| English Name | Research Code | Highest Development Stage | Company | Indication | Clinical Trial |
|---|---|---|---|---|---|
| Human apotransferrin (Sanquin Blood Supply Foundation) | Phase 3 Clinical | Sanquin Plasma Products Bv | Iron Metabolism Disorders; beta-Thalassemia | Details | |
| Delpacibart eteddesiran | AOC-1001; AOC 1001 | Phase 3 Clinical | Avidity Biosciences Inc | Myotonic Disorders; Genetic Diseases, Inborn; Musculoskeletal Diseases; Muscular Disorders, Atrophic; Myotonic Dystrophy; Muscular Atrophy; Heredodegenerative Disorders, Nervous System; Muscular Diseases; Neuromuscular Diseases; Nervous System Diseases; Muscular Dystrophies; Neurodegenerative Diseases; Myotonia | Details |
| CX-2029 | ABBV-2029; ABBV-CX-2029; CX-2029 | Phase 2 Clinical | Abbvie Inc, Cytomx Therapeutics Inc | Solid tumours; Head and Neck Neoplasms; Esophageal Neoplasms; Lymphoma, Large B-Cell, Diffuse; Carcinoma, Non-Small-Cell Lung | Details |
| DYNE-251 | DYNE251 | Phase 2 Clinical | Dyne Therapeutics Inc | Muscular Dystrophy, Duchenne | Details |
| JR-446 | JR-446 | Phase 2 Clinical | Jcr Pharmaceuticals Co Ltd, Medipal Holdings Corp | Mucopolysaccharidosis III | Details |
| JR-441 | JR-441 | Phase 2 Clinical | Jcr Pharmaceuticals Co Ltd | Mucopolysaccharidosis III | Details |
| Lepunafusp alfa | JR-171 | Phase 2 Clinical | Jcr Pharmaceuticals Co Ltd | Mucopolysaccharidosis I | Details |
| PPMX-T003 | PPMX-T003 | Phase 2 Clinical | Perseus Proteomics Inc | Leukemia; Polycythemia Vera | Details |
| Trontinemab | RO-7126209; RG-6102 | Phase 2 Clinical | F. Hoffmann-La Roche Ltd | Alzheimer Disease | Details |
| INA-03 | INA03 | Phase 1 Clinical | Inatherys | Leukemia; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Myeloid, Acute | Details |
| KK-2260 | KK2260; KK-2260 | Phase 1 Clinical | Kyowa Kirin Co Ltd | Solid tumours; Esophageal Neoplasms; Squamous Cell Carcinoma of Head and Neck | Details |
| ABX-1100 | ABX-1100 | Phase 1 Clinical | Aro Biotherapeutics Co | Glycogen Storage Disease Type II | Details |
| English Name | Research Code | Highest Development Stage | Company | First Brand Name | First Approved Country | First Indication | First Approved Company | First Approved Date | Indication | Clinical Trial |
|---|---|---|---|---|---|---|---|---|---|---|
| Pabinafusp alfa | JR-141 | Approved | Jcr Pharmaceuticals Co Ltd | Japan | Mucopolysaccharidosis II | Jcr Pharmaceuticals Co Ltd | 2021-03-23 | Mucopolysaccharidosis II | Details |
