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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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API CSV14행 · 1개 프로그램
OverviewToxicologyClinical

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항목
CGTStandard DatabasestalePhase 2
adeno-associated virus vector AAV- CNGA3 (adeno-associated virus vector AAV- CNGA3)
MeiraGTx UK II Ltd·Achromatopsia
3 trials
Overview
Program
adeno-associated virus vector AAV- CNGA3
Overview
Company
MeiraGTx UK II Ltd
Overview
Modality
CGT
Overview
Target
Achromatopsia
Overview
Indication
Achromatopsia
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
Safety and Toxicology of Ocular Gene Therapy with Recombinant AAV Vector rAAV.hCNGA3 in Nonhuman Primates.. The purpose of this study was to examine the toxicity and side effects of a recombinant adeno-associated virus 8 (AAV8) vector, aimed to treat cyclic nucleotide gated channel alpha 3 ( CNGA3 )-linked achromatopsia, after a single subretinal administration in cynomolgus macaques. Animals were…
Clinical
Safety signal
Safety and Toxicology of Ocular Gene Therapy with Recombinant AAV Vector rAAV.hCNGA3 in Nonhuman Primates.. The purpose of this study was to examine the toxicity and side effects of a recombinant adeno-associated virus 8 (AAV8) vector, aimed to treat cyclic nucleotide gated channel alpha 3 ( CNGA3 )-linked achromatopsia, after a single subretinal administration in cynomolgus macaques. Animals were…
Clinical
Program phase
PHASE_2