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 | rAAV2-CBSB-hRPE65 |
Overview Company | University of Pennsylvania |
Overview Modality | CGT |
Overview Target | Amaurosis of Leber |
Overview Indication | Amaurosis of Leber; Retinal Diseases |
Overview Phase | PHASE_1 |
Overview Status | RECRUITING |
Overview Content status | Limited Data |
Overview Data Confidence | Data Confidence · Low |
Overview Development Signal | Development Signal · Watch |
Overview Approval status | Investigational |
Toxicology Major finding | Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial.. Leber congenital amaurosis (LCA) is a group of autosomal recessive blinding retinal diseases that are incurable. One molecular form is caused by mutations in the RPE65 (retinal pigment epithelium-specific 65-kDa) gene. A recomb… |
Clinical Safety signal | Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial.. Leber congenital amaurosis (LCA) is a group of autosomal recessive blinding retinal diseases that are incurable. One molecular form is caused by mutations in the RPE65 (retinal pigment epithelium-specific 65-kDa) gene. A recomb… |
Clinical Program phase | PHASE_1 |
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 | rAAV2-CBSB-hRPE65 |
Overview Company | University of Pennsylvania |
Overview Modality | CGT |
Overview Target | Amaurosis of Leber |
Overview Indication | Amaurosis of Leber; Retinal Diseases |
Overview Phase | PHASE_1 |
Overview Status | RECRUITING |
Overview Content status | Limited Data |
Overview Data Confidence | Data Confidence · Low |
Overview Development Signal | Development Signal · Watch |
Overview Approval status | Investigational |
Toxicology Major finding | Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial.. Leber congenital amaurosis (LCA) is a group of autosomal recessive blinding retinal diseases that are incurable. One molecular form is caused by mutations in the RPE65 (retinal pigment epithelium-specific 65-kDa) gene. A recomb… |
Clinical Safety signal | Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial.. Leber congenital amaurosis (LCA) is a group of autosomal recessive blinding retinal diseases that are incurable. One molecular form is caused by mutations in the RPE65 (retinal pigment epithelium-specific 65-kDa) gene. A recomb… |
Clinical Program phase | PHASE_1 |
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