8:00 am Check-In & Light Breakfast
8:50 am Chair’s Opening Remarks
Unlocking the Priorities & Investment Opportunities for the Next Generation RNAs to Ensure Better Drugs Reach Patients Faster
9:00 am CircVec – Deploying CircRNA to Enhance Nucleic Acid Medicines
Synopsis
• Circio´s circVec technology is based on a genetic cassette for in situ circRNA production
• circVec extends mRNA half-life by 70x and protein expression level by 15-fold
• How the circVec platform can be applied to enhance DNA and viral format therapeutics for next generation gene and cell therapy
9:30 am Fireside Chat: Unlocking Priorities for the Next Generation RNA Community to Increase Investment for Driving More Therapeutics to the Patients in Need
Synopsis
• What innovations need to come through to unleash the next frontiers in next generation RNA therapeutic development?
• Identifying future trends and opportunities within investment for circular and self-amplifying RNA therapeutics
• Highlighting priority actions for the circular and self-amplifying RNA community to drive success in 2025
10:15 am Morning Break & Networking
Maximizing the Longevity & Potency of Circular & Self-Amplifying RNA Therapies for Optimal Treatment of a Wide Range of Diseases
11:15 am Deep-Dive Workshop: Understanding Circular & Self-Amplifying RNAs Unique Potential as More Durable & Stable Therapeutics for Treating Patients in Need
Synopsis
• Circular RNA and self-amplifying RNA have many differences to linear mRNA, they have greater stability and durability, however, it’s vital to understand how these properties can be taken advantage of for developing efficacious and safe therapeutics to treat a wide array of diseases which are currently undruggable.
This session will deep dive into:
• Evaluating the advantages and disadvantages of circular RNA and self-amplifying RNA payloads over traditional linear mRNA
• Uncovering the optimal applications of next generation RNA therapeutics
• What cross-learnings can be taken from the linear mRNA field to advance new and innovative RNA therapeutics
12:30 pm Lunch & Networking
Advancing Durable Therapeutics Towards the Clinic by Refining Preclinical Strategies for more Streamlined Translation
1:30 pm In Vivo Reversible Programming of T cells with SAIL’s Targeted NP-eRNA Platform for Treatment of Autoimmune Diseases & Beyond
Synopsis
• Enabling in vivo reversible programming of T cells with SAIL’s targeted LNP-eRNA technology platform
• Replacing a complex, costly, and genome-integrative cell therapy product with an off-the-shelf, and non-integrative IV injectable product by encoding a CAR specific for hCD19 directly in the patient’s T cells in vivo
• Advancing a first-generation in situ CAR product for AID to human clinical studies while in parallel advancing next-generation in vivo reprogramming medicines
New Data
2:00 pm Circular RNA: Transforming a Promising Technology into Cutting-Edge Therapeutics
Synopsis
• Introduction to RiboX Therapeutics, a globally operated biotech company pioneering fully engineered circular RNA (circRNA) therapeutics. RiboX’s proprietary therapeutic platforms include a plug-and-play circular RNA platform, an ionizable lipid platform and unique assets in active LNP targeting
• Preclinical proof of concept study demonstrating the advantages of circRNA as a therapeutic modality
• Brief overview and development status of RiboX’s clinical stage circRNA therapeutic, marking the first circRNA to enter clinical development
New Data
New Company
2:30 pm Afternoon Break & Networking
Advancing Self-Amplifying RNA Therapies with Optimized Designed & Improved Immunogenicity for More Successful Approvals
3:00 pm Second Generation Self-Replicating RNA & its Utility for Vaccine Applications & Beyond
Synopsis
• Replicate has designed 2nd generation self-replicating RNAs by vectorizing new members of the alphavirus family, along with non-coding region optimization
• Vaccines from 2nd generation srRNAs are effective at ultra-low doses
• Clinical testing of a 2nd generation srRNA vaccine resulted in strong immunogenicity in a naive population without SAEs or DLTs
3:30 pm Myeloid Cell Targeting Cancer Immunotherapy via Localized Gene Delivery with Alphavirus Replicon Particles (VRP) Expressing IL-12
Synopsis
• VRP containing a saRNA vector were developed to reprogram the immunosuppressive tumor microenvironment in solid tumors.
• In preclinical studies, intratumoral injection of VRP efficiently delivered saRNA expressing IL-12, reduced immunosuppressive cell populations, and increased antigenspecific T cells.
• The safety and efficacy of VLPONC-01 will be assessed in a phase I trial as a neoadjuvant treatment in combination with pembrolizumab
4:00 pm STX-003: mRNA-based Cancer Therapy for Tumor-Specific Expression of IL-12 via Programmable Genetic Circuits
Synopsis
• STX-003 is a systemically delivered immune oncology drug targeting solid tumors and metastatic disease
• Programmable sensors embedded in STX-003 allow for cell-selective expression
• Engineered control using these sensors improves STX-003 tolerability without compromising efficacy in pre-clinical animal models