Explore the Agenda
Inaugural AI & mRNA Design Day
8:00 am Check In & Coffee
8:50 am Chair’s Opening Remarks
Leveraging Deep Learning Models & Generative AI to Optimize the Design of Stable & Effective mRNA Sequences
9:00 am The Next Frontier for mRNA Therapeutics – AI Applications in Design, Delivery & Manufacturing
- Using ML/AI in mRNA sequence design
- Applying ML/AI in delivery design and optimization
- Leveraging ML/AI in protein design
- Automation, process optimization, and digitization – Enabling efficiency but also feeding the Design Build-Test-Learn virtuous cycle
9:30 am Sequencing-Powered Data & AI for Next-Generation mRNA Therapeutic Design
- RNA-centric sequencing datasets capture unique features needed for predictive mRNA design models
- Foundation models enable UTR optimization and enhanced structural stability to improve therapeutic efficacy
- Integrated Design-Make-Test workflows provide a robust lab-in-the-loop engine for mRNA therapeutic development
10:00 am Deep Learning Guided Optimization of Translation Efficiency for mRNA Vaccine Development
- Delivered mRNA vaccines benefit from a high protein yield to stimulate an effective immune response
- Training RiboNN, a deep learning model, to predict translation efficiency — a major determinant of protein yield — among numerous cell types
- RiboNN can be used to guide the design of translation-optimized mRNA therapeutics
10:30 am Morning Break & Networking
Harnessing mRNA Language Models to Improve mRNA Design for Vaccine Applications & Biomanufacturing
11:30 am Using mRNA Language Models for Efficient Coding Sequence Design
- AI, especially biological language models, accelerates mRNA design
- mRNA design methods for heterologous expression can be used for biomanufacturing and biopharmaceuticals
- Feedback from experimental data is important for improving mRNA design
12:00 pm Evolving the mRNA Alphabet: Foundational Models for Vaccine Design & Optimization
- Highlighting key foundational mRNA language models
- Illustrating end-to-end mRNA vaccine optimization
- Discussing De novo mRNA vaccine design
12:30 pm Lunch Break & Networking
Boosting mRNA Design Capabilities & Data Set Generation from Structure to Function Across Therapeutic Applications
1:30 pm Advancing mRNA Stability Modeling & Design
- Highlighting RNA sequence space
- Presenting key design features
- Showcasing new structure metric and stability predictor
2:00 pm Biologically Informed AI models for mRNA design
- Domain-specific knowledge can facilitate more efficient training strategies and help improve the accuracy of AI models
- Illustrating this with examples of AI models for mRNA property prediction and design
- Discussing how to expand this strategy to other properties in the context of mRNA therapeutics
2:30 pm AI‑Driven Design of Selectively Translated mRNA Therapeutics for In Vivo CAR-T
- Presenting cell selective translation
- Demonstrating effective dataset generation
- Showcasing training models and designing mRNA
3:00 pm Chair’s Closing Remarks
3:15 pm End of AI & mRNA Design Day
3rd Next Generation mRNA Delivery Day
8:00 am Check In & Coffee
8:50 am Chair’s Opening Remarks
Innovating mRNA-LNP Designs to Achieve Effective & Targeted Payload Delivery for Vaccine & Therapeutic Applications
9:00 am Novel Approach with Bispecific Antibodies to Simplify Conjugation & Improve Receptor Engagement for Effective Targeted Payload Delivery
- Leveraging bispecific antibodies’ dual specificity to boost engagement with extrahepatic tissues and optimize payload delivery
- Removing complex chemical conjugation process by leveraging bispecific antibodies to connect LNPs with cell-specific receptors
- Overcoming traditional conjugation challenges to support easier large-scale production of targeted LNP and accelerate translation from research to clinic
9:30 am Minimalist Nanoparticle Designs for RNA Medicines
- Leveraging artificial intelligence in mRNA and cirRNA development
- Presenting a novel delivery strategy with fresh data insights
10:00 am Round Table Discussion: A Scientific Outlook – Overcoming Challenges of Novel LNP Chemistries & Formulations to Achieve a Successful Patent Outcome for mRNA Delivery
- Dissecting a patent and presenting an overview about the process
- What can be patented in an LNP?
- Designing LNPs with specific patent claims in mind
- How do I know that I have enough information to file a patent?
10:30 am Morning Break & Networking
Beyond the LNP: Unlocking Non-Viral Delivery Methods to Optimize Delivery & Therapeutic Performance
11:30 am Advancing RNA-Based Therapeutics Using a Structural Nanomedicine Approach
- Pioneering a structural nanomedicine platform designed to optimize delivery and therapeutic performance across mRNA and other therapeutic modalities
- Demonstrating examples of enhanced mRNA delivery and efficiency using structural nanomedicine, outperforming conventional LNP
- Highlighting recent program updates with expanded non-clinical data in oncology and CRISPR-based gene editing applications
12:00 pm Optimizing mRNA Delivery with Targeted Elastin-Like Polypeptide-Based LENN Formulations
- LENN comprised of elastin-like polypeptide-epidermal growth factor fusion proteins (ELP-EGF) and cyclodextrin-peptide conjugates form stable mRNA complexes that efficiently deliver their cargo to the cytosol of EGFR+ bladder tumor cells in vitro and in vivo
- Multiple reaction monitoring HPLC-MS lipidomics analysis, pharmacologic inhibition, and EGF blockade experiments indicate that EGF-bearing mRNA LENN enter EGFR+ cells via clathrin-mediated endocytosis, with no significant changes to other lipid pathways that would indicate off-target effects or immune responses
- Addition of 10% glycerol and lyophilization enables the long-term storage of mRNA LENN complexes, with full retention of activity after rehydration
12:30 pm Lunch Break & Networking
Overcoming the Barriers of Delivery Chemistry to Achieve Organ Specific Targeting & Offer a Competitive Edge in the mRNA Market
1:30 pm Oral mRNA Delivery to the Gastrointestinal Tract
- Outlining challenges of delivering mRNA to the gastrointestinal tract
- Presenting a strategy for iterating LNPs in vivo to deliver to the gastrointestinal tract
- Demonstrating an application of oral mRNA=LNP in mouse models
2:00 pm Low-Inflammatory RNABL Delivery Enables Organ-Specific Therapeutic RNA Applications
- Introducing non-inflammatory, biodegradable RNABL dendritic molecules for nucleic acid complexation and in vivo delivery
- Showcasing Organ tunability and safety profile of RNABL formulations
- Demonstrating successful therapeutic applications in animal models