ka lāʻau lapaʻau ʻo ka hōʻike ʻana i ka mea loaʻa BCMA chimeric ma nā pūnae T autologous o ka mea maʻi ma o ka lentivirus-mediated genetic modification in vitro.Ma mua o ka hoʻokomo ʻia ʻana o ka lāʻau lapaʻau cell gene, ua loaʻa i ka mea maʻi nā pūhui ʻelua o cyclophosphamide a me fludarabine no ka mālama mua ʻana.Lapaʻau no ka wehe ʻana i nā pūnaewele T i hoʻololi ʻole ʻia mai ka mea maʻi, a laila hoʻokomo hou i nā pūnaewele T i hoʻololi ʻia i loko o ke kino o ka mea maʻi e ʻimi a pepehi i nā maʻi kanesa hōʻike BCMA.

(11) Libmeldy

Hui: Hoʻomohala ʻia e Orchard Therapeutics.

Ka manawa i ka mākeke: ʻApono ʻia e ka European Union no ka papa inoa ma Dekemaba 2020.

Nā hōʻailona: No ka mālama ʻana i ka metachromatic leukodystrophy (MLD).

Manaʻo: ʻO Libmeldy kahi lāʻau lapaʻau e pili ana i ka lentiviral in vitro gene modification o autologous CD34+ cell.Hōʻike ka ʻikepili maʻi i ka hopena o ka infusion intravenous o Libmeldy i ka hoʻololi ʻana i ke ʻano o ka MLD hoʻomaka mua a me ka hōʻino kino a me ka cognitive i nā maʻi i mālama ʻole ʻia o ka makahiki like.

(12) Benoda

Hui: Kūkulu ʻia e WuXi Junuo.

Ka manawa i ka mākeke: Ua ʻae ʻia e NMPA i Kepakemapa 2021.

Nā hōʻailona: Hoʻomaʻamaʻa i ka lymphoma B-cell nui (r/r LBCL) i hoʻihoʻi hou ʻia i nā maʻi maʻi ma hope o ka laina ʻelua a i ʻole ka ʻōnaehana systemic therapy.

Manaʻo: ʻO Benoda kahi anti-CD19 CAR-T gene therapy, a ʻo ia hoʻi ka huahana kumu o WuXi Junuo.ʻO ia ka lua o ka huahana CAR-T i ʻāpono ʻia ma Kina, koe wale no ka lymphoma B-cell nui i hoʻihoʻi hou ʻia.Eia kekahi, ua hoʻolālā ʻo WuXi Junuo e hoʻomohala iā Ruiki Orenza injection no ka mālama ʻana i nā ʻano hōʻailona ʻē aʻe, e like me ka follicular lymphoma (FL), mantle cell lymphoma (MCL), lymphocytic leukemia (CLL), ka lua-line diffuse nui B-cell lymphoma (DLBCL) a me ka maʻi leukemia lymphoblastic (ALL).

(13) CARVYKTI

Hui: ʻO ka huahana mua a Legend Bio i ʻāpono ʻia.

Ka manawa i ka mākeke: ʻApono ʻia e ka FDA i Pepeluali 2022.

Nā hōʻailona: Lapaʻau ʻana i ka myeloma lehulehu (R/R MM).

Manaʻo: ʻO CARVYKTI (ciltacabtagene autoleucel, i kapa ʻia ʻo Cilta-cel) he CAR-T cell immune gene therapy me ʻelua mau antibodies domain hoʻokahi e huli ana i ka B cell maturation antigen (BCMA).Hōʻike ka ʻikepili ua hōʻike ʻo CARVYKTI i ka helu pane holoʻokoʻa a hiki i ka 98% i nā poʻe maʻi me ka relapsed a i ʻole refractory multiple myeloma i loaʻa i ʻehā a ʻoi aʻe paha nā lāʻau lapaʻau ma mua, me ka proteasome inhibitors, immunomodulators, a me anti-CD38 monoclonal antibodies.

2. In vivo gene therapy ma muli o nā vectors viral

(1) Ke kāne/hānau hou

Hui: Hoʻomohala ʻia e Shenzhen Saibainuo Company.

Ka manawa e kuai ai: Aponoia no ka papa inoa ma Kina i ka makahiki 2003.

Nā hōʻailona: No ka mālama ʻana i ka maʻi squamous cell carcinoma o ke poʻo a me ka ʻāʻī.

Manaʻo: Recombinant kanaka p53 adenovirus injection Gendicine/Jinshengsheng he adenovirus vector gene therapy drug me nā kuleana waiwai naʻauao kūʻokoʻa nona ka Shenzhen Saibainuo Company.Hoʻokumu ʻia ka lāʻau lapaʻau i ke kanaka maʻamau suppressor gene p53 a me ka recombinant recombinant replication-deficient Human adenovirus type 5 i haku ʻia me ke ʻano adenovirus kanaka 5. ʻO ka mua ke kumu nui o ka lāʻau e hoʻokō ai i ka hopena anti-tumor, a ʻo ka mea hope ke hana ma ke ʻano he lawe.Lawe ka adenovirus vector i ka gene therapeutic p53 i loko o ka cell target, a hōʻike i ka tumor suppressor gene p53 i loko o ka cell target.Hiki i ka huahana ke hoʻoponopono i nā ʻano genes anti-cancer a hoʻohaʻahaʻa i nā hana o nā oncogenes like ʻole, ma laila e hoʻonui ai i ka hopena anti-tumor o ke kino a hoʻokō i ke kumu o ka pepehi ʻana i nā ʻōpū.

