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

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프로그램 선택

API CSV14행 · 1개 프로그램
OverviewToxicologyClinical

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항목
CGTStandard DatabasestalePhase 2
rAAV1-CB-hAAT (rAAV1-CB-hAAT)
Beacon Therapeutics·Alpha 1-Antitrypsin Deficiency
2 trials
Overview
Program
rAAV1-CB-hAAT
Overview
Company
Beacon Therapeutics
Overview
Modality
CGT
Overview
Target
Alpha 1-Antitrypsin Deficiency
Overview
Indication
Alpha-1 Antitrypsin 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
Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results.. Recombinant adeno-associated virus (rAAV) vectors offer promise for the gene therapy of α(1)-antitrypsin (AAT) deficiency. In our prior trial, an rAAV vector expressing human AAT (rAAV1-CB-hAAT) provided sustained, vector-derived AAT expression for >1 year. In the curre…
Clinical
Safety signal
Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results.. Recombinant adeno-associated virus (rAAV) vectors offer promise for the gene therapy of α(1)-antitrypsin (AAT) deficiency. In our prior trial, an rAAV vector expressing human AAT (rAAV1-CB-hAAT) provided sustained, vector-derived AAT expression for >1 year. In the curre…
Clinical
Program phase
PHASE_2