Gene Editing Innovation Discussion Day: Friday, March 29

9:00 am Registration & Coffee

9:50 am Chair’s Opening Remarks

9:55 am Understanding the Current Gene Editing Landscape

10:00 am The Current Focus of Gene Editing: Applying Lessons from Ex-vivo Gene Editing to Inform In-vivo Innovations

  • Howard Wu Co-founder and CSO, Full Circles Therapeutics

Synopsis

  • An overview of Full Circle’s gene editing/writing platform technology
  • Unpacking the successes in ex-vivo gene editing
  • Exploring strategies to apply this platform technology to In-vivo applications

10:30 am 10.30am Advanced Bioinformatics for Multiplex Viral Targeting

  • TJ Cradick Chief Scientific Officer, Excision BioTherapeutics Inc.

Synopsis

  • Dual gRNA excisions on viral DNA
  • In-silico probes to analyze on- and off-target effects
  • Computational methods to design the most effective guide RNAs

11:00 am Morning Refreshments & Networking

11:05 am Exploring Optimal Methods for Delivering Editing Tools Specifically and Safely

11:30 am Panel Discussion: Ensuring Transient Delivery with Durable Outcomes

Synopsis

  • Discussing methods to ensure durable outcomes with transient delivery
  • Exploring the safety concerns involved with viral delivery methods
  • Maximising the genetic payload for viral vectors
  • Improving the tropism of non-viral delivery methods
  • Looking into the future with LNP-based Ribonucleoprotein delivery

12:00 pm Next Generation Delivery Systems: Overcoming the Challenges Associated with Viral Methods

Synopsis

  • Minimizing the risk of off target editing and improving precision with appropriate delivery system selection
  • Discussing advantages and disadvantages of viral delivery methods
  • Exploring immunogenicity reduction and improving the safety of gene editing

12:30 pm Lunch & Networking

12:35 pm Recent Gene Editing Technology Innovations

1:30 pm Using a novel CRISPR-based Editing Technology for Template-Free Gene Editing

  • Ravi Iyer Chief Scientific Officer, Script Biosciences

Synopsis

  • Achieving site-specific double-strand breaks
  • Precise 1-3 base insertions and controlled DNA repair
  • Minimising off-target edits in in-vitro and in-vivo studies

2:00 pm Next-Generation Uses for Gene Editing: Using CRISPR to Perform Multiplex Gene Editing

  • Wenning Qin SVP Innovation and Process Development, eGenesis Inc.

Synopsis

  • Using the EGEN Platform to leverage advances on gene editing technologies
  • Translational lessons from CRISPR gene editing
  • Using CRISPR to tackle the worldwide organ shortage

2:30 pm REMEDY Corrects Heterozygous Mutations Without an Exogenous Donor DNA Template

Synopsis

  • REMEDY does not require an exogenous DNA template to be provided, no AAV needed
  • REMEDY can be used in numerous cell types
  • REMEDY corrected multiple heterozygous mutations such as Cystic Fibrosis, VCP mutations and more

3:00 pm Chairs Closing Remarks

  • Meisam Kararoudi Principal Investigator, Center for Childhood Cancer, Nationwide Children's Hospital