8:00 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
The largest 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. Therefore, during this workshop you will explore cutting-edge delivery strategies which enhance RNA stability, improve targeting precision, and optimize encapsulation for safe and specific tissue and cell targeting.
This session will deep dive into:
- Optimizing delivery strategies for efficient RNA encapsulation and specific tissue targeting
- Uncovering the promise of LNPs and AAVs for targeted delivery of circular and self-amplifying RNA therapeutics
- Delving into novel delivery vehicles for enhancing endosomal escape of RNA therapeutics
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
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