Friday, September 25Friday, 25 September 2026 - EST (Eastern Time, UTC-05:00

• Provide an overview of the scientific and clinical rationale for targeting tau biology in Alzheimer’s disease, including how tau-lowering approaches may fit within the evolving treatment landscape.
• Describe the rationale for MAPT gene silencing with siRNA as a precision approach designed to reduce production of tau protein, with potential relevance across Alzheimer’s disease and related tauopathies.
• Highlight Arrowhead’s TRiM and CNS-SC platform approaches, including TfR1-mediated systemic delivery to the CNS and translational evidence supporting CNS delivery and target engagement.
• Review the ARO-MAPT-SC development program, including key preclinical and translational findings and the design of the ARO-MAPT-SC-1001 Phase 1/2a study.

• Differentiated tissue-selective delivery enabled by LEAD™ — SanegeneBio’s proprietary LEAD™ (Ligand and Enhancer Assisted Delivery) platform enables targeted siRNA delivery to a broad range of extrahepatic tissues and cell types, expanding the therapeutic potential of RNAi for obesity, cardiometabolic, and autoimmune diseases.
• Preclinical validation of potential best-in-class RNAi therapeutics — Leveraging the LEAD™ platform, SanegeneBio has generated compelling preclinical proof-of-concept across multiple hepatic and extrahepatic targets, demonstrating potent gene silencing, durable efficacy, and differentiated tissue selectivity with potential best-in-class profiles.
• Clinical validation supporting platform differentiation — Across multiple clinical programs, SanegeneBio’s siRNA therapeutics have demonstrated strong safety, efficacy, and durability, providing clinical validation of the LEAD™ platform and reinforcing the potential of RNAi medicines to transform the treatment landscape for obesity, cardiometabolic, and autoimmune diseases.

• PGN-EDODM1 represents an advanced peptide therapeutic engineered with enhanced properties for treating Duchenne muscular dystrophy type 1 (DMD1), incorporating optimized cell-penetrating sequences and improved stability that enable efficient delivery to skeletal and cardiac muscle tissues, with preclinical studies demonstrating superior dystrophin restoration, functional muscle improvement in animal models, favorable pharmacokinetic profiles, and a well-characterized safety profile that supports progression into clinical development where the therapeutic aims to address the underlying pathophysiology of DMD1 and provide meaningful clinical benefit to patients suffering from this progressive and life-threatening neuromuscular disorder.

This presentation explores next-generation siRNA manufacturing strategies that address growing industry demand for scalable, cost-effective production. By integrating enzymatic ligation and thermostable enzyme technologies, we demonstrate pathways to improve efficiency, reduce cost of goods, and enable flexible manufacturing at scale.

• Transition from conventional SPOS to hybrid enzymatic ligation for improved yield, purity, and sustainability

• Design and screening of thermostable ligases to enable high-temperature, high-efficiency ligation

• Impact of elevated temperature on impurity control, fragment selectivity, and ligation performance

• Scalable manufacturing strategies (C-to-C, C-to-P, P-to-P) to optimize cost, robustness, and process efficiency