Conference Day Two- November 16, 2023
8:00 am Morning Networking Coffee
8:50 am Chair’s Opening Remarks
Breaking the Mold: Showcasing Novel & Next Generation Platforms to Advance Non-Viral Delivery of RNA Therapies
9:00 am Utilizing Recent Innovations in Non-Viral Delivery of RNA With LNPs, Polymers & Antibodies to Inform the Development of Your Own Delivery System
Synopsis
- Examining LNP delivery of 2nd generation self-replicating RNA to upgrade your LNP platform
- Developing your own polymers with a look at polymer delivery of 1st generation self-replicating RNA
- Determining future directions for nanoparticle delivery using lessons learned from antibody siRNA conjugates
9:30 am Peptide Nanoparticle (PNP) Delivery Platforms for the Next Generation of RNA Vaccines and Therapeutics
Synopsis
- The peptides in our PNPs do not stimulate the innate immune system and are less toxic to cells and tissues than lipid nanoparticle formulations
- PNP/mRNA conjugates are structurally and functionally compatible with both freeze dried and spray dried formulations
- Our carefully constructed patent protections provide a clear path for licensing and utilization of Phoreus PNP platforms in a wide variety of commercial applications
9:45 am Non-Viral Delivery of RNA & Genome Editors
Synopsis
- Discussion of progress mRNA delivery to lung
- Discussion of the development of targeting mRNA delivery to hematopoietic stem cells
- Development of next generation LNP and PNP formulations
10:15 am In Vitro and In Vivo Lipid Nanocrystal (LNC) Delivery of RNAi Oligonucleotides
Synopsis
- Lipid nanocrystals (LNCs) – characteristics and capabilities
- Recent in vitro and in vivo data with small oligos
- Future directions and opportunities
10:45 am Morning Break & Networking
11:15 am Panel Discussion: Discussing the Future of Non-Viral Delivery of RNA & Emerging Systems of Delivery Beyond LNPs
Synopsis
Join us for a thought-provoking panel discussion as experts delve into the future of non-viral delivery whilst exploring emerging systems of delivery beyond lipid nanoparticles (LNPs). Gain valuable insights and stay ahead in the rapidly evolving field of drug delivery.
- What are the ground-breaking advancements in non-viral delivery systems that are opening new frontiers for therapeutic delivery?
- How can you exploit the untapped potential of emerging delivery systems to revolutionize the way RNA is delivered to target cells?
- How are novel non-viral delivery technologies enabling precise and targeted drug delivery to improve therapeutic efficacy while minimizing side effects?
11:45 am Biodegradable Poly(amido)amine-Based Nanoparticles for Efficient Delivery of Nucleic Acids
Synopsis
- Optimizing the nanoparticle composition, formulation, and product characterization
- Promoting cellular uptake, endosomal escape, intracellular release, and efficiency of payload delivery
- Implementing the nanoparticles for vaccine and therapeutic applications
12:15 pm Networking Lunch
Beyond the mRNA Horizon: Creating Bespoke Non-Viral Delivery Systems to Solve the Delivery Challenges Faced by siRNA Payloads
1:15 pm Big Things Come in Small Packages: Non-Viral Delivery of siRNA across BBB for CNS Indications
Synopsis
- Enabling novel, non-invasive, targeted delivery of RNA to CNS for therapeutic applications
- Promoting targetspecific delivery in CNS to deliver RNA to the desired location
- Exploring various CNS delivery systems for efficacious delivery of RNA
1:45 pm Targeted Delivery of siRNA to Extrahepatic Tissues via a Novel Platform Using Protein-RNA Conjugate
Synopsis
- Discovering the first-in-kind protein-RNA conjugate (PRC) that enables targeted delivery of siRNA to extrahepatic cells
- Combining siRNA to a tumor receptor specific protein to generate many therapeutic candidates in the oncology sector
- Augmenting T-cell infiltration and recruitment of proinflammatory macrophages using KB101 to successfully inhibit tumor growth
2:15 pm Employing Conjugation Chemistry to Achieve Therapeutic Delivery of siRNAs to Reach Desired Tissues
Synopsis
- Modifying siRNA chemistry to better complex with conjugate delivery system
- Gearing conjugation chemistry for targeted siRNA delivery to elicit desired therapeutic effect
- Improving potency, specificity and safety of siRNAs for effective delivery to target site
2:45 pm Afternoon Networking Break
Streamlining Up & Down-Stream Process Development for Seamless Development of Non-Viral Nanoparticles to Accelerate Entry into Clinic
3:15 pm Panel Discussion: Increasing Collaboration Between Functions Such as Discovery, Formulation, Optimization & CMC to Accelerate Entry into Clinic
Synopsis
The successful development of a non-viral delivery system needs an immense amount of cross-functional collaboration between not just chemistry and biology focused teams, but also between functions such as discovery, formulation, process development, and CMC. Hence, it is important to develop a robust strategy to integrate various teams and increase collaborations.
This panel will delve into:
- Promoting cross-functional co-operation between teams to design an efficacious nanoparticle from discovery to CMC
- Incorporating scalability as an important criterion during early formulation to ensure smooth CMC processing down the line
- Integrating the chemical (delivery system) and biological (payload) aspects of a nanoparticle by encouraging cooperation between the respective teams