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Kwanan nan, an amince da magungunan jiyya guda uku don tallatawa, wato: (1) A ranar 21 ga Yuli, 2022, PTC Therapeutics, Inc. (NASDAQ: PTCT) ta sanar da cewa Hukumar Turai ta amince da maganin kwayoyin halittar AAV Upstaza™. an yarda don talla).(2) A ranar 17 ga Agusta, 2022, Hukumar Abinci da Magunguna ta Amurka (FDA) ta amince da maganin halittar Bluebird Bio's Zynteglo (betibeglogene autotemcel, beti-cel) don maganin beta thalassaemia.Yarda da maganin a Amurka babu shakka "taimako a cikin dusar ƙanƙara" ga Bluebird Bio, wanda ke cikin rikicin kuɗi.(3) A ranar 24 ga Agusta, 2022, BioMarin Pharmaceutical (BioMarin) ya sanar da cewa Hukumar Turai ta amince da tallan yanayin ROCTAVIAN ™ (valoctocogene roxaparvovec), maganin ƙwayar cuta don hemophilia A, don kula da marasa lafiya da ba su da tarihin FVIII factor inhibitors da marasa lafiya na AAV5 na baya-bayanan henophilia. ophilia A genetherapy yarda don talla).Ya zuwa yanzu, an amince da magungunan jiyya guda 41 don tallatawa a duk duniya.

Gene shine ainihin rukunin kwayoyin halitta wanda ke sarrafa halaye.Sai dai kwayoyin halittar wasu kwayoyin cuta, wadanda suka hada da RNA, kwayoyin halittar mafi yawan halittu sun kunshi DNA.Yawancin cututtuka na kwayoyin halitta suna faruwa ne ta hanyar hulɗar da ke tsakanin kwayoyin halitta da muhalli, kuma yawancin cututtuka na iya warkewa ko rage su ta hanyar maganin kwayoyin halitta.Ana ɗaukar magungunan ƙwayoyin halitta a matsayin juyin juya hali a fagen magani da kantin magani.Faɗin magungunan ƙwayoyin cuta sun haɗa da magungunan da suka dogara da magungunan DNA da aka gyaggyarawa (kamar ƙwayoyin cuta na tushen vivo genetherapy magunguna, in vitro genetherapy drugs, tsirara magungunan plasmid, da dai sauransu) da kuma RNA kwayoyi (kamar antisense oligonucleotide kwayoyi, siRNA kwayoyi, da mRNA genetherapy, da dai sauransu);ƙunƙuntaccen ma'anar magungunan Genetherapy galibi sun haɗa da magungunan DNA na plasmid, magungunan ƙwayoyin cuta waɗanda suka dogara da ƙwayoyin cuta, magungunan ƙwayoyin cuta waɗanda suka dogara da ƙwayoyin cuta, tsarin gyaran kwayoyin halitta da magungunan ƙwayoyin cuta waɗanda aka gyara ta cikin vitro.Bayan shekaru na ci gaba mai raɗaɗi, magungunan ƙwayoyin cuta sun sami sakamako mai ban sha'awa na asibiti.(ban da allurar DNA da allurar mRNA), an yarda da magungunan jiyya guda 41 don tallatawa a duniya.Tare da ƙaddamar da samfura da haɓakar haɓakar fasahar jiyya ta ƙwayoyin halitta, maganin ƙwayoyin cuta na gab da kawo ƙarshen ci gaba cikin sauri.

duniya 1

Rabe-raben jiyya na kwayoyin halitta (Tsarin Hoto: Jingwei Biological)

Wannan labarin ya lissafa hanyoyin kwantar da hankula guda 41 waɗanda aka amince da su don tallatawa (ban da allurar DNA da rigakafin mRNA).

1. In vitro genetherapy

(1) Strimvelis

Kamfanin: GlaxoSmithKline (GSK) ne ya haɓaka.

Lokacin kasuwa: Tarayyar Turai ta amince da shi a watan Mayu 2016.

Alamomi: Don maganin matsanancin rashin ƙarfi na rigakafi (SCID).

Bayani: Babban tsarin wannan farfaganda shine fara samun nasu kwayoyin halitta na hematopoietic na majiyyaci, fadadawa da al'adarsu a cikin vitro, sannan a yi amfani da retrovirus don gabatar da kwafin kwayar halittar ADA (adenosine deaminase) mai aiki a cikin kwayoyin jikinsu na hematopoietic, kuma a karshe canja wurin gyare-gyaren kwayoyin halitta na hematopoietic.Ana shigar da ƙwayoyin hematopoietic a baya cikin jiki.Sakamakon asibiti ya nuna cewa shekarun rayuwa na shekaru 3 na marasa lafiya ADA-SCID da aka yi wa Strimvelis ya kasance 100%.

(2) Zalmoxis

Kamfanin: MolMed, Italiya ne ke samarwa.

Lokacin zuwa kasuwa: Samu izinin tallan sharadi na EU a cikin 2016.

Alamomi: Ana amfani da shi don maganin adjuvant na tsarin rigakafi na marasa lafiya bayan dashen kwayar cutar hematopoietic.

Bayani: Zalmoxis wani allogeneic T cell kashe kansa gene immunotherapy gyara ta retroviral vector.1NGFR da HSV-TK Mut2 kwayoyin kashe kansa suna ba da damar mutane su yi amfani da ganciclovir a kowane lokaci don kashe ƙwayoyin T da ke haifar da mummunan martani na rigakafi, hana ci gaba da lalacewa na GVHD wanda zai iya faruwa, da kuma mayar da aikin rigakafi a cikin marasa lafiya tare da HSCT na haploidentical bayan tiyata Escort.

