Lentiviral Vector Gene Therapy - The GuardOne Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease
Status:
Recruiting
Trial end date:
2023-12-01
Target enrollment:
Participant gender:
Summary
This is a multinational, open-label study to assess the safety and efficacy of AVR-RD-02 in
approximately 8 to 16 subjects (male or female) who are ≥18 and ≤45 years of age and
postpubertal at Screening with a confirmed diagnosis of Type 1 Gaucher disease (based on
clinical phenotype, genotyping, and deficient GCase enzyme activity in whole blood).