Conference Day Two
Tuesday, April 18 2023

7:30am Registration & Morning Coffee

8:20 am Chair’s Opening Remarks

Setting Industry Standards & Genome Editing Applications

8:30 am Addressing the Importance of Setting Standards in Excipients

  • Anni Chang Senior Scientist - Scientific Liaison, US Pharmacopeia


  • Outlining a USP introduction and standard setting process
  • Identifying lipid standards and on-going work focusing on phospholipids
  • Discussing emerging standards context and applicability to LNP test methods

9:00 am Non-Viral Delivery of RNA & Genome Editing Tools


  • Discussing broadly non-viral formulations for in vivo delivery of RNA to a range of tissues
  • Developing new lipid formulations for delivery of RNA to the lung
  • Evaluating new formulations for delivery of RNA to hematopoietic stem cells

9:30 am Genome Editing Using LNPs: New Methods & Insights


  • How do LNPs used for gene editing applications differ from traditional mRNA LNPs?
  • New characterization technique that guide optimization of LNPs for gene editing applications
  • Understanding how nucleic acid size can modulate LNP loading

10:00 am
Morning Break & Speed Networking


Enjoy a morning coffee while networking with fellow experts

10:40 am
Track A:

LNP Discovery & Characterization

Track Chair: Roger Adami, Vice President, Pharmaceutical Sciences and Business Development, Nitto BioPharma

10:45 am Cryo-Transmission Electron Microscopy: An Essential Characterization Technique For Nucleic Acid- Based Therapeutics & Vaccines


  • Cryo-TEM directly observes individual particles in their near-native state, and simultaneously providing information on multiple characteristics
  • Demonstrating how cryo-TEM can aid in formulation development, process development, batch or lot comparisons, and long-term stability / storage condition assessments
  • Discussing the challenges of using this technique to

characterize LNPs

11:00 am Translational PK/PD & Immunogenicity of LNP Encapsulated mRNA Therapeutics with Repeated Dosing

  • Tao Niu Associate Director - Modeling & Simulations, Vertex Pharmaceuticals


  • PK/PD of the mRNA and expressed protein in preclinical species and human
  • Pharmacokinetics, particularly accumulation of LNP Lipids with repeated dosing
  • Immunogenicity of various components in the LNP encapsulated mRNA therapy with repeated dosing

11:30 am Nonclinical Safety Strategy Landscape of mRNA Vaccines to GoSafe & GoFast in Clinics


  • Understanding that mRNA platform-based vaccines have changed the vaccine development landscape
  • Delving into nonclinical safety assessment as a critical step to ensure the safety of mRNA vaccines and exploring challenges in the guidelines
  • Can we look beyond more classical approaches and customize the nonclinical safety strategy?

12:00 pm Assessment of LNP Quality & Encapsulation Efficiency by Nano-flow Cytometry


  • 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  

12:15 pm Panel Discussion: Extra-Hepatic Delivery of LNP
Moderated by: Manuel Sanchez-Felix, Senior Fellow, Novel Delivery Technologies, NIBR

  • Paul Peng Director - Oligonucleotide Technical Development, Biogen
  • Manuel Sanchez-Felix Associate Director, Novartis AG
  • Neha Kaushal Principal Scientist, Sanofi mRNA Center of Excellence
  • Xiaoyong Lu Director - Chemistry, Ribonucleic Acid Delivery & Intellectual Property inventions, Sirnaomics Inc.
  • Lloyd Jeffs Senior Director - Clinical Manufacturing Solutions, Precision NanoSystems


It’s important to understand how we get the LNP or the cell type we want, without having everything accumulated in the liver.

Your panelists will discuss:

  • How to move outside the liver
  • Liver shielding techniques
  • De-targeting the liver so LNP is not being actively accumulated

12:45 pm End of Track

Track B:

LNP Formulation & Process Development
Track Chair: Mohit Gupta, Senior Scientist, Tessera Therapeutics

10:45 am Enhancing Stability & Shelf Life


Session Reserved for Avanti Polar Lipids

11:00 am Wacker Biotech – Your Partner for Manufacturing of Nucleic Acids from R&D to Commercial

  • Hagen Richter Manager Nucleic Acids, Wacker Chemie Pensionskasse


  • Company Presentation WACKER & Wacker Biotech
  • Overview Wacker Biotech Capabilities in Nucleic Acid Manufacturing
  • Overview WACKER Capabilities in R&D Services Around Nucleic Acid Manufacturing  

11:30 am Utilizing Freeze / Spray Drying for LNPs


  • Exploring uses of freeze / spray drying for improved stability
  • Preventing damage and protecting nanoparticle integrity
  • How can we make formulation more stable?

12:00 pm Characterization of Lipid Nanoparticles Using Biophysical Methods

  • Salette Martinez Senior Scientist - Biophysical Analytical Development, Beam Therapeutics


  • Overview of biophysical methods for LNP characterization
  • Applying novel analytical tools to study LNP case study 1: RNA encapsulation
  • Applying novel analytical tools to study LNP case study 2: protein binding to LNP

Track C:

LNP Scale Up & Manufacturing
Track Chair: Sujit Jain, Director, External Manufacturing, SalioGen Therapeutics

10:45 am Helix Biotech – LNP Development


  • LNPs have emerged as a promising platform for gene therapy and gene editing, with potential applications covering a wide range of diseases and conditions
  • Helix Biotech has developed manufacturing systems and built new facilities specifically to support the development, manufacturing, and analysis of emerging RNA and DNA LNP Drug Products
  • From discovery and development to scaling up with GMP manufacturing, hear about recent developments and lessons learned from an LNP CDMO

11:00 am Panel Discussion: Meeting Demand: Scaling Up Your LNP
Moderated by: Lubo Nechev