(3) Invossa-K

Kamfanin: Kamfanin TissueGene (KolonTissueGene) ya haɓaka.

Lokacin kasuwa: An amince da jeri a Koriya ta Kudu a cikin Yuli 2017.

Alamomi: Don maganin cututtukan ciwon gwiwa na degenerative.

Bayanan Bayani: Invossa-K shine maganin kwayoyin halitta na allogeneic wanda ya shafi chondrocytes na mutum.Kwayoyin Allogeneic an gyare-gyare ta hanyar kwayoyin halitta a cikin vitro, kuma sel da aka gyara zasu iya bayyanawa da ɓoye abubuwan haɓaka girma β1 (TGF-β1) bayan allurar intra-articular.β1), don haka inganta alamun osteoarthritis.Sakamakon asibiti ya nuna cewa Invossa-K na iya inganta ciwon gwiwa sosai.Hukumar kula da magunguna ta Koriya ta Kudu ta soke lasisin a shekarar 2019 saboda masana'anta sun yi kuskuren yiwa sinadaran da aka yi amfani da su alama.

(4) Zynteglo

Kamfanin: Kamfanin bluebird bio (bluebird bio) na Amurka ya haɓaka.

Lokacin kasuwa: Tarayyar Turai ta amince da ita a cikin 2019, kuma FDA ta amince da ita a cikin Agusta 2022.

Alamomi: Don maganin β-thalassemia mai dogaro da jini.

Bayani: Zynteglo shine maganin lentiviral in vitro genetherapy, wanda ke amfani da vector na lentiviral don gabatar da kwafin aikin na al'ada na β-globin gene (βA-T87Q-globin gene) a cikin kwayoyin halitta na hematopoietic da aka cire daga marasa lafiya., sa'an nan kuma shigar da waɗannan gyare-gyaren kwayoyin halitta masu autologous hematopoietic stem cell baya cikin majiyyaci.Da zarar majiyyaci yana da kwayar halittar βA-T87Q-globin ta al'ada, za su iya samar da furotin na HbAT87Q na yau da kullun, wanda zai iya ragewa ko kawar da buƙatar ƙarin jini yadda ya kamata.Jiyya ce ta lokaci ɗaya da aka ƙera don maye gurbin ƙarin ƙarin jini na rayuwa da magunguna na tsawon rai ga marasa lafiya masu shekaru 12 zuwa sama.

(5) Skysona

Kamfanin: Kamfanin bluebird bio (bluebird bio) na Amurka ya haɓaka.

Lokacin zuwa kasuwa: EU ta amince da tallata a watan Yuli 2021.

Alamomi: Don maganin farkon cerebral adrenoleukodystrophy (CALD).

Bayani: Maganin kwayoyin halittar Skysona shine kawai maganin kwayoyin halitta na lokaci daya da aka amince da shi don maganin farkon adrenoleukodystrophy na cerebral (CALD).Skysona (elivaldogene autotemcel, Lenti-D) wani hematopoietic stem cell lentiviral in vitro gene far Lenti-D.Tsarin gabaɗaya na jiyya shine kamar haka: ana fitar da ƙwayoyin hematopoietic ta atomatik daga majiyyaci, an canza su a cikin vitro ta hanyar lentivirus ɗauke da kwayar halittar ABCD1 na ɗan adam, sannan a mayar da su cikin majiyyaci.Don kula da marasa lafiya a ƙarƙashin shekaru 18 tare da maye gurbin ABCD1 da CALD.

(6) Kymriah

Kamfanin: Novartis ya haɓaka.

Lokacin kasuwa: FDA ta amince da ita a watan Agusta 2017.

Alamomi: Jiyya na precursor B-cell m lymphoblastic cutar sankarar bargo (ALL) da koma baya da kuma refractory DLBCL.

Bayani: Kymriah magani ne na lentiviral in vitro genetherapy, maganin CAR-T na farko da aka amince da shi a duniya, yana nufin CD19 kuma yana amfani da ma'aunin motsa jiki na 4-1BB.Farashi shine $475,000 a Amurka da $313,000 a Japan.

(7) Yaskarta

Kamfanin: Kite Pharma, reshen Gileyad ne ya haɓaka.

Lokacin zuwa kasuwa: FDA ta amince da shi a watan Oktoba 2017.

Alamomi: Don maganin sake dawowa ko refractory babban lymphoma B-cell.

Jawabai: Yescarta magani ne na retroviral in vitro.Ita ce maganin CAR-T na biyu da aka amince da shi a duniya.Yana hari CD19 kuma yana amfani da ƙimar kudin CD28.Farashin a Amurka shine $ 373,000.

(8) Takardu

Kamfanin: Gileyad (GILD) ne ya haɓaka.

Lokacin kasuwa: FDA ta amince da shi a cikin Yuli 2020.

Alamomi: Don sake dawowa ko refractory mantle cell lymphoma.

Bayani: Tecartus magani ne mai sarrafa kansa na CAR-T wanda ke nufin CD19, kuma shine magani na CAR-T na uku da aka amince don tallatawa a duniya.

(9) Breyanzi

Kamfanin: Bristol-Myers Squibb (BMS) ya haɓaka.

Lokacin kasuwa: FDA ta amince da shi a cikin Fabrairu 2021.

Alamomi: Relapsed ko refractory (R/R) babban B-cell lymphoma (LBCL).

Jawabai: Breyanzi magani ne na in vitro wanda ya dogara da lentivirus, kuma magani na CAR-T na huɗu da aka amince don tallatawa a duniya, yana nufin CD19.Yarda da Breyanzi wani ci gaba ne ga Bristol-Myers Squibb a fagen rigakafin rigakafi ta salula, wanda Bristol-Myers ya samu lokacin da ya sami Celgene akan dala biliyan 74 a cikin 2019.

