Gwero: WuXi AppTec

Zaka zaposachedwa, gawo la chithandizo cha RNA chawonetsa njira yophulika-m’zaka 5 zokha zapitazi, njira zochiritsira 11 za RNA zavomerezedwa ndi FDA, ndipo chiŵerengerochi chikuposa ngakhale chiŵerengero cha machiritso a RNA ovomerezedwa kale!Poyerekeza ndi machiritso achikhalidwe, chithandizo cha RNA chimatha kupanga njira zochiritsira zomwe zikuyenda bwino kwambiri malinga ndi momwe jini limayendera.Kumbali inayi, mankhwala ambiri a RNA akadalipo kuti athetse matenda osowa, ndipo chitukuko cha mankhwala oterechi chikukumana ndi zovuta zambiri zokhudzana ndi kukhazikika kwa mankhwala, chitetezo, ndi kubereka.M'nkhani ya lero, aGulu lokhutira la WuXi AppTec liwunika momwe chithandizo cha RNA chikuyendera chaka chatha, ndikuyembekezera tsogolo la gawo lomwe likubwerali ndi owerenga.

▲ 11 RNA mankhwala kapena katemera wavomerezedwa ndi FDA m'zaka 5 zapitazi

Kuchokera ku matenda osowa kwambiri kupita ku matenda wamba, maluwa ambiri amaphuka

Mu mliri watsopano wa korona, katemera wa mRNA adabadwa modzidzimutsa ndipo walandira chidwi chochuluka kuchokera kumakampani.Pambuyo paKupambana pakupanga katemera wa matenda opatsirana, vuto lina lalikulu lomwe ukadaulo wa mRNA ukukumana nawo ndikukulitsa kuchuluka kwa ntchito yochizira ndi kupewa matenda ambiri.
Mwa iwo,katemera wa khansa payekha ndi gawo lofunikira laukadaulo wa mRNA, ndipo tawonanso zotsatira zabwino zachipatala za katemera angapo a khansa chaka chino.Mwezi uno, katemera wa khansa payekha wopangidwa ndi Moderna ndi Merck, wophatikizidwa ndi PD-1 inhibitor Keytruda,kuchepetsa chiopsezokubwereza kapena kufa kwa odwala omwe ali ndi siteji ya III ndi IV ya melanoma pambuyo pochotsa chotupa chonse ndi 44% (poyerekeza ndi Keytruda monotherapy).Kutulutsa kwa atolankhani kunanena kuti aka ndi koyamba kuti katemera wa khansa ya mRNA awonetse mphamvu pochiza khansa ya melanoma pamayesero achipatala osasintha, chomwe ndi chochitika chodziwika bwino pakupanga katemera wa khansa ya mRNA.
Kuphatikiza apo, katemera wa mRNA amathanso kupititsa patsogolo kuchiritsa kwa ma cell.Mwachitsanzo, kafukufuku wa BioNTech adawonetsa kuti ngati odwala ayamba kulowetsedwa ndi mlingo wochepa wa mankhwala a CLDN6 omwe amatsata CAR-T.BNT211, ndiyeno kubayidwa ndi katemera wa mRNA wokhotakhota CLDN6, amatha kulimbikitsa maselo a CAR-T mu vivo pofotokozera CLDN6 pamwamba pa ma antigen-presenting cells.Kukulitsa, potero kumawonjezera mphamvu ya anticancer.Zotsatira zoyambilira zidawonetsa kuti odwala 4 mwa 5 omwe adalandira chithandizo chophatikiza adapeza kuyankha pang'ono, kapena 80%.

