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프로그램 비교

타깃 · 모달리티 · 임상 근거 · 비임상 맥락으로 최대 5개 프로그램을 나란히 비교합니다. · 다음 갱신 D-6 · 마지막 9월 2일

현재 선택: 1 · 임상 갱신 필요 1

프로그램 상세에서 관심 등록 후 2개 이상 모으면 여기서 한 번에 비교할 수 있습니다.

API CSV14행 · 1개 프로그램

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항목
CGTStandard DatabasestalePhase 2
PEG-ADA ERT (PEG-ADA ERT)
Orchard Therapeutics·ADA-SCID
7 trials
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