(10)Abba

Kamfanin: Haɗin gwiwar Bristol-Myers Squibb (BMS) da bluebird bio.

Lokacin kasuwa: FDA ta amince da shi a cikin Maris 2021.

Alamomi: Maimaituwa ko refractory mahara myeloma.

Bayanan Bayani: Abecma shine tushen lentivirus a cikin maganin kwayoyin halittar vitro, farkon maganin tantanin halitta na CAR-T a duniya wanda ke nufin BCMA, kuma magani na CAR-T na biyar wanda FDA ta amince da shi.Ka'idar miyagun ƙwayoyi ita ce bayyana mai karɓa na BCMA na chimeric akan ƙwayoyin T na majiyyaci ta hanyar gyare-gyaren kwayoyin halittar lentivirus a cikin vitro.Kafin jiko na kwayar cutar kwayar halitta, mai haƙuri ya karbi mahadi guda biyu na cyclophosphamide da fludarabine don maganin farko.Jiyya don cire ƙwayoyin T da ba a canza su ba daga majiyyaci, sannan a saka sel T da aka gyara a baya cikin jikin majiyyaci don nema da kashe ƙwayoyin cutar kansa na BCMA.

(11) Libmeldy

Kamfanin: Orchard Therapeutics ne ya haɓaka.

Lokacin kasuwa: Tarayyar Turai ta amince da yin lissafin a watan Disamba 2020.

Alamomi: Don maganin metachromatic leukodystrophy (MLD).

Bayani: Libmeldy magani ne na kwayoyin halitta wanda ya dogara da lentiviral in vitro gyare-gyaren kwayoyin halittar CD34+.Bayanai na asibiti sun nuna cewa jiko guda ɗaya na Libmeldy yana da tasiri wajen gyaggyarawa tsarin MLD na farko da kuma mummunan motsi da rashin fahimta a cikin marasa lafiya marasa lafiya na shekaru ɗaya.

(12) Benoda

Kamfanin: WuXi Junuo ya haɓaka.

Lokacin kasuwa: NMPA ta amince da shi bisa hukuma a cikin Satumba 2021.

Alamomi: Jiyya na relapsed ko refractory babban B-cell lymphoma (r / r LBCL) a cikin manya marasa lafiya bayan layi na biyu ko fiye da tsarin tsarin.

Bayani: Benoda maganin kwayoyin halittar CD19 CAR-T ne, kuma shine ainihin samfurin WuXi Junuo.Shi ne samfurin CAR-T na biyu da aka amince da shi a kasar Sin, sai dai babban lymphoma na B-cell da ya koma baya.Bugu da kari, WuXi Junuo kuma yana shirin samar da allurar Ruiki Orenza don maganin wasu alamomi daban-daban, wadanda suka hada da lymphoma follicular (FL), lymphoma cell cell lymphoma (MCL), cutar sankarar jini ta lymphocytic na yau da kullun (CLL), layin na biyu yana yaduwa babban lymphoma B-cell (DLBCL) da kuma cutar sankarar lymphoblastic (ALL).

(13) CARVYKTI

Kamfanin: samfur na farko da aka amince da Legend Bio.

Lokacin kasuwa: FDA ta amince da shi a cikin Fabrairu 2022.

Alamomi: Maganin sake dawowa ko refractory mahara myeloma (R/R MM).

Bayani: CARVYKTI (ciltacabtagene autoleucel, ana kiranta da Cilta-cel) magani ne na rigakafin ƙwayoyin cuta na CAR-T tare da ƙwayoyin rigakafi guda biyu waɗanda ke niyya da antigen B cell maturation (BCMA).Bayanan sun nuna cewa CARVYKTI ya nuna adadin amsa gabaɗaya har zuwa 98% a cikin marasa lafiya tare da sake dawowa ko refractory myeloma da yawa waɗanda suka karɓi jiyya huɗu ko fiye da baya, gami da masu hana proteasome, immunomodulators, da anti-CD38 monoclonal antibodies.

2. In vivo genetherapy dangane da ƙwayoyin cuta

(1) Jinsi/mayar haihuwa

Kamfanin: Kamfanin Shenzhen Saibainuo ne ya haɓaka.

Lokacin kasuwa: An amince da jeri a China a cikin 2003.

Alamomi: Don maganin ciwon daji na kai da wuyansa.

Bayani: Recombinant human p53 allurar adenovirus Gendicine/Jinshengsheng magani ne na adenovirus vector therapy tare da haƙƙin mallakar fasaha mai zaman kansa mallakar Kamfanin Shenzhen Saibainuo.Maganin yana kunshe da al'adar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar ƙwayar cuta ta 5 ta nau'in adenovirus na nau'in 5. Na farko shine babban tsarin maganin don aiwatar da maganin ciwon daji, kuma na ƙarshe yana aiki a matsayin mai ɗauka.Alamar adenovirus tana ɗaukar kwayar cutar p53 zuwa cikin tantanin halitta da aka yi niyya, kuma yana bayyana kwayar cutar ƙwayar cuta ta p53 a cikin tantanin halitta.Samfurin na iya haɓaka nau'ikan ƙwayoyin rigakafin cutar kansa da ƙasa-kayyade ayyukan oncogenes daban-daban, ta haka yana haɓaka tasirin cutar kansa da kuma cimma manufar kashe ciwace-ciwace.

(2) Rikicin

Kamfanin: Kamfanin Latvia Latima ne ya haɓaka.