(2) Rigvir

Hui: Hoʻomohala ʻia e ka hui Latvian Latima.

Ka manawa e kuai ai: Aponoia ma Latvia i ka makahiki 2004.

Nā hōʻailona: No ka mālama ʻana i ka melanoma.

Manaʻo: ʻO Rigvir kahi lāʻau lapaʻau i hoʻokumu ʻia ma kahi ECHO-7 enterovirus vector i hoʻololi ʻia, i hoʻohana ʻia ma Latvia, Estonia, Polani, Armenia, Belarus a me nā wahi ʻē aʻe, a ke hoʻopaʻa inoa ʻia nei me ka EMA o ka European Union..Ua hōʻoia nā hihia lapaʻau i nā makahiki he ʻumi i hala aku nei he palekana a maikaʻi ʻo Rigvir oncolytic virus, a hiki ke hoʻomaikaʻi i ke ola ola o nā maʻi melanoma e 4-6 mau manawa.Eia kekahi, ua kūpono ka lāʻau lapaʻau no nā ʻano maʻi maʻi ʻē aʻe, e like me ka maʻi maʻi colorectal, ka maʻi pancreatic, ka maʻi maʻi maʻi.ka ma'i 'a'ai, ka ma'i 'a'ai, ka ma'i 'a'ai prostate, ka ma'i 'a'ai, ka ma'i 'a'ai, lymphosarcoma, etc.

(3) Oncorine/Ankerui

Hui: Hoʻomohala ʻia e Shanghai Sunway Biotechnology Co., Ltd.

Ka manawa i ka mākeke: Apono ʻia no ka papa inoa ʻana ma Kina i ka makahiki 2005.

Nā hōʻailona: Hoʻomaʻamaʻa ʻana i nā ʻōpū o ke poʻo a me ka ʻāʻī, ka maʻi maʻi ate, ka maʻi maʻi pancreatic, ka maʻi maʻi ʻāʻī a me nā maʻi maʻi ʻē aʻe.

Manaʻo: ʻO Oncorine kahi huahana oncolytic virus gene therapy e hoʻohana ana i ka adenovirus ma ke ʻano he vector.Hiki i ka oncolytic adenovirus i loaʻa ke hoʻopili kikoʻī i nā maʻi ʻaʻole a i ʻole nā ​​​​gene p53 maʻamau, e hoʻoulu ai i ka lysis cell tumor, a laila e pepehi ai i nā pūnaewele tumo.me ka hoʻopōʻinoʻole i nā pūnaewele maʻamau.Hōʻike nā hopena maʻi i ka palekana a me ka maikaʻi o Anke Rui no nā ʻano maʻi maʻi ʻino.

(4) Glybera

Hui: Hoʻomohala ʻia e uniQure.

Ka manawa i ka mākeke: Apono ʻia ma ʻEulopa ma 2012.

Nā hōʻailona: Hoʻomaʻamaʻa i ka lipoprotein lipase deficiency (LPLD) me nā maʻi koʻikoʻi a i ʻole ka hoʻi hou ʻana o ka maʻi pancreatitis ʻoiai ka ʻai ʻana i ka momona.

Manaʻo: ʻO Glybera (alipogene tiparvovec) kahi lāʻau lapaʻau gene ma muli o AAV ma ke ʻano he vector.Ke hoʻohana nei kēia lāʻau lapaʻau i ka AAV ma ke ʻano he vector e hoʻololi i ka therapeutic gene LPL i loko o nā ʻiʻo ʻiʻo, i hiki ai i nā cell pili ke hana i kekahi nui o ka lipoprotein lipase.Ua hoʻopau ʻia ka lāʻau lapaʻau i ka makahiki 2017, a ʻo nā kumu no ka wehe ʻana i ka papa inoa e pili ana paha i ʻelua mau kumu: ke kumu kūʻai kiʻekiʻe a me ke koi palena palena.ʻO ke kumukūʻai maʻamau o ka lāʻau lapaʻau hoʻokahi e like me ka 1 miliona US kālā, a hoʻokahi wale nō mea maʻi i kūʻai a hoʻohana iā ia i kēia manawa.ʻOiai ua hoʻihoʻi ka hui ʻinikua olakino iā ia no 900,000 US kālā, he kaumaha nui hoʻi ia no ka hui ʻinikua.Eia kekahi, ʻo ka hōʻailona no ka lāʻau lapaʻau he mea liʻiliʻi loa, me ka nui o ka hopena ma kahi o 1 i ka 1 miliona a me kahi kiʻekiʻe o ka misdiagnosis.

