ADC 1032 · Antibody 4683 · CGT 2713 · 다음 갱신 D-8 · 마지막 7월 21일
Multiple myeloma · BCMA
LBCL, FL · CD19
Hyperlipidemia, ASCVD, HeFH (LDL-C lowering) · PCSK9 (siRNA)
ALL, DLBCL, FL · CD19
Hyperlipidemia, ASCVD, HeFH · PCSK9
Breast Cancer; Metastatic Breast Cancer · Lung Cancer
Spinal muscular atrophy (SMA) Type 1–3, pediatric · SMN1 (AAV9)
HER2 Mutant Non-small Cell Lung Cancer; HER2-positive Metastatic Breast Cancer · HER2
Duchenne muscular dystrophy (DMD) — ambulatory / label-expanded populations · Micro-dystrophin (AAVrh74)
Advanced Solid Tumors Cancer · Non Small Cell Lung Cancer
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)
Advanced or Metastatic Solid Malignancies · Advanced or Metastatic Solid Malignancie
Diffuse Large B-Cell Lymphoma (DLBCL) · Diffuse Large B-Cell Lymphoma (DLBCL)
FDA-approved indication · See label
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구조모아 (StructureMoa)항암 chemical structure spider web · 2D 구조식 맵핑
갱신 기록 없음 · 14일 주기 · 방문 143 / 4,777