Puna: WuXi AppTec

ʻO nā makahiki i hala iho nei, ua hōʻike ke kahua o ka RNA therapy i kahi ʻano pahū-i loko o nā makahiki 5 i hala iho nei, ua ʻae ʻia nā lāʻau lapaʻau RNA he 11 e ka FDA, a ua ʻoi aku kēia helu ma mua o ka huina o nā lāʻau lapaʻau RNA i ʻae ʻia!Hoʻohālikelike ʻia me nā lāʻau lapaʻau kuʻuna, hiki i ka RNA therapy ke hoʻomohala koke i nā lāʻau lapaʻau i hoʻopaʻa ʻia me ka helu kūleʻa kiʻekiʻe ke ʻike ʻia ke kaʻina gene o ka pahuhopu.Ma ka ʻaoʻao ʻē aʻe, ʻo ka hapa nui o nā lāʻau lapaʻau RNA e loaʻa wale ana no ka mālama ʻana i nā maʻi maʻamau, a ke kū nei ka hoʻomohala ʻana o ia mau lāʻau lapaʻau i nā pilikia he nui e pili ana i ka lōʻihi o ka therapy, palekana, a me ka lawe ʻana.Ma ka ʻatikala o kēia lā, ʻo kaE nānā ka hui maʻiʻo o WuXi AppTec i ka holomua ma ke kahua o ka RNA therapy i ka makahiki i hala, a e nānā i mua i ka wā e hiki mai ana o kēia kahua e kū nei me ka poʻe heluhelu.

▲ Ua ʻae ʻia nā lāʻau lapaʻau RNA 11 e ka FDA i nā makahiki 5 i hala.

Mai nā maʻi kakaʻikahi a hiki i nā maʻi maʻamau, ʻoi aku ka nui o nā pua

I ke ahulau lei hou, ua hānau ʻia ka lāʻau lapaʻau mRNA mai kahi ʻole a ua loaʻa ka manaʻo nui mai ka ʻoihana.Ma hope o kaʻO ka holomua i ka hoʻomohala ʻana i nā maʻi maʻi maʻi maʻi, kahi paʻakikī nui e kū nei i ka ʻenehana mRNA ʻo ka hoʻonui ʻana i ke ʻano o ka noi no ka mālama ʻana a me ka pale ʻana i nā maʻi hou aʻe.
Iwaena o lakou,ʻO nā lāʻau lapaʻau maʻi maʻi pilikino kahi kahua noi nui o ka ʻenehana mRNA, a ua ʻike pū mākou i nā hopena lapaʻau maikaʻi o kekahi mau lāʻau lapaʻau maʻi kanesa i kēia makahiki.ʻO kēia mahina wale nō, ua hoʻomohala pū ʻia ka maʻi maʻi maʻi kanaka hoʻokahi e Moderna a me Merck, i hui pū ʻia me ka PD-1 inhibitor Keytruda,hoemi i ka pilikiao ka hoʻi hou ʻana a i ʻole ka make ʻana i nā maʻi me ka pae III a me IV melanoma ma hope o ka wehe ʻana o ka maʻi maʻi e 44% (hoʻohālikelike ʻia me Keytruda monotherapy).Ua hōʻike ʻia ka paʻi paʻi ʻana ʻo kēia ka manawa mua i hōʻike ʻia kahi maʻi maʻi maʻi mRNA i ka maikaʻi i ka mālama ʻana i ka melanoma ma kahi hoʻokolohua hoʻokolohua randomized, ʻo ia kahi hanana nui i ka hoʻomohala ʻana i nā maʻi maʻi kanesa mRNA.
Eia kekahi, hiki i nā lāʻau lapaʻau mRNA ke hoʻonui i ka hopena therapeutic o ka cell therapy.No ka laʻana, ua hōʻike ʻia kahi noiʻi a BioNTech inā e hoʻokomo mua ʻia nā mea maʻi me kahi haʻahaʻa haʻahaʻa o CLDN6-targeted CAR-T therapy.BNT211, a laila hoʻokomo ʻia me kahi maʻi maʻi mRNA e hoʻopili ana i ka CLDN6, hiki iā lākou ke hoʻoulu i nā cell CAR-T in vivo ma o ka hōʻike ʻana i ka CLDN6 ma ka ʻili o nā cell antigen-presenting.Hoʻonui, e hoʻonui ai i ka hopena anticancer.Ua hōʻike ʻia nā hopena mua he 4 o 5 mau maʻi i loaʻa i ka lāʻau hoʻohui i loaʻa i kahi pane hapa, a i ʻole 80%.

