Conference Day One

7:20 am Registration & Networking

7:55 am Chair’s Opening Remarks

8:00 am Session Reserved for BioNTech

Showcasing Clinical Updates to Accelerate the Next Wave of mRNA Vaccines for Infectious Diseases & Oncology

8:30 am Local Delivery of mRNA Therapeutics for Treatment of Respiratory Diseases

Synopsis

  • Unlocking local delivery of mRNA therapeutics into the upper and lower respiratory tract to enable production of the target protein, directly at the site of desired pharmacological activity
  • Addressing the technical challenges of inhaled mRNA delivery and how to overcome them
  • Revealing the first clinical trial results using mRNA for immunomodulation in the respiratory tract

9:00 am Updates & Interim Data of mRNA-Based Cancer Vaccines to Treat EBV-Related Cancers

Synopsis

  • Present breakthroughs in the treatment of EBV-related cancers, including NPC and NKT lymphoma
  • Highlight safety and efficacy based on ongoing enrollment and interim data
  • Demonstrate the transformative potential of mRNA therapeutics in the treatment of viral-related malignancies

9:30 am Session Reserved for Cytiva

10:00 am Morning Break & Speed Networking

TRANSLATIONAL DEVELOPMENT TRACK

Chair Moderation:

  • Samuel Deutsch Executive Vice President, Research and Early Development, Nutcracker Therapeutics

Unlocking Novel RNA Technology Approaches to Expand Vaccine & Therapeutic Application

11:00 am Key Differences of Natural vs Chemically Modified mRNA in Terms of Translation & Immune Sensor Activation

Synopsis

  • Understanding how bioproduction of mRNA in eukaryotic cells bear different types of nucleoside modifications
  • What is the impact of such endogenous modification on the translation efficiency and immune activation?
  • Showcasing preclinical results using such modified mRNA

11:30 am Round Table Discussion: Reimagining the Practical Use of mRNA Beyond Vaccines & its Applications

Synopsis

  • How easy is it to use mRNA in comparison to traditional medicines for drugs already in market?
  • Which assays are required to determine viability for certain indications?
  • How can these be translated successfully into pre-clinical development?

12:00 pm Trans-Amplifying RNA as a Versatile Vaccine Platform Having Unique Advantages for Improving Pandemic Preparedness

Synopsis

  • Showcasing in vitro taRNA development and optimisation
  • Sharing In vivo experiences with taRNA
  • Providing future perspectives on the potential contribution of taRNA for improved pandemic preparedness

12:30 pm Session Reserved for Helix Biotech

CMC
TRACK

Chair Moderation:

  • Cristina Bruno Programme Manager, Consultant - mRNA Technology Transfer, World Health Organization - WHO

Translating CMC Methods to Advance mRNA Products from Bench to Clinic with Ease

11:00 am Advancing CMC Strategies for Self-Amplifying RNA to Achieve a Transition from Discovery through to Clinic

  • Iulia Oita Chemistry, Manufacturing & Controls Manager, Ziphius Vaccines NV

Synopsis

  • How to optimise CMC methods for self-amplifying RNA whilst de-risking failures
  • Examining platform requirements for a scalable production process to meeting the needs at each phase
  • Maximising volume and speed to ensure future clinical success

11:30 am Democratising mRNA Therapeutics with Novel Cap Analogs & Modified NTPs

  • May Guo Chief Commercial Officer, Areterna

Synopsis

  • Showcasing novel cap analogs and their discovery
  • Unravelling novel modified NTPs
  • Leveraging mRNA capping assays and paired capped/ upcapped mRNA standards

12:00 pm mRNA-Based CRISPR/Cas9 Delivery for Gene Editing in Muscle Stem Cells

  • Simone Spuler Professor; Co-founder, Max-Delbruck-Center for Molecular Medicine (MDC)

Synopsis

  • Exploring muscle wasting and muscular dystrophy
  • Assessing clinical trial, ATMP and regulatory considerations
  • Advancing CMC and ex-vivo editing

12:30 pm Reduction of Double-Strand mRNA in Process Development

Synopsis

Session Reserved: CATUG

12:40 pm Lunch Break & Networking

Translating Preclinical to Clinical Success in the Context of mRNA-Based Therapeutics & Vaccines

Chair Moderation:

1:45 pm Beyond COVID Vaccines – mRNA Therapeutics & Lean Strategies for their Non-Clinical (Safety) Assessments

Synopsis

  • Introduction into special considerations for mRNA therapeutics with regards to their non-clinical PD/DMPK and safety assessments
  • Introduction of the platform toxicology concept
  • Challenges and opportunities for mRNA therapeutics in the non-clinical (safety) space

2:15 pm Round Table Discussion: Deep Diving Chronic Dosing with mRNA Therapy to Suit the Treatment Options of Different Diseases

Synopsis

  • Comparing the therapeutic risk-reward profile of each mode of delivery along with combination therapies to understand which diseases can withstand which side effects
  • Overcoming the difficulties of efficacious mRNA payloads to penetrate deep tissue
  • Do we fully understand kinetics of IV injections and off-target effects of lipid compositions?
  • How can we deliver large payloads safely with optimal dosing concentrations?

