Exclusive Summary

• Validated Target with Novel Delivery: Targets Glucosylceramide synthase (GCS) using the proprietary FsiRNA-6.0 platform, enabling Substrate Reduction Therapy (SRT) for Gaucher and Fabry diseases without the need for complex delivery vehicles.
• Systemic Reach Beyond the Liver: Unlike traditional GalNAc-siRNA limited to hepatocytes, UB091 utilizes FsiRNA's self-delivery mechanism to achieve pan-expressive knockdown, effectively addressing the multi-organ systemic nature of these lysosomal storage disorders.
• Superior Compliance Potential: Offers a significant upgrade over daily oral pills or frequent enzyme replacement infusions (ERT) by leveraging the long-lasting gene silencing effects of siRNA, promising potentially infrequent subcutaneous dosing and high orphan drug market value.

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