Conference Day Two
Thursday, June 26
8:00 am Light Breakfast & Check-In
8:55 am Chair’s Opening Remarks
Pioneering Next-Gen Formulation Techniques to Create Accessible Yet Viable Novel Delivery Systems
9:00 am PANEL DISCUSSION: Navigating Formulation, CMC, & Regulatory Challenges: Overcoming Barriers to Adopt New Delivery Systems
Synopsis
- What challenges do new delivery systems pose for formulation, CMC, and process development, and how can we overcome these to produce a new system at scale, speed and low costs?
- How can we design a CMC platform that can quickly adapt to the complexity of scaling up a novel formulation while maintaining consistent quality, stability, and performance?
- What results do we need to see to justify the investment in adapting a platform to a next-gen delivery system that introduces additional workload in formulation, CMC, and regulatory compliance?
9:45 am Engineering HDT Bio’s Thermostable LION/repRNA Lyophilized Vaccine to Increase Accessibility & Enable Localized Distribution
Synopsis
- Engineering HDT Bio’s non-LNP-based LION/repRNA lyophilized vaccine stored at 2-8°C and 25°C, facilitating practical storage and distribution, particularly in low resource settings
- Comprehensive screening of mono- and disaccharides (e.g., sucrose, trehalose, maltose) as lyoprotectants for the lyophilized vaccine, assessing their impact on particle size, repRNA integrity, and in vitro potency post-lyophilization
- Evaluation of in vivo biodistribution and efficacy of the LION/repRNA vaccine
10:15 am Overcoming Barriers in Lipid Based Nanoparticles for Stable Vaccines
Synopsis
- How to overcome significant challenges faced during discovery and development of novel lipid-based nanoparticles for stable vaccines
- Novel analytical tools that can help us to identify the root cause
- Optimize formulations and process to overcome these challenges
10:45 am Morning Break & Networking
Revolutionizing Cancer Treatment & Oral Nucleic Acid Delivery with Advanced Nanoparticle Design Strategies for Targeted Therapy & Enhanced Bioavailability
11:45 am Revolutionizing Cancer Treatment with Layer-by-Layer Nanoparticles for Targeted Tumor Therapy
Synopsis
- Designing versatile nanoparticles with high loading capacity for combinatory therapeutics, enabling effective delivery of RNA, proteins, and small molecules, such as doxorubicin and cisplatin
- Enhancing in vivo stability and enabling staged cargo release, ensuring optimal therapeutic efficacy while minimizing side effects
- Customizing surface chemistries for targeted tumor delivery, utilizing pH-sensitive polymers and specific ligands for selective binding to aggressive tumor cell receptors
12:15 pm Paving the Way for Oral Nucleic Acid Delivery with Advanced Formulation Strategies
Synopsis
- Addressing key challenges in oral delivery, including stability, bioavailability and efficient gastrointestinal absorption to unlock new therapeutic potential for nucleic acids
- Developing a versatile platform formulation for siRNA and mRNA to enhance stability and maximize efficacy in oral administration
- Showcasing stability and efficacy data that brings us one step closer to making oral nucleic acid delivery a reality
12:45 pm Lunch Break & Networking
Revolutionizing Nanoparticle Design with PEG-Free & Poloxamer-Coated LNPs with Reduced Immunogenicity & Safer, More Targeted Delivery
1:45 pm Redefining Nanoparticle Delivery: PEG-Free LNP Formulation for Targeted & Extrahepatic Applications
Synopsis
- Replacing PEG lipids with natural gangliosides to mitigate immune responses, lower toxicity and improve the overall safety of nanoparticle delivery systems
- Enabling targeted delivery to bone marrow, lungs and other non-hepatic tissues and cells without the need for antibody conjugation, overcoming key translational hurdles
- Characterizing particle size, encapsulation efficiency and in vitro/in vivo performance to drive enhanced transfection, biodistribution and therapeutic impact
2:15 pm Development & Evaluation of a 3-component LNP With Poloxamer Coating
Synopsis
- Developing a formulation of 3-component LNP coated with fast shedding off stealth polymer
- Optimizing the formulation to achieve comparable efficacy with standard LNP for in vivo expression of mAbs
- Evaluating the anti-PEG antibody and cytokine level to improve the safety and repeated dose efficacy of the novel formulation
2:45 pm Afternoon Networking Break
Optimizing Non-Lipid-Based Delivery Systems for Enhanced Stability, Targeting Precision, & Scalability to Deliver Novel Therapeutic Solutions to Patients
3:45 pm Revolutionizing Intracellular Delivery with Feldan’s Advanced Peptide- Based Platform for Clinical Success
Synopsis
- Pioneering endosomal escape through rationally designed peptide shuttles, enabling efficient delivery of small molecules, proteins, enzymes and oligonucleotides across diverse cell types
- Delivering functional biomolecules in different animal models
- Demonstrating clinical potential of the lead program targeting basal cell carcinoma through in vivo data and progression to first-in-human trial
4:15 pm Unlocking Targeted Delivery with Peptide Systems by Overcoming Stability Challenges & Avoiding Degradation to Induce Therapeutic Interventions in Desired Tissues
Synopsis
- Addressing peptide stability issues through chemical modifications and new formulation approaches, enhancing their shelf-life and efficacy in vivo
- Improving targeting precision by optimizing peptide sequences and conjugation strategies, ensuring specific delivery to disease sites with minimal off-target effects
- Exploring hybrid peptide platforms to combine the advantages of peptides with other nanoparticle systems, enabling more effective delivery for therapeutic success