830개 · ADC 882 · Antibody 4071 · CGT 2714 · 자동 수집 갱신 권장 · 9월 2일
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LBCL, FL · CD19
ALL, DLBCL, FL · CD19
Unresectable or metastatic melanoma after PD-1 blockade · Polyclonal tumor neoantigens (TIL)
Relapsed or refractory multiple myeloma · BCMA
Spinal muscular atrophy (SMA) Type 1–3, pediatric · SMN1 (AAV9)
Duchenne muscular dystrophy (DMD) — ambulatory / label-expanded populations · Micro-dystrophin (AAVrh74)
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)
Multiple Myeloma; Recurrent Multiple Myeloma · BCMA
Myeloma-Multiple; Myeloma, Plasma-Cell · BCMA
Carcinoid; Neuroendocrine · Carcinoid
Solid Tumors · B7-H3
B Cell Malignancies · CD19
Lymphoma, Non-Hodgkin; Diffuse Large B Cell Lymphoma · CD19
Malignant Glioma; Glioblastoma · EGFR
Leber's Hereditary Optic Neuropathy · Leber's Hereditary Optic Neuropathy
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