(5) Imlygic

Hui: Kūkulu ʻia e Amgen.

Ka manawa i ka mākeke: Ma 2015, ua ʻae ʻia no ka papa inoa ʻana ma ʻAmelika Hui Pū ʻIa a me ʻEulopa.

Nā hōʻailona: Lapaʻau ʻana i nā ʻeha melanoma ʻaʻole hiki ke wehe loa ʻia e ka ʻoki ʻana.

Manaʻo: Imlygic he genetically modified (ka holoi ʻana i kāna mau ʻāpana gene ICP34.5 a me ICP47, a me ka hoʻokomo ʻana i ke kanaka granulocyte-macrophage colony-stimulating factor GM-CSF gene i loko o ka maʻi) attenuated herpes simplex virus type 1 (HSV-1) oncolytic virus, ʻo ka lāʻau oncolytic mua i ʻae ʻia e FDA.ʻO ke ʻano o ka lawelawe ʻana he intralesional injection.Hiki i ka hoʻokomo pololei ʻana i nā ʻeha melanoma ke hana i ka haki ʻana o nā pūnaewele tumo a hoʻokuʻu i nā antigens i loaʻa i ka tumo a me GM-CSF e hoʻolaha i nā pane pale pale anti-tumor.

(6) Luxturna

Hui: Hoʻomohala ʻia e Spark Therapeutics, kahi hui ʻo Roche.

Ka manawa e kūʻai aku ai: Ua ʻae ʻia e ka FDA ma 2017, a laila ʻae ʻia no ke kūʻai aku ma ʻEulopa i 2018.

Nā hōʻailona: No ka mālama ʻana i nā keiki a me nā pākeke me ka nalo ʻana o ka ʻike ma muli o ka hoʻololi ʻana i ke kope pālua o ka gene RPE65 akā me nā helu kūpono o nā cell retinal viable.

Manaʻo: ʻO Luxturna kahi lāʻau lapaʻau i hoʻokumu ʻia ma AAV i lawelawe ʻia e ka subretinal injection.Hoʻohana ka gene therapy i ka AAV2 ma ke ʻano he mea lawe e hoʻokomo i kahi kope hana o ka gene RPE65 maʻamau i loko o nā keʻena retinal o ka mea maʻi, no laila e hōʻike nā cell pili i ka protein RPE65 maʻamau e uku ai i ka hemahema o ka protein RPE65 o ka mea maʻi, a laila e hoʻomaikaʻi ai i ka ʻike o ka mea maʻi.

(7) Zolgensma

Hui: Hoʻomohala ʻia e AveXis, kahi hui o Novartis.

Ka manawa i ka mākeke: ʻApono ʻia e ka FDA i Mei 2019.

Nā hōʻailona: Hoʻomaʻamaʻa ʻana i nā maʻi maʻi ma lalo o 2 mau makahiki (Spinal Muscular Atrophy, SMA).

Manaʻo: ʻO Zolgensma kahi lāʻau lapaʻau e pili ana i ka vector AAV.ʻO kēia lāʻau lapaʻau ʻo ia wale nō ka hoʻolālā lapaʻau no ka spinal muscular atrophy i ʻae ʻia no ke kūʻai ʻana ma ka honua.ʻaoʻao, he holomua holomua.Ke hoʻohana nei kēia gene therapy i ka vector scAAV9 e hoʻokomo i ka gene SMN1 maʻamau i nā maʻi ma o ka infusion intravenous, e hana ana i ka protein SMN1 maʻamau, ma laila e hoʻomaikaʻi ai i ka hana o nā cell i hoʻopili ʻia e like me nā neuron motor.I ka hoʻokaʻawale ʻana, koi ʻia nā lāʻau SMA Spinraza a me Evrysdi i ka dosing hou ʻana no ka manawa lōʻihi, me Spinraza i lawelawe ʻia ma ke ʻano he spinal injection i kēlā me kēia ʻehā mahina, a me Evrysdi, kahi lāʻau lapaʻau waha i kēlā me kēia lā.

(8) Delytact

Hui: Hoʻomohala ʻia e Daiichi Sankyo Company Limited (TYO: 4568).

Ka manawa e kuai ai: Ka ʻae ʻia ʻana mai ke Kuhina Ola, Labour a Welfare (MHLW) i Iune 2021.

Nā hōʻailona: No ka mālama ʻana i ka glioma malignant.