Lokacin kasuwa: An amince da shi a Latvia a cikin 2004.

Alamomi: Don maganin melanoma.

Bayani: Rigvir magani ne na kwayoyin halitta wanda aka yi amfani da shi ta hanyar ECHO-7 enterovirus vector, wanda aka yi amfani da shi a Latvia, Estonia, Poland, Armenia, Belarus da sauran wurare, kuma ana yin rajista tare da EMA na Tarayyar Turai..Abubuwan da suka shafi asibiti a cikin shekaru goma da suka gabata sun tabbatar da cewa Rigvir oncolytic virus yana da lafiya kuma yana da tasiri, kuma yana iya inganta yawan rayuwar marasa lafiya na melanoma da sau 4-6.Bugu da ƙari, maganin ya kuma dace da wasu nau'o'in ciwon daji, ciki har da ciwon daji na colorectal, ciwon daji na pancreatic, ciwon daji na mafitsara.ciwon daji, ciwon koda, ciwon prostate, ciwon huhu, ciwon mahaifa, lymphosarcoma, da dai sauransu.

(3) Oncorine/Ankerui

Kamfanin: Shanghai Sunway Biotechnology Co., Ltd. ya haɓaka.

Lokacin kasuwa: An amince da jeri a China a cikin 2005.

Alamomi: Maganin ciwace-ciwacen kai da wuya, ciwon hanta, kansar pancreatic, kansar mahaifa da sauran cututtukan daji.

Bayani: Oncorine shine samfurin maganin ƙwayoyin cuta na oncolytic ta amfani da adenovirus azaman vector.Adenovirus oncolytic da aka samu zai iya yin kwafi musamman a cikin ciwace-ciwacen da ba su da kyau ko kuma na al'ada na p53, yana haifar da ƙwayar ƙwayar cuta, ta haka ne ke kashe ƙwayoyin tumor.ba tare da lalata ƙwayoyin al'ada ba.Sakamakon asibiti ya nuna cewa Anke Rui yana da lafiya mai kyau da inganci ga nau'ikan ciwace-ciwacen daji iri-iri.

(4) Glybera

Kamfanin: UniQure ne ya haɓaka.

Lokacin kasuwa: An amince da shi a Turai a cikin 2012.

Alamomi: Maganin rashi lipoprotein lipase (LPLD) tare da mai tsanani ko maimaita lokuta na pancreatitis duk da ƙuntataccen abincin mai mai.

Bayani: Glybera (alipogene tiparvovec) magani ne na maganin kwayoyin halitta wanda ya dogara da AAV a matsayin vector.Wannan farfesa yana amfani da AAV a matsayin vector don canja wurin jiyya na LPL na warkewa a cikin ƙwayoyin tsoka, don haka sel masu dacewa zasu iya samar da wani adadin lipoprotein lipase, Yana taka rawa wajen kawar da cututtuka, kuma wannan maganin yana da tasiri na dogon lokaci bayan gudanarwa ɗaya (tasirin zai iya wuce shekaru da yawa).An soke maganin a cikin 2017, kuma dalilan da aka cire ta na iya kasancewa da alaƙa da abubuwa biyu: tsada mai yawa da ƙarancin buƙatun kasuwa.Matsakaicin farashin magani guda ɗaya ya kai dalar Amurka miliyan 1, kuma majiyyaci ɗaya ne ya saya ya yi amfani da shi ya zuwa yanzu.Duk da cewa kamfanin inshorar likitanci ya mayar masa da dalar Amurka 900,000, hakan kuma babban nauyi ne ga kamfanin inshora.Bugu da ƙari, alamun maganin yana da wuyar gaske, tare da adadin abubuwan da suka faru na kusan 1 a cikin 1 miliyan da kuma yawan rashin ganewar asali.

(5) Ilygic

Kamfanin: Amgen ne ya haɓaka.

Lokacin kasuwa: A cikin 2015, an amince da shi don jeri a cikin Amurka da Tarayyar Turai.

Alamomi: Maganin ciwon melanoma waɗanda ba za a iya cire su gaba ɗaya ta hanyar tiyata ba.

Bayani: Imlygic shine gyare-gyaren kwayoyin halitta (share nau'in nau'in ICP34.5 da ICP47, da kuma shigar da granulocyte-macrophage colony-stimulating factor GM-CSF gene a cikin kwayar cutar) yana rage ƙwayar cutar ta herpes simplex irin 1 (HSV-1) oncolytic virus, na farko da aka yarda da kwayar cutar ta FDA.Hanyar gudanarwa shine allurar intralesional.Allurar kai tsaye a cikin raunuka na melanoma na iya haifar da fashewar ƙwayoyin tumor da kuma saki antigens da aka samo daga tumor da GM-CSF don inganta amsawar rigakafin ƙwayar cuta.

(6) Luxturna

Kamfanin: Spark Therapeutics, wani reshen Roche ne ya haɓaka.

Lokaci don kasuwa: FDA ta amince da ita a cikin 2017, sannan kuma an amince da ita don tallace-tallace a Turai a cikin 2018.

Alamomi: Don kula da yara da manya tare da asarar hangen nesa saboda maye gurbi a cikin kwafin nau'in nau'in RPE65 guda biyu amma tare da isassun lambobi na ƙwayoyin retinal masu dacewa.

Jawabai: Luxturna magani ne na tushen AAV wanda ake gudanarwa ta hanyar allurar subretinal.Tsarin kwayoyin halitta yana amfani da AAV2 a matsayin mai ɗaukar hoto don gabatar da kwafin aiki na al'ada RPE65 na al'ada a cikin sel na retinal na mai haƙuri, don haka sel masu dacewa suna bayyana furotin RPE65 na al'ada don ramawa ga lahani na furotin RPE65 na mai haƙuri, don haka inganta hangen nesa mai haƙuri.

