Conference Day Two: Wednesday, April 16
7.15 Check-In & Coffee with Light Breakfast
8:15 am Chair’s Opening Remarks
Uncovering Opportunities for Partnering, Collaborating & Licensing to Fast-Track Efficacious & Cell Specific LNPs Towards the Patients in Need
8:30 am Panel Discussion: Forging New Alliances in the LNP Industry with Strategic Partnerships & Collaborative Opportunities to Accelerate More LNPs onto the Market
Synopsis
- How to identify complementary strengths and weaknesses in other biopharma companies to optimize partnership capabilities
- How to decide who invests in which resources and who gains which returns
- What thought processes and strategies should be put in place for determining successful partnerships between biotech/biotech and biotech/pharma?
9:00 am Navigating Licensing Agreement Challenges to Capitalize on Using More Novel Lipids for Improved Targeted Delivery
Synopsis
- Effectively navigating deal terms to ensure accomplishment of partnership goals
- Showcasing strategies for accelerating to the final point in the transaction process
- Aligning the key economics and qualities of a good licensing partnership to enable commercial success on both sides of the deal
9:30 am LNP Downstream Processes: From Dialysis to TFF & Buffer Exchange Impact
Synopsis
- Small scale downstream purification processes
- Choice of exchange buffers
- TFF concentration and diafiltration process
10.00 LNP Awards & Morning Networking Break
CHAIR - Magali Hickey, Independent Consultant
Uncovering Strategies to Identify LNP Structure & Encapsulation Efficiency for Improving LNP Analysis
11.00 am Optimizing LNP Analysis using Small Angle X-Ray Scattering, Cryo-EM & LNP
Research-Grade Test Materials for More Efficacious Therapeutics
- Leveraging Small small angle x-ray scattering for understanding the relationship between LNP formulation and structure
- Coupling small angle x-ray scattering and Cryo-EM for improved LNP analysis
- Producing, homogeneity testing and characterizing of a NIST LNP/RNA research-grade test material
Thomas Cleveland, Research Physicist, NIST
11.30 am Roundtable Discussion: Defining an LNP Analytical Roadmap to Standardize Testing Methods for Full Characterization of LNPs
- Evaluating whether understanding LNP size is sufficient, or if more understanding of LNP shape is required
- How to orthogonal analytical testing strategies to gain whole LNP understanding?
- How to gain both qualitative and quantitative data for deeper understanding of how many LNPs have payload completely encapsulated?
Erika Jensen, Principal Scientist, Pfizer
Lipa Shah, Director, Novel Delivery Systems, Novartis
12.00 pm Lunch Break & Networking
Improving LNP Characterization by Delving in Advanced Tools & Potency Assays for Fast-Tracking Regulatory Compliant & Stable LNPs towards the Market
13.00 pm Delving into Gaps of LNP Characterization to Achieve Improved Nucleic Acid Delivery
- Evaluating advanced tools used for deeper characterization of LNP delivery vehicles
- Discussing how to better inform design of these LNPs using various advanced characterization tools
- Bridging the gaps for improved understanding of LNP characteristics for designing more
effective delivery systems
Yan Xia, Head, Nanotechnology Formulations, Axelyf
13.30 pm Session Details to be Revealed
13.45 pm Delving into Potency Assay Strategies for LNP-mRNA Therapies to Enable Accurate Potency & Efficacy Testing for Rare Diseases
- Challenges of developing functional assays for rare diseases
- Phase appropriate strategy for potency assay development
- Controlling variability in cell-based potency assay
Rumpa Bhattacharjee, Senior Scientist II, ReCode Therapeutics
2:15 pm Afternoon Networking Break
The Future of LNPs: Illuminating Next Generation LNPs for Delivering Nucleic Acid Therapies to the Patients in Need
3:15 pm Beyond the liver: Engineering the Fusogenix PLV Platform for Extra-Hepatic Delivery of RNA & DNA
Synopsis
- Development of a fusion-powered delivery system for RNA and DNA
- Optimization of formulation to achieve extra-hepatic delivery with maximum tolerability
- PLV-mediated delivery of RNA and DNA to tissues and cells of the lung, eye, and muscle
3:45 pm Development of SHARP: A Novel Next-Generation Non-Viral Delivery Technology for mRNA
Synopsis
- The innovative SHARP (Single-component Homogeneous Amphiphilic Routed Particles) delivery technology demonstrates exceptional stability comparing to the four-component lipid nanoparticle systems
- SHARP technology paves the way for functionally delivering nucleic acids beyond the conventional lipid nanoparticle systems
- SHARP efficiently delivers mRNA cargo to liver as well as to extrahepatic organs