A Gaucher Disease Gene Therapy Trial With FLT201

Key Inclusion Criteria:

• 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

Key Exclusion Criteria:

• 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 participantunsuitable for the study

• Positive pregnancy test or lactating

• History of hematopoietic stem cell transplant (HSCT)/bone marrow transplant or anysolid organ transplant.

• History of receiving any gene therapy or cell therapy.

• History of total splenectomy. Note: Additional protocol defined Inclusion andExclusion criteria apply