Kuphatikiza pa chithandizo cha mRNA, oligonucleotide therapy ndi RNAi therapy yapezanso zotsatira zabwino pakukulitsa kuchuluka kwa matenda.Pochiza matenda a chiwindi a B, pafupifupi 30% ya odwala sangathe kuzindikira hepatitis B pamwamba antigen ndi hepatitis B virus DNA mu vivo atagwiritsa ntchito antisense oligonucleotide therapy.bepirovirsen yopangidwa pamodzi ndi GSK ndi Ioniskwa masabata 24.Odwala ena, ngakhale patatha milungu 24 atasiya chithandizo, zizindikiro za matenda a chiwindi a B zinali zosaonekabe m’thupi.
Mwachidziwitso, chithandizo cha RNAiVIR-2218 yopangidwa molumikizana ndi Vir Biotechnology ndi Alnylam idaphatikizidwandi interferon α, komanso mu mayesero a chipatala cha 2, pafupifupi 30% ya odwala matenda a chiwindi a B adalepheranso kuzindikira hepatitis B pamwamba antigen (HBsAg).Kuphatikiza apo, odwalawa adapanga ma antibodies motsutsana ndi mapuloteni a hepatitis B, kuwonetsa kuyankha kwamphamvu kwa chitetezo chamthupi.Kutengera zotsatira izi palimodzi, makampaniwa akuwonetsa kuti chithandizo cha RNA chingakhale chinsinsi chochiza matenda a hepatitis B.
Ichi chikhoza kukhala chiyambi chabe cha mankhwala a RNA a matenda wamba.Malinga ndi payipi ya R&D ya Alnylam, ikupanganso njira zochiritsira za RNAi zochizira matenda oopsa, matenda a Alzheimer, komanso steatohepatitis osaledzeretsa, ndipo tsogolo liyenera kuyembekezera.

Kuthetsa vuto la kuperekedwa kwa mankhwala a RNA

Kupereka chithandizo cha RNA ndi chimodzi mwazolepheretsa kugwiritsa ntchito kwake.Asayansi akupanganso matekinoloje osiyanasiyana kuti apereke chithandizo cha RNA ku ziwalo ndi minofu ina kupatula chiwindi.
Imodzi mwa njira zomwe zingatheke ndi "kumanga" ma RNAs achire ndi mamolekyu enieni a minofu .Mwachitsanzo, Avidity Biosciences posachedwapa yalengeza kuti nsanja yake yaukadaulo imatha kuphatikiza ma antibodies a monoclonal ndi oligonucleotides kuti agwirizane.kumanga bwino siRNAs.kutumizidwa ku minofu ya chigoba.Kutulutsidwa kwa atolankhani kunanena kuti iyi ndi nthawi yoyamba yomwe siRNA ikhoza kuyang'aniridwa bwino ndikuperekedwa ku minofu ya minofu ya anthu, yomwe ndi gawo lalikulu la chithandizo cha RNA.

Chithunzi chojambula: 123RF

Kuphatikiza pa ukadaulo wa antibody conjugation, makampani angapo omwe amapanga lipid nanoparticles (LNP) nawonso "akukweza" zonyamula zotere.Mwachitsanzo, ReCode Therapeuticamagwiritsa ntchito yapadera Selective Organ Targeting LNP Technology (SORT) kuti apereke mitundu yosiyanasiyana yamankhwala a RNA m'mapapo, ndulu, chiwindi ndi ziwalo zina.Chaka chino, kampaniyo idalengeza kutsirizidwa kwa ndalama za US $ 200 miliyoni Series B, ndi madipatimenti akuluakulu a Pfizer, Bayer, Amgen, Sanofi ndi makampani ena akuluakulu ogulitsa mankhwala omwe akugwira nawo ntchito.Kernal Biologics, yomwe idalandiranso $ 25 miliyoni mu Series A ndalama chaka chino, ikupanganso LNPs zomwe sizikuchuluka m'chiwindi, koma zimatha kupereka mRNA kuti zigwirizane ndi maselo monga ubongo kapena zotupa zenizeni.
Orbital Therapeutics, yomwe inayamba chaka chino, imatenganso kupereka chithandizo cha RNA monga njira yofunikira yachitukuko.Mwa kuphatikiza ukadaulo wa RNA ndi njira zoperekera, kampaniyo ikuyembekeza kupanga nsanja yapadera yaukadaulo ya RNA yomwe imatha kukulitsa kulimbikira ndi theka la moyo wamankhwala opangira RNA ndikuwapereka kumitundu yosiyanasiyana yama cell ndi minofu.