(7) Zolgensma

Kamfanin: AveXis, wani reshen Novartis ne ya haɓaka.

Lokacin kasuwa: FDA ta amince da shi a watan Mayu 2019.

Alamomi: Jiyya na atrophy na muscular na kashin baya (Spinal Muscular Atrophy, SMA) marasa lafiya a karkashin 2 shekaru.

Bayani: Zolgensma magani ne na kwayoyin halitta wanda ya danganci vector AAV.Wannan magani shine kawai tsarin jiyya na lokaci ɗaya don atrophy na muscular na kashin baya da aka amince don tallatawa a duniya.shafi, babban ci gaba ne.Wannan maganin kwayoyin halitta yana amfani da vector na scAAV9 don gabatar da al'ada na SMN1 na al'ada a cikin marasa lafiya ta hanyar jiko na ciki, yana samar da furotin SMN1 na al'ada, don haka inganta aikin ƙwayoyin da aka shafa kamar su neurons.Sabanin haka, magungunan SMA Spinraza da Evrysdi suna buƙatar maimaita allurai na tsawon lokaci, tare da Spinraza ana gudanar da shi azaman allurar kashin baya kowane wata huɗu, da Evrysdi, maganin baka na yau da kullun.

(8) Tsarkakewa

Kamfanin: Daiichi Sankyo Company Limited ne ya haɓaka (TYO: 4568).

Lokaci don kasuwa: Yarda da sharadi daga Ma'aikatar Lafiya, Ma'aikata da Jin Dadin Jama'a (MHLW) a cikin Yuni 2021.

Alamomi: Don maganin cutar glioma.

Bayani: Delytact shine samfurin maganin ƙwayoyin cuta na oncolytic na huɗu da aka amince da shi a duniya kuma samfurin ƙwayar cuta na oncolytic na farko da aka amince da shi don maganin glioma.Delytact wata cuta ce da aka yi amfani da ita ta hanyar kwayoyin cutar ta herpes simplex irin 1 (HSV-1) cutar oncolytic da Dr. Todo da abokan aiki suka kirkira.Delytact yana gabatar da ƙarin maye gurbi a cikin G207 genome na ƙarni na biyu na HSV-1, yana haɓaka zaɓaɓɓen kwafinsa a cikin ƙwayoyin cutar kansa da shigar da martanin rigakafin ƙwayar cuta, yayin da yake kiyaye babban martabar aminci.Delytact shine farkon ƙarni na uku na oncolytic HSV-1 a halin yanzu a cikin kimantawa na asibiti.Amincewar Delytact a Japan ya dogara ne akan gwajin asibiti na mataki na 2 mai hannu ɗaya.A cikin marasa lafiya tare da maimaita glioblastoma, Delytact ya sadu da farkon ƙarshen rayuwa na shekara guda, kuma sakamakon ya nuna cewa Delytact ya yi aiki fiye da G207.Ƙarfi mai ƙarfi da aikin antitumor mafi girma.Wannan yana da tasiri a cikin nau'ikan nau'ikan ƙari waɗanda suka haɗa da nono, prostate, schwannoma, nasopharyngeal, hepatocellular, colorectal, m na gefen jijiya kumburi da ciwon daji na thyroid.

(9) Tafiya

Kamfanin: PTC Therapeutics, Inc. ya haɓaka (NASDAQ: PTCT).

Lokacin kasuwa: EU ta amince da shi a cikin Yuli 2022.

Nunawa: Don rashi L-amino acid decarboxylase (AADC), an yarda da shi don kula da marasa lafiya watanni 18 da haihuwa.

Bayani: Upstaza™ (eladocagene exuparvovec) magani ne a cikin vivo ta hanyar amfani da nau'in ƙwayar cuta mai alaƙa da adeno 2 (AAV2) azaman vector.Mai haƙuri yana fama da rashin lafiya saboda maye gurbi a cikin kwayar halittar da ke ɓoye enzyme AADC.AAV2 yana ɗaukar kwayar halitta mai lafiya wanda ke ɓoye enzyme AADC.Ana samun sakamako na warkewa a cikin nau'in ramuwa na kwayoyin halitta.A cikin ka'idar, kashi ɗaya yana da tasiri na dogon lokaci.Ita ce farkon da aka fara sayar da maganin ƙwayoyin cuta kai tsaye a cikin kwakwalwa.Izinin tallan ya shafi duk ƙasashe membobin EU 27, da Iceland, Norway da Liechtenstein.

(9) Roctavian

Kamfanin: BioMarin Pharmaceutical (BioMarin) ya haɓaka.

Lokacin kasuwa: EU ta amince da shi a watan Agusta 2022.

Alamomi: Don kula da manya marasa lafiya tare da hemophilia mai tsanani A ba tare da tarihin hana FVIII factor da AAV5 antibody korau.

