Vironexis Biotherapeutics Inc.
This is a Phase 1/2, first-in-human, open-label, dose-escalating trial designed to assess the safety and efficacy of VNX-101 in patients with relapsed or refractory CD19-positive hematologic malignancies.
VNX-101 is an investigational adeno-associated virus (AAV) gene therapy developed to express a secreted anti-CD19/anti-CD3 scFv diabody (termed GP101). GP101 binds both cluster of differentiation (CD)19 and CD3, inducing T-cells to kill both benign and malignant B-cells. Following a single intravenous (IV) infusion, the vector induces the liver and key tissues to continuously secrete GP101 into the bloodstream, resulting in long-term, consistent serum levels of GP101. Potential advantages of VNX-101 over autologous CAR-T therapy include it is off-the-shelf, provides a gentle onset of action, d…
Inclusion Criteria: * Age: Part 1: 18-90 years of age, Part 2: 13-90 years of age * Relapsed or refractory CD-19 positive leukemia or lymphoma as defined in the protocol * CD19-positive expression * AAV specified capsid total antibody \<1:400 * Protocol-specified ranges for renal, liver, cardiac and pulmonary function * Protocol-specified ranges for hematology parameters Exclusion Criteria: * Hepatoxicity (AST or ALT \> 2x upper limit of normal) * History of thrombotic microangiopathy or cardiomyopathy, or evidence of sensory neuropathy * Pregnant or nursing (lactating) women * Acute Graft…
Adeno-associated viral vector encoding the CD3/CD19 Bi-Specific T-Cell Engager (AAV.CD3/CD19), Single IV infusion
Adeno-associated viral vector encoding the CD3/CD19 Bi-Specific T-Cell Engager (AAV.CD3/CD19), Single IV infusion
Adeno-associated viral vector encoding the CD3/CD19 Bi-Specific T-Cell Engager (AAV.CD3/CD19), Single IV infusion
Adeno-associated viral vector encoding the CD3/CD19 Bi-Specific T-Cell Engager (AAV.CD3/CD19), Single IV infusion