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 NIH funding for technologies to deliver RNA and genome editors to specific organs
Synopsis
- Examples and results from the NIH Somatic Cell Genome Editing Consortium
- Prize competition for delivery technologies to target genome editors to specific organs and across the blood-brain barrier
- Other NIH funding opportunities for RNA delivery
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
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
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
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 A novel method using ultrasound to characterize LNP
Synopsis
- Ultrasonic Pulsed Doppler (USPD) is a novel method employing Ultrasonic backscatter to characterize nanoparticles
- USPD can be used for batch measurements of nanoparticle concentration and size distribution or can be incorporated into existing equipment for in situ real-time measurements.
- Data suggests that USPD can be used to measure LNP cargo uptake.