2:45 pm Harnessing mRNA Therapeutic Platforms to Accelerate Product Development

  • Samuel Deutsch Executive Vice President, Research and Early Development, Nutcracker Therapeutics

Synopsis

  • Describing different platform components from design to manufacturing, and their importance to accelerating product development
  • Leveraging critical elements in protein and mRNA design to drive clinically relevant PK and purity behaviours in vivo
  • Transitioning from in vitro to rodents to non-human primates to best predict clinical efficacy in heterogeneous populations

3:15 pm Impingement Jets Mixing for Scalable Production of Lipid Nanoparticles

  • Matthias Lubbert Principle Engineer - Process, KNAUER Wissenschaftliche Geräte GmbH

Synopsis

  • Showcasing KNAUER’s NanoProducer Systems with Impingement Jet Mixing have shown great success for large-scale production of mRNA vaccine
  • Successfully using laboratory Scale NanoScaler for method development and small batches
  • Achieving scalability between both ranges with our range of Impingement Jets Mixers

Unravelling mRNA-Delivery Thermostability to Produce Best-In-Class Drug Formulations

Chair Moderation

  • Cristina Bruno Programme Manager, Consultant - mRNA Technology Transfer, World Health Organization - WHO

1:45 pm Combating Thermostability with the World’s First Approved Lyophilised mRNA-Based Vaccine

  • Swarnendu Kaviraj Associate Director Research and development, Gennova Biopharmaceuticals

Synopsis

  • Developing the first self-amplifying mRNA vaccine to gain European approval
  • Benchmarking the convenience of 2-8 degrees Celsius storage and supply, pharmacoeconomics and reachability
  • Advancing needle free delivery
  • Showcasing self-sufficiency of raw materials, ease of manufacturing and stockpiling

2:15 pm A Novel Unit Operation Successfully Drying mRNA/ LNP by Mass Transfer

Synopsis

  • Presenting a unique LaminarPace ambient drying, based on mass transfer principle
  • Turning liquid biopharmaceuticals into thermostable dry powders
  • Examining how the well-defined powder may be reconstituted or used for novel administration routes

2:45 pm Advances in Development of Thermostable mRNA-LNP Formulations

Synopsis

  • Showcasing development of the first mRNA vaccine developed by Abogen in China, but approved in Indonesia for emergency authorisation use
  • Sharing technical insights into Abogen’s mRNA COVID vaccine which can be stored at 2-8 degrees Celsius whilst similar vaccines from BioNTech-Pfizer and Moderna require much lower storage temperatures of -70 and -20 degrees Celsius
  • Highlighting Abogen’s recent progress in development of frozen and lyophilised mRNA-lipid nanoparticle formulations for conventional use

3:15 pm Thermostability Q&A – Ask Speakers Your Burning Questions Following Their Presentations

3:30 pm
Afternoon Break & Poster Session

Synopsis

As the landscape of innovation that is supercharging the next wave of efficacious mRNA vaccines and therapeutics, it is more important than ever to collaborate and learn for the growth of the mRNA field. Join our dedicated poster session to share your latest data and have first look into what your peers are working on!

Delving into Novel Applications of mRNA-Based Therapeutics to Expand Potential Beyond Infectious Diseases

4:00 pm Small but Mighty: How Digital Technology can Support Small Batch Manufacturing of mRNA Therapeutics

Synopsis

Session details TBC

4:30 pm Targeted mRNA Therapeutics for Treatment of Kidney Diseases

Synopsis

  • Mercurna’s technology platform combines unique cell-targeting moieties, with a lipid-based delivery vehicle to deliver engineered mRNA with high specificity
  • Active targeting strategies through optimised technology on targeted LNPs
  • Showcasing examples on how Mercurna leverages its platform for development of targeted mRNA therapeutics to treat kidney diseases

5:00 pm Is “GMP Grade” Good Enough? Choosing Materials for Producing mRNA Therapeutics

Synopsis

Session Reserved: Thermo Fisher Scientific

5:30 pm Delivery of Synthetic Tropoelastin mRNA for the Regeneration of Elastic Tissues in Rare Diseases

Synopsis

  • Showcasing protein replacement therapy using synthetic mRNA
  • Accelerating mRNA therapeutics for rare diseases
  • Unleashing targeted delivery of synthetic TE mRNA

6:00 pm Chair’s Closing Remarks

6:05 pm End of Conference Day One