医药行业创新药平台技术系列报告：CAR~T快速发展为治愈带来希望-230201（21页）.pdf

1、SS0880521070001 101020304 kUlYuWpZlV9Y8X8VpWcV7NdN9PoMoOmOmPiNrRnPlOoOnO7NpPyRxNnOpRwMqRoM201 3RNA TherapyGene TherapyGene EditingCell Therapy(non-genetically modified)RNA/working gene/DNADNA()mRNA小核酸 ASO RNAi基因疗法细胞治疗(经过基因改造)/CAR-T CAR-NK TCR-T(TIL NKZFNTALEN CRISPR基因编辑细胞治疗(未经基因改造)广义的“基因治疗”+01 4CAR-

2、TT(CAR-T)TCRT(TCR-T)(TIL)(NK)CAR-TNature ReviewsCAR-T2020859CART2019290TCRTMHC-TTPubmed01 5CAR-T/1.T2.TCAR3.T CRSMHCCAR-T/CAR-TCAR-NK/NK1.NK2.NK3.CAR-NKPubmed01 6TIL/1.IL-22.2-33.OKT3IL-24.5-610111.2.TTTTCR3.TCRTTCRT4.TCR-T/MHCMHCPubmed01 7CARCAR-TTCARScFvTCARKymriahYescartaTecartusBreyanziAbecmaCAR

3、VYKTICD19CD19CD19CD19CD19BCMABCMA/Kite/KiteBMSBMS/scFvmurine murine murine FMC63 murine VHH4-1BB(CD137)CD28CD284-1BB4-1BB4-1BB4-1BBTCD3 zetaCD3 zetaCD3 zetaCD3 zetaCD3 zetaCD3 zetaCD3 zeta$47.5$37.3$37.3$41.0129$41.95$46.52017.08(FDA)2017.10(FDA)2020.07(FDA)2021.02(FDA)2021.09(CN)2021.03(FDA)2022.2.

4、28(FDA)Pubmed01 802 9CAR-T2CAR-T1CAR-TTCAR-T2(On-Target Off-Tumor)CAR-TCAR-T3(CRS)CAR-T100100000CAR-T/1CAR-TCAR-T3CAR-T1CAR-TCAR-T1.5CAR-T0.4CAR-T2CAR-TCAR-TKymriahYescarta201747.537.3Kymriah1000Yescarta15004Pubmed02 10CAR-T212(On-Target Off-Tumor)3(CRS)1CAR-T341CAR-T2CAR-T-CAR-TCAR-CARTCARscFvCAR-2

5、019298.5253.4225.7168.088.782.9NHL79.5 50%DLBCL68.262.356.736.527.9CAR-TCAR-TCAR-TPubmed02 11outpatientCAR-T=+Kymriah47.5Yescarta37.345Break-evenKymriah44%CRS4045curetodayhealio02 12CAR-T2.20.060.070.762.642.784.564.745.630.445.876.951.760.871.643.978.232.171.272.630.79KymriahYescartaTecartusBreyanz

6、iAbecmaCarvyktiCAR-T201720182019202020212022Q1-3FDA2017201820192020202122H12022Q1-32017.08NovartisKymriah676278474587263397yoy1167%266%71%24%-11%2017.10GileadYescarta7264456563695506823yoy3671%73%23%23%160%2020.07GileadTecartus-44176136217yoy300%82%2021.02BMSB