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
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
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
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