Manaʻo: ʻO Delytact ka hā o ka oncolytic virus gene therapy product i ʻāpono ʻia ma ka honua holoʻokoʻa a ʻo ka hua mua oncolytic virus i ʻāpono ʻia no ka mālama ʻana i ka glioma malignant.ʻO Delytact kahi maʻi maʻi herpes simplex type 1 (HSV-1) i hoʻomohala ʻia e Kauka Todo a me nā hoa hana.Hoʻokomo ʻo Delytact i kahi hoʻololi hoʻopau hou i loko o ka genome G207 o ka lua o ka hanauna HSV-1, e hoʻonui ana i kāna hoʻopiʻi koho ʻana i nā maʻi maʻi maʻi a me ka hoʻokomo ʻana i nā pane pale anti-tumor, ʻoiai e mālama ana i kahi kūlana palekana kiʻekiʻe.ʻO Delytact ka mua o ke kolu o ka hanauna oncolytic HSV-1 i kēia manawa i ka loiloi lapaʻau.Ua hoʻokumu ʻia ka ʻae ʻana o Delytact ma Iapana ma kahi hoʻokolohua lapaʻau Phase 2 lima hoʻokahi.I nā poʻe maʻi me ka glioblastoma hou, ua hālāwai ʻo Delytact i ka hopena mua o ke ola hoʻokahi makahiki, a ua hōʻike nā hopena i ʻoi aku ka maikaʻi o Delytact ma mua o G207.ʻO ka hoʻopiʻi ikaika a me ka hana antitumor kiʻekiʻe.He mea maikaʻi kēia i nā hiʻohiʻona maʻi maʻi me ka umauma, prostate, schwannoma, nasopharyngeal, hepatocellular, colorectal, malignant peripheral nerve sheath tumors a me ka maʻi maʻi thyroid.

(9) Upstaza

Hui: Hoʻomohala ʻia e PTC Therapeutics, Inc. (NASDAQ: PTCT).

Ka manawa i ka mākeke: Apono ʻia e ka EU ma Iulai 2022.

Hōʻike: No ka hemahema L-amino acid decarboxylase (AADC), i ʻae ʻia no ka mālama ʻana i nā maʻi 18 mahina a ʻoi aku.

Manaʻo: ʻO Upstaza™ (eladocagene exuparvovec) he lāʻau lapaʻau in vivo gene me ka hoʻohana ʻana i ka adeno-associated virus type 2 (AAV2) ma ke ʻano he vector.Ua maʻi ka mea maʻi ma muli o ka hoʻololi ʻana i ka gene e hoʻopili ana i ka enzyme AADC.Lawe ʻo AAV2 i kahi gene olakino e hoʻopili ana i ka enzyme AADC.Loaʻa ka hopena therapeutic ma ke ʻano o ka uku genetic.Ma ke kumumanaʻo, hoʻokahi ka hopena no ka manawa lōʻihi.ʻO ia ka lāʻau lapaʻau mua i kūʻai ʻia i loko o ka lolo.Pili ka ʻae kūʻai aku i nā mokuʻāina lālā 27 EU, a me Iceland, Norewai a me Liechtenstein.

(9) Roctavian

Hui: Hoʻomohala ʻia e BioMarin Pharmaceutical (BioMarin).

Ka manawa i ka mākeke: Apono ʻia e ka EU ma ʻAukake 2022.

Nā hōʻailona: No ka mālama ʻana i nā poʻe maʻi me ka hemophilia A koʻikoʻi me ka ʻole o ka moʻolelo o ka FVIII factor inhibition a me AAV5 antibody negative.

Manaʻo: Hoʻohana ʻo Roctavian (valoctocogene roxaparvovec) i ka AAV5 ma ke ʻano he vector a hoʻohana i ka HLP mea hoʻolaha kikoʻī o ke akepaʻa kanaka e hoʻokele i ka hōʻike ʻana o ka helu coagulation kanaka ʻewalu (FVIII) me ka waihona B i holoi ʻia.ʻO ka hoʻoholo a ke Komisina ʻEulopa e ʻae i ke kūʻai aku ʻana o ka valoctocogene roxaparvovec e pili ana i ka ʻikepili holoʻokoʻa o ka papahana hoʻomohala lāʻau lapaʻau.Ma waena o lākou, ua hōʻike ʻia ka pae III hoʻokolohua GENEr8-1 i ka hoʻohālikelike ʻia me ka ʻikepili o ka makahiki ma mua o ke kau inoa ʻana, ma hope o ka infusion hoʻokahi o ka valoctocogene roxaparvovec, ʻoi aku ka haʻahaʻa haʻahaʻa o ke kahe koko makahiki (ABR), ka liʻiliʻi o ka hoʻohana ʻana i ka recombinant factor VIII (F8) hoʻomākaukau protein, a i ʻole ka piʻi nui o ka hana F8 i ke koko.Ma hope o nā pule 4 o ka mālama ʻana, ua hoʻemi ʻia ka hoʻohana F8 makahiki a me ka ABR e koi ana i ka mālama ʻana e 99% a me 84%, i kēlā me kēia, kahi ʻokoʻa nui (p<0.001).Ua maikaʻi ka ʻaoʻao palekana, ʻaʻohe kumuhana i loaʻa i ka F8 factor inhibition, malignancy, a i ʻole thrombotic side effects, a ʻaʻole i hōʻike ʻia nā hanana koʻikoʻi koʻikoʻi (SAEs) pili i ka lāʻau.

3. Nā lāʻau liʻiliʻi nucleic acid

(1) Vitravene

Hui: Hoʻokumu pū ʻia e Ionis Pharma (ʻo Isis Pharma ma mua) a me Novartis.

