Day Two

Wednesday, December 13

8:00 am
Morning Registration & Coffee

8:45 am Chair’s Opening Remarks

  • Maja Sedic Director, Toxicology, ReNAgade Therapeutics

Enhancing Assessment of Immunogenicity & Toxicity for Safer Therapeutic Interventions

9:00 am Strategic Consideration to Leverage the Translatability From Preclinical Assessments to Clinical Trial Through Implementation of Appropriate Assays

  • Jian Liu Chief Scientist Director of Toxicology, Nitto BioPharma

Synopsis

  • In vitro cytokine release assay in hPBMC and in vivo toxicity assessment with characterization of drug exposure
  • Immune response under repeated dosing modality of PEGylated LNP through systemic delivery and mitigation strategies
  • Regulatory perspective of toxicity assessment on the LNP with different API entities

9:30 am Leveraging Animal Models for Comprehensive Immunogenicity & Toxicity Assessment

  • Jake Brenner Associate Director & Assistant Professor - Medicine, University of Pennsylvania

Synopsis

  • Administering the therapeutic intervention to animal models and monitoring their immune responses, including cytokine production, immune cell activation, and antibody generation
  • Assessing the biodistribution of the therapeutic intervention in animal models using imaging techniques or tissue sampling
  • Evaluating the potential systemic and local toxic effects of the therapeutic intervention in animal models through histopathology, organ function tests, and biomarker analysis

Exploring the Structural Analysis of LNP & Its Impact on Immunogenicity for Amplified Therapeutic Potential

10:15 am Exploring the Effect of LNP Composition on Immunogenicity

  • Esmaiel Jabbari Professor of Chemical and Biomedical Engineering, University of South Carolina

Synopsis

  • Investigating the impact of different lipid nanoparticle compositions on the immunogenicity of therapeutic interventions
  • Comparing the immunogenicity profiles of different LNP compositions in preclinical and clinical studies
  • Exploring the impact of LNP composition on the duration and magnitude of immune responses to mRNA and other payloads

10:45 am
Morning Refreshments & Speed Networking

11:45 am Exploring Novel Biodegradable LNPs & Their Effect on Pharmacokinetics & Toxicity

Synopsis

  • Investigating the effect of LNP chemistry on pharmacokinetics and toxicity
  • Developing new ionizable lipids for rapid clearance
  • Comparing optimal LNP compositions for RNA vaccines and therapeutics

Optimizing Therapeutic Safety: Unveiling the Impact of Diverse LNP Payloads on Immunogenicity & Toxicity

12:15 pm The Effect of DNA as an LNP Payload on Immunogenicity & Toxicity

Synopsis

  • Investigating the impact of using DNA as a payload within LNP delivery systems on immunogenicity and toxicity
  • Assessing the potential immune responses triggered by the presence of DNA components within LNPs
  • Assessing the role of different LNP formulations and DNA payload designs in modulating the immune response and minimizing adverse reactions

12:45 pm Non-clinical & Clinical Safety Profiles of Two LNP-Adjuvanted Vaccines

Synopsis

  • Characterization of the immune response in nonclinical species following administration of two different LNP-adjuvanted vaccines
  • Non-clinical toxicity profiles of two different vaccines with the same LNP adjuvant
  • Disparate clinical safety profiles of two vaccines adjuvanted with the same LNP

1:15 pm
Lunch & Networking

2:15 pm Round Table Discussion: Maximizing Benefits: Unravelling the Impact of Diverse Payloads on Immunogenicity &Toxicity for Safer & More Effective Therapies

Synopsis

Payloads to consider – mRNA, RNA, DNA, SiRNA, CRISPR etc.

  • Differences and similarities across different payloads
  • How is the payload impacting the immunogenic response
  • Understanding the toxicity of the payload
  • How are we reducing/enhancing immunogenic responses
  • Innovations in alternative payloads being considered for drug development

3:00 pm Impact of LNP Based Therapies on Off-Target Effects for Targeted Therapeutic Interventions

  • RAKESH DIXIT President Chief Executive Officer, Bionavigen

Synopsis

  • Investigating the potential off-target effects of LNP delivery systems in therapeutic interventions
  • Assessing unintended interactions and effects of LNPs with non-target cells or tissues
  • Investigating the potential immunogenicity triggered by off-target effects of LNPs, leading to unwanted immune responses

3:30 pm Chair’s Closing Remarks

3:45 pm Close of Summit