ArticlePharmaceutical research2025
Preclinical Evaluation of AAV8-R338L Gene Therapy for Hemophilia: Efficacy, Pharmacokinetics, Distribution, Excretion and Toxicity in Mouse Models and Non-human Primates.
Article in Pharmaceutical research, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 2 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
2 citing papers in PubMed.
- Decoding Nonlinearities in AAV-Based Gene Therapy Using PBPK Modelling.The AAPS journal · 2026Article
- CRISPLD2 protects against liver inflammation and fibrosis via GRP78 to repress HMGB1/TLR4 axis-mediated STING palmitoylation.Hepatology communications · 2026Article
Corrections and comments
- Erratum issued
Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundVGB-R04, an investigational gene therapy employs an engineered AAV8 capsid-based vector to deliver a liver-specific expression cassette encoding the high-activity FIX Padua variant (R338L) of human coagulation factor IX for Hemophilia B. The pharmacodynamics, Pharmacokinetic, immunogenicity and safety of VGB-R04 needs to be evaluated for non-clinical research.
methodsThe pharmacodynamics, Pharmacokinetic, immunogenicity and safety study of VGB-R04 via intravenous injection in mice and cynomolgus monkeys were conducted to support an investigational new drug (IND) application. The endpoints included pharmacodynamic biomarkers, in-life measurements. Anti-AAV8 neutralizing antibodies and anti-hFIX Padua protein antibody test, viral shedding, and tissue bio-distribution were analyzed.
resultsThe peak concentration was noted one hour after injection, subsequently exhibiting a distinct decline over time. The elimination rate of target genes in mouse blood exceeded that in cynomolgus monkeys. The concentration in liver tissues was indicating distinct liver tissue tropism. The elimination rate of target genes in mouse liver exceeded that in cynomolgus monkeys. The plasma concentration of hFIX Padua protein exhibited a dose-dependent elevation in mice. Cynomolgus monkeys exhibited significant elevation in plasma concentrations of hFIX Padua protein at 4 × 10
conclusionThe studies demonstrated the pharmacodynamic advantages, pharmacokinetic profile with target distribution and potential safety of VGB-R04 in mice and cynomolgus monkeys after a single administration. The results support the gene therapy for future clinical studies.
Indexed as
Identifiers
41073832What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.