Ka manawa i ka mākeke: Apono ʻia e FDA a me EU EMA ma 1998 a me 1999.

Nā hōʻailona: No ka mālama ʻana i ka cytomegalovirus retinitis i nā maʻi maʻi HIV.

Manaʻo: ʻO Vitravene kahi lāʻau antisense oligonucleotide a ʻo ka lāʻau oligonucleotide mua i ʻae ʻia no ke kūʻai aku ʻana ma ka honua.I ka hoʻomaka ʻana o ka mākeke, ua wikiwiki loa ka noi mākeke no nā lāʻau anti-cytomegalovirus;a laila ma muli o ka hoʻomohala ʻana o ka antiretroviral therapy ikaika loa, ua hāʻule nui ka nui o nā hihia cytomegalovirus.Ma muli o ke koi haʻahaʻa haʻahaʻa, ua hoʻokuʻu ʻia ka lāʻau lapaʻau ma 2002 a me 2006 Withdrawal ma nā ʻāina EU a me ka US.

(2) Macugen

Hui: Hoʻokumu pū ʻia e Pfizer a me Eyetech.

Ka manawa i ka mākeke: Apono ʻia no ka papa inoa ʻana ma United States i ka makahiki 2004.

Nā hōʻailona: No ka mālama ʻana i ka macular degeneration pili i ka makahiki neovascular.

Manaʻo: ʻO Macugen kahi lāʻau oligonucleotide i hoʻololi ʻia i hoʻololi ʻia e hiki ke hoʻopaʻa a hoʻopaʻa i ka mea ulu endothelial vascular (VEGF165 isoform), a lawelawe ʻia e ka intravitreal injection.

(3) Defitelio

Hui: Hoʻomohala ʻia e Jazz.

Ka manawa i ka mākeke: ʻApono ʻia e ka European Union ma 2013, a ʻae ʻia e ka FDA i Malaki 2016.

Nā hōʻailona: No ka mālama ʻana i ka maʻi hepatic venule occlusive e pili ana me ka renal a i ʻole pulmonary dysfunction ma hope o ka hemopoietic stem cell transplantation.

Manaʻo: ʻO Defitelio kahi lāʻau oligonucleotide, kahi hui o nā oligonucleotides me nā waiwai plasmin.Ua hoʻihoʻi ʻia i ka makahiki 2009 no nā kumu kalepa.

(4) Kynamro

Hui: Hoʻokumu pū ʻia e Ionis Pharma a me Kastle.

Ka manawa e kūʻai aku ai: Ua ʻae ʻia ma ʻAmelika Hui Pū ʻIa ma ke ʻano he lāʻau makua makua ʻole ma 2013.

Nā hōʻailona: No ka mālama ʻana i ka hypercholesterolemia ʻohana homozygous.

Manaʻo: ʻO Kynamro kahi lāʻau antisense oligonucleotide, he antisense oligonucleotide e kuhikuhi ana i ke kanaka apo B-100 mRNA.Hāʻawi ʻia ʻo Kynamro e like me 200 mg subcutaneously i hoʻokahi manawa i ka pule.

(5) Spinraza

Hui: Hoʻomohala ʻia e Ionis Pharmaceuticals.

Ka manawa e kuai ai: Aponoia e ka FDA i Dekemaba 2016.

Nā hōʻailona: No ka mālama ʻana i ka spinal muscular atrophy (SMA).

Manaʻo: ʻO Spinraza (nusinersen) kahi lāʻau antisense oligonucleotide.Hiki iā Spinraza ke hoʻololi i ka RNA splicing o SMN2 gene ma ka hoʻopaʻa ʻana i ka pūnaewele splicing o SMN2 exon 7, a laila e hoʻonui ai i ka hana ʻana o ka protein SMN holoʻokoʻa.I ʻAukake 2016, ua hoʻohana ʻo BIOGEN Corporation i kāna koho e kiʻi i nā kuleana honua o Spinraza.Ua hoʻomaka ʻo Spinraza i kāna hoʻokolohua lapaʻau mua i nā kānaka ma 2011. I nā makahiki 5 wale nō, ua ʻae ʻia e ka FDA ma 2016, e hōʻike ana i ka ʻike piha ʻana o ka FDA i kona pono.Ua ʻae ʻia ka lāʻau lapaʻau no ke kūʻai aku ʻana ma Kina i ʻApelila 2019. ʻO ka pōʻai holoʻokoʻa holoʻokoʻa o Spinraza ma Kina ma lalo o 6 mau mahina.He 2 makahiki a me 2 mahina mai ka manawa i apono mua ia ai o Spinraza ma United States.ʻO ia ʻano blockbuster haole rare maʻi lāʻau hou aia i ka wikiwiki loa o ka papa inoa ʻana ma Kina.ʻO kēia ma muli o ka "Notice on the Release of the First List of Overseas New Drugs Urgently Needed for Clinical Research" i hoʻopuka ʻia e ka Center for Drug Evaluation ma Nowemapa 1, 2018, i hoʻokomo ʻia i loko o ka pūʻulu mua o 40 kī nui nā lāʻau lapaʻau hou no ka loiloi wikiwiki, a ʻo Spinraza i koho ʻia i loko.

