Explore the Agenda
8:00 am Check In & Light Refreshments
8:50 am Chair’s Opening Remarks
Pioneering Complement-Targeted Gene Therapies for Durable Therapeutic Options with Reduced Patient Burden
9:00 am Driving the Development of Gene Therapies for Complement-Mediated Diseases to Deliver Sustained Patient Benefit
- Investigating gene therapy approaches capable of providing long-term complement control
- Navigating delivery, safety, and expression durability challenges to maximize therapeutic impact
- Translating learnings from the development pipeline into successful clinical and commercial outcomes
NEW SPEAKING COMPANY
9:30 am Advancing Gene Therapy Approaches for Complement-Mediated Diseases to Achieve Long-Term Therapeutic Benefit with Reduced Dosing Requirements
- Evaluating the potential of gene therapies to provide sustained complement modulation
- Overcoming delivery, durability and safety challenges to maximize therapeutic benefit across systemic and tissue-specific diseases
- Sharing lessons learned from development pipelines to accelerate the translation of gene therapy approaches into approved treatments
10:00 am Morning Break & Networking
Expanding Complement-Based Approaches Across Emerging Modalities Including siRNA & Next-Generation Biologics to Increase Treatment Options
11:00 am Engineering Next-Generation Biologics to Enhance Selectivity, Durability & Clinical Impact in Complement Therapeutics
- Developing novel biologics, including bispecific antibodies, fusion proteins and engineered complement regulators, to improve therapeutic performance
- Optimizing pharmacokinetics, tissue targeting and mechanism of action to enhance efficacy while minimizing treatment burden
- Leveraging advances in protein engineering to create differentiated therapies across complement-mediated diseases
NEW SPEAKING COMPANY
11:30 am Harnessing siRNA Technologies to Achieve Precise Complement Modulation Across an Increased Range of Indications
- Exploring the advantages of siRNA-based approaches for reducing complement protein production and enabling durable pathway inhibition
- Assessing target selection, dosing strategies and safety considerations to optimize efficacy across complement-mediated diseases
- Demonstrating how next-generation RNA technologies are expanding treatment options and addressing unmet patient needs
12:00 pm Clinical Development Progress of VSA012, an siRNA Molecule Targeting CFB
- VSA012 profile, a highly potent and selective inhibitor of CFB
- Phase 2 data of in PNH in naïve and refractory patients
- Preliminary data in IgAN for VSA012
NEW SPEAKING COMPANY
12:30 pm Deepening Understanding of Complement Biology in Ophthalmology to Enable Targeted Intervention & Improve Patient Outcomes
- Investigating the role of complement dysregulation across ophthalmologic diseases and its contribution to disease onset, progression and vision loss
- Identifying biomarkers that could predict disease development, patient risk and treatment response to support earlier intervention strategies
- Elucidating relevant targets within the complement cascade that offer the greatest therapeutic potential within ocular diseases
1:00 pm Lunch Break & Networking
Validating Novel Cascade Targets & Exploring Intracellular Complement to Navigate Future Innovation
2:00 pm Advanced Assay Strategies for the Validation of Novel Complement Targets
- Detailing target-agnostic assay methodologies for characterizing complement inhibitors in early discovery
- Elucidating strategies to enhance assay sensitivity and enable robust, cross-species evaluation of inhibitor potency and mechanism of action
- Illustrating the broad, disease-agnostic application of these techniques
NEW SPEAKING COMPANY
2:30 pm Harnessing C1q in Ophthalmic Neurodegenerative Diseases
- Elucidating the role of C1q in ophthalmic neurodegenerative diseases
- Integrating preclinical and clinical data to validate C1q’s impact on neurodegeneration
- Leveraging advanced imaging for longitudinal assessment of neurodegenerative progression and therapeutic response
3:00 pm Round Table Discussion: Defining the Role of Intracellular Complement to Reveal Novel Targets for Future Complement Therapeutics
- Evaluating growing evidence for intracellular complement as a key regulator of cellular function in health and disease
- Characterizing how intracellular and extracellular complement pathways interact to shape disease biology and therapeutic responses
- Identifying technological limitations, critical knowledge gaps, and research priorities that will accelerate the development of next-generation complement medicines
NEW SPEAKING ORGANIZATION