Explore the Agenda
8:00 am Check-in, Coffee & Networking
8:50 am Chair’s Opening Remarks
Evaluating RNA Modalities to Enable Confident Platform Selection & Improve Indication Fit
9:00 am Panel: Contrasting Circular vs. Self-Amplifying RNA to Match Modalities to Use-Cases
- Weigh strengths and trade-offs in potency, durability, manufacturability, and safety to guide platform decisions
- Compare fit-for-purpose advantages across CAR-T, protein replacement, combination therapies, autoimmune, and CNS applications to reduce risk
- Define where each modality delivers a strategic edge and where constraints limit applicability for smarter portfolio planning
9:30 am Roundtable: Aligning Platform Selection to Clinical, Biological & Commercial Objectives
- Debate real-world trade-offs across timeline, tissue targeting, safety, manufacturability, and IP boundaries to inform strategy
- Evaluate modality-to-indication fit through case scenarios to improve development efficiency and reduce failure risk
- Align stakeholder priorities (clinical, regulatory, BD, IP) to ensure long-term portfolio viability and investment readiness
10:00 am Morning Break & Networking
Engineering Amplified RNA Architectures to Maximize Potency, Durability & Therapeutic Reach Across Vaccines and Beyond
10:30 am Unlocking Low-Dose, Long-Duration Expression with Fully Modified saRNA to Transform Vaccine & Therapeutic Performance
- Drive potent, durable protein expression at low doses by using modified nucleotides that preserve saRNA activity and maximize translational efficiency
- Suppress innate immune activation to improve tolerability and dosing flexibility, enabling safer multi-dose regimens and expanding therapeutic use cases
- Combine chemical modification, RdRp engineering, and optimized multi-gene architectures to achieve optimal saRNA performance
11:00 am Harnessing Cascade Amplification & Therapeutic Payload (CATP) Systems to Unlock Potent, Low-Dose & Multi-Payload saRNA Therapeutics
- Achieve strong therapeutic expression at lower doses using a two-step amplification system that combines saRNA with defective viral replication to bypass current safety– efficacy limits
- Enable multi-gene and complex therapeutic logic through a dual-amplification system capable of supporting larger and more sophisticated payloads beyond traditional vaccine applications
- Improve targeting precision and reduce off-target liver accumulation with advanced delivery design that supports tissue- and cell-type–specific uptake for safer, more predictable translation
12:00 pm Networking Lunch
Applying Circular & Self-Amplifying RNA to Enable In Vivo CAR-T & Replace Complex Ex Vivo Cell Therapies with Scalable Immune Modulation
1:00 pm Programming T Cells In Vivo with Targeted Nanoparticles Encapsulating eRNATM-encoded hCD19 CAR for Transient Activation to Achieve Deep B-cell Depletion and Immune Reset
- Applying Circular & Self- Amplifying RNA to Enable In Vivo CAR-T & Replace Complex Ex Vivo Cell Therapies with Scalable Immune Modulation
- In vivo CAR-T cell engineering has the potential to transform the care of patients with autoimmune diseases by simplifying and broadening access to treatments with potential for cures
- Sail’s in vivo CAR-T drug candidate (DC) has been engineered and extensively optimized to achieve an optimal dose efficiency profile and ultimately therapeutic index in patients with autoimmune conditions. As a result, Sail’s in vivo CAR-T DC is able to achieve deep tissue depletion of B cells and effectively reset the B cell compartment at low dose levels
- Sail’s in vivo CAR-T DC has the potential to replace complex, costly, and genome-integrative cell therapy products with an off-the-shelf and non-integrative IV injectable product that avoids the safety risks associated with lymphoablative conditioning
1:30 pm Advancing Multi-Gene RNA Therapies to Unlock Durable In-Vivo CAR-T Treatments for Eosinophilic Conditions
- Enable deeper, multi-pathway disease control in eosinophilic indications by using multi-gene RNA constructs that co-deliver multi-CARs and cytokine payloads
- Achieve durable, low-immunogenic expression suitable for repeat dosing through a synthetic RNA architecture optimized for chronic therapeutic applications
- Leverage modular multi-gene RNA design to support versatile therapeutic strategies and maintain flexibility for future development needs
2:15 pm Afternoon Networking Break
Redesigning Circular & Self-Amplifying RNA to Unlock Durable & Tissue-Selective Therapeutics Beyond Oncology & Autoimmunity
2:30 pm circVec: A Circular RNA Vector–Driven Expression System for NextGeneration Cell and Gene Therapy
- circVec, a novel gene expression platform developed by Circio, utilizes the high intra-cellular stability of circular RNA (up to 75 times longer half-life than mRNA) to achieve enhanced protein expression from viral and non-viral DNA vectors
- The low decay rate of circular RNA leads to high accumulation over time, particularly in specific tissues, such as heart, spleen, and eye, which enables safer and/or more effective gene and cell therapy in these areas
- Low accumulation of circRNA in hepatic tissue and overall lower stimulation of the UPR pathway compared to mRNA-based vectors suggest reduced tox and cellular stress upon systemic injection of circVec-based vectors
3:00 pm Engineering 300-nt Circular RNAs to Unfold Pathogenic RNA Structures as a Novel Therapeutic Strategy for Infectious & NonInfectious Diseases
- Reverse structure-driven pathogenicity by applying hybridization-based circRNAs that block the formation of aberrant RNA structures underlying ALS, muscular dystrophies, and viral replication
- Expand circRNA utility well beyond vaccines and protein replacement by demonstrating a new non-canonical mode of action with in vitro and in vivo proof across multiple disease classes
- Strengthen translational readiness through insights into purity thresholds, linear– circular separation, and delivery considerations unique to small, non-coding circRNA modalities
3:30 pm Redesigning Self-Amplifying RNA to Enable Durable, Low-Dose In Vivo Therapeutic Reprogramming
- Extend therapeutic expression from days to months by redesigning intracellular control of self-amplifying RNA to support sustained, low-frequency dosing beyond vaccine-optimized kinetics
- Expand saRNA applicability into chronic and tissue-specific indications by overcoming rapid clearance and homogeneous expression through synthetic biology modification of UTRs and replicase machinery
- Enable scalable, cost-effective alternatives to ex vivo cell therapies and complex biologics by positioning in vivo saRNA-driven reprogramming as a practical therapeutic platform for osteoporosis, ophthalmology, and rare diseases