구조모아 (StructureMoa)항암 chemical structure spider web
방문타깃 · 모달리티 · 임상 근거 · 비임상 맥락으로 최대 5개 프로그램을 나란히 비교합니다. · 다음 갱신 D-6 · 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 | PEG-ADA ERT |
Overview Company | Orchard Therapeutics |
Overview Modality | CGT |
Overview Target | ADA-SCID |
Overview Indication | Severe Combined Immunodeficiency Due to ADA Deficiency |
Overview Phase | PHASE_2 |
Overview Status | ACTIVE |
Overview Content status | Standard Database |
Overview Data Confidence | Data Confidence · Medium |
Overview Development Signal | Development Signal · Emerging |
Overview Approval status | Investigational |
Toxicology Major finding | Metabolite and thymocyte development defects in ADA-SCID mice receiving enzyme replacement therapy.. Deficiency of adenosine deaminase (ADA, EC3.5.4.4), a housekeeping enzyme intrinsic to the purine salvage pathway, leads to severe combined immunodeficiency (SCID) both in humans and mice. Lack of ADA results in the intracellular accumulation of toxic metabolites which have effects on T cell develo… |
Clinical Safety signal | Metabolite and thymocyte development defects in ADA-SCID mice receiving enzyme replacement therapy.. Deficiency of adenosine deaminase (ADA, EC3.5.4.4), a housekeeping enzyme intrinsic to the purine salvage pathway, leads to severe combined immunodeficiency (SCID) both in humans and mice. Lack of ADA results in the intracellular accumulation of toxic metabolites which have effects on T cell develo… |
Clinical Program phase | PHASE_2 |
타깃 · 모달리티 · 임상 근거 · 비임상 맥락으로 최대 5개 프로그램을 나란히 비교합니다. · 다음 갱신 D-6 · 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 | PEG-ADA ERT |
Overview Company | Orchard Therapeutics |
Overview Modality | CGT |
Overview Target | ADA-SCID |
Overview Indication | Severe Combined Immunodeficiency Due to ADA Deficiency |
Overview Phase | PHASE_2 |
Overview Status | ACTIVE |
Overview Content status | Standard Database |
Overview Data Confidence | Data Confidence · Medium |
Overview Development Signal | Development Signal · Emerging |
Overview Approval status | Investigational |
Toxicology Major finding | Metabolite and thymocyte development defects in ADA-SCID mice receiving enzyme replacement therapy.. Deficiency of adenosine deaminase (ADA, EC3.5.4.4), a housekeeping enzyme intrinsic to the purine salvage pathway, leads to severe combined immunodeficiency (SCID) both in humans and mice. Lack of ADA results in the intracellular accumulation of toxic metabolites which have effects on T cell develo… |
Clinical Safety signal | Metabolite and thymocyte development defects in ADA-SCID mice receiving enzyme replacement therapy.. Deficiency of adenosine deaminase (ADA, EC3.5.4.4), a housekeeping enzyme intrinsic to the purine salvage pathway, leads to severe combined immunodeficiency (SCID) both in humans and mice. Lack of ADA results in the intracellular accumulation of toxic metabolites which have effects on T cell develo… |
Clinical Program phase | PHASE_2 |
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