Post-Conference Focus Day

Thursday 7 April, 2022

9:45 am
Coffee and Registration

10:10 am Chair’s Opening Remarks

Delving into Delivery

10:20 am Delivery Considerations for RNA Editing Payloads

Synopsis

  • Exploring delivery options for RNA editing payloads
  • Tailoring delivery of RNA editing payloads to the target disease
  • Highlighting ShapeTX AAVid capsid discovery platform enables tissue-specific delivery of gene-encoded RNA editing payloads

10:50 am Unlocking Therapeutic RNA Editing

  • Paul Bolno, MD President and Chief Executive Officer, Wave Life Sciences

Synopsis

  • Building a best-in-class chemistry tool box to create AIMers
  • Leveraging endogenous ADAR enzymes with AIMers to simplify the approach to RNA editing
  • Understand how we have systemized our RNA editing platform based on discovery-stage learnings and have shown applicability across targets and tissues

11:20 am Morning Q&A and Panel Discussion – Improving Delivery Performance

Synopsis

  • Your chance to ask this morning’s presenters follow up questions about their case studies
  • Dive deeper into tailoring delivery of RNA editing
  • Better understand the technology platforms

11:50 am Interactive Discussion Groups:

Synopsis

This session facilitates in-depth discussions among participants in an informal environment. After splitting into groups, participants will discuss key challenges and opportunities around their chosen topic with a table moderator

  • Non-viral and viral vectors
  • Overcoming toxicity challenges
  • Dosage
  • Preventing Immunogenicity

12:30 pm
Lunch and Networking

1:30 pm A New Tool for the Delivery of RNA Therapeutics to Specific Target Cells

  • Joey Verdi Co-founder and Chief Scientific Officer, Hepione Therapeutics

Synopsis

  • Understanding cell-type specific targeted delivery of cargo
  • Discussing easily modifiable and versatile technology platform
  • Divulging a lipid-based delivery system

2:00 pm Pre-Clinical and Clinical Delivery to Myeloid Cells and CNS

  • Nagy Habib Head of Research and Development, MiNa Therapeutics

Synopsis

  • Discussing Nov 340 liposomes for myeloid delivery to bone marrow and tumour associated macrophages
  •  Understanding transferrin receptor aptamer for CNS delivery
  • Learning how small activating RNA are used to upregulate gene expression

2:30 pm Afternoon Q&A and Panel Discussion – Improving Delivery Mechanisms

Synopsis

Your chance to ask this afternoon’s presenters follow up questions about their case studies

2:50 pm Interactive Discussion Groups:

Synopsis

This session facilitates in-depth discussions among participants in an informal environment. After splitting into groups, participants will discuss key challenges and opportunities around their chosen topic with a table moderator

3:30 pm Chair’s Closing Remarks

3:40 pm End of Conference