This segment focuses on the research, development, and clinical trials of ex vivo lentiviral gene therapies for lysosomal storage disorders. AVROBIO utilizes a proprietary platform to modify a patient's own hematopoietic stem cells (HSCs) with a functional copy of the defective gene. This approach aims to provide a long-term therapeutic benefit by correcting the genetic defect. The segment includes clinical trials for Fabry disease (AVR-RD-01), Gaucher disease (AVR-RD-02), Pompe disease (AVR-RD-03), cystinosis (AVR-RD-04), and Hunter syndrome (AVR-RD-05). The company is also developing AVR-RD-06 for Gaucher disease type 3. The goal is to improve the quality of life for patients suffering from these rare genetic conditions by providing potentially curative treatments. This segment's market positioning is in the rare disease space, with a competitive advantage in its ex vivo gene therapy platform. Future opportunities include expanding the pipeline to other LSDs and securing regulatory approvals for commercialization. This segment is subject to rigorous clinical trial regulations and requires partnerships with patient advocacy groups and clinical research organizations.