7:30 am
Registration & Morning Coffee

8:30 am Chair’s Opening Remarks

  • Gilles Besin Vice President, Head, Discovery Research, Affinivax Inc.

Advancing the Formulation of Your LNP to Further Its Therapeutic Potential

8:45 am Roundtable Discussion:In-Depth Discussion of the LNP Landscape & Future Opportunities

  • Mohamed Elsayed Senior Research Fellow, Biotechnology Discovery Research, Eli Lilly & Co
  • P.J. Brooks Program Director, NCATS, NIH
  • Rui Zhang Associate Director, LNP Formulation, Tessera Therapeutics
  • Jane Wang Head of LNP Formulation, Tessera Therapeutics
  • Luis Brito Vice President, Delivery Platform, Beam Therapeutics

Synopsis

  • An insightful summary of the current position of the LNP field following the use of LNPs in the COVID-19 vaccine
  • Discussing the current limitations and challenges of LNPs, including difficulty scaling up GMP manufacturing and issues with maintaining the stability of LNPs
  • Highlighting key questions to be addressed throughout the duration of the conference
  • Analyzing future opportunities in the field and summarizing the large pharmaceutical potential of LNPs

9:45 am Discussing the Composition of LNPs With Respect to Their Efficacy & Function

Synopsis

  • Laying out the importance of choosing the correct components of LNPs
  • Discussing the optimal ionizable lipid pKa level to enhance efficacy
  • Analyzing the ratios of LNP components and how modulation of these ratios affects LNP characteristics
  • Evaluating operating and formulation parameters which influence the physiochemical characteristics of the particles, including the flow rate ratio (FRR) and total flow rate (TFR)
  • Optimizing your LNP formulation for mRNA, siRNA and larger nucleic acids

10:15 am Delivering LNP Beyond the Liver: Enhancing Formulation of LNPs With Target Destination in Mind

Synopsis

  • Comparing the formulation parameters required for delivery to the liver, cardiology, central nervous system, and muscles
  • Discussing advancements in formulation technology required to successfully progress the delivery of LNPs beyond the liver
  • Reviewing the therapeutic potential of LNPs when delivery beyond the liver is possible

10:45 am Smoothing Your LNP Tech Transfer & Establishing Robust CDMO Partnerships with Exelead

Synopsis

  • Understanding how CDMOs can reduce costs and development time for pharmaceutical companies via tech transfer
  • What to look for in CMDO tech transfers: including experience in all stages of manufacturing, methods of drug development techniques, appropriate facilities, cost efficient, and efficient supply chain, regulatory support, expertise with complex products and an understanding of international standards and requirements
  • Developing a tech transfer framework and plan
  • How to maintain smooth communication between the vendor and manufacturer

11:15 am
Structured Networking & Morning Coffee Break

Synopsis

This session is the ideal opportunity to get face-to-face time with many of the brightest minds working in the LNP field and establish meaningful business relationships to pursue for the rest of the conference.

12:15 pm Reviewing the Ability to Test Thousands of Nanoparticles in vivo Using DNA Barcodes

  • James Dahlman Associate Professor, Georgia Institute of Technology

Synopsis

  • Evaluating the use of a high-throughput DNA barcoding system which has provided the ability to test thousands of nanoparticles in vivo
  • Discussing how this breakthrough technology could dramatically accelerate the discovery and understanding of nanoparticle drug delivery
  • Analyzing species-dependent difference in nanoparticle delivery and the effects
    on efficacy and function
  • Reviewing exciting progress suggesting that LNPs can target non-liver tissues without targeting ligands, and the implications of this discovery

12:45 pm Addressing the Key Challenge Associated With LNPs – Enhancing Long Term Stability to Maximize Drug Product Quality & Shelf-Life

Synopsis

  • Understanding the effects of freezing on LNP suspensions
  • Evaluating the additional stress of drying in the lyophilization process
  • Discussing the mechanisms of instability during storage
  • Considering formulation components for combatting instability in the dried state

1:15 pm Flexible LNP Formulation, Process Development & Manufacturing Solutions

Synopsis

  • A brief overview of the manufacturing challenges faced by LNP innovators
  • Discussing the importance of flexibility in a CDMO partner to match the speed and flexibility promise of LNPs

1:45 pm
Lunch & Networking

Enhancing the Formulation of Your LNP to be the Winning Vehicle

2:45 pm Discussing the Lessons Learnt From the SARS-CoV-2 Vaccine & Current LNPs & Evaluating the Future Directions for this Novel Technology

  • Anthony Barry Executive Director, Biotherapeutics and Technologies, Pfizer

Synopsis

  • Examining some of the key challenges with the COVID-19 vaccine
  • Discussing novel approaches to mitigate known challenges
  • Identifying and evaluating new techniques to adapt current formulations to address challenges and expand the potential utility of the mRNA modality

3:15 pm Using LNPs for mRNA-based Therapy of Liver Cancer

  • Dan Duda Associate Professor, Harvard Medical School

Synopsis

  • Discussing the potential therapeutic use of LNPs produced for the COVID-19 vaccine for alternative therapeutic modalities
  • Zooming in on the potential to use LNPs for mRNA-based therapy of liver cancer
  • Evaluating the future use of this technology in oncology

3:45 pm Molecular Design Principles for Lipid Nanoparticle Mediated Delivery of mRNA

Synopsis

  • AexFactor: How Lipid Ionization Characteristics Drive for Structure Activity Relationships and Biodistribution
  • AexBoost: LNP Assembly Techniques that Multiply Potency

4:15 pm
Afternoon Networking & Refreshment

4:45 pm Refining the Balance Between Dosing & Toxicity With Respect to Formulation

Synopsis

  • Reviewing previous clinical data which highlights toxicity as a safety hazard when using lipid-based vectors
  • Identifying formulation parameters required to overcome challenges in the toxicity of lipid-based vectors
  • Discussing the implications of using higher and more frequent dosage for alternative therapeutic usage on issues with toxicity and immunity

5:15 pm Beyond the Liver: the CMC development journey of the first systemic LNP-mRNA

Synopsis

  • A brief overview of the manufacturing, analytical and stability challenges encountered
  • Discussing the ability to go above the manufacturable particle size limit in GMP environment

5:30 pm Utilizing PEGylation as a Strategy to Improve Nanoparticle-based Drug & Gene Delivery

Synopsis

  • Discussing the implications of high-throughput workflow for LNP formulation and characterization
  • Analyzing the impact of PEG-lipid on the physiochemical properties and cellular interactions of LNPs
  •  Evaluating the challenges associated with translation to scalable microfluidic formulation method

6:00 pm Chair’s Closing Remarks

6:15 pm Networking Drinks Reception

Synopsis

After the formal presentations have finished for the afternoon, the networking carries on. The Networking Drinks Reception will allow you to continue connecting with your peers in a relaxed atmosphere, explore our exhibition booths and forge new and existing relationships.