By Fineline Cube 
Sichuan R&B Biotech, a China-based biotech firm, announced receiving clinical approval from the US Food and Drug Administration (FDA) to initiate a global, multi-center Phase I/II study for its gene therapy ZS805. This marks a significant milestone in the development of novel treatments for Fabry disease.
Fabry Disease and ZS805 Gene Therapy
Fabry disease is a rare X-linked inherited lysosomal storage disorder caused by mutations in the α-Gal A gene. These mutations lead to structural and functional abnormalities in the enzyme, resulting in the accumulation of sphingolipids (Gb3) in the body. ZS805, a recombinant adeno-associated virus (rAAV)-based gene therapy, is designed with the world’s smallest liver-specific promoter and an optimized alpha Gal A gene with a signal peptide. This enables specific expression and efficient secretion of the alpha Gal A protein in liver cells, effectively reducing the concentration of the key biomarker Lyso-GL3 in serum.
Advantages Over Current Treatments
Compared to enzyme replacement therapy currently used in clinical practice, ZS805 offers a one-time treatment with long-term or even lifelong efficacy. This avoids the need for frequent medication, providing a potentially transformative treatment option for patients with Fabry disease.-Fineline Info & Tech