(6) Exondys 51

Hui: Hoʻomohala ʻia e AVI BioPharma (i kapa hou ʻia ʻo Sarepta Therapeutics).

Ka manawa i ka mākeke: ʻApono ʻia e ka FDA i Kepakemapa 2016.

Nā hōʻailona: No ka mālama ʻana i ka Duchenne muscular dystrophy (DMD) me DMD gene mutation ma exon 51 skipping gene.

Manaʻo: ʻO Exondys 51 kahi lāʻau antisense oligonucleotide.Hiki i ka antisense oligonucleotide ke hoʻopaʻa i ka exon 51 kūlana o ka pre-mRNA o ka DMD gene, ka hopena i ka hoʻokumu ʻana o ka mRNA makua.ʻO ka excision, no laila e hoʻoponopono hapa i ka mRNA heluhelu kiʻi, kōkua i ka mea maʻi e synthesize i kekahi mau ʻano hana o ka dystrophin i ʻoi aku ka pōkole ma mua o ka protein maʻamau, a laila e hoʻomaikaʻi ai i nā hōʻailona o ka mea maʻi.

(7) Tegsedi

Hui: Hoʻomohala ʻia e Ionis Pharmaceuticals.

Ka manawa i ka mākeke: Apono ʻia e ka European Union no ke kūʻai aku ʻana ma Iulai 2018.

Nā hōʻailona: No ka mālama ʻana i ka transthyretin amyloidosis (hATTR).

Manaʻo: ʻO Tegsedi kahi lāʻau antisense oligonucleotide e huli ana i ka transthyretin mRNA.ʻO ia ka lāʻau lapaʻau mua i ʻae ʻia no ka mālama ʻana i ka hATTR.ʻO ke ʻano o ka hoʻokele ʻana he subcutaneous injection.Hoʻemi ka lāʻau lapaʻau i ka hana ʻana o ka protein ATTR ma ka huli ʻana i ka mRNA o transthyretin (ATTR), a loaʻa iā ia kahi lakio pōmaikaʻi maikaʻi i ka mālama ʻana i ka ATTR.ʻAʻole pili ka pae maʻi a me ka hele ʻana o ka cardiomyopathy.

(8) Onpattro

Hui: Hoʻokumu pū ʻia e Alnylam a me Sanofi.

Ka manawa i ka mākeke: ʻApono ʻia no ka papa inoa ʻana ma United States i 2018.

Nā hōʻailona: No ka mālama ʻana i ka transthyretin amyloidosis (hATTR).

Manaʻo: ʻO Onpattro kahi lāʻau lapaʻau siRNA e huli ana i ka transthyretin mRNA, e hōʻemi ana i ka hana ʻana o ka protein ATTR i loko o ke ake a me ka hōʻiliʻili ʻana o nā waihona amyloid i nā aʻalolo peripheral ma o ka huli ʻana i ka mRNA o transthyretin (ATTR)., ma laila e hoʻomaikaʻi ai a hoʻomaha i nā hōʻailona maʻi.

(9) Givlaari

Hui: Hoʻomohala ʻia e Alnylam Corporation.

Ka manawa i ka mākeke: ʻApono ʻia e ka FDA i Nowemapa 2019.

Nā hōʻailona: No ka mālama ʻana i ka porphyria hepatic acute (AHP) i nā pākeke.

Manaʻo: ʻO Givlaari kahi lāʻau siRNA, ka lua o ka lāʻau siRNA i ʻae ʻia no ke kūʻai ʻana ma hope o Onpattro.Hāʻawi ʻia ka lāʻau lapaʻau ma lalo o ka ʻili a kuhikuhi i ka mRNA no ka hoʻohaʻahaʻa ʻana i ka protein ALAS1.Hiki i ka mālama ʻana i kēlā me kēia mahina ʻo Givlaari ke hoʻohaʻahaʻa i ke kiʻekiʻe o ka ALAS1 i loko o ke akepaʻa, ma laila e hoʻemi ai i nā pae o ka neurotoxic ALA a me PBG i ka pae maʻamau, a laila hoʻomaha i nā hōʻailona maʻi o ka mea maʻi.Ua hōʻike ka ʻikepili i nā mea maʻi i mālama ʻia me Givlaari he 74% ka emi ʻana o ka nui o nā maʻi lapalapa i hoʻohālikelike ʻia i ka hui placebo.

(10) Vyondys53

Hui: Hoʻomohala ʻia e Sarepta Therapeutics.

Ka manawa i ka mākeke: ʻApono ʻia e ka FDA i Dekemaba 2019.

Hōʻike: No ka mālama ʻana i nā maʻi DMD me ka dystrophin gene exon 53 splice mutation.

