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

8:15 am Chair’s Opening Remarks

  • Andreas Kuhn Senior VP - RNA Biochemistry & Manufacturing, BioNTech

Scalable Manufacturing & Global Development of mRNA- Based Vaccines & Therapeutics

8:30 am Landscaping mRNA- Based Vaccine Technologies for the Future

  • Arun Kumar Preclinical Vaccines Development Lead, Coalition for Epidemic Preparedness Innovations (CEPI)

Synopsis

  • Global mRNA vaccine development landscape is very diverse
  • mRNA- based vaccines are powerful tools to revolutionize the way of developing vaccines in both developing and developed world
  • CEPI’s role in building up mRNA vaccine development ecosystem

9:00 am mRNA: Removing Bottlenecks in Process Development & Manufacturing

  • Rok Sekirnik Head Process Development for mRNA and pDNA, BIA Separations

Synopsis

  • mRNA manufacturing is not yet a platform approach. Standardized products along the processes are needed
  • mRNA is a relatively large molecule, which leads more challenges for downstream purification
  • mRNA is intrinsically unstable and prone to degradation
  • High raw material cost is limiting mRNA commercialization

9:30 am Advancing Strategies & CMC for mRNA- Based Therapeutic Development

Synopsis

  • How to optimise mRNA design to promote CD4 T cells immune response for mRNA therapeutics
  • Accelerating novel purification platforms for standardising production of mRNA- based therapeutics
  • Implementation of a scalable and flexible mRNA manufacturing platform and delivery supporting the Sanofi internal portfolio

10:00 am Engineering the Future of mRNA Therapies

Synopsis

  • Challenges and limitations of current mRNA technology
  • Applications for a developing technology and the tools to do it
  • The importance of process and supply

10:15 am | Morning Break & Structured Networking

Novel Applications of mRNA- Based Vaccines & Therapeutics Beyond Infectious Diseases

11:15 am Local Immunotherapy with Cytokine-Encoding mRNAs for Immuno-Oncology

Synopsis

  • Achieving anti-tumor immunity with an mRNA-encoded cytokines mixture
  • Exploring the mechanistic details of a combined cancer attack with mRNA-based therapeutics
  • Translational aspects of preclinical data

11:45 am Targeting Immunomodulation with mRNA- Based Therapeutics

Synopsis

  • How Ethris is employing its modified mRNA, formulation and inhaled delivery technology platforms towards development of mRNA therapeutics for treatment of lung diseases
  • Utilizing inhaled mRNA therapeutics to modulate immune responses in the lung provides novel opportunities to treat autoimmune diseases and respiratory viral infections beyond vaccines

12:15 pm Manufacturing Considerations of mRNA- Based Therapeutics

  • Henrik Ihre Global Director Strategic Technologies, Cytiva

Synopsis

  • New API modalities require new manufacturing processes
  • The optimal mRNA processes may depend on the application – for example, a pandemic vaccine versus a personalized therapy
  • Fibro-based chromatography for large target molecules can boost productivity

12:45 pm Discovering Novel Methods to Promote Protein Replacement in Chronic End-Stage Disease

Synopsis

  • Achieving robust and efficient mRNA delivery in severely damaged organs
  • Devising mRNA- based therapeutic methods to combat fibrosis and cirrhosis of the liver in vivo
  • Discovering tools for multiple cell-specific targeting for chronic endstage diseases and/or organ failure

1:15 pm | Networking Lunch

Driving the Vision & Success of Next Generation mRNA- Based Vaccines for Infectious Diseases

2:15 pm Lipid-Encapsulated mRNA Delivery of a Monoclonal Antibody with Neutralizing Activity Against Chikungunya Virus: Results From a Phase 1 Trial

