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

  • George Tachas Director of Drug Discovery & Patents, Antisense Therapeutics

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

  • Jia Tay Senior Director Program Management, Triplet Therapeutics

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

  • Julia Alterman Assistant Professor , RNA Therapeutics Institute, UMass Chan Medical School

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

  • Alfica Sehgal Senior Vice President & Head of Discovery, Translation Discovery, CAMP4 Therapeutics

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

  • Alicia Secor President & Chief Executive Officer, Atalanta Therapeutics
  • Lisa Shafer Chief Scientific Officer, Cerebral Therapeutics

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

3:00 pm End of Day Two