Conference Day Two |
Wednesday, December 3, 2025
8:00 am Registration & Coffee
8:50 am Chair’s Opening Remarks
Focussing on the Advancement of Preclinical Research & Advanced Technology in the Complement Field to Drive Innovation & Reduce Translational Hurdles
9:00 am Exploring Complement’s Role in Transplantation
Synopsis
- Understanding how the complement pathway triggers that drive classical pathway activation which contributes to immediate and delayed graft function
- Examining emerging clinical experience with complement inhibitors in transplantation, showing improvement in graft survival
- Discussing the next steps in optimizing complement inhibition strategies to achieve long-term graft function
9:30 am Panel Discussion: Uncovering the Latest Technology in Preclinical Modelling Through the Promise of Humanized Complement Models in Drug Development
Synopsis
- Evaluating the utility of humanized complement models for testing complement inhibitors
- Delving into their advantages for human targeted agents
- Addressing key challenges such as incomplete humanization, low human protein expression and functional assay development
- Understanding how humanized complement models can be used to inform complement inhibitor dose prediction
10:00 am Repurposing Preclinical Models for Mechanistic Insights & Target Validation to Uncover the Next Generation of Complement Inhibitors
Synopsis
- Reframing the primary value of preclinical models to focus on the validation of specific aspects of a drugs mechanism of action
- Deep-diving into an example to show how humanized complement models can show the effective inhibition of C3 and C3-driven Kidney model
- Confirming target engagement, particularly for clinically validated targets where the functional outcome is more directly anticipated
10:30 am Morning Break & Networking
Showcasing Novel Modalities for Enhanced Precision & Patient Comfort to Improve Adherence & Efficacy in Neurological & Retinal Indications
11:00 am Navigating Safety, Efficacy & Delivery for Ocular Gene Therapies to Treat Complement-Mediated Retinal Diseases
Synopsis
- Exploring the critical need to prioritize complement targets with established clinical safety and efficacy profiles to mitigate the risks associated with sustained, irreversible therapeutic action
- Focussing on the complexities of achieving sufficient and consistent protein expression levels from gene therapy vectors to match the complement inhibition level seen with traditional biologics, particularly when targeting the immune-privileged organs like the eye
- Highlighting advancements and ongoing programs and explore how understanding complement biology can inform the design of more effective and targeted gene therapies
12:00 pm Precision Medicine Solutions for Complement-Mediated Retinal Disease
Synopsis
- Exploring the critical need to prioritize complement targets with established clinical safety and efficacy profiles to mitigate the risks associated with delivery to the eye
- Focussing on the complexities of achieving sufficient complement inhibition when targeting the immune-privileged organs like the eye
- Highlighting advancements and ongoing programs and explore how understanding complement biology can inform the design of more effective and targeted therapies
12:30 pm Panel Discussion: Discussing the Next-Generation of Novel Modalities to be Used for Enhanced Delivery & Broader Impact in CNS & Ophthalmic Indications
Synopsis
- Addressing the critical challenge of achieving sufficient complement inhibitor exposure in the CNS, a major reason for past failures with peripherally acting drugs
- Exploring novel delivery modalities like gene therapy, shuttle technologies that are in early development, to unlock the therapeutic potential of complement inhibition neurodegenerative diseases
- Highlighting the significant appeal and ongoing development of gene therapies for chronic ophthalmic conditions driven by complement
1:00 pm Lunch & Networking
2:00 pm ADX-038, a Long-Acting CFB siRNA Therapeutic for Multiple Complement Mediated Diseases
Synopsis
- Exploring the current shift towards their use as add-on therapies in hematology where C5 inhibition alone is rarely sufficient
- Delving into the strategic decisions for Factor B when used in addition to expensive C5 inhibitors
- Exploring the current shift towards their use as add-on therapies in hematology where C5 inhibition alone is rarely sufficient
2:30 pm Optimizing Clinical Outcomes Utilizing Complement Mediators in Ophthalmic Indications: Are All Complement Pathways Equal?
Synopsis
- Assess potential distinct roles for the Classical and Alternative Pathways in Age-Related Macular Degeneration (AMD)
- Describe the clinical outcomes with distinct complement approaches (Factor B, C3, C5, C1q)
- Debate the role of local versus systemic complement in AMD
3:00 pm Afternoon Refreshments
Examining Progress in Safety & Efficacy Studies & Vaccination Considerations to Inform Regulatory Strategy for Complement Inhibitors
3:30 pm Roundtable: Exploring Current Regulatory Challenges in Complement Inhibitors form IND to Approval
Synopsis
Join this roundtable to have open and insightful discussions on regulatory experiences with your fellow complement colleagues to gain actionable insights and learnings to bring back to your team. Join the table for the regulatory interaction most relevant to your stage of development.
INTERACT Meeting
Pre-IND Meeting
End of Phase1/2 Meeting
Pre-BLA Meeting
4:00 pm Panel Discussion: Understanding the Current Attitudes Towards Mandatory Vaccinations Before Dosing a Patient with Complement Inhibitors
Synopsis
- Re-evaluating the blanket vaccination requirements for complement inhibitors considering the emergence of novel, non-C5 complement inhibitors that show a lower infection risk compared to earlier C5 antibodies
- Discussing whether a blanket-approach remains justified, or whether a more nuance, risk stratified approach is warranted
- Navigating vaccination in acute vs chronic complement-mediated conditions, to mitigate infection risk without compromising rapid therapeutic intervention