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

Synopsis

  • 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

Synopsis

  • 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

Synopsis

  • 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

Morning Break & Speed Networking

Synopsis

Enjoy a morning coffee while networking with fellow experts

10:40 am
Track A:

LNP Discovery, Biology & Characterization

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

Synopsis

  • 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 AD, Vertex Pharmaceuticals

Synopsis

  • 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

Synopsis

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

Synopsis

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


Track B:

Formulation Considerations & Stability Implications

10:45 am Enhancing Stability & Shelf Life

Synopsis

Session Reserved for Avanti Polar Lipids

11:00 am Panel: Discussion Discussing the Importance of Storage Capabilities & Storage Conditions
Moderated by: Zohreh Amoozgar

Synopsis

  • How to ensure your formulation is viable to provide the population with the best therapeutic available
  • How much does lack of storage capabilities, or requiring conditions that are not feasible limit progression?
  • Identify formulation design, and processing improvements to allow storage at more routine temperatures

11:30 am Utilizing Freeze / Spray Drying for LNPs

  • Sune Andersen Principal Scientist - Spray Drying, Janssen Research & Development, LLC

Synopsis

  • 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 Outlining Characterization of Ionizable Lipids & LNP Stability

Synopsis

  • Discussing forced degradation of ionizable lipid
  • Exploring stability of LNP’s when formulated with known impurity
  • Outlining the subsequent interactions with encapsulated payload

10:15 am Track C

LNP Manufacturing & CMC

10:45 am Lipid Assembly & Scale Up

Synopsis

Session Reserved for Helix Biotech

11:00 am Panel Discussion: Meeting Demand: Scaling Up Your LNP
Moderated by: Lubo Nechev
  • Lubomir Nechev Sr. Vice President, CMC Development, Alnylam Pharmaceuticals
  • Dennis Lee Senior Program Officer, Bill & Melinda Gates Foundation

Synopsis

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

11:30 am GMP Manufacturing, Fill & Finish, Tech transfer

Synopsis

Session Reserved for Knauer

11:45 am Lipid Assembly & Scale Up

Synopsis

Session Reserved for Arrantabio

12:15 pm Q&A with Expert Speakers

12:30pm Networking Lunch

Track A:

LNP For Targeted Delivery

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

Synopsis

  • 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 Professor and Vice President for Research and Development, Tel-Aviv University

Synopsis

  • 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

Synopsis

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

Track B:

Downstream Process Development

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

  • Jayant Arora Principal Scientist - Product & Process Development Group Leader, Johnson & Johnson

Synopsis

  • 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

Synopsis

  • 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 and Analytical Sciences, Omega Therapeutics

Synopsis

  • 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 – LNP Manufacturing & CMC

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

Synopsis

  • 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

Synopsis

  • 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 & Process Control, Entos Pharmaceuticals

Synopsis

  • 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 Exploring Delivery & Therapeutic Approaches: In Vivo Reprogramming Tools for CAR-T Cells Directly In Vivo

Synopsis

  • 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

  • Priya Karmali Vice President, Technology Innovation and Development, Arcturus Therapeutics

Synopsis

  • 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

Synopsis

  • 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