  • Sujit Jain Director - External Manufacturing, SalioGen Therapeutics
  • Lubo Nechev Senior Vice President - Chemistry, Manufacturing & Controls Development & Process, Analytical Sciences, Alnylam Pharmaceuticals
  • Jason Coleman Clinical Application Scientist Lead, Precision NanoSystems


  • CMC strategies for LNP
  • Scaling up pending on your demand
  • Accelerating medicines for patients

11:30 am Advancing LNP Formulation: Next Generation Impingement Jets Mixing Systems


  • From formulation to GMP production: up-scaling of LNP formulation
  • Expanded functionality of the IJM NanoScaler: two-channel eluent heater 
  • Flexibility and automation of LNP formulation: automated high throughput screening and sample collection

11:45 am End to End Process Development & Manufacturing Solutions for Lipid Nanoparticles


  • Address supply chain challenges in non-GMP and GMP LNP manufacturing
  • Scalability from benchtop to GMP manufacturing
  • Flexibility to tailor to client needs for pre-clinical and clinical drug products

12:15 pm Q&A with Expert Speakers

12:30pm Networking Lunch

Track A:

LNP Discovery & Characterization

Track Chair: Roger Adami, Vice President, Pharmaceutical Sciences and Business Development, Nitto BioPharma

1:30 pm Delivering Genome Editors to Target Tissues Other Than The Liver: Current Progress & New Opportunities


  • Non-viral delivery of somatic cell genome editing across the BBB
  • Implementing independent validation
  • Identifying cell-type specific delivery

2:00 pm Targeted LNPs for Cell Type Specific Delivery of RNAs

  • Dan Peer Vice President - Research & Development, Tel-Aviv University


  • Discussing cell types specific coating of LNPs with targeting moieties
  • Examples of LNPs payloads such as siRNAs, mRNAs and sgRNAs
  • Exploring scale up and CMC challenges and opportunities

2:30 pm Brain Targeting LNPs for ASO Delivery in CNS

  • Paul Peng Director - Oligonucleotide Technical Development, Biogen


  • Designing brain targeting LNPs (BLNPs)
  • Identifying in vitro/in vivo correlation challenges
  • Exploring animal studies to show improved biodistribution using BLNPs

Track B:

LNP Formulation & Process Development

1:30 pm Roundtable Discussion: Future of Thermostable LNP Drug Products: Challenges & Opportunities

  • Jayant Arora Associate Director - Non Viral Gene Delivery, Johnson & Johnson


  • Strategies to enable thermostable LNP drug products and their impact on the current state of the supply chain
  • Various challenges pertaining manufacturability, supply chain and regulatory aspects associated with above mentioned strategies
  • Beyond LNP: alternatives to LNPs to attain scalable, stable and efficacious non-viral delivery of RNA constructs

2:00 pm LNP Process Development Considerations


  • LNP mixing technologies
  • Impact of mixing parameters on product attributes
  • Role of structural characterization towards process understanding

2:30 pm Outlining LNP Stability Considerations

  • Jeffrey Atkinson Executive Director - Process Development & Analytical Sciences, Omega Therapeutics


  • Understanding the challenges of freeze/thaw cycles from manufacturing to clinic
  • Evaluating product freeze/thaw stability for product understanding
  • Managing freeze/thaw challenges

Track C:

Scale Up & Manufacturing
Track Chair: Director, External Manufacturing, SalioGen Therapeutics

1:30 pm Partnering With Suppliers & Managing the Supply Chain, Developing Procurement Strategies for Accelerated Drug Development

  • David Karakas Senior Director - Procurement & Strategic Sourcing, Tessera Therapeutics
  • Ryan Beck Director, Supply Chain, Tessera Therapeutics


  • Maturity – impact of internal and external capabilities on transition from R&D to tox to launch
  • Capabilities – supplier capabilities and impact on procurement strategies
  • Partnering – Ideas on how to build a strong relationship across Supply Chain and Procurement

2:00 pm Roundtable Discussion: Delving into Fill-Finish Techniques


  • Barriers faced in finishing the process of packaging the drug for distribution
  • Exploring the importance of fill finish to move the product from one region to the other, storage and protection
  • Reducing the risk of contamination of your product during the fill finish

2:30 pm Exploring An Overview of GMP Manufacturing of LNPs

  • Ping Wee Lead Scientist - Formulations and Process Control, Entos Pharmaceuticals


  • Outlining considerations for beginning GMP manufacturing for LNPs
  • Performing GMP manufacturing
  • Quality Control testing of GMP LNP products

3:00pm Afternoon Break & Networking

The LNP-Payload Journey, Cell Therapy & Re-Establishing Mindsets

3:45 pm Adapting LNP System to Payloads Beyond mRNA – DNA

4:15 pm Reprogramming CAR-T Cells Directly In Vivo


  • Directly inserting a targeting moiety via SF or nanobodies into the nanoparticle itself
  • Not relying on this specific delivery to the lipid particle itself but target engagement
  • Targeted delivery through a targeting moiety

4:45 pm Capstan In Vivo Platform – Transforming Cell Therapy


  • Using LNP for in vivo Cell Engineering
  • Exploring the untapped potentials of LNP-CAR for curative immunotherapies including fibrosis, oncology, autoimmune etc
  • Scaling this up into a robust and viable platform

5:15 pm Enabling World Wide Access to State of the Art Targeted LNPs for the Development of Vaccines against a Range of Infectious Diseases


  • Engineering and design considerations for mRNA LNP vaccines developed for LMIC countries
  • Innovative business strategies to ensure access to low and middle income countries
  • Planning for local manufacturing and distribution

5:45 pm Chair’s Closing Remarks

5:50 pm End of Conference Day Two