An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease

Exclusion Criteria13

• Diagnosis of a significant CNS disease other than GD2 that may be a cause for the patient's GD symptoms or may confound study objectives.
• Achieved independent gait.
• Severe visceral symptoms of GD which, in the opinion of the Investigator, would pose an unacceptable risk to the patient or interfere with the patient's ability to comply with study procedures or interfere with the conduct of the study.
• Clinically active infection (including HIV, HBV, HCV or syphilis).
• For those receiving enzyme replacement therapy and/or substrate reduction therapy and/or ambroxol for Gaucher disease, stable treatment ≤2 months before enrollment.
• Use of strong inhibitors or inducers of cytochrome CYP3A4 or P-glycoprotein (P-gp) medications, herbals, or over-the-counter agents.
• Any type of prior gene or cell therapy.
• Immunizations (live vaccines) in the prior 4 weeks.
• Use of systemic immunosuppressant or corticosteroid therapy other than protocol-specified (topical preparations for dermatological conditions are allowed).
• Patients with anti-AAV9 neutralizing antibody titer over 1:5.
• Brain MRI (magnetic resonance imaging) showing clinically significant abnormality considered to prevent intracisternal injection.
• Contraindication to sedation during surgery or imaging studies (PET).
• Presence of other significant medical conditions that would create an unacceptable risk to the patient or interfere with the patient's ability to comply with study procedures or interfere with the conduct of the study.