Kunobva: WuXi AppTec

Makore achangopfuura, ndima yeRNA therapy yakaratidza kuputika maitiro-.mumakore mashanu apfuura chete, 11 RNA kurapa kwakatenderwa neFDA, uye iyi nhamba inotopfuura hwerengedzo yezvakambobvumidzwa kurapa kweRNA!Kuenzaniswa nekurapa kwechinyakare, RNA therapy inogona kukurumidza kugadzira marapirwo anonangwa ane budiriro yepamusoro sekureba kutevedzana kwemajini kwechinangwa kuchizivikanwa.Nekune rimwe divi, marapirwo mazhinji eRNA achiri kuwanikwa chete kurapa zvirwere zvisingawanzo, uye kuvandudzwa kwemishonga yakadaro kuchiri kutarisana nematambudziko akawanda maererano nekusimba kwekurapa, kuchengetedzeka, uye kusununguka.Muchinyorwa chanhasi, theChikwata chezvemukati cheWuXi AppTec chichaongorora kufambira mberi mumunda weRNA therapy mugore rapfuura, uye kutarisira ramangwana reiyi ndima iri kubuda nevaverengi.

▲ 11 RNA marapiro kana majekiseni akatenderwa neFDA mumakore mashanu apfuura.

Kubva pazvirwere zvisingawanzoitiki kune zvirwere zvakajairika, maruva akawanda anotumbuka

Mune denda idzva rekorona, iyo mRNA yekudzivirira yakazvarwa pasina pese uye yakagamuchira kutariswa kwakapararira kubva kuindasitiri.Mushure mebudiriro mukugadzirwa kwemishonga yekudzivirira chirwere chinotapukira, rimwe dambudziko guru rakatarisana nemRNA tekinoroji nderekuwedzera chiyero chekushandiswa kwekurapa nekudzivirira zvimwe zvirwere.
Pakati pavo,majekiseni egomarara ega ega inzvimbo yakakosha yekushandisa yemRNA tekinoroji, uye takaonawo migumisiro yakanaka yekiriniki yemishonga yakawanda yegomarara gore rino.Mwedzi uno chete, iyo yega yega yekudzivirira cancer yakabatanidzwa neModerna neMerck, yakasanganiswa nePD-1 inhibitor Keytruda,kuderedza ngoziyekudzokazve kana kufa muvarwere vane nhanho III uye IV melanoma mushure mekupedzisira bundu resection ne44% (zvichienzaniswa neKeytruda monotherapy).Kuburitswa kwenhau kwakaratidza kuti aka ndekekutanga kuti mRNA vaccine yegomarara iratidze kushanda mukurapa melanoma muyedzo yekiriniki isina kujairika, inova chiitiko chakakosha mukugadzirwa kwemRNA cancer yekudzivirira.
Pamusoro pezvo, mishonga yekudzivirira mRNA inogona zvakare kusimudzira kurapa kwesero therapy.Semuenzaniso, chidzidzo chakaitwa neBioNTech chakaratidza kuti kana varwere vakatanga kuisirwa nedosi yakaderera yeCLDN6-yakanangana neCAR-T kurapwa.BNT211, yobva yaiswa jekiseni nemRNA vaccine encoding CLDN6, inogona kukurudzira CAR-T masero mu vivo nekuratidza CLDN6 pamusoro pema antigen-presenting cells.Amplification, nekudaro kuwedzera anticancer mhedzisiro.Mhedzisiro yekutanga yakaratidza kuti 4 kubva pavashanu varwere vakagamuchira kurapwa kwemusanganiswa vakawana mhinduro isina kukwana, kana 80%.

