구조모아 (StructureMoa)항암 chemical structure spider web
방문타깃 · 모달리티 · 임상 근거 · 비임상 맥락으로 최대 5개 프로그램을 나란히 비교합니다. · 다음 갱신 D-5 · 마지막 9월 2일
현재 선택: 1개 · 임상 갱신 필요 1개
프로그램 상세에서 관심 등록 후 2개 이상 모으면 여기서 한 번에 비교할 수 있습니다.
표가 넓으면 좌우로 스크롤하세요. 핵심 비교 모드에서는 중요 항목만 표시됩니다.
| 항목 | |
|---|---|
Overview Program | AAV9-GLB1 |
Overview Company | National Human Genome Research Institute (NHGRI) |
Overview Modality | CGT |
Overview Target | Lysosomal Diseases |
Overview Indication | Lysosomal Diseases; Gangliosidosis |
Overview Phase | PHASE_2 |
Overview Status | RECRUITING |
Overview Content status | Standard Database |
Overview Data Confidence | Data Confidence · Medium |
Overview Development Signal | Development Signal · Emerging |
Overview Approval status | Investigational |
Toxicology Major finding | Human GLB1 knockout cerebral organoids: A model system for testing AAV9-mediated GLB1 gene therapy for reducing GM1 ganglioside storage in GM1 gangliosidosis.. GM1 gangliosidosis is an autosomal recessive neurodegenerative disorder caused by the deficiency of lysosomal β-galactosidase (β-gal) and resulting in accumulation of GM1 ganglioside. The disease spectrum ranges from infantile t… |
Clinical Safety signal | Human GLB1 knockout cerebral organoids: A model system for testing AAV9-mediated GLB1 gene therapy for reducing GM1 ganglioside storage in GM1 gangliosidosis.. GM1 gangliosidosis is an autosomal recessive neurodegenerative disorder caused by the deficiency of lysosomal β-galactosidase (β-gal) and resulting in accumulation of GM1 ganglioside. The disease spectrum ranges from infantile t… |
Clinical Program phase | PHASE_2 |
타깃 · 모달리티 · 임상 근거 · 비임상 맥락으로 최대 5개 프로그램을 나란히 비교합니다. · 다음 갱신 D-5 · 마지막 9월 2일
현재 선택: 1개 · 임상 갱신 필요 1개
프로그램 상세에서 관심 등록 후 2개 이상 모으면 여기서 한 번에 비교할 수 있습니다.
표가 넓으면 좌우로 스크롤하세요. 핵심 비교 모드에서는 중요 항목만 표시됩니다.
| 항목 | |
|---|---|
Overview Program | AAV9-GLB1 |
Overview Company | National Human Genome Research Institute (NHGRI) |
Overview Modality | CGT |
Overview Target | Lysosomal Diseases |
Overview Indication | Lysosomal Diseases; Gangliosidosis |
Overview Phase | PHASE_2 |
Overview Status | RECRUITING |
Overview Content status | Standard Database |
Overview Data Confidence | Data Confidence · Medium |
Overview Development Signal | Development Signal · Emerging |
Overview Approval status | Investigational |
Toxicology Major finding | Human GLB1 knockout cerebral organoids: A model system for testing AAV9-mediated GLB1 gene therapy for reducing GM1 ganglioside storage in GM1 gangliosidosis.. GM1 gangliosidosis is an autosomal recessive neurodegenerative disorder caused by the deficiency of lysosomal β-galactosidase (β-gal) and resulting in accumulation of GM1 ganglioside. The disease spectrum ranges from infantile t… |
Clinical Safety signal | Human GLB1 knockout cerebral organoids: A model system for testing AAV9-mediated GLB1 gene therapy for reducing GM1 ganglioside storage in GM1 gangliosidosis.. GM1 gangliosidosis is an autosomal recessive neurodegenerative disorder caused by the deficiency of lysosomal β-galactosidase (β-gal) and resulting in accumulation of GM1 ganglioside. The disease spectrum ranges from infantile t… |
Clinical Program phase | PHASE_2 |
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