A Gaucher Disease Gene Therapy Trial With FLT201

Open to people ages 18 years and up

• Aged ≥18 years at time of screening.
• Clinical diagnosis of Gaucher disease type 1
• Stable hemoglobin concentration at baseline
• Stable platelet count at baseline
• Receiving ERT or SRT without interruption for at least 2 years

• Diagnosed or suspected Gaucher disease type 2 or type 3
• Positive for AAVS3 neutralizing antibodies.
• Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study
• Positive pregnancy test or lactating
• History of hematopoietic stem cell transplant (HSCT)/bone marrow transplant or any solid organ transplant.
• History of receiving any gene therapy or cell therapy.

• History of total splenectomy. Note: Additional protocol defined Inclusion and

  Exclusion criteria apply