7:55 am
Breakfast & Registration

8:55 am Chairs Opening Remarks

  • David Corey Professor, UT Southwestern Medical Center

Investigating Current Challenges & Opportunities in Oligonucleotide Delivery Within the CNS

9:00 am Revealing Tips & Tricks to Achieve Deeper Brain Penetration at Lower Doses When Delivering Oligonucleotide Therapies


  • Dissecting challenges with translating results from pre-clinical models to humans
  • Understanding the impact of dosing regimens
  • Investigating the impact of different modifications of oligonucleotides

9:30 am Exploring Efficient On-Target Delivery of Oligonucleotides: In Vitro & In Vivo Performance of Sapreme’s Endosomal Escape Enhancers for Various Tissues and Targets

  • Miriam Bujny Chief Development Officer, Sapreme Technologies


  • Learning how Sapreme’s endosomal escape enhancers optimize cytoplasmic release to reveal full efficacy
  • Uncovering the delivery of oligonucleotides and protein therapeutics with broad applicability
  • Investigating targeting through ligand-conjugation to increase cell/tissuespecificity

10:00 am
Morning Break & Networking

11:00 am A Landscape Analysis of Oligonucleotide-Based Therapies


  • Breaking down the oligonucleotide drug and trial landscape – a look into diseases, delivery systems & nucleic acid modifications across modalities
  • Comparing the drug & trial landscape within the CNS & beyond
  • A look into the future of the field & upcoming activity

Revealing Key Toxicity & Safety Considerations of Oligonucleotide Therapeutics for CNS Disorders

11:30 am Challenges in Designing Pre-Clinical Toxicity Studies for Oligonucleotides & Their Clinical Relevance

  • Meena Stoke Vice President of Bioanalytical, DMPK & Biomarker Development, Stoke Therapeutics


  • Lessons learned from approved oligonucleotide therapeutics (ONTs)
  • Lack of specific regulatory guidance for development of ONTs
  • Considerations for development of ONTs

12:00 pm
Lunch Break

Enhancing Drug Delivery Across the Blood Brian Barrier for More Clinically Meaningful Oligonucleotide Therapeutics

1:00 pm Exploring Antibodies, Small Molecules & Nanoparticles for Increased Movement of Oligonucleotide Therapeutics Across the Blood Brain Barrier


  • Uncovering considerations for each therapeutic modality from a therapeutic perspective
  • Exploring molecule design considerations for moving across the blood brain barrier more readily
  • Are there recommendations to consider for antibodies, small molecules, nanoparticles and peptides?

1:30 pm Audience Discussion: Looking Beyond Intrathecal Administration: Are We Able to Modify Oligonucleotide Chemistry for Intravenous Delivery Across the Blood Brain Barrier?


Achieving intravenous delivery of oligonucleotide therapeutics across the blood brain barrier has long been an attractive prospect for biopharma however despite years of trying to effectively deliver beyond the BBB, a plethora of barriers still remain.

Join this interactive audience discussion to deep dive into the success, failures and challenges of delivering oligonucleotide therapeutics across the blood brain barrier and leave with the knowledge you need to strategically inform and optimize your
intravenous delivery approach.

2:00 pm
Afternoon Break & Networking

Revealing How to Strategize Oligonucleotide Pipeline Expansion into the CNS

2:30 pm Spotlight on SynaptixBio – A Virtual Approach to Developing an Oligonucleotide Therapy for a CNS Disorder


  • TUBB4A Leukodystrophy and it’s suitability for an oligonucleotide therapeutic approach
  • SynaptixBio’s virtual strategy to outsourcing and developing a therapeutic
  • Exploring the pros and cons of applying a virtual model to development – learnings from a start-up biotechnology company

Exploring Emerging Therapeutic Approaches When Using Oligonucleotides Against CNS Diseases

3:00 pm Diversifying Therapeutic Strategies for Glioblastoma & Alzheimer’s Disease


  • Diverse non-coding RNAs regulate all biological functions in a cell and expand the landscape of therapeutic targets in the CNS pathologies
  • Malignant brain tumors are a major unmet need in neuro-oncology, and oligonucleotides as future therapeutics for glioblastoma
  • Oligonucleotides could provide neuroprotection for neurodegenerative diseases such as Alzheimer’s

3:30 pm End of Day One