Manaʻo: ʻO Vyondys 53 kahi lāʻau antisense oligonucleotide.Kuhi ʻia ka lāʻau oligonucleotide i ke kaʻina splicing o ka dystrophin mRNA precursor.Ma ke kaʻina hana ʻole o ka dystrophin mRNA precursor, ua hoʻokaʻawale ʻia ka Exon 53 o waho, ʻo ia hoʻi, ʻaʻole i loaʻa ma ka mRNA makua, a ua hoʻolālā ʻia e hana i kahi protein dystrophin i ʻoki ʻia akā e hana mau ana, e hoʻomaikaʻi ai i ka hiki ke hoʻoikaika kino i nā maʻi.

(11) Waylivra

Hui: Hoʻomohala ʻia e Ionis Pharmaceuticals a me kāna ʻoihana ʻo Akcea Therapeutics.

Ka manawa i ka mākeke: ʻApono ʻia e ka European Medicines Agency (EMA) i Mei 2019.

Hōʻike: Ma ke ʻano he lāʻau hoʻohui i kahi meaʻai i hoʻomalu ʻia i nā poʻe maʻi makua me ka familial chylomicronemia syndrome (FCS).

Manaʻo: ʻO Waylivra kahi lāʻau antisense oligonucleotide, ʻo ia ka lāʻau lapaʻau mua i ʻae ʻia no ka mālama ʻana i ka FCS ma ka honua.

(12) Leqvio

Hui: Kūkulu ʻia e Novartis.

Ka manawa i ka mākeke: Apono ʻia e ka EU i Dekemaba 2020.

Nā hōʻailona: No ka mālama ʻana i ka hypercholesterolemia mua o nā pākeke (ʻohana heterozygous a me non-familial) a i ʻole dyslipidemia hui ʻia.

Manaʻo: ʻO Leqvio kahi lāʻau lapaʻau siRNA e kuhikuhi ana i ka PCSK9 mRNA.ʻO ia ka lāʻau lapaʻau siRNA hoʻohaʻahaʻa cholesterol mua loa o ka honua (LDL-C).ʻO ke ʻano o ka hoʻokele ʻana he subcutaneous injection.Hana ʻia ka lāʻau lapaʻau e ka RNA interference e hoʻohaʻahaʻa i nā pae protein PCSK9, ka mea e hoʻohaʻahaʻa i nā pae LDL-C.Hōʻike ka ʻikepili lapaʻau hiki iā Leqvio ke hōʻemi i ka LDL-C ma kahi o 50% i nā poʻe maʻi ʻaʻole hiki ke hoʻemi ʻia nā pae LDL-C i nā pae i hoʻopaʻa ʻia ʻoiai ʻo ka nui o ka ʻae ʻia o nā statins.

(13) Oxlumo

Hui: Hoʻomohala ʻia e Alnylam Pharmaceuticals.

Ka manawa i ka mākeke: ʻApono ʻia e ka EU i Nowemapa 2020.

Nā hōʻailona: No ka mālama ʻana i ka hyperoxaluria type 1 (PH1).

Manaʻo: ʻO Oxlumo kahi lāʻau lapaʻau siRNA e huli ana i ka hydroxy acid oxidase 1 (HAO1) ​​mRNA, i lawelawe ʻia ma lalo o ka ʻili.Ua hoʻomohala ʻia ka lāʻau lapaʻau me ka hoʻohana ʻana i ka ʻenehana conjugation ESC-GalNAc hou loa i hoʻonui ʻia e Alnylam, kahi e hiki ai i nā siRNA hoʻokele subcutaneously me ka hoʻomau a ʻoi aku ka maikaʻi.Hoʻopili ka lāʻau lapaʻau i ka hoʻohaʻahaʻa a i ʻole ka inhibition o ka hydroxy acid oxidase 1 (HAO1) ​​mRNA, e hōʻemi i ke kiʻekiʻe o ka glycolate oxidase i loko o ke akepaʻa, a laila hoʻopau i ka substrate i koi ʻia no ka hana ʻana o ka oxalate a hōʻemi i ka hana ʻana o ka oxalate e kāohi i ka holomua o ka maʻi a hoʻomaikaʻi i nā hōʻailona maʻi.

(14) Viltepso

Hui: Hoʻomohala ʻia e NS Pharma, kahi hui o Nippon Shinyaku.

Ka manawa i ka mākeke: ʻApono ʻia e ka FDA i ʻAukake 2020.

Nā hōʻailona: No ka mālama ʻana i ka Duchenne muscular dystrophy (DMD) me DMD gene mutation ma exon 53 skipping gene.

Manaʻo: ʻO Viltepso kahi lāʻau lapaʻau phosphorodiamide morpholino oligonucleotide.Hiki i kēia lāʻau lapaʻau oligonucleotide ke hoʻopaʻa i ke kūlana exon 53 o ka pre-mRNA o ka DMD gene, ka hopena i ka hoʻokumu ʻana o ka mRNA makua.Hoʻopau hapa ʻia ka exon, a laila hoʻoponopono ʻāpana i ke kiʻi heluhelu mRNA, e kōkua ana i ka mea maʻi e synthesize i kekahi mau ʻano hana o ka dystrophin i ʻoi aku ka pōkole ma mua o ka protein maʻamau, a laila e hoʻomaikaʻi ai i nā hōʻailona o ka mea maʻi.

