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항목
CGTStandard DatabasestalePhase 3
Adjuvant IM Therapy (Adjuvant IM Therapy)
Ultragenyx Pharmaceutical Inc·MPS IIIA
31 trials
Overview
Program
Adjuvant IM Therapy
Overview
Company
Ultragenyx Pharmaceutical Inc
Overview
Modality
CGT
Overview
Target
MPS IIIA
Overview
Indication
MPS IIIA; Sanfilippo Syndrome
Overview
Phase
PHASE_3
Overview
Status
RECRUITING
Overview
Content status
Standard Database
Overview
Data Confidence
Data Confidence · Medium
Overview
Development Signal
Development Signal · Emerging
Overview
Approval status
Investigational
MoA
Mechanism
target Mendelian randomization (MR), mediation MR, transcriptome-based machine learning, and single-cell RNA-seq were integrated to prioritize targets and pathway
MoA
Biomarker
HER2-Positive
PK/PD
Species
Mouse
PK/PD
Animal (cat.)
Mouse, In vitro
PK/PD
Experiment
PK
Toxicology
Species
Mouse
Toxicology
Animal (cat.)
Mouse, In vitro
Toxicology
Major finding
Chemo-Immunotherapy, a Combination Approach for the Treatment of HER2-Positive Breast Cancer in a Mouse Model.. The discovery of tumor-associated antigens has been a significant milestone for cancer therapy as the current conventional therapies (radiotherapy, chemotherapy) were found to be insufficient in terms of cancer treatment. Human epidermal growth factor receptor (HER2) is a tumor-associate…
Toxicology
CRS
Reported
Clinical
Safety signal
Chemo-Immunotherapy, a Combination Approach for the Treatment of HER2-Positive Breast Cancer in a Mouse Model.. The discovery of tumor-associated antigens has been a significant milestone for cancer therapy as the current conventional therapies (radiotherapy, chemotherapy) were found to be insufficient in terms of cancer treatment. Human epidermal growth factor receptor (HER2) is a tumor-associate…
Clinical
Selected reported efficacy
3%
Clinical
Result source
ClinicalTrials.gov NCT01147068
Clinical
Program phase
PHASE_3
Clinical
Trial ref
NCT01147068
Preclinical
Animal (cat.)
Mouse, In vitro