Bayani: Roctavian (valoctocogene roxaparvovec) yana amfani da AAV5 a matsayin vector kuma yana amfani da HLP mai haɓaka hanta na ɗan adam don fitar da bayyanar coagulation na mutum takwas (FVIII) tare da share yankin B.Shawarar da Hukumar Tarayyar Turai ta yanke don amincewa da tallan valoctocogene roxaparvovec ya dogara ne akan jimillar bayanan shirin ci gaban asibiti na miyagun ƙwayoyi.Daga cikin su, gwajin gwaji na asibiti na III na GENEr8-1 ya nuna cewa idan aka kwatanta da bayanan shekarar da ta gabata kafin yin rajista, bayan jiko ɗaya na valoctocogene roxaparvovec, Abubuwan da ke da alaƙa suna da raguwar yawan zubar jini na shekara-shekara (ABR), ƙarancin amfani da shirye-shiryen furotin na recombinant VIII (F8), ko haɓaka mai girma a cikin ayyukan F8 a cikin jini.Bayan makonni 4 na jiyya, abubuwan da ake amfani da su na F8 na shekara-shekara da ABR da ke buƙatar jiyya an rage su da 99% da 84%, bi da bi, bambancin ƙididdiga (p<0.001).Bayanan martabar aminci ya kasance mai kyau, ba tare da batutuwan da ke fuskantar hanawar F8 ba, malignancy, ko thrombotic sakamako masu lahani, kuma ba a ba da rahoton wasu munanan abubuwan da suka shafi jiyya (SAEs).

3. Kananan magungunan nucleic acid

(1) Vitravene

Kamfanin: Haɗin gwiwa ta hanyar Ionis Pharma (tsohon Isis Pharma) da Novartis.

Lokacin kasuwa: FDA da EU EMA sun amince da su a cikin 1998 da 1999.

Alamomi: Don maganin cytomegalovirus retinitis a cikin marasa lafiya masu cutar HIV.

Bayani: Vitravene magani ne na oligonucleotide na antisense kuma maganin oligonucleotide na farko da aka amince da shi don tallatawa a duniya.A farkon kasuwa, buƙatar kasuwa na magungunan anti-cytomegalovirus ya kasance cikin gaggawa;sa'an nan kuma saboda haɓakar haɓakar maganin rigakafin cutar kansa, adadin ƙwayoyin cutar cytomegalovirus ya ragu sosai.Saboda ƙarancin buƙatun kasuwa, an fitar da maganin a cikin 2002 da 2006 Janyewar a cikin ƙasashen EU da Amurka.

(2)Magu

Kamfanin: Pfizer da Eyetech ne suka haɓaka.

Lokacin kasuwa: An amince da jeri a Amurka a cikin 2004.

Alamomi: Don maganin cututtukan da ke da alaƙa da macular degeneration shekaru neovascular.

Bayani: Macugen wani magani ne na oligonucleotide wanda aka gyara wanda zai iya yin niyya kuma ya ɗaure ga haɓakar haɓakar haɓakar ƙwayoyin cuta (VEGF165 isoform), kuma ana gudanar da shi ta hanyar allurar intravitreal.

(3) Defitelio

Kamfanin: Jazz ne ya haɓaka.

Lokacin kasuwa: Tarayyar Turai ta amince da ita a cikin 2013, kuma FDA ta amince da shi a cikin Maris 2016.

Alamomi: Don maganin cututtukan hanta na hanta da ke hade da rashin aikin koda ko na huhu bayan dashen kwayar cutar hematopoietic.

Bayani: Defitelio magani ne na oligonucleotide, cakuda oligonucleotides tare da abubuwan plasmin.An cire shi a cikin 2009 don dalilai na kasuwanci.

(4) Kynamro

Kamfanin: Ionis Pharma da Kastle ne suka haɓaka.

Lokacin kasuwa: An amince da shi a cikin Amurka azaman maganin marayu a cikin 2013.

Alamomi: Don maganin adjuvant na homozygous familial hypercholesterolemia.

Bayani: Kynamro maganin antisense oligonucleotide ne, maganin antisense oligonucleotide wanda ke nufin apo B-100 mRNA na ɗan adam.Ana gudanar da Kynamro a matsayin 200 MG subcutaneously sau ɗaya a mako.

(5) Spinraza

Kamfanin: Ionis Pharmaceuticals ne ya haɓaka.

Lokacin kasuwa: FDA ta amince da shi a watan Disamba 2016.

Alamomi: Don maganin atrophy na muscular na kashin baya (SMA).

Bayani: Spinraza (nusinersen) maganin antisense oligonucleotide ne.Spinraza na iya canza ɓangarorin RNA na ƙwayar SMN2 ta ɗaure zuwa wurin da aka raba na SMN2 exon 7, ta haka yana haɓaka samar da furotin SMN mai cikakken aiki.A watan Agustan 2016, Kamfanin BIOGEN ya yi amfani da zaɓin sa don samun haƙƙin duniya na Spinraza.Spinraza ya fara gwajin gwaji na farko a cikin mutane a cikin 2011. A cikin shekaru 5 kawai, FDA ta amince da shi a cikin 2016, yana nuna cikakkiyar amincewar FDA game da ingancinta.An amince da maganin don tallata a China a cikin Afrilu 2019. Dukan sake zagayowar amincewar Spinraza a China bai wuce watanni 6 ba.Shekaru 2 da watanni 2 kenan tun lokacin da aka fara amincewa da Spinraza a Amurka.Irin wannan cuta ta waje da ba kasafai ba sabon magani yana cikin saurin jeri a China ya riga ya yi sauri sosai.Wannan kuma ya faru ne saboda "Sanarwa akan Fitar da Jerin Farko na Sabbin Magungunan Ƙasashen Waje da ake Bukatar gaggawa don Bincike na Clinical" wanda Cibiyar Nazarin Magunguna ta bayar a kan Nuwamba 1, 2018, wanda aka haɗa a cikin rukunin farko na 40 key sababbin magunguna na waje don sake dubawa, kuma Spinraza matsayi a ciki.

(6) Fitowa ta 51

Kamfanin: AVI BioPharma ne ya haɓaka (daga baya aka sake masa suna Sarepta Therapeutics).

Lokacin kasuwa: FDA ta amince da ita a watan Satumba 2016.

