Focus Day
Wednesday, April 19 2023

7:30 am Morning Coffee & Networking

Track A:

Discovery, Screening, Biology & Chemistry

Workshop A – 8.30 – 11.30

8:30 am Implications of LNP Immunogenicity
Workshop Leader: David Peritt

  • David Peritt Founder & Chief Scientific Officer, Lupagen
  • Roger Adami Vice President, Pharmaceutical Sciences and Business Development, Nitto BioPharma


As we consider LNP for the delivery of therapeutics for broader use in vivo, how does immunogenicity impact multi-dosing, use for multiple indications and pre-existing immunity from LNP formulated vaccines?

To progress, the industry needs to understand how to create LNP formulations with low immunogenicity to enable re-dosing with clinical confidence.

Track B:

Formulation & Process Development

Workshop D – 8.30 – 11.30

8:30 am Exploring Alternative Delivery Systems – Polymer Nanoparticles
Moderated by: Zohreh Amoozgar


Major breakthroughs in recent years in the field of nanotechnology outlines significant potential for nanomedicine. In the last 24 months, LNPs have stood in the spotlight as a promising delivery vehicle with clinical utility, but not without their drawbacks.

Polymer nanoparticles, with a long-standing history, offer their own advantages for drug development.

We will explore the advantages of polymer nanoparticles as an alternative delivery system to LNPs.

Track C:

CMC, Reference Standards & Scale-Up

Workshop G – 8.30 – 11.30

8:30 am Developing LNPs As An Effective Delivery Technology for Cell Therapy Applications
Workshop Leaders: Julie Shi, Brenna Kelley-Clarke, Melissa Hanson

  • Julie Shi Associate Director, Gene Delivery Process & Analytical Development, Bristol-Myers Squibb Co.
  • Melissa Hanson Senior Principal Scientist - Non-Viral Delivery & Nanoparticle Technology Platform Lead, Bristol-Myers Squibb Co.
  • Brenna Kelley-Clarke Senior Director - Gene Delivery Process & Analytics, Bristol-Myers Squibb Co.


With cell & gene therapies evolving as the future of LNP

development, many hurdles need to be crossed to ensure LNPs are an effective delivery technology:

  • Considerations when developing LNPs for ex vivo vs. in vivo applications
  • LNP scale-up to support cell therapy development
  • Delving into developing efficient formulations for payload transgene delivery
  • Looking into expanded scope of payloads including DNA

11:30 am Lunch & Poster Session

Workshop B – 12.00 – 3.00

12:00 pm Leverage Tools to Overcome the Lack of In Vitro & In Vivo Correlation


Considerations about LNPs in vitro characterization and the translation to in vivo evaluation.

Handling large amounts of data and interpreting this can be challenging, and learning how to navigate this in the LNP field can aid you in selecting the best LNPs.

Your leaders will discuss:

  • Exploration on LNPs functionality in vitro: up take & cargo release
  • Optimizing the in vitro platform early on to screen the different lipids
  • Selecting and rank order the most promising LNPs for in vivo studies
  • Outlining High-throughput analytical methods to support the HTS of LNPs and high-content data analysis to reveal the structural-activity relationship

Workshop E – 12.00 – 3.00

12:00 pm Outlining Available Routes of Administration & Novel Delivery Modes for LNPs


Addressing major bottlenecks in LNP administration methods is key, as well and understanding the implications on efficacy, toxicity, uptake. It is important to understand how LNPs are used for novel delivery methods – what are the benefits of this?

An overview of delivery and novel delivery modes of LNP in different ways: from blood, to inhalation, to BBB.

Workshop H – 12.00 – 3.00

12:30 pm Consistent Reference Standards for Characterization Testing of LNPs
Workshop Leaders: Thomas Cleveland and Mark Lowenthal

  • Thomas Cleveland Physicist, National Institute of Standards & Technology - NIST
  • Mark Lowenthal Research Chemist, National Institute of Standards & Technology - NIST


Finding gold-standard methods for characterization testing on both physical, chemical, and biological functionality of mRNA LNPs is vital for continued success of LNPs.

Join workshop leaders this interactive session to discuss:

  • Determining reference standards for innovative LNP platforms
  • Characterizing both the physical chemical and the biological functionality of mRNA LNPs
  • Establishing reliable methods and consensus methods for determining the potency of these mRNA-LNP products

3:00 pm Afternoon Break & Networking

Workshop C – 3.30 – 6.30

3:30 pm Advancing Gene Editing Applications for LNPs

  • Rahul Keswani Associate Director - Non Viral & Gene Therapy, Arbor Biotechnologies


Looking into the development and potential of LNPs, gene editing is a clear area of interest. Gene editing is attractive beneficial therapeutic option for treating various diseases.

Some barriers arise in the delivery of gene editing components.

LNPs can offer safe, efficacious promise for the future of gene


We will discuss considerations when using LNPs for gene editing,

off-target delivery implications and fine-tuning dosing strategy.

Workshop F – 3.30 – 6.30

3:30 pm Navigating Inherent Instability of LNP-Payload


Stability is one of the largest challenges faced in LNP development. It is important to be able to develop drugs that are more stable and have ease of distribution. Optimal stability of LNPs will enable longer shelf lives, ease of transportation and clinical utility.

Join this interactive workshop to understand the barriers and how can we achieve long-term stability.

Workshop I – 3.30 – 6.30

3:30 pm Delving into Continuous Manufacturing & Scale Up Techniques in LNP
Moderated by: Diane J Burgess

  • Diane Burgess Professor, University of Connecticut
  • Sujit Jain Director - External Manufacturing, SalioGen Therapeutics


Manufacturing LNP-mediated drugs is presented with its own challenges.

Join this interactive workshop where your leaders with discuss:

  • Considerations for continuous manufacturing
  • What are the scale up technologies available?
  • Going from PD manufacturing scale to commercial manufacturing
  • How to address waste volume when produced at scale

6:30 pm End of Focus Day