Pamusoro pekurapa mRNA, oligonucleotide therapy uye RNAi therapy yakawanawo mhedzisiro yakanaka mukuwedzera huwandu hwezvirwere.Mukurapa kwechisingaperi hepatitis B, vangangoita 30% yevarwere havagone kuona hepatitis B pamusoro antigen uye hepatitis B virus DNA in vivo mushure mekushandisa antisense oligonucleotide therapy.bepirovirsen yakabatanidzwa pamwe chete neGSK neIoniskwemavhiki makumi maviri nemana.Mune vamwe varwere, kunyange mavhiki makumi maviri nemana mushure mekumira kurapwa, izvi zviratidzo zvehepatitis B zvakanga zvisati zvaonekwa mumuviri.
Sezvineiwo, iyo RNAi kurapwaVIR-2218 yakabatanidzwa pamwe neVir Biotechnology uye Alnylam yakasanganiswane interferon α, uye muchikamu chechipiri chekliniki miedzo, inenge 30% yevarwere vasingaperi vehepatitis B vakakundikanawo kuona hepatitis B surface antigen (HBsAg).Pamusoro pezvo, varwere ava vakagadzira masoja ekudzivirira chirwere anorwisa hepatitis B, achiratidza mhinduro yakanaka.Kutora mhedzisiro iyi pamwe chete, indasitiri inotaura kuti RNA therapy inogona kunge iri kiyi yekurapa kunoshanda kwehepatitis B.
Uku kunogona kunge kuri kutanga kweRNA kurapwa kwezvirwere zvakajairika.Sekureva kwepombi yeAlnylam's R&D, iri kugadzirawo marapirwo eRNAi ekurapa hypertension, chirwere cheAlzheimer, uye chisiri doro steatohepatitis, uye ramangwana rakakodzera kutarisira.

Kutyora mukati mebhodhoro reRNA kurapwa kuburitsa

Kuendeswa kweRNA kurapa ndeimwe yemabhodhoro anomisa kushandiswa kwayo.Masayendisiti ari kugadzirawo akasiyana matekinoroji ekunyatso kuendesa RNA kurapa kunhengo uye matishu kunze kwechiropa.
Imwe yedzinogona nzira ndeye "kusunga" marapirwo eRNAs ane tishu-chaiwo mamorekuru .Semuenzaniso, Avidity Biosciences nguva pfupi yadarika yakazivisa kuti tekinoroji yayo inogona kubatanidza monoclonal antibodies kune oligonucleotideszvinonyatso sunga siRNAs.inotumirwa kune skeletal muscle.Kuburitswa kwenhau kwakaratidza kuti ino inguva yekutanga iyo siRNA inogona kunyatso kunangwa uye kuendeswa kune yemhasuru tishu, inova budiriro huru mumunda weRNA therapy.

Mufananidzo kunobva: 123RF

Pamusoro peiyo antibody conjugation tekinoroji, akati wandei emakambani ari kugadzira lipid nanoparticles (LNP) ari zvakare "kusimudzira" vatakuri vakadaro.Semuyenzaniso, ReCode Therapeuticinoshandisa yayo yakasarudzika Selective Organ Targeting LNP Technology (SORT) kuendesa akasiyana emhando dzakasiyana dzeRNA kurapa kumapapu, spleen, chiropa uye dzimwe nhengo.Gore rino, kambani yakazivisa kupedzwa kweUS $ 200 miriyoni Series B kutenderera kwemari, ine venture capital madhipatimendi ePfizer, Bayer, Amgen, Sanofi nemamwe makambani makuru emishonga ari kutora chikamu mukudyara.Kernal Biologics, iyo yakagamuchirawo mamiriyoni makumi maviri neshanu emadhora muSeries A mari gore rino, iri kugadzirawo LNPs isingaunganidzi muchiropa, asi inogona kuendesa mRNA kune masero akadai seuropi kana mamota chaiwo.
Orbital Therapeutics, iyo yakatanga gore rino, zvakare inotora kuendeswa kweRNA kurapwa seyakakosha nzira yekusimudzira.Nekubatanidza tekinoroji yeRNA uye nzira dzekutumira, kambani inotarisira kuvaka yakasarudzika RNA tekinoroji chikuva chinogona kuwedzera kushingirira uye hafu yeupenyu hwehunyanzvi hweRNA therapeutics uye kuvaendesa kune akasiyana siyana akasiyana maseru nematishu marudzi.

