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 Session Reserved for ReciBioPharm
10.00 Morning Networking Break
Improving LNP Potency Using Cryo-EM & Assay Methods to Accelerate more Biologically Relevant LNPs towards Approval
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 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
12.00 pm Lunch Break & Networking
Standardizing Analytical Development for LNPs to Create a Clear & Robust Roadmap for Drug Developers Leading to More Regulatory Compliant LNPs
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 Reserved for Innovation Partner
13.45 am Roundtable Discussion: Defining an LNP Analytical Roadmap to Standardize Testing
Methods for Full Characterization of LNPs
- How to analyze LNP size and encapsulation of payload to minimize heterogeneity
- How to identify 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
Sudhakar Garad, Global Head, Pre-formulation, Novel Delivery & Biopharmaceutics, Novartis
2:15 pm Afternoon Networking Break
3:00 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
3:30 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