Conference Day Two: Wednesday, April 16
7.15 Check-In & Coffee with Light Breakfast
8:15 am Chair’s Opening Remarks
Addressing Opportunities & Challenges in IP, Collaboration & Licensing to Fast-Track Efficacious & Cell Specific LNPs Towards the Patients in
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 are innovations in LNP delivery important for achieving potent & efficacious drug products?
· How to identify complementary collaborators and partnerships to enable target specific delivery for a variety of payload types
· How to choose partners & CDMOs to accomplish large-scale manufacturing for accelerating more LNPs onto the market
9:00 am Navigating IP Challenges for Companies Utilizing Lipid Nanoparticle Delivery Systems to Enable Freedom to Operate & Mitigate Infringement Risk
Synopsis
• Providing an overview of key freedom-to-operate challenges facing therapeutic developers operating in this
• How to ensure you have high-quality patent protection earlier in the drug development pipeline
• Covering advanced solution-based strategies for de-risking the therapeutic program from an IP perspective
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
10.15 Morning Networking Break
CHAIR - Magali Hickey, Independent Consultant
Enhancing LNP Analysis by Uncovering Strategies to Identify LNP Structure & Encapsulation Efficiency
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 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 LNPs beyond the basic characterization with dynamic light scattering: What else should be considered for the characterization toolkit?
- Applying orthogonal analytical testing strategies to better understand manufacturing process impact on formulation critical quality attributes
- Gaining deeper understanding of encapsulated LNPs: quantitative and qualitative analytics
Erika Jensen, Principal Scientist, Pfizer
Lipa Shah, Director, Science & Technology, Technical Research & Development 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
1.00 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
1.30 pm Single LNP Characterization for Formulation & Process Development: Understanding Payload & Targeting Molecule Composition on Single LNPs
- Learn how nanoflow cytometry techniques reveal the details of your LNP formulations including
size, concentration (particles/ml) payload distribution, in terms of both loaded and unloaded
particles, and quantification of the payload per particle - Understand the display of surface markers on each LNP to determine how active the targeting molecules attached to your LNP are
- With low sample requirements, quick easy assays, and simple data collection tools from NanoFCM are allowing formulation and process development groups to gather data that provides granular, detailed data regarding the state of the sample
Clayton Deighan, North American Sales & Applications Manager, NanoFCM
1.45 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
2:45 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
3:15 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
3:45 pm CMC & Allied Challenges in LNP Formulation for Ocular Delivery
Synopsis
- Purification of LNP at scale and membrane technology
- Surface modification of LNP and its characterization
- Regulatory landscape and challenges for LNP in ocular space