Day One

Tuesday, December 12

8:00 am
Morning Registration & Coffee

8:45 am Chair’s Opening Remarks

  • Maja Sedic Director, Toxicology, ReNAgade Therapeutics

Innovative Approaches to Immunogenicity Risk Assessment of mRNA-LNP Products to Unlock Therapeutic Potentials

9:00 am Clinical Risk Assessments Considerations for Immunogenicity & Tolerogenicity for LNP Vectored Therapeutics Vaccine


  • Evaluating the potential for the LNP vector to induce antigen-presenting cells that may trigger unwanted immune responses
  • Analyzing the likelihood of the vaccine inducing the production of antibodies against the therapeutic antigen
  • Understanding the persistence of the tolerogenic response induced by the LNP-based vaccine over time

9:30 am Case Study: Approaches to Immunogenicity Risk Assessment of mRNA-LNP Products


  • Addressing specific regulatory guidelines for the identification and mitigation of unwanted immunogenicity risk factors for LNP-mRNA products
  • Defining a strategy utilizing a suite of in vitro immunogenicity/reactogenicity assays that could guide the immunogenicity de-risking by design of LNP-mRNA therapeutics

10:00 am Immunogenicity Risk Assessment of mRNA/LNP Therapies


  • Outlining the importance of the immunogenicity risk assessment for the development of safe mRNA/LNP therapies
  • Summarizing regulatory requirements and providing practical guidance on immunogenicity risk identification and evaluation
  • Emphasizing the impact of immunogenicity risk assessment on the bioanalytical monitoring strategy for all mRNA/LNP components during development

10:30 am
Morning Refreshments & Networking

Bridging The Gap: Strengthening Translatability for Seamless Preclinical-to-Clinical Translation

11:30 am LNP-Based Therapies for Lasting Relief in Monogenic Liver Disorders

  • Nick Weber Principal Scientist - New Programs & Innovation Lead, Vivet Therapeutics


  • LNP and AAV: comparisons in immunogenicity and toxicity and potential for combinatorial approaches
  • An overview of immunogenicity and toxicity in metabolic liver disease, striving for enhanced targeting and uptake
  • Confronting the challenge of efficiently delivering therapeutic agents to liver cells using LNPs

12:00 pm Panel Discussion: Unveiling the Lasting Benefits of LNP-Based Treatment


  • Explore the potential for prolonged well-being as LNP delivery systems enable therapies to deliver benefits that persist long after administration
  • Gain insights into tailoring treatment schedules for maximum patient benefit, ensuring consistent and lasting positive outcomes
  • Assessing the balance between the toxicity of stable versus the efficacy of not stable integrating

12:45 pm
Lunch & Networking

1:45 pm Panel Discussion: Assessing Different Routes of Administration for Optimal Safety Profiles and Therapeutic Effects


Experts in this panel discussion will delve into various routes of administration across topical, intramuscular, inhalation,

intranodal, intranasal, intraocular, intratumoral, intravesical

  • Assessing the effectiveness of each route of administration in delivering the desired therapeutic effect
  • Evaluating the safety profile of different routes of administration, considering potential adverse effects and risks
  • Considering the ease of administration and patient preference to ensure better treatment adherence

Advancing Clinical Safety for Enhanced Patient Safeguarding

2:30 pm Preclinical Safety Evaluation of mRNA Cancer Therapeutics: A Platform Approach

  • Jan Diekmann Senior Director Non-clinical Safety, BioNTech


  • Toxicology assessment of RNA-therapeutics can be split into two parts – assessment of RNA-LNP characteristics and the assessment of translated protein
  • Toxicity data of an RNA-LNP platform facilitating various RNA payloads show a comparable safety profile in non-clinical safety studies
  • Platform-based strategy could reduce the need for toxicity tests involving animal testing (3R)

3:00 pm IND-Enabling Safety Evaluation of mRNA-LNP Cancer Therapeutic

  • Maja Sedic Director, Toxicology, ReNAgade Therapeutics


  • General consideration for Tox assessment of mRNA-LNP
  • Safety evaluation of mRNA-LNP in a context of cancer immunotherapy
  • Enabling intratumoral administration

3:30 pm
Afternoon Refreshments & Poster Session

4:30 pm Clinical Trial Designs for Immunogenicity Assessments


  • Incorporate Immunogenic Adverse Events assessment endpoints in clinical trial protocols and statistical analyses plans
  • Implementing robust systems for collection and medical evaluation of Immunogenic Adverse Events occurring during clinical trials
  • Identifying clinically significant Immunogenic Adverse Events for participants with and without ADA

5:00 pm Characterization of Anti-PEG Antibodies to Support Clinical Development of GLM101, a Liposome-Based Therapeutic Candidate for PMM2-CDG


  • Clinical feedback on liposome product given to patients chronically
  • Optimal strategy on collecting immunogenicity data
  • Exploring the impact of PEG antibodies on clinical performance

5:30 pm Chair’s Closing Remarks

  • Maja Sedic Director, Toxicology, ReNAgade Therapeutics

5:45 pm Close of Day One