Mtundu watsopano wamankhwala a RNA umatuluka panthawi yodziwika bwino

Pofika pa Disembala 21 chaka chino, pankhani ya chithandizo cha RNA, pakhala pali zochitika zoyambira 31 (onani njira yomwe ili kumapeto kwa nkhaniyo kuti mumve zambiri), yokhudzana ndi makampani 30 otsogola (kampani imodzi idalandira ndalama kawiri), ndi ndalama zokwana 1.74 biliyoni za US.Kuwunika kwamakampaniwa kukuwonetsa kuti osunga ndalama ali ndi chiyembekezo chokhudza makampani otsogola omwe akuyembekezeka kuthetsa zovuta zambiri zamankhwala a RNA, kuti athe kuzindikira kuthekera kwa chithandizo cha RNA ndikupindulitsa odwala ambiri.
Ndipo pali makampani omwe akubwera omwe akupanga mitundu yatsopano yamankhwala a RNA.Mosiyana ndi chikhalidwe cha oligonucleotides, RNAi kapena mRNA, mitundu yatsopano ya mamolekyu a RNA opangidwa ndi makampaniwa akuyembekezeka kupyola muzitsulo zamankhwala omwe alipo kale.
RNA yozungulira ndi imodzi mwamalo otentha kwambiri pamsika.Poyerekeza ndi mzere wa mRNA, teknoloji yozungulira ya RNA yopangidwa ndi kampani yodula kwambiri yotchedwa Orna Therapeutics ingapewe kuzindikiridwa ndi chitetezo cha m'thupi ndi exonucleases, zomwe sizimangochepetsa kwambiri immunogenicity, komanso zimakhala zokhazikika.Kuphatikiza apo, poyerekeza ndi RNA yozungulira, mawonekedwe opindika a RNA ozungulira ndi ocheperako, ndipo RNA yozungulira kwambiri imatha kuyikidwa ndi LNP yomweyi, kupititsa patsogolo luso loperekera chithandizo cha RNA.Izi zitha kuthandiza kukonza potency ndi kukhazikika kwamankhwala a RNA.
Chaka chino, kampaniyo idamaliza ndalama zokwana $221 miliyoni za Series B, ndipo idafikiranso kafukufuku ndimgwirizano wachitukuko ndi Merck mpaka US $ 3.5 biliyoni.Kuphatikiza apo, Orna amagwiritsanso ntchito RNA yozungulira kupanga mwachindunji chithandizo cha CAR-T mu nyama, kumaliza umboni walingaliro.
Kuphatikiza pa RNA yozungulira, ukadaulo wodzikulitsa wa mRNA (samRNAs) nawonso umakondedwa ndi osunga ndalama.Tekinoloje iyi imakhazikitsidwa ndi njira yodzikuza ya ma virus a RNA, omwe angapangitse kubwereza kwa ma samRNA mu cytoplasm, kutalikitsa mawu akuti kinetics a mRNA therapeutics, potero amachepetsa kuchuluka kwa kayendetsedwe kake.Poyerekeza ndi mRNA yodziwika bwino, samRNA imatha kukhalabe ndi milingo yofananira yama protein pafupifupi 10-fold low Mlingo.Chaka chino, RNAimmune, yomwe ikuyang'ana pa chitukuko cha ntchitoyi, inalandira US $ 27 miliyoni mu ndalama za Series A.
Tekinoloje ya tRNA ndiyofunikanso kuyang'ana.Thandizo lochokera ku tRNA limatha "kunyalanyaza" kuyimitsidwa kolakwika pamene selo limapanga puloteni, kotero kuti puloteni yokhazikika yokwanira bwino imapangidwa.Chifukwa pali mitundu yocheperako ya ma codons oyimitsa kuposa matenda omwe amagwirizana nawo, chithandizo cha tRNA chili ndi kuthekera kopanga chithandizo chimodzi chomwe chimatha kuchiza matenda ambiri.Chaka chino, hC Bioscience, yomwe imayang'ana kwambiri pa chithandizo cha tRNA, yapeza ndalama zonse za US $ 40 miliyoni mu ndalama za Series A.

Epilogue

Monga njira yochiritsira yomwe ikubwera, chithandizo cha RNA chapeza chitukuko chofulumira m'zaka zaposachedwa, ndipo njira zambiri zochiritsira zavomerezedwa.Kuchokera ku zomwe zachitika posachedwa m'makampaniwa, zitha kuwoneka kuti njira zochiritsira za RNA zotsogola zikukulitsa matenda osiyanasiyana omwe mankhwalawa amatha kuchiza, kuthana ndi zopinga zosiyanasiyana popereka zomwe akufuna, ndikupanga mamolekyu atsopano a RNA kuti athe kuthana ndi zolephera zamankhwala omwe alipo pokhudzana ndi mphamvu komanso kulimba.zovuta zambiri.Munthawi yatsopanoyi yamankhwala a RNA, makampani otsogola awa atha kukhala gawo lalikulu pamsika pazaka zingapo zikubwerazi.

Zogwirizana nazo:

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

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