Pre-Conference Workshop Day
Monday | December 11, 2023
LNP Immunogenicity
Workshop A
9:00 am Modulating Immunogenicity at The Design Phase
Synopsis
Addressing immunogenicity at the design phase plays a crucial role in enhancing therapeutic outcomes and reducing adverse reactions. The design phase offers opportunities to optimize the selection and design of therapeutic targets, considering factors such as antigenicity and immune recognition. This session provides immune
evasion strategies can be incorporated into the therapeutic intervention design, aimed at reducing immune activation and promoting tolerogenic responses.
Join us in this interactive workshop to learn about:
- Incorporating immune evasion strategies into the design of therapeutic interventions to reduce immunogenicity
- Optimizing the selection and design of the therapeutic target to minimize the potential for immune recognition and response
- Modifying the composition or structure of the therapeutic payload or delivery system to enhance biocompatibility and reduce immune activation
- Utilizing alternative delivery systems, such as stealth nanoparticles or viral vectors with reduced immunogenicity, to minimize immune recognition
- Conducting comprehensive in vitro and in vivo immunogenicity assessments during the design phase to identify and address potential immune triggers
LNP Toxicity
Workshop C
9:00 am Optimizing Repeat Dosing & Widening The Therapeutic Window
Synopsis
It is important to address key considerations and strategies for optimizing repeat dosing and widening the therapeutic window of an LNP based therapeutic intervention. By carefully evaluating pharmacokinetics, individualizing dosing strategies, and leveraging innovative approaches, we can enhance treatment outcomes while maintaining safety and tolerability. Continuous monitoring and collaboration with regulatory agencies are also crucial for ensuring the longterm benefit-risk balance of the therapy.
Throughout this workshop, we will uncover:
- Understanding PKPD of the therapeutic intervention: Evaluating the rate of clearance, distribution, and target engagement to determine the optimal dosing regimen for repeat administration
- Assessing safety and tolerability: Monitoring and evaluating any potential cumulative toxicity or adverse effects associated with repeat dosing, to maximize therapeutic window
- Pharmacogenomics considerations: Considering genetic variations that may impact drug metabolism, response, or tolerability when determining the appropriate repeat dosing regimen for individual patients
- Dose escalation and de-escalation: Adjusting the dose based on the patient’s response and tolerability to optimize efficacy while minimizing the risk of toxicity or suboptimal treatment outcomes
- Biomarker-guided dosing: Utilizing biomarkers or surrogate endpoints to guide dosing decisions and assess treatment response, enabling more precise dosing adjustments
12:00 pm Networking Lunch
LNP Immunogenicity
Workshop B
1:00 pm Striking the Balance Between Wanted & Unwanted Immunogenicity
Synopsis
Immunogenicity plays a crucial role in various fields such as vaccine development, therapeutic protein production, and gene therapy. This session will equip you with the skills needed to strike a balance between wanted and unwanted immunogenicity and emphasises its position as a critical challenge that requires careful consideration and strategic approaches.
In this session, we will explore:
- Delineating the specific immune reactions necessary for efficacy from those that may lead to adverse events or reduced treatment effectiveness
- Developing and utilizing agents that can modulate immune responses, to strike a balance between activation and suppression of the immune system
- Incorporating adjuvants to enhance the desired immune response, improve vaccine efficacy, and potentially reduce the risk of unwanted immunogenicity
- Exploring the use of immunomodulatory drugs or biologics to regulate the immune system and fine-tune immune responses
- Leveraging advancements in genomics, biomarkers, and patient profiling to tailor immunotherapies
LNP Toxicity
Workshop D
1:00 pm Evaluating Potential Immunogenic Adverse Events and Characterizing Anti-Drug Antibodies, and Their Impact on Potential Immunogenic Adverse Events
Synopsis
This hands-on workshop offers attendees clear examples for characterizing anti-drug antibody (ADA) outcomes in clinical trials and assessing their impact on significant clinical results in line with regulatory expectations and immunogenicity guidelines. Participants will collaborate in teams to discuss these examples, presenting summaries of their discussions on data analysis, result interpretation, and communicating key messages regarding immunogenic risk. This includes integrating immunogenicity clinical data for dossiers, comprehending how immunogenic response risks are reported from trials, identifying potential immunogenic adverse events, and ultimately securing the safety of treated individuals.
Join us to learn about:
- Clinical trial design: how and when to collect samples to characterize anti-drug antibody (ADA). How to analyze and report the results of ADA from clinical trials, in alignment with regulatory agencies immunogenicity guidelines
- Statistical Analysis Plan: examples on how to assess the impact of ADA on clinically meaningful outcomes
- Collection of Data: how to collect Injection Site Reactions (ISR) and/or Infusion Related Reactions (IRR) for each administered dose, and how to use the data in the programmed algorithms for analyses and reporting
- Medical Evaluation: Utilizing standardized assessments to evaluate all safety events, identify if they are potential acute or delayed immunogenic reactions against the therapeutic drug
- Assessment of clinically significant impact by presence of ADA on potential Immunogenic Adverse Events
- Identify the risk of potential Immunogenic Adverse Events and their significant impact on clinical outcomes for each program to inform risk-benefit considerations