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CGT

10개 · ADC 1068 · Antibody 5456 · CGT 2744 · 다음 갱신 D-14 · 마지막 7월 13일

인기최근 업데이트큐레이션임상 많은갱신 필요ORR최신Phase 3FDA승인프로그램 10개
CGTSHOTFDACuratedFDABiologicKitevedotin plus bendamustine a…Approved

axicabtagene ciloleucel (Yescarta, axi-cel)

LBCL, FL · CD19

#CD19#CGT#Approved#FDA승인#바이오모아추천+5
효능 ORR 92%임상시험 66상태 APPROVED
CGTSHOTFDACuratedFDABiologicNovartisApproved

tisagenlecleucel (Kymriah, tisa-cel)

ALL, DLBCL, FL · CD19

#CD19#CGT#Approved#FDA승인#바이오모아추천+5
효능 ORR 82.3%임상시험 48상태 APPROVED
CGTAAVAHOTFDACuratedFDABiologicNovartisSMN1 cDNA under hybrid CMV …Approved

onasemnogene abeparvovec (Zolgensma, AVXS-101, onasemnogene abeparvovec-xioi)

Spinal muscular atrophy (SMA) Type 1–3, pediatric · SMN1 (AAV9)

#SMN1AAV9#CGT#Approved#FDA승인#바이오모아추천+5
임상시험 26상태 APPROVED
CGTAAVBHOTFDACuratedFDABiologicSarepta TherapeuticsMicro-dystrophin cassetteApproved

delandistrogene moxeparvovec (Elevidys, SRP-9001, delandistrogene moxeparvovec-rokl)

Duchenne muscular dystrophy (DMD) — ambulatory / label-expanded populations · Micro-dystrophin (AAVrh74)

#Micro-dystrophinAAVrh74#CGT#Approved#FDA승인#바이오모아추천+5
임상시험 12상태 APPROVED
CGTSHOTFDACuratedFDABiologicFerring PharmaceuticalsHuman interferon alfa-2bApproved

nadofaragene firadenovec (Adstiladrin, rAd-IFNα/Syn3, nadofaragene firadenovec-vncg)

High-risk BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) · IFNα2b (adenoviral)

#IFN2badenoviral#CGT#Approved#FDA승인#바이오모아추천+5
효능 ORR 55%임상시험 10상태 APPROVED
CGTBHOTFDACuratedFDABiologicVertexApproved

exagamglogene autotemcel (Casgevy, exa-cel, CTX001)

Sickle cell disease with recurrent VOCs; transfusion-dependent β-thalassemia · BCL11A enhancer (CRISPR/Cas9) → HbF

#BCL11AenhancerCRISPRCas9HbF#CGT#Approved#FDA승인#바이오모아추천+5
임상시험 9상태 APPROVED
CGTAAVBHOTFDACuratedFDABiologicBioMarinB-domain deleted FVIIIApproved

valoctocogene roxaparvovec (Roctavian, BMN 270, valoctocogene roxaparvovec-rvox)

Severe hemophilia A in adults · Factor VIII (AAV5)

#FactorVIIIAAV5#CGT#Approved#FDA승인#바이오모아추천+5
임상시험 8상태 APPROVED
CGTAAVBHOTFDACuratedFDABiologicCSL BehringFactor IX Padua variant (hy…Approved

etranacogene dezaparvovec (Hemgenix, AMT-061, CSL222, etranacogene dezaparvovec-drlb)

Hemophilia B (congenital Factor IX deficiency) in adults · Factor IX (AAV5 Padua)

#FactorIXAAV5Padua#CGT#Approved#FDA승인#바이오모아추천+5
임상시험 7상태 APPROVED
CGTAAVBHOTFDACuratedFDABiologicSpark TherapeuticsHuman RPE65 cDNAApproved

voretigene neparvovec (Luxturna, AAV2-hRPE65v2, voretigene neparvovec-rzyl)

Inherited retinal dystrophy due to confirmed biallelic RPE65 mutations · RPE65 (AAV2)

#RPE65AAV2#CGT#Approved#FDA승인#바이오모아추천+5
임상시험 7상태 APPROVED
CGTCHOTFDACuratedFDABiologicbluebird bioβA-T87Q-globinApproved

lovotibeglogene autotemcel (Lyfgenia, lovo-cel, bb1111)

Sickle cell disease with recurrent vaso-occlusive events · βA-T87Q-globin (lentiviral)

#A-T87Q-globinlentiviral#CGT#Approved#FDA승인#바이오모아추천+5
임상시험 2상태 APPROVED