Ma kahi o ka mRNA therapy, oligonucleotide therapy a me RNAi therapy i loaʻa i nā hopena maikaʻi i ka hoʻonui ʻana i ke ʻano o nā maʻi.I ka mālama ʻana i ka maʻi hepatitis B mau loa, kokoke i 30% o nā mea maʻi ʻaʻole hiki ke ʻike i ka maʻi hepatitis B surface antigen a me ka maʻi hepatitis B DNA i loko o vivo ma hope o ka hoʻohana ʻana i ka antisense oligonucleotide therapy.bepirovirsen i hoʻomohala pū ʻia e GSK a me Ionisno 24 pule.I kekahi poʻe maʻi, ʻoiai he 24 pule ma hope o ka pau ʻana o ka mālama ʻana, ʻaʻole ʻike ʻia kēia mau hōʻailona o ka maʻi hepa B i ke kino.
I ka manawa like, ʻo ka RNAi therapyUa hui pū ʻia ʻo VIR-2218 e Vir Biotechnology a me Alnylamme ka interferon α, a ma ka mahele 2 o na hoao ana, ma kahi o 30% o na ma'i hepa B mau i hiki ole ke ike i ka hepa B surface antigen (HBsAg).Eia kekahi, ua hoʻomohala kēia mau maʻi i nā antibodies e kūʻē i ka protein hepatitis B, e hōʻike ana i kahi pane ʻōnaehana pale maikaʻi.I ka hui pū ʻana i kēia mau hopena, hōʻike ka ʻoihana ʻo ka RNA therapy ke kī i kahi lāʻau lapaʻau no ka maʻi hepatitis B.
ʻO kēia paha ka hoʻomaka ʻana o ka therapy RNA no nā maʻi maʻamau.Wahi a Alnylam's R&D pipeline, ke hoʻomohala nei ʻo ia i nā lāʻau lapaʻau RNAi no ka mālama ʻana i ke koko kiʻekiʻe, ka maʻi Alzheimer, a me ka steatohepatitis non-alcoholic, a ʻo ka wā e hiki mai ana ke nānā aku.

Ke wehe nei i ka bottleneck o ka lawe ʻana i ka therapy RNA

ʻO ka hāʻawi ʻana i ka RNA therapy kekahi o nā bottlenecks e kaupalena ana i kāna noi.Ke hoʻomohala nei ka poʻe ʻepekema i nā ʻenehana like ʻole e hāʻawi kikoʻī i ka therapy RNA i nā kino a me nā ʻiʻo ʻē aʻe ma waho o ke ake.
ʻO kekahi o nā ala hiki ke "hoʻopaʻa" i nā RNA therapeutic me nā molekole kikoʻī kikoʻī.No ka laʻana, ua hoʻolaha aku ʻo Avidity Biosciences e hiki i kāna paepae ʻenehana ke hoʻohui i nā antibodies monoclonal i oligonucleotides ihoʻopaʻa pono i nā siRNA.hoʻouna ʻia i ka ʻiʻo iwi.Ua hōʻike ʻia ka paʻi paʻi ʻana ʻo ia ka manawa mua e hiki ke hoʻokō maikaʻi ʻia ʻo siRNA a hāʻawi ʻia i ka ʻiʻo ʻiʻo o ke kanaka, he mea koʻikoʻi nui ia ma ke kahua o ka RNA therapy.

