Explore the Agenda
Inaugural AI & mRNA Design Day
7:50 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 Bioinformatic Design of long mRNA – Unlocking the 8-12kb Frontier
- Long mRNAs (8-12kb) unlock new therapeutic modalities impossible with 2-5kb constructs (large protein replacement, gene editing, self-amplifying RNA, polycistronic mRNA, tissue targeting, etc.)
- Designing longer mRNA sequences requires not only greater computational power but also new, scalable algorithms AND the integration of stringent manufacturing constraints (repeat sequences, dsRNA-forming structures, plasmid instability, etc.)
- While human cells naturally produce long mRNAs, the in vitro transcription (IVT) process scales poorly with increasing mRNA length. Is it time to mimic eukaryotic cells to enable the production of longer mRNAs?
12:10 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
12:40 pm Lunch Break & Networking
Boosting mRNA Design Capabilities & Data Set Generation from Structure to Function Across Therapeutic Applications
1:45 pm Advancing mRNA Stability Modeling & Design
- Highlighting RNA sequence space
- Presenting key design features
- Showcasing new structure metric and stability predictor
2:15 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
2:45 pm Chair’s Closing Remarks
3:00 pm End of AI & mRNA Design Day
6:00 pm Scale with Confidence Reception Hosted by Leon-Nanodrugs
3rd Next Generation mRNA Delivery Day
8:20 am Check In & Coffee
9:20 am Chair’s Opening Remarks
Innovating mRNA-LNP Designs to Achieve Effective & Targeted Payload Delivery for Vaccine & Therapeutic Applications
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 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
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 Impact of mRNA Design on In Vivo Murine mRNA-LNP CD19 CAR in a Syngeneic Mouse Model
More Information to Come Soon!
2:00 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
2:30 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