VGB-R04 / Shanghai Vitalgen BioPharma - LARVOL DELTA

Preclinical Evaluation of AAV8-R338L Gene Therapy for Hemophilia: Efficacy, Pharmacokinetics, Distribution, Excretion and Toxicity in Mouse Models and Non-human Primates. (PubMed, Pharm Res) - "The 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."

Journal • PK/PD data • Preclinical • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Pharmacokinetics, Biodistribution, Immunogenicity, and Model-Informed-Based PK/PD Model of a Next-Generation Advanced Novel Gene Therapy for Hemophilia. (PubMed, Mol Pharm) - "This study aims to develop and validate a population pharmacokinetic and pharmacodynamics model (PK/PD) of VGB-R04 gene therapy for prediction of clinical dose...The model's adequacy and robustness were assessed by VPC and bootstrap methods. Utilizing these data, we formulated a semimechanistic pharmacokinetic/pharmacodynamic quantitative model at a dosage of 4 × 1012 vg/kg to enhance clinical translation, optimize clinical decision-making, and inform personalized therapy, despite the absence of suitable quantitative published data for developing pharmacokinetic models in gene therapy."

Journal • PK/PD data • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Safety and Tolerability of VGB-R04 in Patients With Haemophilia B (clinicaltrials.gov) - P1 | N=3 | Recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital | Trial completion date: Mar 2023 ➔ Dec 2025 | Trial primary completion date: Mar 2023 ➔ Mar 2025

Trial completion date • Trial primary completion date • Hematological Disorders • Hemophilia • Rare Diseases

Safety and Tolerability of VGB-R04 in Patients With Haemophilia B (clinicaltrials.gov) - P1 | N=3 | Recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Disorders • Hemophilia • Rare Diseases

Lead-in Study of VGB-R04 Gene Therapy for Hemophilia B-- An Observational Survey Analysis Study (clinicaltrials.gov) - P=N/A | N=20 | Not yet recruiting | Sponsor: Shanghai Vitalgen BioPharma Co., Ltd.

New trial • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

A Study to Evaluate the Safety and Efficacy of VGB-R04 in Adult Hemophilia B Patients (clinicaltrials.gov) - P1/2 | N=26 | Not yet recruiting | Sponsor: Shanghai Vitalgen BioPharma Co., Ltd.

New P1/2 trial • Hematological Disorders • Hemophilia • Rare Diseases