Puna kiʻi: 123RF

Ma waho aʻe o ka ʻenehana conjugation antibody, ʻo kekahi mau hui e hoʻomohala ana i nā nanoparticles lipid (LNP) e "hoʻomaikaʻi" i kēlā mau mea lawe.ʻo kahi laʻana, ReCode Therapeutichoʻohana i kāna Selective Organ Targeting LNP Technology (SORT) e hoʻopuka i nā ʻano ʻano like ʻole o ka therapy RNA i nā māmā, spleen, ate a me nā mea ʻē aʻe.I kēia makahiki, ua hoʻolaha ka hui i ka hoʻopau ʻana o kahi $ 200 miliona Series B puni kālā, me nā keʻena ʻoihana ʻoihana o Pfizer, Bayer, Amgen, Sanofi a me nā hui lāʻau lapaʻau nui ʻē aʻe e komo ana i ka hoʻopukapuka.ʻO Kernal Biologics, ka mea i loaʻa he $ 25 miliona ma ke kālā Series A i kēia makahiki, ke hoʻomohala nei hoʻi i nā LNP i hōʻiliʻili ʻole i loko o ke akepaʻa, akā hiki ke hāʻawi i ka mRNA i nā cell target e like me ka lolo a i ʻole nā ​​kiko kikoʻī.
ʻO Orbital Therapeutics, ka mea i hoʻomaka i kēia makahiki, lawe pū i ka hāʻawi ʻana i ka RNA therapy ma ke ʻano he alakaʻi hoʻomohala koʻikoʻi.Ma ka hoʻohui ʻana i ka ʻenehana RNA a me nā mīkini hoʻopuka, manaʻo ka hui e kūkulu i kahi kahua ʻenehana RNA kūʻokoʻa e hiki ke hoʻolōʻihi i ka hoʻomau a me ka hapalua o ke ola o nā therapeutics RNA hou a hāʻawi iā lākou i nā ʻano ʻano cell a me nā ʻano kiko.