(15) Amvuttra (vutrisiran)

Hui: Hoʻomohala ʻia e Alnylam Pharmaceuticals.

Ka manawa i ka mākeke: ʻApono ʻia e ka FDA i Iune 2022.

Nā hōʻailona: No ka mālama ʻana i ka amyloidosis transthyretin hereditary me ka polyneuropathy (hATTR-PN).

Manaʻo: ʻO Amvuttra (Vutrisiran) kahi lāʻau lapaʻau siRNA e huli ana i ka transthyretin (ATTR) mRNA, ka mea i lawelawe ʻia e ka subcutaneous injection.Hoʻolālā ʻia ʻo Vutrisiran e pili ana i ka Alnylam's Enhanced Stabilization Chemistry (ESC) -GalNAc conjugated delivery platform me ka hoʻonui ʻana i ka mana a me ka paʻa metabolic.Hoʻokumu ʻia ka ʻae ʻana o ka lāʻau lapaʻau ma ka ʻikepili 9-mahina mai kāna haʻawina ʻo Phase III (HELIOS-A), me nā hopena holoʻokoʻa e hōʻike ana i ka hoʻomaikaʻi ʻana o ka therapy i nā hōʻailona o ka hATTR-PN, me ka ʻoi aku o 50% o nā maʻi e hoʻohuli a hoʻōki i ka holomua.

4. Nā lāʻau lapaʻau gene therapy ʻē aʻe

(1) Rexin-G

Hui: Hoʻomohala ʻia e Epeius Biotech.

Ka manawa e kuai ai: Aponoia e ka Philippine Food and Drug Administration (BFAD) ma 2005.

Nā hōʻailona: No ka mālama ʻana i nā maʻi maʻi maʻi kūʻē i ka chemotherapy.

Manaʻo: ʻO Rexin-G kahi mea i hoʻopaʻa ʻia i ka nanoparticle injection.Hoʻokomo ia i ka cyclin G1 mutant gene i loko o nā cell target ma o retroviral vector e pepehi pono i nā ʻōpū paʻa.ʻO ke ʻano o ka lawelawe ʻana he intravenous infusion.Ma ke ʻano he lāʻau lapaʻau i hoʻopaʻa ʻia i ka maʻi maʻi e ʻimi ikaika a luku i nā sela maʻi maʻi metastatic, loaʻa iā ia kekahi hopena i nā poʻe maʻi ʻaʻole pono e kūʻē i nā lāʻau maʻi maʻi ʻē aʻe, e komo pū ana me nā lāʻau olaola.

(2) Neovasculgen

Hui: Hoʻomohala ʻia e ka Human stem cell institute.

Wā hoʻopaʻa inoa: Apono ʻia no ka hoʻopaʻa inoa ʻana ma Rūsia ma Dekemaba 7, 2011, a laila hoʻopaʻa inoa ʻia ma Ukraine ma 2013.

Nā ʻōlelo hōʻike: No ka mālama ʻana i nā maʻi ʻāʻī o peripheral arterial, e komo pū ana me ka ischemia lālā koʻikoʻi.

Manaʻo: ʻO Neovasculgen kahi DNA plasmid-based gene therapy kahi i kūkulu ʻia ai ka gene vascular endothelial growth factor (VEGF) 165 ma luna o ka iwi kuamoʻo plasmid a hoʻokomo ʻia i loko o nā maʻi.

(3) Collategene

Hui: Hoʻokumu pū ʻia e ke Kulanui o Osaka a me nā ʻoihana ʻoihana ʻoihana.

Ka manawa papa inoa: ʻApono ʻia e ke Kuhina Ola, Labour a Welfare no Iapana no ka papa inoa ʻana ma ʻAukake 2019.

Nā hōʻailona: Hoʻomaʻamaʻa i ka ischemia koʻikoʻi o ka ʻaoʻao haʻahaʻa.

Manaʻo: ʻO Collategene kahi lāʻau lapaʻau i hoʻokumu ʻia i ka plasmid, ʻo ia ka lāʻau lapaʻau ʻōiwi Iapana mua i hana ʻia e AnGes.ʻO ka mea nui o kēia lāʻau lapaʻau he plasmid olohelohe i loaʻa i ke kaʻina gene o ke kanaka hepatocyte growth factor (HGF).Inā hoʻokomo ʻia ka lāʻau lapaʻau i loko o nā ʻiʻo o nā lālā haʻahaʻa, ʻo ka HGF i hōʻike ʻia e hāpai i ke kūkulu ʻana i nā kīʻaha koko hou a puni nā kīʻaha koko i pani ʻia.Ua hōʻoia nā hoʻokolohua lapaʻau i kona pono i ka hoʻomaikaʻi ʻana i nā maʻi ʻeha.

HOPE


Ka manawa hoʻouna: Nov-10-2022