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

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항목
CGTStandard DatabasestalePhase 3
FIX (Factor IX, Factor IX (FIX))
CSL Behring·Hemophilia B
225 trials
Overview
Program
Factor IX (FIX)
Overview
Company
CSL Behring
Overview
Modality
CGT
Overview
Target
Hemophilia B
Overview
Indication
Hemophilia B
Overview
Phase
PHASE_3
Overview
Status
ACTIVE
Overview
Content status
Standard Database
Overview
Data Confidence
Data Confidence · Medium
Overview
Development Signal
Development Signal · Emerging
Overview
Approval status
Investigational
Technology
Vector
AAV based gene therapy for hemophilia B pertains to dose-dependent vector-related i
MoA
Mechanism
targets the tissue factor pathway
PK/PD
Species
NHP, Mouse
PK/PD
Animal (cat.)
Human, Mouse, NHP
PK/PD
Experiment
Biodistribution
Toxicology
Species
NHP, Mouse
Toxicology
Animal (cat.)
Human, Mouse, NHP
Toxicology
Major finding
Prospective collection of bleeding rate and factor usage in participants with hemophilia A and B in a noninvestigational product study.. We report efficacy and select safety outcomes of current factor prophylaxis of adults with moderately severe to severe hemophilia A (HA) or hemophilia B (HB) in a large, prospective, noninvestigational product study. This study established prospective efficacy da…
Clinical
Safety signal
Prospective collection of bleeding rate and factor usage in participants with hemophilia A and B in a noninvestigational product study.. We report efficacy and select safety outcomes of current factor prophylaxis of adults with moderately severe to severe hemophilia A (HA) or hemophilia B (HB) in a large, prospective, noninvestigational product study. This study established prospective efficacy da…
Clinical
Selected reported efficacy
5.2%
Clinical
Result source
ClinicalTrials.gov NCT02713204
Clinical
Program phase
PHASE_3
Clinical
Trial activity
No active/completed counts
Clinical
Trial ref
NCT02713204