Conference Day Two- November 16, 2023

8:00 am Morning Networking Coffee

8:50 am Chair’s Opening Remarks

  • Susan Rosenbaum Founder, Chairwoman & Chief Executive Officer, Lauren Sciences

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

  • Andrew Geall Co-founder & Chief Development Officer, Replicate Bioscience

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

  • Michael Coe Chief Scientific Officer, Phoreus Biotech

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

  • Susan Rosenbaum Founder, Chairwoman & Chief Executive Officer, Lauren Sciences

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

  • Jay Kim Co-Founder & Chief Executive Officer, K2B Therapeutics

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

  • Mano Manoharan Senior Vice President - Innovation Chemistry and Distinguished Scientist, Alnylam Pharmaceuticals

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

  • Susan Rosenbaum Founder, Chairwoman & Chief Executive Officer, Lauren Sciences
  • Andrew Geall Co-founder & Chief Development Officer, Replicate Bioscience
  • Liping Zhou Senior Director, Advanced Drug Delivery, AstraZeneca

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

3:45 pm Chair’s Closing Remarks & End of the 2nd Annual Non-Viral RNA Delivery Systems Summit

  • Susan Rosenbaum Founder, Chairwoman & Chief Executive Officer, Lauren Sciences