Alamomi: Don maganin Duchenne muscular dystrophy (DMD) tare da maye gurbin DMD a cikin exon 51 skipping gene.

Bayani: Exondys 51 maganin antisense oligonucleotide ne.The antisense oligonucleotide zai iya ɗaure zuwa exon 51 matsayi na pre-mRNA na DMD gene, haifar da samuwar mRNA balagagge.Excision, ta haka wani ɗan gyara tsarin karatun mRNA, yana taimaka wa majiyyaci don haɗa wasu nau'ikan dystrophin na aiki waɗanda suka fi guntu na furotin na yau da kullun, don haka inganta alamun haƙuri.

(7) Tegsedi

Kamfanin: Ionis Pharmaceuticals ne ya haɓaka.

Lokacin kasuwa: Tarayyar Turai ta amince da tallan a watan Yuli 2018.

Alamomi: don maganin gadon gado na transthyretin amyloidosis (hATTR).

Bayani: Tegsedi maganin antisense oligonucleotide ne wanda ke nufin transthyretin mRNA.Ita ce maganin farko da aka amince da shi a duniya don maganin hatTR.Hanyar gudanarwa shine allurar subcutaneous.Magungunan yana rage samar da furotin na ATTR ta hanyar yin niyya ga mRNA na transthyretin (ATTR), kuma yana da ƙimar fa'ida mai kyau a cikin kula da ATTR.Babu matakin cuta ko kasancewar cardiomyopathy bai dace ba.

(8) Tafiya

Kamfanin: Alnylam da Sanofi ne suka haɓaka.

Lokacin kasuwa: An amince da jeri a cikin Amurka a cikin 2018.

Alamomi: don maganin gadon gado na transthyretin amyloidosis (hATTR).

Jawabai: Onpattro magani ne na siRNA wanda ke niyya da transthyretin mRNA, wanda ke rage samar da furotin ATTR a cikin hanta da tarin amyloid adibas a cikin jijiyoyi na gefe ta hanyar niyya mRNA na transthyretin (ATTR)., don haka ingantawa da kuma kawar da alamun cututtuka.

(9) Givlaari

Kamfanin: Alnylam Corporation ne ya haɓaka.

Lokacin kasuwa: FDA ta amince da ita a watan Nuwamba 2019.

Alamomi: Don maganin m hepatic porphyria (AHP) a cikin manya.

Bayani: Givlaari sirna ce, sirna na biyu magani da aka amince don tallatawa bayan Onpattro.Ana gudanar da maganin ta hanyar subcutaneously kuma ana kai hari ga mRNA don lalata furotin ALAS1.Jiyya na Givlaari na wata-wata na iya dagewa da rage matakin ALAS1 a cikin hanta, ta haka ne za a rage matakan neurotoxic ALA da PBG zuwa kewayon al'ada, don haka Rage alamun cutar mara lafiya.Bayanan sun nuna cewa marasa lafiya da aka yi musu magani tare da Givlaari sun sami raguwar 74% a cikin adadin cututtukan cututtuka idan aka kwatanta da rukunin placebo.

(10) Vyondys53

Kamfanin: Sarepta Therapeutics ne ya haɓaka.

Lokacin kasuwa: FDA ta amince da shi a cikin Disamba 2019.

Nunawa: Don kula da marasa lafiya na DMD tare da dystrophin gene exon 53 splice maye gurbi.

Bayani: Vyondys 53 maganin antisense oligonucleotide ne.Maganin oligonucleotide yana hari akan tsarin rarrabawar dystrophin mRNA precursor.A cikin kaikaice tsari na dystrophin mRNA precursor, Exon 53 na waje an raba shi da wani yanki, watau ba a kan mRNA balagagge ba, kuma an tsara shi don samar da furotin dystrophin da aka yanke amma har yanzu yana aiki, don haka inganta ƙarfin motsa jiki a cikin marasa lafiya.

(11) Waylivra

Kamfanin: Ionis Pharmaceuticals ne suka haɓaka da nasa na Akcea Therapeutics.

Lokacin kasuwa: Hukumar Kula da Magunguna ta Turai (EMA) ta amince da ita a watan Mayu 2019.

Nunawa: A matsayin ƙarin magani ga abinci mai sarrafawa a cikin manya marasa lafiya tare da ciwo na chylomicronemia na iyali (FCS).

Bayani: Waylivra magani ne na oligonucleotide, wanda shine magani na farko da aka amince da shi don maganin FCS a duniya.

(12) Leqvio

Kamfanin: Novartis ya haɓaka.

Lokacin kasuwa: EU ta amince da shi a cikin Disamba 2020.

Alamomi: Don kula da babban matakin hypercholesterolemia (heterozygous familial da wadanda ba na iyali) ko gauraye dyslipidemia.

Bayani: Leqvio sirna ce ta sirna da ke nufi PCSK9 mRNA.Ita ce farkon sirna mai rage cholesterol (LDL-C) a duniya.Hanyar gudanarwa shine allurar subcutaneous.Magungunan yana aiki ta hanyar tsangwama na RNA zuwa ƙananan matakan furotin na PCSK9, wanda hakan yana rage matakan LDL-C.Bayanan asibiti sun nuna cewa Leqvio na iya rage LDL-C da kusan 50% a cikin marasa lafiya waɗanda matakan LDL-C ba za a iya rage su zuwa matakan da aka yi niyya ba duk da matsakaicin jurewar allurai na statins.

(13) Oxlumo

Kamfanin: Alnylam Pharmaceuticals ne ya haɓaka.

Lokacin kasuwa: EU ta amince da shi a cikin Nuwamba 2020.