Ke puka mai nei kahi ʻano hou o ka RNA therapy i ka manawa mōʻaukala

Ma ka lā 21 o Dekemaba i kēia makahiki, ma ke kahua o ka RNA therapy, ua loaʻa he 31 mau hanana kālā mua (e ʻike i ke ʻano ma ka hopena o ka ʻatikala no nā kikoʻī), e pili ana i nā ʻoihana ʻoki 30 (ua loaʻa ʻelua kālā i hoʻokahi hui), me ka huina kālā o 1,74 biliona kālā US.Hōʻike ka loiloi o kēia mau ʻoihana e ʻoi aku ka manaʻo o nā mea hoʻopukapuka e pili ana i kēlā mau ʻoihana ʻokiʻoki i manaʻo ʻia e hoʻoponopono i nā pilikia he nui o ka RNA therapy, i mea e ʻike pono ai i ka hiki o ka RNA therapy a pōmaikaʻi i nā mea maʻi.
A aia nā ʻoihana e hiki mai ana e hoʻomohala i nā ʻano therapeutic RNA hou loa.ʻAʻole like me nā oligonucleotides kuʻuna, RNAi a i ʻole mRNA, nā ʻano hou o nā molekala RNA i hoʻomohala ʻia e kēia mau ʻoihana e manaʻo ʻia e haki i ka bottleneck o nā lāʻau lapaʻau.
ʻO Circular RNA kekahi o nā wahi wela o ka ʻoihana.Ke hoʻohālikelike ʻia me ka linear mRNA, hiki i ka ʻenehana RNA circular i hoʻomohala ʻia e kahi hui ʻokiʻoki i kapa ʻia ʻo Orna Therapeutics hiki ke pale i ka ʻike ʻia e ka ʻōnaehana immune innate a me exonucleases, ʻaʻole ia e hōʻemi nui i ka immunogenicity, akā ʻoi aku ka kiʻekiʻe.Eia kekahi, ke hoʻohālikelike ʻia me ka RNA linear, ʻoi aku ka liʻiliʻi o ka conformation o ka circular RNA, a ʻoi aku ka nui o ka RNA circular hiki ke hoʻouka ʻia me ka LNP like, e hoʻomaikaʻi ana i ka hoʻopuka ʻana o ka RNA therapy.Hiki i kēia mau hiʻohiʻona ke kōkua i ka hoʻomaikaʻi ʻana i ka ikaika a me ka lōʻihi o nā lāʻau lapaʻau RNA.
I kēia makahiki, ua hoʻopau ka hui i kahi US $ 221 miliona Series B puni kālā, a ua hōʻea pū i kahi noiʻi aka launa pū ʻana me Merck a hiki i ka US$3.5 biliona.Eia kekahi, hoʻohana pū ʻo Orna i ka RNA circular e hana pololei i ka therapy CAR-T i nā holoholona, ​​​​e hoʻopau ana i kahi hōʻoia omanaʻo.
Ma waho aʻe o ka RNA circular, ua makemake nui ʻia ka ʻenehana mRNA (samRNAs) hoʻonui iā ia iho e nā mea hoʻopukapuka.Hoʻokumu ʻia kēia ʻenehana i ka mīkini hoʻoikaika ponoʻī o nā maʻi RNA, hiki ke hoʻoulu i ka replication o nā kaʻina samRNA i loko o ka cytoplasm, e hoʻolōʻihi i ka ʻōlelo kinetics o ka mRNA therapeutics, a laila e hōʻemi i ka pinepine o ka hoʻokele.Ke hoʻohālikelike ʻia me ka mRNA laina kuʻuna, hiki i ka samRNA ke mālama i nā pae hōʻike protein like ʻole ma kahi o 10-folds haʻahaʻa haʻahaʻa.I kēia makahiki, ua loaʻa iā RNAimmune, ka mea e pili ana i ka hoʻomohala ʻana i kēia kahua, he US $ 27 miliona ma ke kālā Series A.
Pono ka ʻenehana tRNA e nānā i mua.Hiki i ka tRNA-based therapy ke "hōʻole" i ka codon stop hewa i ka wā e hana ai ke kelepona i ka protein, no laila e hana ʻia ka protein holoʻokoʻa maʻamau.Ma muli o ka liʻiliʻi loa o nā ʻano codons stop ma mua o nā maʻi pili, hiki i ka tRNA therapy ke hoʻomohala i hoʻokahi lāʻau lapaʻau hiki ke mālama i nā maʻi he nui.I kēia makahiki, hC Bioscience, e kālele ana i ka tRNA therapy, ua hoʻonui i ka huina o US $40 miliona ma ke kālā Series A.

Epilogue

Ma ke ʻano he ʻano lapaʻau e puka mai nei, ua hoʻokō ʻo RNA therapy i ka hoʻomohala wikiwiki i nā makahiki i hala iho nei, a ua ʻae ʻia nā lāʻau lapaʻau he nui.Mai nā hanana hou loa i ka ʻoihana, hiki ke ʻike ʻia ke hoʻonui ʻia nei nā maʻi RNA ʻokiʻoki i ka nui o nā maʻi e hiki ai i ia mau lāʻau lapaʻau ke mālama, ka lanakila ʻana i nā bottlenecks like ʻole i ka hāʻawi ʻia ʻana, a me ka hoʻomohala ʻana i nā molekala RNA hou e lanakila ai i nā palena o nā lāʻau lapaʻau e pili ana i ka pono a me ka lōʻihi.mau pilikia.I kēia au hou o ka RNA therapy, hiki i kēia mau ʻoihana ʻokiʻoki ke lilo i mea nui o ka ʻoihana i nā makahiki e hiki mai ana.

Nā huahana pili:

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

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