  • Allison August VP, Clinical Development, Infectious Diseases, Moderna

Synopsis

  • This phase 1, first-in-human, randomized, placebo-controlled, proof-ofconcept trial conducted from January 2019 to June 2020 evaluated the safety and pharmacology of mRNA-1944, a lipid nanoparticle-encapsulated messenger RNA encoding the heavy and light chains of a CHIKV-specific monoclonal neutralizing antibody, CHKV-24 (NCT03829384)
  • The primary outcome was to evaluate the safety and tolerability of escalating doses of mRNA-1944 administered via intravenous infusion in healthy participants aged 18–50 years
  • The secondary objectives included determination of the pharmacokinetics of mRNA encoding for CHKV-24 immunoglobulin heavy and light chains and ionizable amino lipid component and the pharmacodynamics of mRNA-1944 as assessed by serum concentrations of mRNA encoding for CHKV-24 immunoglobulin G (IgG), plasma concentrations of ionizable amino lipid and serum concentrations of CHKV-24 IgG
  • To our knowledge, mRNA-1944 is the first mRNA-encoded monoclonal antibody showing in vivo expression and detectable ex vivo neutralizing activity in a clinical trial and may offer a treatment option for CHIKV infection

2:45 pm Accelerated Development of Self-Amplifying mRNA (saRNA) Vaccines

  • Lloyd Jeffs Senior Director of Pharmaceutical Development , Precision NanoSystems

Synopsis

  • Self-amplifying RNAs (saRNA) have the potential to elicit effective immune response at lower doses compared to non-replicating mRNA vaccines
  • saRNAs are inherently more fragile than mRNA and are prone to degradation and needs to be packaged in lipid nanoparticles (LNP) to be used as vaccine candidates
  • Typical high shear LNP manufacturing methods affect the potency; hence, low shear robust manufacturing methods are needed for rapid screening of preclinical vaccine candidates and then to swiftly advance the lead candidates to GMP-enabling studies as required
  • Demonstrating the utility of a genomic medicine toolkit, including a proprietary lipid reagent library in a microfluidic-based nanoparticle manufacturing platform using a unique vector design

3:15 pm Reconceptualizing Vaccine Approaches Across the Development Pipeline

Synopsis

  • What opportunities/advantages do mRNA- based vaccines offer against infectious diseases and how to accelerate success?
  • Learning from current pandemic and past infectious diseases for designing more potent vaccines
  • Regulatory standards for saRNA vaccines (revisiting criteria beyond VNT)

3:45 pm The mRNA Landscape & Beyond

Synopsis

• An overview of the RNA drug and trial landscape
• An in-depth look at the mRNA drug and trial landscape, delivery systems
and nucleic acid modifications
• A look into the future of mRNA and upcoming activity

4:00 pm | Afternoon Networking Break

4:30 pm Rapid Commercialization & Technology Transfer of mRNA Vaccines & Therapeutics

Synopsis

  • Recent development and manufacturing technologies for mRNA pipeline therapeutics and lyophilized vaccines
  • Current challenges and success in large-scale production of mRNA vaccines
  • Global technology transfer, process validation rapid commercialization, and supply strategies

5:00 pm Panel Discussion

Synopsis

With mRNA- based vaccines now established for certain infectious diseases, it is now important to realise the opportunities of such mRNA- based vaccine therapeutic applications and the surrounding regulatory requirements. From development to commercialization, we will explore aspects of mRNA- based vaccines beyond Sars-COVID-19.

  • What does the future look like for the next generation of mRNA- based vaccines for infectious diseases?
  • Where do we expect innovations to come from and what challenges are ahead of us?
  • What regulatory requirements need to be addressed to overcome the challenges of mRNA- based vaccines 2.0?

5:30 pm Engineering and Results of Next-Generation COVID-19 Vaccines – Sponsored by Codex DNA

Synopsis

  • Design and synthesis of COVID-19 vaccines
  • Rapid synthesis and cloning of COVID vaccine variants using the BioXP
  • Broad neutralization across several SARS-coV-2 strains from preclinical data

5:45 pm The Therapeutic Potential of oRNA

Synopsis

  • Engineered circular RNA (oRNA) has significant advantages over mRNA
  • Ease of production allows “omics” approach to expression optimization
  • IV delivery to non-hepatic tissues opens new therapeutic areas

6:15 pm Chair’s Closing Remarks & End of Conference Day One