*All times listed are Central European Time (CET)*

9:00 am Chair’s Opening Remarks

New & Emerging Delivery Technologies for mRNA- Based Vaccines & Therapeutics

9:15 am Approaches Towards Next Generation mRNA Delivery Systems

Synopsis

  • Design of mRNA delivery systems, from molecular organization to clinical validation
  • CMC and GMP criteria for successful clinical translation of mRNA delivery systems
  • Coherencies for development of next generation mRNA delivery systems

9:45 am Keys to Successful mRNA Manufacturing from Development, Purification and Lipid Formulation to Facility Design

  • Julian Mochayedi Strategic Marketing Manager – mRNA solutions, Merck KGaA
  • Nargisse El Hajjami Senior Consultant - Novel Modalities BioProcessing Strategy Operationalization, Merck KGaA

Synopsis

  • Key factors in mRNA manufacturing and facility design to ensure success
  • How our PCR-based technology can accelerate mRNA development and manufacturing
  • Design principles and critical parameters to enable the rapid development of lipids for RNA formulation

10:15 am Harnessing an Alternative Non-Viral Polymer- Based Drug Delivery System for mRNA- Based Therapeutics

Synopsis

  • How to create a successful polymer-based delivery system for mRNAbased therapeutics to extend across a broad spectrum of disease applications
  • How to modulate one-step formulations of polymer- based materials to extend stability and for efficient storage
  • Polymer based drugs- Can they truly resolve challenges of delivery compared to LNP’s?

10:45 am mRNA Modifications that Matter: Modified Uridine as a Therapeutic Tool

Synopsis

  • In vitro transcribed (IVT) mRNA has become an effective and scalable therapeutic modality for a wide range of disease indications
  • It has been shown that modifications to wild-type uridine may improve the efficacy of these therapeutics
  • To aid in the synthesis of these compounds, TriLink is developing an extensive platform to manufacture modified uridine and mRNA at both GMP quality and scale.

11.00 am | Morning Networking Break

Achieving Effective Cell & Tissue Specificity for mRNA- Based Therapeutics

12:15 pm Tissue Specific Targeting of mRNA to Increase Therapeutic & Vaccine Efficacy

  • Drew Weissman Professor - Vaccine Research, University of Pennsylvania, Perelman School of Medicine

Synopsis

Digital Presentation

  • Analysing cellular and tissue characteristics for successful uptake of mRNA- based therapeutics and vaccines
  • How to overcome the challenges of tissue specificity whilst increasing mRNA- based therapeutic efficacy?
  • Expanding the use of mRNA- based therapeutics and vaccines for brain, bone marrow and beyond

12:45 pm Monitoring Functional Cellular Uptake of mRNA Using Native-Like Fluorophores & Challenges with Expanding the Methodology to In Vivo

Synopsis

  • Exploring novel methods of monitoring mRNA uptake and distribution in cells that does perturb natural uptake mechanisms and expression of corresponding proteins
  • How to successfully quantify uptake and biodistribution of mRNA using fluorescence microscopy?
  • How to advance mRNA tracking to in vivo investigations to facilitate increased tissue penetration and translatability?

1:15 pm Integrating a Fragmented mRNA- Based Therapies Supply Chain

Synopsis

  • A brief overview of the manufacturing challenges faced by mRNA innovators
  • Bringing the complex multistep process under one roof
  • The importance of flexibility in a CDMO partner to match the speed and flexibility promise of mRNA therapeutics

1:30 pm Programming mRNA to Enable Cell-Type Specific Target Expression

Synopsis

  • Designing programmable mRNAs for different applications
  • Building mRNA- based logic circuits to overcome the challenges of cell specificity
  • Translating observations from in vitro systems to in vivo models to display the power of cell-type classifiers for mRNA- based therapeutics

2.00 pm | Networking Lunch

Future Applications & Considerations to Expand mRNA- Based Therapeutics Opportunities

3:00 pm Strategies of mRNA- Based Therapeutics to Replace Enzymes for Rare Diseases

Synopsis

  • Exploring mRNA construct requirements to meet therapeutic thresholds for rare diseases
  • Unravelling the challenges expression vs delivery for successful mtranslation of mRNA- based therapeutics
  • How to predict efficacy from pre-clinical models to unleash the therapeutic benefit for patients with rare diseases?

3:30 pm Plan Now or Fail Later: Why Raw Materials Matter

  • Darwin Asa Market Development Manager, ThermoFisher Scientific

Synopsis

  • Important criteria to consider for choosing materials to make an mRNA
    vaccine or therapeutic
  • Changes in requirements for materials as projects move from discovery to
    development to commercial manufacture
  • Examples of companies who successfully chose proper materials and
    examples of those who didn’t

4:00 pm Exploring the Broader Application of mRNA- Based Therapies for Age-Related Degenerative Diseases

Synopsis

  • Redefining the potential of mRNA technology to go beyond infectious diseases
  • How to fine tune mRNA- based therapeutics to create a balance across subtle phenotypes
  • Exploring methods to develop mRNA- based technologies to combat age-related degenerative diseases

4.30 pm | Afternoon Networking Break

5:00 pm Panel Discussion

Synopsis

With an increase in investment and development of mRNA technology platforms and upcoming biotechs this calls for further clarity to standardise frameworks for future innovations. It is critical within the mRNA space to streamline practices for mRNA- based therapeutics across the development value chain and overcome key bottlenecks for better clinical translation.

  • How to accelerate platforms for future innovative technologies, applications and commercial opportunities?
  • How to standardise mRNA technology platforms and its potential applications?
  • How to identify and successfully address the key bottlenecks from early discovery to clinical translation for mRNA- based therapeutics?

5:30 pm Computational Approaches to Develop Next-Generation mRNA- Based Therapeutic Delivery Methods

Synopsis

  • Exploring the power of biophysical modelling to improve mRNA- based therapeutic efficacy and delivery
  • Adapting computational approaches to promote mRNA stability and maximise cellular delivery
  • How to transform computational methods for hybrid LNP and polymeric systems for easy release and target specificity

6:00 pm Chair’s Closing Remarks & End of mRNA- Based Therapeutics Summit Europe