Conference Day Two

Day One

Day Two

8:30 am Registration & Morning Coffee

Evaluating, Optimizing, & Engineering Different Delivery Systems to Ensure They Fit the Target & Editor

9:00 am Chair’s Opening Remarks

  • John Zuris Director - Editing Technologies, Editas Medicine

9:15 am Predicting How the Delivery System Affects the Expression of the Gene Editor

  • Kelly Banas Principal Investigator, ChristianaCare Gene Editing Institute

Synopsis

  • Deducing the characteristics of delivery systems that influence the gene editor’s expression levels in vivo
  • Overcoming off-target editing with appropriate delivery system selection
  • Increasing gene editor expression to reduce the need for repeat or large dosing

9:45 am Panel Discussion: Contesting the Viability of Viral Vectors for Gene Editing Delivery

  • Kelly Banas Principal Investigator, ChristianaCare Gene Editing Institute
  • Philip John (P.J.) Brooks Deputy Director - Office Rare Diseases Research, NCATS , NIH, Division of Rare Disease Research Innovation, NCATS NIH
  • Robert Bell Senior Vice President & Head of Research, Ascidian Therapeutics
  • Sourav Choudhury Head of AAV Technologies, Sanofi Genomic Medicines Unit
  • John Zuris Director - Editing Technologies, Editas Medicine

Synopsis

  • Considering the off-target editing and immunogenicity of viral vectors and whether these bottlenecks are solvable
  • Discussing the viability and reliability of self-inactivating viral vectors
  • Understanding the effects of random incorporation of viral genetic components into the genome and continued expression of the gene editor

10:45 am Optimizing AAVs for Efficient Delivery of Prime Editors

  • Jessie Davis Scientist II, Exploratory Delivery Technologies, Prime Medicine

Synopsis

  • Understanding when AAVs are preferential to LNPs for delivery of prime editors
  • Discussing Prime Medicines’ approach
  • Streamlining editors to reduce their size to be appropriate for the desired delivery system

11:15 am Morning Break & Networking Poster Session

Synopsis

Learn from your peers about their latest scientific insights and gain up-to-date data from companies sharing their exciting work. Contribute to the conversation and share your cutting-edge research to your fellow gene editing delivery community to:

  • Communicate your breakthrough discoveries to a vast audience of experts
  • Learn how others are tackling similar challenges to you

12:15 pm Evaluating Delivery Systems for Genome Editing Machinery to Diverse Cells & Tissues Via Different Routes of Administration

  • Philip John (P.J.) Brooks Deputy Director - Office Rare Diseases Research, NCATS , NIH, Division of Rare Disease Research Innovation, NCATS NIH

Synopsis

  • Discussing delivery systems developed and validated by the NIH Somatic Cell Genome Editing Consortium
  • Comparing the pros and cons of viral vectors versus nonviral delivery agents for different targets
  • Introducing the NIH TARGETED Challenge: Programmable Delivery Systems for multiple targets, and nonviral delivery across the blood-brain barrier

12:45 pm Comparing In Vivo Behaviour of Different Gene Editors & Delivery Systems for Patient Safety

  • Saumyaa Saumyaa Associate Director - Cell , Gene Therapy & Editing Safety, AstraZeneca

Synopsis

  • Understanding the characteristics of different delivery systems and editors that affect their safety
  • Exploring offsetting immune pathways within the cells with the cargo to improve safety profile
  • Engineering modifications of different delivery systems to reduce adverse health effects 

1:15 pm Networking Lunch

Examining High Throughput Discovery, GMP Scale-Up, & Regulatory Compliance to Accelerate into the Clinic

2:15 pm Exploring High Throughput Discovery of Prime Editors In Vivo

  • Marine Hatit Senior Scientist I – LNP Discovery Lead, Prime Medicine

Synopsis

  • Understanding how to accelerate the discovery of delivery systems that are scalable
  • Designing different experiments to assess how variable conditions affect the viability of LNPs
  • Maximizing encapsulation efficiency

2:45 pm Designing and Developing LNP Characterization Techniques for Gene Editing Delivery

Synopsis

  • Discussing standard characterization techniques to determine size, PDI, cargo concentration, morphology, LNP concentration, and encapsulation efficiency
  • Understanding in vitro and in vivo potency to determine the efficacy of the delivery vehicle
  • Developing advanced characterization techniques to determine cargo instability, and structure-activity relationships

3:15 pm Chair’s Closing Remarks

  • John Zuris Director - Editing Technologies, Editas Medicine

3:30 pm End of Inaugural Gene Editing Delivery Summit

CONFERENCE DAY ONE
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