Conference Day Two
8:20 am Chair’s Opening Remarks
Optimizing Clinical Development Strategies to Ensure Commercial Success with Complement Therapeutics
8:30 am Devloping Biomarkers to Inform PK/PD & Patient Populations in Clinical Trials
Synopsis
- In vitro and in vivo models to inform biomarker development in the clinic
- Identifying biomarkers to elucidate complement therapeutic MOA and PD effects in clinical trials
- Identifying the most useful patient population to see efficacy in trial
9:00 am Delivering Clinically Actionable Complement Diagnostics
Synopsis
- Current diagnostic challenges result in limited clinical utility
- To be clinically actionable, biomarker data must be reliable and accurate, available and interpretable
- Quality multiplexed data delivered at POC is key
9:10 am Panel Discussion: Utilizing Preclinical Findings to Influence and Improve Clinical Trial Design in Complement-Based Drug Development
Synopsis
- Strategies for translating preclinical findings into actionable clinical trial plans, including dose selection, patient population identification, and endpoint determination
- Biomarkers identified in preclinical studies that can be used for patient stratification, monitoring drug response, and predicting outcomes
- Adaptive clinical trial designs that allow flexibility from insights from preclinical stages
9:40 am Morning Break and Networking
Mitigating Toxicity Effects of Immunosuppression with Complement Inhibitors
10:40 am The Clinical Promise of Tissue Targeted Complement Therapeutics
Synopsis
- Exploring Q32’s approach in utilizing tissue targeted therapies for complement mediated diseases
- ADX-097’s ability to block complement activity whilst persevering natural immune responses
- Exploring the benefits and the future promise of tissue targeted therapeutics in the complement industry.
11:10 am Localised & Long-Term Delivery of Complement Regulators Using Gene Therapies
Synopsis
- Utilising localised gene therapies to navigate toxicity effects
- Reviewing the benefits of a long-term treatment
- Targeting complement Factor H for the treatment of Geographic Atrophy/Dry AMD
New Modalities of Complement Therapeutics Demonstrating Clinical Promise
11:40 am Next-Generation Complement-Targeted Gene Therapies in Complement Mediated Diseases
Synopsis
- Reviewing Kriya’s gene therapies that precisely target dysregulation and hyperactivity of the complement system
- Detailing the advantages of using gene therapy to target Geographic Atrophy and other ocular diseases
- Looking forward to the next steps for Kriya
12:39 pm
Optimization of Reactions & Workups to Accelerate Speed to Market
12:40 pm CMAC Data Lab: Cyberphysical Systems and Digital Transformation of CMC
Synopsis
Key topics that will be addressed from CMAC’s research programme:
- Overview of predictive toolbox development for crystallisation (CCS) and drugproduct (MCS+)
- Building the data fabric to support product and process development
- Datafactories and automated workflows to accelerate development
- Challenges & opportunities for industrial digital technologies in CMC
1:10 pm Lunch and Networking
1:30 pm Harnessing RNAi for the Development of Complement Therapeutics
Synopsis
- Mechanism and properties of Alynlam’s RNA interference drugs
- Applications of Cemdisiran, a C5 targeting siRNA
- Exploring RNAi for other complement pathway targets
Exploring the Non-Canonical Functions of Complement & Identifying Future Therapeutic Opportunities
2:00 pm Neuronal Pentraxin Nptx2 Regulates Complement Activity & Restrains Microglia-mediated Synapse Loss in Neurodegeneration
Synopsis
- Complement C1q-dependent synapse elimination by glial cells in Alzheimer’s disease (AD) mouse models.
- Nptx2 binds C1q and restrains complement activity and synapse loss in neurodegeneration.
- Nptx2 concentration is decreased in CSF from AD and frontotemporal dementia patients, which correlates with elevated levels of C1q and activated C3
2:30 pm Expanding the Complement Therapeutic Areas: Unlocking the Potential of Gene Therapy & Intracellular Complement
Synopsis
- New opportunities in targeting complement emerge with the discovery of non-canonical roles of complement
- Recent developments in gene therapy hold promise for the future of complement mediated therapies
- Technological and knowledge advancements seem poised to expand complement therapies beyond the rare disease space
3:00 pm Assessing Whether Complements Role in Neurology Highlights a New Opportunity for Neurodegenerative & Neuropsychiatric Diseases
Synopsis
- Evidence from GWAS that structural variants of C4 confer a greater risk of developing schizophrenia
- Using target genetic manipulation to mechanistically link C4 overexpression to aberrant circuit wiring
- Utilising these techniques for the general the study of complement-associated neurological diseases such as age-related synapse elimination and Alzheimer’s disease models
3:30 pm Afternoon Break
4:00 pm Panel Discussion: What are the Future Applications of Complement Therapeutics; What Will the Landscape Look Like in 5 Years’ Time?
Synopsis
- Reflecting on all the incredible insights shared today and our key takings, where do we see the complement landscape going?
- With an increasing appreciation of complement’s role in other conditions, do we see commercial opportunity in tackling neurological diseases?
- Will emerging modalities and tissue specific therapies reduce safety and toxicity effects of immunosuppressants?
- With regulatory floodgates open will there be a flood of new approved therapeutics?