Day One

Wednesday, January 25 2023

8:00am | Registration & Coffee

8:40am | Chair’s Opening Remarks

  • Pierre Côte Head of Strategic Partnerships & innovation Management, mRNA Center of Excellence Sanofi

Seizing the RNA Opportunity in Cell Therapies

8:45 am Industry Leaders Fireside Chat: Setting a Trailblazing Fire to Ignite the CTx Field with RNA-Editing

  • Michael Singer Chief Scientific Officer, Cartesian Therapeutics
  • Birgit Schultes Senior Vice President, Cell Therapies, Intellia Therapeutics
  • Pierre Côte Head of Strategic Partnerships & innovation Management, mRNA Center of Excellence Sanofi
  • Amy Becker Director of Immunology, Orna Therapeutics


An executive panel discussion from the industry leaders of the field to set the scene on RNA-based CTx.
Ask your questions live to understand the expert’s thoughts on key topics including:

  • Getting up to scratch for RNA and CTx field and the differences in gene editing to take actionable learnings into RNA CTx developments
  • Sharing recent successes and failures of RNA-based CTx to indicate future directions
  • Exploring novel manufacturing, editing and analytical techniques to enhance potency and efficacy of RNAbased CTx

9:30 am Sharing an Overview of Key Developments & Players in the RNA-Based CTx Space & the Future Prospectus

  • Dharini Shah Senior Research Director, Immunology, Tidal (a Sanofi Company)


  • Providing a comprehensive overview of the preclinical pipeline of RNA-based CTx and revealed clinical trials
  • Sharing insights into the different approaches, opportunities, and current trends to elucidate future direction for RNA-edited CTx
  • Comparing the efficacy and safety of viral and non-viral delivery techniques

10:00am | Morning Refreshments & Speed Networking

Optimizing Delivery to a Targeted Cell Type for Cell Therapy

11:00 am In Situ CAR Therapy Using oRNA™ Immunotropic Lipid Nanoparticles

  • Amy Becker Director of Immunology, Orna Therapeutics


  • Developing a simplified strategy to produce circular RNA (oRNA) and improve durability of CAR expression over conventional mRNA in immune cells
  • Engineering immunotropic lipids to deliver LNPs to immune cells for release of immunoquiescent, unmodified oRNA
  • Sharing how our expression screening platform (FoRCETM) has yielded novel IRESs that elevate CAR expression and drive significant improvements in tumor regression (in vivo

11:30 am Improving Ex Vivo Immune Cell Engineering Using LNP-Mediated Delivery of CRISPR/Cas9 mRNA & sgRNA

  • Aaron Prodeus Associate Director, Cell Engineering, Intellia Therapeutics


  • Discussing how LNPs can be used to efficiently deliver mRNA to T-cells and other immune cell types with minimal toxicity
  • Developing LNP mediated delivery of CRISPR/Cas9 to achieve high editing efficiency and improved phenotype and potency of TCR-T and CAR-T cells
  • Achieving sequential editing of T-cells with LNPs to enable multiplex editing with minimal to no translocations across target genes

12:00 pm Panel Discussion: Optimizing Non-Viral Gene Transfer to Fast-Track Efficient & Scalable RNA Transfection to Different Cell Therapies

  • Albert Kwok Co-Founder, CSO, Nuntius Therapeutics
  • Aaron Prodeus Associate Director, Cell Engineering, Intellia Therapeutics
  • Amy Becker Director of Immunology, Orna Therapeutics


  • Mapping out major cell therapy subtypes and their capability for RNA delivery
    Discussing key technologies and their relative advantages and drawbacks for gene delivery
    Taking learnings from vaccine delivery to improve formulation with enhanced efficiency and deliverability in vivo
    and ex vivo

12:30pm | Lunch & Networking

Optimizing Cell & RNA Characterization to Streamline Analytical Assessments

1:30 pm Leveraging Efficient Analytical Techniques to Meet Quality Attributes Rapidly & Meet Regulatory Expectations in RNA CTx

  • Emily English Senior Director of Quality, Cartesian Therapeutics


  • Defining key quality attributes for RNA CTx and modifications and manufacturing methodologies to improve long-term purity of RNA to prevent expression of off-target receptors
  • Understanding proxy variables to integrate online techniques to automate quality assessment, minimize wait times and prevent material loss
  • Sharing Cartesian Therapeutics’ path from discovery to fully-integrated clinical-stage biotech

Demonstrating Safety & Toxicity to Accelerate Through Clinical Investigations

2:00 pm Accelerating RNA CTx Development from Bench to Bedside (& Back) for Use in Oncology & Beyond


  • Debating optimal pathways to the clinic for RNA CTx and cGMP considerations to assess before embarking on clinical development
  • Assessing the best-in-class ways to measure pharmacokinetic and pharmacodynamic properties of RNA CTx
  • Highlighting unique safety and toxicology considerations for this class of drugs and the types of animal models to accelerate to the clinic

2:30 pm Combining Adenovirus Oncolytic Therapy With Circular RNAs: Future Perspectives Based on Clinical Experience


  • Demonstrating promising clinical efficacy and capacity to increase tumor infiltration on anti PD-1-resistant advanced melanoma
  • Providing clinical efficacy of ONCOS-102 correlated with virus persistence in the tumor and stable remodelling of the tumor microenvironment
  • Harnessing expression of highly stable circular RNAs encoding additional immuno-modulatory payloads to further increase efficacy of oncolytic adenoviruses

3:00 pm Roundtable Discussion: Leveraging Key Learnings From Vaccines & Traditional CTx Clinical Development to Accelerate RNA CTx to the Clinic


  • Practical and highly interactive breakout-style roundtables where attendees can crowd-source solutions and share their opinions around how to fast-track RNA CTx to the clinic. This is a valuable opportunity for you to voice your experiences and debate best practice while identifying unique solutions.

3:30 pm End of Day One