Conference Day One

7:45 am Registration & Morning Coffee

Advancing Targeted Extra-Hepatic Delivery to Specific Tissues & Organs to Widen the Therapeutic Index of Gene Editing

8:45 am Chair’s Opening Remarks

  • John Zuris Director - Editing Technologies, Editas Medicine

9:00 am Panel Discussion: Assessing the State of Play & Visualizing the Future: Challenges & Opportunities for Extra-Hepatic In Vivo Gene Editing


  • Examining the current landscape of in vivo gene editing delivery
  • Delving into the critical bottlenecks preventing extra-hepatic delivery and identifying the side effects of overdosing to the liver
  • Setting a roadmap to accelerate drugs to patients

10:00 am Harnessing Mobile Genetic Elements to Write DNA Sequences with RNA Beyond the Liver

  • Wes Salomon Director - Delivery Biology, Tessera Therapeutics


  • Exploring LNPs as a versatile delivery system for RNA cargo, well-suited for the all-RNA system of Gene Writers
  • Uncovering LNPs’ systemic administration and biology towards organs other than the liver
  • Optimizing and quantifying delivery to establish therapeutically relevant extra-hepatic delivery

10:30 am Morning Break & Speed Networking


Our speed networking is the ideal opportunity to get face-to-face time with the brightest minds working in gene editing delivery and introduce yourself to the attendees that you would like to have more in-depth conversations with. Benchmark against industry leaders and establish meaningful business relationships to pursue for the conference and beyond.

11:30 am Identifying Delivery System Modifications to Avoid the Liver & Target the Desired Tissue for Maximum Efficacy


  • Uncovering how to identify proteins that target the tissue of choice by rational and random design
  • Understanding whether the protein can be fragmented to reduce the size of the therapeutic package
  • Assessing whether this method can eliminate extra-hepatic delivery

12:00 pm Roundtable Discussion: Exploring the Viability of Engineering Delivery Systems That Hit Only One Cell/Tissue Type

  • John Zuris Director - Editing Technologies, Editas Medicine


  • Assessing the current advances in engineering tissue specificity
  • Debating whether precise in vivo gene delivery is a pipe dream or viable
  • Sharing experiences with new advances to maximize the therapeutic window of in vivo gene editors

12:45 pm Networking Lunch

1:45 pm Exploring Pulmonary Delivery for Gene Editing for Therapeutic Efficacy

  • Xue Anna Cheng Director - R&D Pulmonary Drug Delivery, Feldan Therapeutics


  • Introducing novel peptide-based delivery strategies to target pulmonary tissues
  • Assessing delivery to airway cells using different animal models
  • Discussing therapeutic potential of gene editor delivery to the lungs

2:15 pm Crossing the Blood Brain Barrier with Targeting, Safety & Efficacy

  • Susan Rosenbaum Founder, Chairman & Chief Executive Officer, Lauren Sciences LLC


  • Delivery targeted only to central nervous system
  • Avoiding off-target effects, including hepatic accumulation
  • Effectively targeting and treating specific brain diseases

2:45 pm Optimizing Ocular Delivery to Accelerate In Vivo Therapy into the Clinic for Rare Retinal Disorders

  • Robert Bell Senior Vice President & Head of Research, Ascidian Therapeutics


  • Assessing strategies to advance ocular gene editing delivery
  • Understanding how the engineering differentiates from systemic delivery
  • Addressing safety concerns

3:15 pm Afternoon Networking Break

Discussing Next Generation & Novel Gene Editors for Increased Editing Targeting

4:00 pm Inserting Transposable Elements into Genomes to Take Advantage of Natural Systems

  • Jerrin George HFSP Postdoctoral Fellow, Columbia University Irving Medical Center Sternberg Lab


  • Discussing the benefits of transposable elements relative to CRISPR/Cas9 in the context of size and tissue accessibility
  • Overcoming the continuous diversification of transposable elements to optimize their therapeutic use
  • Targeting transposable elements to the desired DNA sequence and cell type

4:30 pm Exploring Novel Cas Nucleases to Widen the Targeting Index of CRISPR/Cas Systems


  • Discovering new Cas nucleases with more DNA sequence targets to widen the targeting ability of gene editors
  • Understanding why different Cas nucleases target different sequences to aid rational design
  • Minimizing off-target editing with novel nucleases 

5:00 pm Chair’s Closing Remarks

  • John Zuris Director - Editing Technologies, Editas Medicine

5:15 pm End of Conference Day One