Day Two

Thursday, January 26 2023

8:00am | Coffee & Networking

8:45am | Chairman’s Opening Remarks

The Next Generation of Precise RNA Cell Engineering to Enable Durable Therapeutic Impact

8:50 am Presenting SLEEK: a Highly Efficient Knock-In Technology for NextGeneration Cellular Medicines

  • John Zuris Director, Editing Technologies, Editas Medicine


  • Illuminating a greater than 90% knock-in efficiency across clinically relevant cell types to ensure guaranteed robust expression of single and multiple transgene cargos
  • Demonstrating equal efficiency with both viral and non-viral DNA donor templates to provide a manufacturing advantage for enhanced scalability
  • Weaponizing the next generation of iPSC-derived NK cells (iNKs) with robust tumor killing and unprecedented persistence in vivo

9:20 am Engineering mRNA to enable Cell-Type Specific RNA therapies


  • Utilizing synthetic biology to enable cell type specific expression
  • Encoding sensors within mRNAs to reduce tox and improve therapeutic efficacy
  • Sharing insights from preclinical program to engineer certain types of immune

9:50 am Leverage RNA Medicine to Expand Delivery to a Larger Range of Cell and Tissue Types

10:00am | Morning Break & Networking

Driving Durable Therapeutic Responses From Transient Effects to Yield In Vivo

10:30 am Panel Discussion: Opportunities & Challenges to Optimize In Vivo RNA Engineering

  • Haig Aghanjanian Co-Founder & Vice President of Research, Capstan Therapeutics
  • David Peritt Founder & Chief Scientific Officer, Lupagen
  • Dharini Shah Senior Research Director, Immunology, Tidal (a Sanofi Company)


  • Elucidating the opportunities for in vivo cell therapy to minimize current ex vivo bottlenecks and the role of RNA engineering in producing versatile therapies
  • Achieving a transient vs. longer term effect for different indications with RNA modification
  • Maintaining consistent in vivo production of RNA to yield long-term responses

11:00 am Developing In Vivo Production of CAR T cells For the Treatment of Heart Disease: An Industry Perspective

  • Haig Aghanjanian Co-Founder & Vice President of Research, Capstan Therapeutics


  • Enabling off-the-shelf immunotherapies with tight control of dosage and of activity of engineering cells
  • Exploring the applications beyond oncology to inflammation, fibrosis and inflammation-related disorders
  • Outlining Capstan’s journey and future plans to expand to the clinic

11:30 am Advancing mRNA-based Therapeutics for In Vivo Cellular Reprogramming: An Academic Perspective

  • Hamideh Parhiz Research Assistant Professor of Medicine, Perelman School of Medicine, University of Pennsylvania


  • Utilizing highly efficient CD4+ T-cell targeting and genetic recombination using engineered CD4+ cell homing mRNA LNPs
  • Demonstrating efficacy in reducing fibrosis and restoring cardiac function in mice
  • Discussing applications to produce CAR-T cells in vivo using modified mRNA to treat various diseases

12:00 pm Mastermind Session: Making Modifications for Persistence and Durability of RNA Expansion to Consistently Express a Construct of Interest

  • Ashlesha Odak Senior Scientist, Immune Cell Engineering, Strand Therapeutics


  • Test your thinking, and troubleshoot key industry challenges on your tables,
    moderated by Strand Therapeutics
  • Discussing the pros and cons of different RNA types
  • Leveraging techniques to provide stable production of RNAs to maintain potency
    with RNA transiency brainstorming the best approaches to improve durability for
    different cell types

12:30pm | Lunch Break

Integrating Engineering with Online Techniques to Automate Construct Design

1:30 pm Fast-Tracking the Digital Revolution by Screening for Long Non-Coding RNA (lncRNAs) to Understand the Roles of A-to-I RNA Editing for CTx Application

  • Shizuka Uchida Professor & Co- Director, Center for RNA Medicine, Department of Clinical Medicine, Aalborg University


  • Expanding the use of RNA-sequencing data for protein coding gene and lncRNAs and RNA-editing patterns to seize future therapeutic opportunities
  • Re-analyzing and combining published RNA-seq data to screen and share disease-related lncRNAs to the public via a knowledge database to improve online access
  • Spotlighting common RNA modifications in disease, such as A-to-I RNA editing and applications for treating cardiovascular disease and beyond

Navigating the IP Landscape to Secure Future Investment
Chaired by: Pierre Côte Head of Strategic Partnerships & innovation Management, mRNA Center of Excellence Sanofi

2:00 pm Understanding the Patent Landscape to Inform Early Delivery Technology Development & Set You Up For Success


  • Reviewing the patent landscape, key license holders and cutting-edge technologies dominating the space to indicate the industry’s future trajectory
  • Providing exclusive IP insight to inform the beginning of the technology journey to build value and develop strategies to operate with technologies freely
  • Innovating existing new technologies to build on differentiation and avoid infringement of existing patents

2:30 pm Panel Discussion: The Science Speaks for Itself – But What Else Is Required to Secure an Investment?

  • Gregory Fiore Chief Executive Officer, Exacis Biotherapeutics
  • Dan Shores Partner, Rothwell Figg
  • Artavazd Arumov Associate, Qiming Venture Partners
  • Pierre Côte Head of Strategic Partnerships & innovation Management, mRNA Center of Excellence Sanofi


  • What do investors look for to invest in RNA-based cell therapies?
  • How best to work holistically with scientists, investors and lawyers to secure an investment?
  • To what extent does founders legacy play on investment opportunity?

3:00 pm End of Day Two