Prisys Biotechnology tshwj xeeb hauv kev tshawb fawb tsis yog -tib neeg primate (NHP) thiab kev tshawb fawb kev txhais lus. Tsim cov lipid nanoparticles (LNPs) rau kev xa khoom zoo thiab nyab xeeb ntawm mRNA kho rau cov ntaub so ntswg thiab cov kabmob sib txawv tseem yog qhov nyuaj. Ib qho kev sib tw tseem ceeb hauv kev xa khoom ntawm mRNA yog txhais cov kev tshawb pom ua ntej los ntawm cov qauv nas mus rau cov tsis yog -tib neeg primates (NHPs), uas muaj ntau dua physiological thiab pharmacokinetic zoo sib xws nrog tib neeg.
Kab lus no qhia txog ob txoj kev tshawb fawb uas qhia txog kev ua kom zoo dua LNPs rau mRNA xa hauv NHP qauv thiab ntsuas lawv cov peev xwm txhais lus.
Hauv thawj daim ntawv, Cov Kws Tshawb Fawb tau pom tias tib lub LNPs thauj khoom nrog mRNA encoding tib neeg ornithine transcarbamylase (hOTC), lub siab enzyme koom nrog hauv lub voj voog urea, tau txo qis protein ntau hauv daim siab ntawm NHPs dua li cov nas, qhia txog hom - nyob ntawm qhov sib txawv ntawm LNP kev ua. Lawv tau tshawb xyuas ntxiv txog yam uas cuam tshuam rau LNP kev ua tau zoo hauv ntau hom, tsom rau LNP loj thiab polyethylene glycol (PEG) lipid cov ntsiab lus.
Lawv pom tias LNPs me me muaj cov qib protein ntau dua hauv NHP lub siab dua li LNPs loj dua, uas yog qhov txawv ntawm cov qauv pom hauv nas. Lawv kuj pom tias qhov zoo tshaj plaws PEG lipid cov ntsiab lus rau LNPs me me hauv NHPs yog 2.2-2.8%, uas yog siab dua li tus nqi siv rau cov nas. Los ntawm kev kho LNP qhov loj me thiab PEG lipid cov ntsiab lus, lawv muaj peev xwm nce qib protein qhia hauv NHP daim siab los ntawm 8-fold piv rau thawj LNPs.
Hauv daim ntawv thib ob, Cov Kws Tshawb Fawb tau siv lawv cov LNPs optimized xa mRNA encoding human factor IX (hFIX), cov ntshav txhaws, rau NHPs. Lawv tau ua tiav thiab kho cov theem ntawm hFIX qhia hauv cov ntshav ntawm NHPs rau ntau tshaj 6 lub hlis, tsis muaj cov tsos mob ntawm toxicity lossis immunogenicity. Nov yog thawj daim ntawv tshaj tawm txog ntev -mRNA kev qhia hauv NHPs siv LNPs, thiab nws qhia txog lub peev xwm ntawm Prisys Biotechnology's LNPs rau kev kho mob hemophilia thiab lwm yam kab mob caj ces.
Los ntawm kev muab cov kev pabcuam CRO ua ntej raws li tsis yog{0}} tib neeg primates, Prisys Biotechnology tau cog lus rau thaj chaw ntawm mRNA kho thiab nqa cov tshuaj tshiab mRNA rau lub khw. Los ntawm kev ua kom zoo LNPs rau kev xa nrog Prisys Biotech's NHPs, cov kws tshawb fawb tau kov yeej qhov teeb meem loj hauv kev txhim kho preclinical ntawm cov tshuaj mRNA thiab ua txoj hauv kev rau kev sim tshuaj. Tam sim no lawv tab tom ua ntau qhov kev tshawb fawb soj ntsuam siv nws cov LNPs rau ntau qhov kev qhia. Prisys Biotechnology tseem koom tes nrog kev kawm thiab kev lag luam cov neeg koom tes los tshawb nrhiav cov ntawv thov tshiab thiab cov hauv kev ntawm NHPs rau mRNA therapeutics.
- Biodegradable Lipids Ua kom nrawm nrawm Lipid Nanoparticles rau Kev xa khoom ntawm RNAi Therapeutics
- Optimizing Lipid Nanoparticles rau kev xa hauv Primates
