8:30 am Check-In Opens & Light Breakfast
8:50 am Chair’s Opening Remarks
Unleashing Computational Models for Enhancing RNA Structure Prediction to Accelerate Therapeutically Relevant RNA Discovery for Driving Development of More Efficacious Therapies
9:00 am NUPACK: Analysis & Design of Nucleic Acid Structures, Devices, & Systems
Synopsis
- NUPACK is a growing software suite for the analysis and design of nucleic acid structures, devices, and systems, serving researchers in the emerging disciplines of molecular programming, nucleic acid nanotechnology, synthetic biology, and across the life sciences
- NUPACK algorithms have pioneered the treatment of complex and test tube ensembles containing arbitrary numbers of interacting strand species, providing crucial tools for capturing concentration effects essential to analyzing and designing intermolecular interactions that are a hallmark of these fields
- The NUPACK cloud web app leverages a scalable hybrid cloud compute cluster and the all-new NUPACK 4 scientific code base, including: enhanced physical models, dramatic speedups, increased scalability for large complexes, and diverse hard and soft sequence constraints for design
9:30 am Roundtable Discussion: Accelerating Accurate RNA Structure-Function Prediction by Harnessing AI/ML Methodologies to Identify Therapeutically Relevant RNAs
Synopsis
- How do RNA structure predictions influence design of therapeutic agents and delivery for more targeted and efficacious therapies?
- How to improve datasets for training AI/ML algorithms for more accurate structure relationship prediction
- Understanding the features that impact RNA structure folding predictions using machine learning algorithms
10:00 am Morning Break & Networking
Integrating Computational Tools for Generating RNA Delivery Vehicles with Greater Tissue Specificity Leading to Reduced Toxicity
11:00 am Harnessing Computational Tools for Re-Targeting of Virus-Like Particles (VLP) Delivering RNA Medicines
Synopsis
- Viral-like particles (VLPs) are important therapeutic delivery tools for DNA and RNA, with innate tropism
- Using combinations of 3d structure prediction and physics models to facilitate reprogramming the molecular affinities of VLP binding proteins, in order to facilitate targeted delivery beyond the wild type tissue type targets
- Novel binding proteins are designed for a variety of related viral vectors and the results are validated experimentally
11:30 am Decoding the Language of Delivery with Directed Evolution & AI-Guided Protein Design
Synopsis
- Introduction of the FORGETM platform which leverages unbiased directed evolution and AI-guided protein design to discover novel ligands for tissue-selective delivery
- Application of FORGETM in the discovery of ligands targeting skeletal muscle, adipose tissue, and CNS with in vivo proof-of-concept delivering various oligonucleotide payloads
- Next research frontier focused on subcellular delivery by utilizing AI to design multi-functional delivery proteins capable of tissue selectivity and subcellular localization
12:00 pm Panel Discussion: Driving Safer Delivery of RNA Therapeutics & Vaccines by Leveraging Computational Tools with Better Prediction of Targeting & Specificity
Synopsis
- How to optimize machine learning algorithms for better prediction of LNP targeting and specificity
- How to develop computational tools for incorporating addition of targeting moieties on LNPs
- How can AI/ML indicate impact of lipid properties and RNA modifications on LNP performance?
- Uncover computational strategies for delivery vehicles beyond LNPs for selective and precise delivery of RNA medicines
12:30 pm Lunch & Networking
Navigating AI/ML for Fine-Tuning Non-Coding RNA Design for Advancing Specific & Targeted of RNA Therapies Towards Approval
1:30 pm Uncovering the Power of Machine Learning for Advancing Drug Development of Divalent SiRNAs to Treat Neurological Diseases
Synopsis
- Leveraging a computational algorithm for designing and prioritizing siRNA constructs to take forward as lead candidates
- Taking advantage of computational tools, machine learning and proprietary scoring models for identifying therapeutically relevant siRNAs for treatment of neurodegenerative treatments
- Harnessing computational strategies for target prioritization and understanding RNAs expressed in target tissues
2:00 pm Roundtable Discussion: Implementing Computational Strategies for Designing Non-Coding RNA Sequences to Achieve Gene Editing & Expression Regulation
Synopsis
- Investigating how AI/ML can be used to optimize non-coding RNA sequences for tasks such as gene editing or regulating gene expression
- How to use AI/ML to identify cleavage probability of microRNA and siRNAs
- Highlighting the potential of computational tools to predict non-coding RNA interactions with the RNA target
2:30 pm Training Foundation Models for Efficient & Precise RNA editing
Synopsis
- The transformer-based foundation model Helix significantly improves predictions of RNA editing outcomes at single-base resolution and specificity
- These advancements bring us one step closer to reliably generating gRNAs that achieve superior editing and specificity in vivo
- Our goal is to create a seamless pipeline that enables in silico generation of gRNAs with excellent editing efficacy and specificity “zero-shot”. Helix gets us closer to realizing this vision
3:00 pm Afternoon Break & Networking
Accelerating RNA Medicines Towards the Market by Navigating Ownership Challenges & Optimizing Translation with Improved AI & Machine Learning Algorithms
3:30 pm Fireside Chat: Unleashing the Potential of Computational Models for Predicting Clearance, Toxicity & Dosing to Accelerate Therapeutic Translation into the Clinic
Synopsis
- How to leverage AI/ML to gain better predictive power of human exposure and clearance from high throughput in vitro assays
- Shorten RNA design cycles by utilizing clinical and toxicology datasets to better understand translation and improve in vivo prediction
- How to fast-track from RNA discovery to the clinic by integrating AI/ML into preclinical strategies?
4:00 pm Protecting Innovation in AI-Driven RNA & Delivery Vehicle Design by Navigating Ownership Challenges & Improving Patentability
Synopsis
- Unleashing the opportunities for protecting new IP resulting from AI and the AI platforms themselves
- Navigating challenges with ownership when working with vendors and leveraging opensource platforms
- Highlighting current standards concerning inventorship and patentable subject matter for the utilization of AI in RNA and delivery vehicle design