8:50 am Chair’s Opening Remarks

Creating a Forum for Pre-Competitive Collaboration Amongst Drug Developers

9:00 am Opening Address: Navigating the IP Landscape for Lipid Nanoparticle Technology in the mRNA Space

Synopsis

  • Sharing a brief history of intellectual property (IP) in this space
  • Understanding the current IP and competitive landscape for LNP technology
  • Strategies for freedom to operate, design-around, and licensing
  • IP protection strategies for new innovations

9:30 am What’s Next? Next Generation LNPs to Support Novel Therapies With Collaboration

  • Heinrich Haas Vice President, RNA Formulation & Drug Delivery, BioNTech

Synopsis

  • Translating research into reality – consideration for next gen LNP for other vaccines
  • Strategies to improve particular technologies for next gen LNP Lessons learned so far to enhance our considerations during formulation and scale-up for LNP
  • Leveraging pre-competitive collaboration to drive science forward

Improving Process Development for Advanced LNPs

10:00 am Assessment of LNP Quality and Encapsulation Efficiency by Nano-flow Cytometry

Synopsis

  • Quantify particle concentrations and size distribution >40nm label-free
  • Simultaneously identify encapsulated or surface-presented material in loaded subpopulations using fluorescence
  • Multi-parameter measurement of a range of nanoparticles with flexible labelling of targetable material

10:15 am Panel Discussion: Comparing & Evaluating Different Mixing Techniques With Respect to Scalability, Stability & Cost Effectiveness

  • Dai Hayward Chief Executive Officer, Micropore Technologies
  • Sharon Wang SVP of Technical Development, Ultragenyx Pharmaceuticals
  • Aalok Shah Director, Formulation & Drug Delivery, Strand Therapeutics

Synopsis

Discuss with expert panelists the advantages and disadvantages of various mixing technologies on allowing a seamless LNP synthesis, including:

  • Slow filtration
  • Microfluidics
  • T-stream
  • Micropore Technology’s mixing technology

11:00 am
Morning Networking & Refreshments

11:45 am Assessing the Effects of Microfluidic Operating Parameters to Increase LNP Scalability & Stability

  • Dimitrios Lamprou Reader for Pharmaceutical Engineering, Queen’s University Belfast

Synopsis

  • Explaining the advantages of using microfluidics as a mixing technique
  • Evaluating the differences between microfluidics and “common” methods formanufacturing LNPs
  • Discussing the manufacture of LNPs for the delivery of biologics
  • Reviewing microfluidic manufacturing methods, including 3D printing
  • Discussing the effects of encapsulation of nucleic acids, scalability of the microfluidic method, effects of the choice of structural lipid, coating lipid and buffer on characteristics of LNP when using microfluidics
  • Understanding the scalability challenges with microfluidics and areas to focus future technology innovations on

12:15 pm Providing the Optimal Self-Assembly of Lipid Nanoparticles in a Ring Micromixer

Synopsis

  • Understanding the hydrodynamic characterization of a micromixer
  • Identifying the regions in which mixing takes place faster than self-assembling, and the implications of this
  • Defining the flow conditions required to obtain lipid nanoparticles with the desired characteristics

12:45 pm Translating the Discovery and design of RNA-Based LNP into Scalable Genomic Medicines

  • Lloyd Jeffs Senior Director, Pharmaceutical Development, Precision Nanosystems

Synopsis

  • Discussing the ability to rapidly and reproducibly iterate and tune particle critical quality attributes (CQA), allows nanomedicine researchers to discover novel RNA-based lipid nanoparticle (LNP) formulations and opens up possibilities to develop both more effective drugs and to develop grounding breaking therapeutics for previously untreatable diseases and conditions
  • Exploring the experiences and insights from the front lines of RNA vaccine research, highlighting the challenges and solutions faced when developing RNA vaccines from the earliest stages through to the clinic
  • Explaining how the Precision NanoSystems toolkit has enabled the development of a broad range of nanoparticle formulations, starting with a target product profile and determining CQA, key process parameters for scale-up, and analytical development and qualification

1:15 pm
Networking Lunch

Scaling Up LNP Manufacturing With CMC & GMP in Mind

2:15 pm Outlining Regulatory CMC Challenges for LNP Process Development

Synopsis

  • Update on the current regulatory guidance
  • Advising on establishing regulatory approval
  • Outlining Critical Quality Attributes specific to lipid nanoparticles including physiochemical properties such as particle size, charge distribution, encapsulation efficiency and the release of the drug from the LNP
  • Advising on clinical method validations, elemental impurities, residual solvents and setting specifications

2:45 pm Roundtable Discussion: Understanding Process Control Considerations to Support Regulatory Submissions

  • Sumit Luthra Head Global CMC Drug Product Development, mRNA CoE, Sanofi
  • Chandra Webb Senior Director, Drug Product Regulatory Strategy , Pfizer

Synopsis

As the industry progresses at lightning speed, standardization and regulatory framework are going to be critical for future success. This roundtable will be an opportunity for attendees to form working groups to discuss considerations regarding robust process control approaches to deliver quality for LNP-products. We will share feedback at the end of the session.

  • Conceptual introduction of process impact on product quality and controls critical to regulatory success
  • Introduction of regulatory expectations regarding process description
  • Roundtable exercise putting together a description of a well controlled process in context of a pre-defined CQA
  • Sharing agency expectation on how to summarize process control strategy

3:30 pm oRNA: A Superior Coding RNA Payload for LNP Processing & Scale-up

  • Allen Horhota Senior Director, Head of Delivery, Orna Therapeutics

Synopsis

  • Understanding the properties of oRNA, and how oRNA (circular RNA) is a nextgeneration protein-coding RNA with significant biological, manufacturing, and scale benefits over traditional mRNA for LNP processing
  • oRNA is simple to make, pure, and homogeneous, resulting in more consistent formulations and uniform drug products

4:00 pm Quality Attributes and Extended Characterization of Gene Vectors Using Light Scattering

  • John Champagne Senior Applications Scientist & Northeast Regional Manager , Wyatt Technology

Synopsis

  • Quantification of AAV multi-CQAs in one single SEC-MALS run
  • Quantification of LNP payload, size distribution, concentration, morphology, and stability by SEC/FFF-MALS
  • Extended characterization of lentivirus and adenovirus by FFF-MALS

4:30 pm
Afternoon Networking

5:00 pm Discussing Global Technology Transfer and Commercialization of Next-Gen mRNA Therapeutics & Vaccines

Synopsis

  • The novel process development and manufacturing technologies for mRNA therapeutics and vaccines
  • Current challenges and success in large-scale process development of latest mRNA vaccines
  • Global technology transfer, process validation and rapid commercialization

5:30 pm Asking the Question: How to Scale up LNPs With Control From Lab to GMP?

Synopsis

  • Defining implementation of characterization methods to enable robust validation
  • Establishing quality control tests and analysis to achieve GMP manufacturing

6:00 pm Chair’s Closing Remarks & End of Summit