8:30 am Check-In & Light Breakfast
8:50 am Chair’s Opening Remarks
Optimizing Self-Amplifying RNA Construct Design & Delivery to Achieve Greater Stability & Reduced Cytotoxicity for More Durable Therapies
9:00 am Engineering Self-Amplifying mRNA Structure to Improve Vaccine Product
Synopsis
- We can modulate self-amplifying mRNA structure properties through sequence design
- Stabilization of RNA structure leads to improved self-amplifying mRNA stability in solution and in LNP
- Self-amplifying mRNA stability and translation balance can be achieved through iterative design-learning cycles
New Data
New Company
9:30 am Developing Safe & Effective Delivery Technologies for Self-Amplifying RNA Vaccines & Therapeutics
Synopsis
- Discussing HDT bio’s LION™ delivery technology – a cationic nanocarrier that is structurally and functionally distinct from LNPs
- Leveraging LION for enabling self-amplifying RNA encoded mAb therapies
- Thermostable formulations of self-amplifying RNA vaccines
New Company
10:00 am Morning Break & Networking
Navigating Delivery for Circular & Self-Amplifying RNAs to Achieve Specific Targeting Leading to Enhanced Potency
11:00 am Deep-Dive Workshop: Accomplishing Improved Encapsulation & Targeting of RNA Therapeutics by Uncovering Innovative Delivery Strategies for More Potent & Effective Therapies
Synopsis
A major bottleneck inhibiting the full potential of all RNA therapeutics is delivery. With self-amplifying and circular RNA payloads typically being larger than those of linear mRNA, this complicates their encapsulation. Furthermore, while current lipid nanoparticles (LNPs) have demonstrated safety and efficacy of mRNA vaccines, their use has been restricted to intramuscular administration. During this workshop, we will discuss the limitations of current delivery technologies and how alternative delivery strategies may enhance RNA stability, improve tolerability and optimize targeting precision for safe and tissue-specific delivery.
This session will deep dive into:
- Exploring alternative technologies for efficient RNA delivery and targeted tissue engagement
- Investigating the potential of LNPs and alternate technologies for targeted delivery of circular and self-amplifying RNA therapeutics
- Discuss critical quality attributes that drive safe and effective delivery technologies
12:15 pm Lunch & Networking
Advancing Durable Therapeutics Towards the Clinic by Refining Preclinical Strategies for more Streamlined Translation
1:15 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
1:45 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
2:15 pm Afternoon Break & Networking
Advancing Self-Amplifying RNA Therapies with Optimized Designed & Improved Immunogenicity for More Successful Approvals
3:00 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
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
New Data!
New Company!
4:00 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