654개 · ADC 882 · Antibody 4071 · CGT 2714 · 자동 수집 갱신 권장 · 9월 2일
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ALL, DLBCL, FL · CD19
Unresectable or metastatic melanoma after PD-1 blockade · Polyclonal tumor neoantigens (TIL)
Spinal muscular atrophy (SMA) Type 1–3, pediatric · SMN1 (AAV9)
High-risk BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) · IFNα2b (adenoviral)
Sickle cell disease with recurrent VOCs; transfusion-dependent β-thalassemia · BCL11A enhancer (CRISPR/Cas9) → HbF
Severe hemophilia A in adults · Factor VIII (AAV5)
Hemophilia B (congenital Factor IX deficiency) in adults · Factor IX (AAV5 Padua)
Inherited retinal dystrophy due to confirmed biallelic RPE65 mutations · RPE65 (AAV2)
Sickle cell disease with recurrent vaso-occlusive events · βA-T87Q-globin (lentiviral)
Relapsed Non Hodgkin Lymphoma; Relapsed Adult ALL · CD19
Myeloma-Multiple; Myeloma, Plasma-Cell · BCMA
Carcinoid; Neuroendocrine · Carcinoid
B Cell Malignancies · CD19
Malignant Glioma; Glioblastoma · EGFR
Leber's Hereditary Optic Neuropathy · Leber's Hereditary Optic Neuropathy
Recurrent Glioblastoma; Recurrent Malignant Glioma · CD19
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