7:30 am
Breakfast & Registration
8:25 am Chair’s Opening Remarks
Uncovering the Clinical Research Propelling Oligonucleotide Therapeutics Development Forwards in 2022 & Beyond
8:30 am miR Replacement Therapy: Developing microRNA-Based Drugs for Depression
Synopsis
- Exploring depression as a polygenic disorder linked to miRs expression
- Uncovering precision medicine with neuron specific targeting using non-invasive brain delivery technology
- Investigating oligonucledite chemical modifications for immune response optimization
9:00 am Learning How Systemically Delivering Antisense Oligonucleotides Targeting CD49d RNA (ATL1102) Significantly Reduces New Active Brain Lesions in Patients With Relapse Remitting-Multiple Sclerosis
Synopsis
- Analyzing Pharmacodynamics: Focus on circulating white blood cell observations
- Comprehensively analyzing safety data and pharmacokinetics
- Deep diving into the pharmacology of ASOs in the brain
9:30 am Exploring Oligonucleotide Therapeutics for CNS Repeat Expansion Disorders: Spotlight on Huntington’s Disease
Synopsis
- Key highlights from progress within our ASO approach against Huntington’s Disease
- Lessons learned from our work to improve our drug development strategy moving forwards
- Illustrating distribution across the brain in pre-clinical models
10:00 am
Morning Break & Networking
Solving the Translational Drug Development Gap: From Pre-Clinical Models to Clinically Effective Therapeutics
11:00 am Understanding How Route of Administration Impacts siRNA Delivery in the Central Nervous System
Synopsis
- Comparison of parenchymal, ventricular, and intrathecal siRNA administration in the CNS of multiple species
- Effect of ROA on deep brain penetration and oligonucleotide retention
- Relationship between accumulation and silencing and the implications for compound duration of effect
Zoning in on Oligonucleotide Targets: Learning Tips & Tricks for More Streamlined Therapeutic Discovery
11:30 am Investigating Exon-Skipping ASOs to Enhance Adult Hippocampal Neurogenesis in Psychiatric, Neurological & Neurodegenerative Disease
Synopsis
- A novel pathway for modulating adult hippocampal neurogenesis
- Identification of ASOs targeting this pathway
- Potential applications and future directions
12:00 pm
Lunch & Networking
1:00 pm Discovering Gene Upregulation Using Regulatory RNA
Synopsis
- Overview of CAMP4 platform which allows gene upregulation within the CNS
- Illustrating meaningful effect in haploinsufficient diseases with oligonucleotide therapeutics
- Deep diving into oligonucleotides for frontotemporal dementia and epilepsy
1:30 pm Exploring Targeting miRNA in the CNS
Synopsis
- Overview of Regulus platform for selecting miRNA targets in CNS disease
- Choosing in vivo and in vitro models to support lead finding
- Approaches and considerations to improve indication-focused anti-miRNA delivery
2:00 pm Uncovering Next Generation Drug Delivery Strategies for More Precise Weaponization of Oligonucleotides Against the CNS
Synopsis
- Analyzing current administration routes and assessing their suitability for use with varying oligonucleotide modalities
- Examining novel delivery technologies and ranking them for ease of use, functionality and value
- Scrutinizing medical devices, gene therapy delivery, lipid nanoparticles, cellpenetrating peptides, antibodies and more to understand their application within the CNS
Hearing About Oligonucleotide Therapeutics From a Business Angle: What You Need to Know
2:30 pm Panel Discussion: Business Development Perspectives on the Oligonucleotides Space
Synopsis
- Exploring historical trends in business development and financing for oligonucleotide therapeutics
- Uncovering the challenges and opportunities these trends present for those attempting to innovate within this disease area
- Explaining what investors need to see in order to invest
- Perspectives on the need to demonstrate both mechanistic understanding and a regulatory pathway through to the market