Conference Day Two
Thursday, August 22

8:00 am Morning Coffee & Networking

8:55 am Chair’s Opening Remarks

Empowering Extra-Hepatic Delivery With Confidence Through Advanced LNP Technologies

9:00 am Panel Discussion: Revolutionizing Drug Delivery: Next-Gen Lipid-Based Therapies

Synopsis

  • Pushing the boundaries of lipid-based delivery systems beyond the realm of LNPs to explore advancements in tailoring lipid nanoparticles for precise drug targeting and enhance therapeutic efficacy
  • Highlighting the emergence of different intelligent lipid formulations responding to specific physiological cues to accelerate personalized lipid-based therapies
  • Exploring how each next-gen lipid-based therapies go beyond conventional drug delivery barriers overcoming challenges, such as targeted delivery and specificity

9:45 am PEG Alternatives for Increased Therapeutic Success With LNPs Outline

  • Hema Choudhary Postdoctoral Fellow, Innovative Genomics Institute University of California, Berkeley

Synopsis

  • Developing novel polymers for substituting PEG in LNPs
  • Showcasing breakthrough synthesis technologies and formulations.
  • Outperforming traditional PEG-stabilized LNPs in in-vivo transfection and non-immunogenicity

10:15 am Opportunities & Challenges for Extrahepatic Delivery of Therapeutic mRNA

Synopsis

  • Physicochemical properties of lipid-nanoparticles impact on their delivery capabilities depending on the administration route
  • Discussion of extrahepatic delivery and therapeutic opportunities in the lung
  • Challenges for preclinical translation associated with LNP delivery capabilities

10:45 am Morning Refreshment Break & Networking

11:15 am Uncovering the Potential of LNPs as an Innovative Therapy for Solid Tumors

  • Jieni Xu Principal Scientist, LNP Formulation, Kernal Biologics

Synopsis

  • Delving into the strategies employed by LNPs to navigate the complex tumor microenvironment, enhancing their ability to infiltrate solid tumors and deliver therapeutic payloads with precision
  • Unleashing the versatility of LNPs as a potent delivery tool for various therapeutic modalities, from chemotherapy agents to gene therapies, aimed at addressing the unique challenges posed by solid tumor indications
  • Discussing how LNPs contribute to the emerging field of precision oncology, tailoring drug delivery to individual tumor characteristics, paving the way for more effective and personalized treatment strategies

11:45 am A next generation PEG-free LNP technology that actively targets myeloid cells through selective binding to the myeloid lectin DC-SIGN receptor

Synopsis

  • Pullulan LNPs are PEG-free and selectively targets macrophages and dendritic cells through specific binding to the C-type lectin receptor DC-SIGN.
  • PEG-free Pullulan LNP could avoid PEG-dependent undesirable reactions such as allergy, anaphylaxis, and accelerated blood clearance
  • Pullulan LNPs are ideal for tumor environment modulation, cancer vaccines, and infectious disease vaccines

12:15 pm Elucidating & Overcoming PEG-Directed Immunity Against PEGylated LNPs

  • Sam Lai Professor, UNC Eshelman School of Pharmacy, University of North Carolina

Synopsis

  • What is the prevalence and magnitude of pre-existing immunity against polyethylene glycol (PEG), a polymer used in vast majority of LNP formulations?
  • Clinical evidence on LNP-induction of anti-PEG antibodies (APA), and impact on LNP medications
  • Methods to overcome pre-existing APA and mitigate PEG-specific immunogenicity and hypersensitivity

12:45 pm Lunch Break & Networking

1:45 pm Formulation Characterization of DNA-mRNA Dual-Payload LNPs for Photoreceptor-Specific Delivery

  • Shashank Shukla Principal Scientist - Drug Delivery, SalioGen Therapeutics

Synopsis

  • Novel dual cargo formulation
  • Use of specialized techniques like AF4, CryoEM and others to characterize novel LNPs
  • Targeting of photoreceptors via these LNPs

2:15 pm Beyond the Liver: CureVac’s Novel LNP Platform for Spleen Targeting

Synopsis

  • Unveiling CureVac’s innovative approach: explore the cutting-edge design of lipid nanoparticle (LNP) formulations tailored for precise spleen targeting
  • Beyond liver-focused delivery: discover how CureVac’s groundbreaking strategy extends the reach of LNPs
  • Gaining valuable perspective by sharing the rationale behind CureVac’s spleen-targeting LNPs

Unleashing the Power of Next-Gen Lipid Nanocarriers & Payloads

2:45 pm In Vivo Engineering of Cells Using Targeted Lipid Nanoparticles

Synopsis

  • Capstan’s novel targeted LNP (tLNP) platform purpose-built for preferential delivery of RNA to target cells
  • tLNP platform enables two distinct approaches in vivo: direct engineering of immune cells and targeting pathogenic cells
  • Versatility of this non-viral, redosable and scalable platform allows for in vivo RNA-based therapies across broad range of diseases

3:15 pm Afternoon Break & Refreshments

3:45 pm The Endosomal Escape Vehicle Platform Enhances Lipid Nanoparticle-Mediated Delivery of Genomic Medicines

Synopsis

  • Lipid nanoparticle (LNP) technology is promising for delivering nucleic acid therapeutics like mRNA vaccines and gene editing tools. Despite challenges such as limited tissue targeting and low delivery efficiency, our proprietary Endosomal Escape Vehicle (EEV™) technology enhances LNP delivery of genomic medicines.
  • Our research shows that EEV-modified LNPs (EEV-LNPs) greatly enhance mRNA delivery and gene editing efficacy compared to standard LNPs in vitro. Through mechanistic studies, we’ve elucidated EEV-LNP’s uptake mechanism, revealing its ability to target diverse cell types and tissues. These findings underscore the potential of our EEV-LNP platform for enhancing genomic medicine delivery, particularly in mRNA and gene editing applications.

4:15 pm Roundtable Discussion: Beyond Limits: Mastering the Delivery of Complex Payloads With LNPs

Synopsis

  • Oligonucleotide orchestration: Unleashing the potential of LNPs in orchestrating the precise delivery of oligonucleotides, navigating the challenges associated with the complex structures of RNA-based therapeutics
  • Peptide Prowess: Showcasing the versatility of LNPs in delivering large peptides, discussing their role in overcoming barriers to efficient transport and enhancing the therapeutic impact of peptide-based drugs
  • Gene therapy symphony: Exploring the harmonious integration of LNPs in gene therapy, demonstrating their ability to efficiently deliver and express genetic payloads, including CRISPR-Cas9 systems and other gene therapies, for revolutionary therapeutic outcomes

4:45 pm Advancing Therapeutics Through Innovative Delivery Platforms

Synopsis

  • Versatile polymer architectures: Exploring the diverse landscape of polymer-based delivery platforms, from nanoparticles to micelles, showcasing their adaptability for encapsulating a wide range of therapeutic agents, including drugs, genes, and imaging agents
  • Controlled release strategies: Unraveling the sophisticated mechanisms of polymer-based controlled release, highlighting how these delivery platforms can provide sustained and targeted drug delivery, optimizing therapeutic outcomes while minimizing side effects
  • Biocompatibility and customization: Discussing the significance of polymer biocompatibility and the ability to customize properties, such as size, surface charge, and degradation rates, to tailor delivery platforms for specific medical applications, from cancer therapy to regenerative medicine

5:15 pm Chair’s Closing Remarks

5:20 pm End of Conference