Day One
Tuesday, December 12
8:00 am
Morning Registration & Coffee
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
- Amy Rosenberg Senior Director Immunology, Epivax
Synopsis
- 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
- Sophie Tourdot Immunogenicity Sciences Lead, Pfizer
Synopsis
- 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
- Joanna Grudzinska Drug Metabolism & Pharmacokinetics Project Manager, Bayer AG
Synopsis
- 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
Synopsis
- 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
- Olga Lihoradova Distinguished Scientist, Sanofi
- Virna Cortez-Retamozo Head Of Group Onco-Pharmacology, Genzyme
- Zohreh Amoozgar Principal scientist and lab head, Sanofi
Synopsis
- 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
- Olga Lihoradova Distinguished Scientist, Sanofi
- Esmaiel Jabbari Professor of Chemical and Biomedical Engineering, University of South Carolina
- Virna Cortez-Retamozo Head Of Group Onco-Pharmacology, Genzyme
- Daryl Drummond Chief Scientific Officer, Akagera Medicines
- Jake Brenner Associate Director & Assistant Professor - Medicine, University of Pennsylvania
- Rui Zhang Director, Formulation, Stylus Medicine
Synopsis
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
Synopsis
- 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
Synopsis
- 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
- Maria-Dolores Vazquez-Abad Medicine Team Lead Clinical Immunogenicity, Pfizer
Synopsis
- 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
- Hicham Alaoui Chief Scientific Officer, Glycomine
Synopsis
- Clinical feedback on liposome product given to patients chronically
- Optimal strategy on collecting immunogenicity data
- Exploring the impact of PEG antibodies on clinical performance