8:00 am Check In & Morning Coffee

8:55 am Chair’s Opening Remarks

  • Francis Poulin Vice President - Analytical Sciences, Sail Biomedicines

Addressing Investment, Collaboration & Breakthrough Opportunities to Accelerate Next Generation RNA Medicine Discovery & Development Towards Therapeutic Reality

9:00 am Fireside Chat: Bridging the Gap Between Pharma, Biotech & Investors to Support the Development of Upcoming Next Generation RNA Innovations

Synopsis

This discussion-based session will unite specialists from the investment, pharma, and biotech sectors to facilitate the growth and development of the field. Hear investors’ perspectives on evaluating investment opportunities and allocating resources strategically as these stakeholders assess opportunities to invest and collaborate to drive translation of these innovative therapies to benefit patients and improve treatment outcomes.

  • Addressing the importance of collaboration and investment in advancing pharmaceutical and biotech innovations
  • Understanding the need to bridge the gap between pharma, biotech, and investors
  • Identifying challenges and risks associated with investing in these industries
  • Discussing strategies for mitigating risks and maximizing returns on investments
  • Exploring emerging areas of investment interest, such as automation techniques and personalized medicine
  • Predicting future trends and opportunities in the pharmaceutical and biotech sectors

10:00 am Diving into the Exhilarating World of tRNAs & Their Transformative Potential in Treating Genetic Diseases

Synopsis

  • Explore how tRNAs modulate mRNA function to restore or increase protein production and cure disease​
  • Understand how tRNAs address a significant number of diseases that are not amenable to traditional gene therapy
  • Identify how tRNA therapeutics combine ideal aspects, without the pitfalls, of other gene and mRNA therapies
  • Demonstrating how tRNA therapies offer significant market opportunities

10:30 am Morning Break & Speed Networking

Synopsis

This session is your opportunity to get face-to-face time with many of the brightest minds working in the next generation RNA therapeutic development field, and establishmeaningful business relationships to pursue for the rest of the conference

Enhancing Characterization, Safety & Potency to Develop & Manufacture Effective Self- Amplifying RNA Therapeutics for Patients

11:30 am Showcasing the Current Landscape of Self-Amplifying RNA Therapeutics

  • Rachel Groppo Director, Self-Amplifying RNA Team Lead, The Janssen Pharmaceutical Companies of Johnson & Johnson

Synopsis

  • Overview of saRNA applications, with a focus on vaccines and therapeutic interventions 
  • Explore the challenges associated with delivering saRNA and ongoing efforts to address them 
  • Highlighting any recent milestones and breakthroughs in saRNA research 

12:00 pm Next Generation RNA Vaccines in the Clinic Demonstrate Unprecedented Efficacy & Safety

Synopsis

  • Discussing methods for analyzing saRNA molecules, including sequencing, mass spectrometry, and structural characterization
  • Exploring the potential toxicities associated with saRNA therapeutics
  • Discussing methods for assessing the potency of saRNA molecules

12:30 pm A Novel saRNA Backbone for Reduced Cytotoxicity & Enhanced Payload Expression

  • William Jia Chief Scientific Officer & Co-founder, Virogin Biotech Ltd

Synopsis

  • We have developed few strategies with novel saRNA backbones:
  • To reduce dsRNA-induced toxicity and to increase payload expression through mutagenesis
  • To regulate saRNA amplification and payload translation in a cell-type-specific manner
  • HPV mRNA/saRNA therapeutic vaccines were developed and demonstrated both prophylactic and therapeutic efficacy in an HPV+ animal tumor model

12:40 pm Optimizing the Design & Delivery of Self-Amplifying RNA to Improve Therapeutic Index

Synopsis

  • Self-amplifying RNA has generated significant interest for vaccine and therapeutic applications
  • Improved efficacy of self-amplifying RNA drug products vs other modalities
  • Genetic circuits improve spatial control over payload delivery

1:10 pm Lunch Break & Networking

Unlocking the Widespread Potential of RNA Therapeutics with Improved Sequence Design, Target Selection & In Vivo Delivery

2:10 pm Targeted mRNA for in vivo Reprogrammed Off-the-Shelf CAR-T Therapies

  • Dharini Shah Senior Director - Research & Immunology, Sanofi

Synopsis

  • Autologous CAR T cell products have been transformational in hematological malignancies; however, manufacturing complexities limit patient access
  • Sanofi’s (Tidal) novel platform offers potential and promise of in vivo delivery of CARencoding mRNA as a novel therapeutics
  • Transforming CAR-therapies by active in vivo reprogramming using novel targeted delivery approaches and advanced RNA therapeutic

2:40 pm Unlocking the Widespread Potential of RNA Therapeutics via Short Activating RNA Development

  • Anthony Saleh Co-Founder & Chief Executive Officer, MiRecule Inc.

Synopsis

  • Discussing strategies for designing and optimizing saRNA sequences
  • Exploring considerations for specificity, potency, and target selection
  • Assessing the therapeutic potential of saRNA in gene activation and regulation
  • Evaluating applications in treating genetic disorders and rare diseases

3:10 pm Deep Dive into Phospholipids in Vaccines & How to Optimize Their Functions

Synopsis

  • Introduction to Phospholipids
  • Vaccine systems with phospholipids (LNPs and adjuvant carriers)
  • Function of lipids in LNPs
  • Commercial Examples

3:40 pm Afternoon Break & Scientific Poster Session

Synopsis

Present your latest findings and gain insight into the cutting-edge work of your peers in this dedicated poster session. Engage in meaningful discussion with your peers, and knowledge-share to streamline the advancements of this exciting field.

Achieving Better Stability & Efficacy in Pre-Clinical & Clinical Settings with Advanced RNA Delivery Systems

4:30 pm Exploring the Development & Optimization of RNA Delivery Systems to Advance Next Generation RNA Therapeutics

Synopsis

  • Delving into the methods of characterizing your RNA delivery systems to understand their performance under different conditions
  • Optimizing the interaction between your RNA delivery systems and cellular machinery that can lead to RNA degradation to improve the stability and efficacy of RNA delivery systems

5:00 pm Unlocking the Potential of PBAE Nanoparticles: Advancements in mRNA Delivery for Targeted Immunization

  • Stephany Tzeng Assistant Research Professor, The Johns Hopkins University School of Medicine

Synopsis

  • PBAEs can be synthesized with a wide range of chemical structures that have different properties
  • PBAEs can be screened in a high-throughput manner to discover structures that can deliver mRNA to immune cells
  • Optimized PBAE/mRNA nanoparticles can transfect immune cells selectively over other cell types after in vivo administration, without requiring ex vivo cell manipulation or manufacturing
  • PBAE/mRNA nanoparticles can co-deliver multiple sequences and/or other types of nucleic acids, including nucleic acid-based adjuvants
  • Nanoparticles containing mRNA sequences alone or along with adjuvants can safely and effectively immunize animals against an antigen(s) of interest

5:30 pm End of Conference Day One