Alamomi: Don maganin farko na hyperoxaluria nau'in 1 (PH1).

Jawabai: Oxlumo magani ne na siRNA wanda ke niyya hydroxy acid oxidase 1 (HAO1) ​​mRNA, wanda ake gudanarwa ta subcutaneously.An ƙirƙira maganin ta amfani da sabuwar fasahar haɗin gwiwa ta ESC-GalNAc ta Alnylam ta haɓaka, wanda ke ba da damar siRNAs da ake sarrafa su ta hanyar dagewa da inganci.Magungunan miyagun ƙwayoyi yana ƙaddamar da lalacewa ko hanawa na hydroxy acid oxidase 1 (HAO1) ​​mRNA, yana rage matakin glycolate oxidase a cikin hanta, sa'an nan kuma ya cinye abin da ake bukata don samar da oxalate kuma yana rage samar da oxalate don sarrafa ci gaban cututtuka da inganta alamun cututtuka a cikin marasa lafiya.

(14) Viltepso

Kamfanin: NS Pharma, wani reshen Nippon Shinyaku ne ya haɓaka.

Lokacin kasuwa: FDA ta amince da ita a watan Agusta 2020.

Alamomi: Don maganin Duchenne Muscular dystrophy (DMD) tare da maye gurbin DMD a cikin exon 53 skipping gene.

Bayani: Viltepso magani ne na phosphorodiamide morpholino oligonucleotide.Wannan maganin oligonucleotide zai iya ɗaure zuwa exon 53 matsayi na pre-mRNA na kwayar halittar DMD, wanda ya haifar da samuwar mRNA balagagge.An cire wani bangare na exon, ta haka wani bangare yana gyara firam ɗin karatun mRNA, yana taimaka wa majiyyaci don haɗa wasu nau'ikan dystrophin na aiki waɗanda suka fi guntu na furotin na yau da kullun, don haka inganta alamun majiyyaci.

(15) Amvuttra (vutrisiran)

Kamfanin: Alnylam Pharmaceuticals ne ya haɓaka.

Lokacin kasuwa: FDA ta amince da shi a watan Yuni 2022.

Alamomi: don lura da manya na gado transthyretin amyloidosis tare da polyneuropathy (hATTR-PN).

Bayani: Amvuttra (Vutrisiran) sirna magani ce da ke nufin transthyretin (ATTR) mRNA, wanda allurar subcutaneous ke gudanarwa.An ƙirƙira Vutrisiran bisa Alnylam's Ingantacciyar Haɓakawa Chemistry (ESC) -GalNAc haɗaɗɗiyar dandamalin isar da ƙarfi da kwanciyar hankali na rayuwa.Yarda da maganin ya dogara ne akan bayanan watanni 9 daga nazarin asibiti na Phase III (HELIOS-A), tare da sakamakon gaba ɗaya yana nuna cewa maganin ya inganta alamun hatTR-PN, tare da fiye da 50% na marasa lafiya suna juyawa ko dakatar da ci gaba.

4. Sauran magungunan maganin kwayoyin halitta

(1) Rexin-G

Kamfanin: Epeius Biotech ne ya haɓaka.

Lokacin kasuwa: Hukumar Abinci da Magunguna ta Philippine (BFAD) ta amince da ita a cikin 2005.

Alamomi: Don maganin ci-gaban ciwon daji waɗanda ke da juriya ga chemotherapy.

Bayani: Rexin-G allurar nanoparticle ce da aka ɗora ta akan gado.Yana gabatar da cyclin G1 mutant gene zuwa cikin sel masu niyya ta hanyar retroviral vector don kashe ƙaƙƙarfan ciwace-ciwace.Hanyar gudanarwa shine jiko na cikin jini.A matsayin maganin da aka yi niyya da ƙari wanda ke nema da kuma lalata ƙwayoyin cutar daji na metastatic, yana da wani tasiri akan marasa lafiya waɗanda basu da tasiri akan sauran magungunan cutar kansa, gami da ilimin halitta da aka yi niyya.

(2) Neovasculgen

Kamfanin: Cibiyar Human stem cell ce ta haɓaka.

Lokacin jeri: An amince da jeri a Rasha a ranar 7 ga Disamba, 2011, sannan aka jera a Ukraine a cikin 2013.

Alamomi: Don maganin cututtukan cututtuka na gefe, ciki har da ischemia mai tsanani.

Bayani: Neovasculgen shine kwayar halittar DNA plasmid wanda aka gina ta hanyar haɓakar haɓakar haɓakar ƙwayoyin cuta (VEGF) 165 a kan kashin baya na plasmid kuma an shigar da shi cikin marasa lafiya.

(3) Collategene

Kamfani: Jami'ar Osaka ne suka haɓaka tare da kamfanoni masu jari.

Lokacin jeri: Ma'aikatar Lafiya, Ma'aikata da walwala ta Japan ta amince da jeri a watan Agusta 2019.

Alamomi: Jiyya na ƙananan ƙarancin ischemia mai tsanani.

Bayani: Collategene magani ne na tushen plasmid, maganin jiyya na farko na Jafananci wanda AnGes ya samar.Babban bangaren wannan magani shine plasmid tsirara wanda ke dauke da jerin kwayoyin halittar hanta na mutum (HGF).Idan an yi amfani da miyagun ƙwayoyi a cikin tsokoki na ƙananan ƙafafu, HGF da aka bayyana zai inganta samuwar sababbin jini a kusa da tasoshin jini da aka rufe.Gwaje-gwaje na asibiti sun tabbatar da tasirinsa wajen inganta ciwon ciki.

KARSHE


Lokacin aikawa: Nuwamba-10-2022