Rudzi rutsva rweRNA therapy inobuda panguva yenhoroondo

Kubva muna Zvita 21 gore rino, mumunda weRNA kurapwa, kwave nezviitiko makumi matatu neshanu zvekutanga-danho remari (ona nzira iri kumagumo echinyorwa kuti uwane rumwe ruzivo), zvinosanganisira makumi matatu emhando yepamusoro makambani (imwe kambani yakawana mari kaviri), nemari yakazara ye1.74 bhiriyoni US madhora.Ongororo yemakambani aya inoratidza kuti vanoisa mari vane tarisiro pamusoro peaya ekucheka-kumucheto makambani anotarisirwa kugadzirisa akawanda matambudziko eRNA therapy, kuitira kuti vanyatsoona kugona kweRNA kurapwa uye kubatsira varwere vazhinji.
Uye kune makambani ari kuuya-uye-anouya achigadzira mhando nyowani dzeRNA therapeutics.Kusiyana neyakajairwa oligonucleotides, RNAi kana mRNA, mhando nyowani dzeRNA mamorekuru akagadzirwa nemakambani aya anotarisirwa kupaza nepakati pemabhodhoro ekurapa aripo.
Circular RNA ndiyo imwe yenzvimbo dzinopisa muindasitiri iyi.Kuenzaniswa nemutsara mRNA, tekinoroji yeRNA tekinoroji yakagadziridzwa nekambani yekucheka-kumucheto inonzi Orna Therapeutics inogona kudzivirira kuzivikanwa neiyo innate immune system uye exonucleases, iyo isingangodzikisi zvakanyanya immunogenicity, asiwo ine yakanyanya kugadzikana.Pamusoro pezvo, zvichienzaniswa neRNA mutsara, iyo yakapetwa conformation yedenderedzwa RNA idiki, uye yakawanda denderedzwa RNA inogona kutakurwa neiyo yakafanana LNP, ichivandudza kuburitsa kweRNA kurapwa.Aya maficha anogona kubatsira kuvandudza potency uye kusimba kweRNA therapeutics.
Gore rino, kambani yakapedza US$221 miriyoni Series B yemari, uye yakasvikawo patsvagiridzo uye.mubatanidzwa webudiriro neMerck unosvika US$3.5 bhiriyoni.Pamusoro pezvo, Orna anoshandisawo denderedzwa RNA kugadzira zvakananga CAR-T kurapwa mumhuka, kupedzisa humbowo hwe.pfungwa.
Pamusoro peRNA yakatenderedzwa, tekinoroji yekuzvisimudzira mRNA (samRNAs) yakafarirwawo nevanoita mari.Iyi tekinoroji yakavakirwa pane yekuzvimisikidza nzira yeRNA mavhairasi, ayo anogona kukurudzira kudzokororwa kweiyo samRNA kutevedzana mucytoplasm, ichirebesa kutaura kinetics yemRNA therapeutics, nekudaro ichidzikisa kuwanda kwekutonga.Kuenzaniswa neyechinyakare mutsara mRNA, samRNA inokwanisa kuchengetedza yakafanana mapuroteni ekutaura mazinga pane angangoita gumi-yakapetwa madhasi.Gore rino, RNAimmune, iyo inotarisa mukusimudzirwa kwendima iyi, yakagamuchira US $ 27 mamiriyoni muSeries A mari.
tRNA tekinoroji yakakoshawo kutarisira.Iyo tRNA-based therapy inogona "kufuratira" isiriyo yekumira codon kana sero richigadzira protein, kuitira kuti yakajairika yakazara-yakareba protein igadzirwe.Nekuti kune mashoma kwazvo marudzi ekumisa codon pane anoenderana nezvirwere, tRNA therapy ine mukana wekugadzira imwe chete kurapa inogona kurapa zvirwere zvakawanda.Gore rino, hC Bioscience, iyo inotarisa pakurapa kwetRNA, yakasimudza huwandu hweUS $ 40 mamiriyoni muSeries A mari.

Epilogue

Senge iri kubuda yekurapa modhi, RNA therapy yakawana kukura nekukurumidza mumakore achangopfuura, uye akawanda marapirwo akabvumidzwa.Kubva pane zvichangobva kuitika muindastiri, zvinogona kuoneka kuti kurapa-kumucheto kweRNA kurapwa kuri kuwedzera huwandu hwezvirwere izvo marapirwo akadaro anogona kurapa, kukunda mabhodhoro akasiyana-siyana mukuunza kwakanangwa, uye kugadzira mamorekuru matsva eRNA kukunda zvisingakwanisi kurapwa aripo maererano nekushanda uye kusimba.matambudziko akawanda.Munguva ino nyowani yeRNA kurapwa, aya makambani ekucheka-cheka anogona kuve akatarisana neindasitiri mumakore mashoma anotevera.

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