Day One
8:00 am Morning Coffee & Check-in
8:55 am Chair’s Opening Remarks
Advancing Extrahepatic Delivery: Current Innovations Enabling Targeted Therapeutics Beyond the Liver for Enhanced Clinical Outcomes
9:00 am Panel Discussion: Turning Setbacks into Strategy, what Clinical Trial Failures Teach us About Building Better Oligonucleotide Therapies
Synopsis
- Unpack the reasons behind trial discontinuations: Was it the oligo, delivery system, or something else?
- Identify overlooked red flags in preclinical models, PK data, or target biology that led to setbacks
- Discuss how teams rebounded, what pivots, partnerships, or reformulations turned failure into future success
10:00 am Delivering to Muscle, Systemic Approaches for Metabolic & Neuromuscular Indications
Synopsis
- Outlining Opportunities with systemic delivery to overcome long-standing barriers in biodistribution and unlocking new therapeutic potential
- Exploring Naked antisense oligos with activity in the muscle
- Novel methods of targeting, previously undruggable targets, expanding the scope of oligonucleotide therapeutics
10:30 am Morning Break & Speed Networking
Synopsis
The ideal opportunity to get face-to-face with many of the leading experts in oligonucleotide development who are working from within discovery all the way through to late-stage clinical development and engage with attendees for important in-depth conversations.
11:30 am Modulation of the Tumor Microenvironment Using Antisense Oligonucleotides
Synopsis
- Tumors actively shape their microenvironment to promote angiogenesis, metastasis, and immune evasion, creating conditions that support their growth and resistance to therapy
- Neuropilin-1 (NRP1) is broadly expressed across multiple cell types within the tumor microenvironment, where it simultaneously facilitates these pro-tumorigenic processes
- Antisense oligonucleotides (ASOs) designed to silence NRP1 expression effectively reduce its levels in key cellular compartments, leading to potent antitumor activity both as a monotherapy and in combination with immune checkpoint inhibitors
12:00 pm Outlining the FORCE Platform: Innovations to Enhance Oligonucleotide Delivery to Muscle Tissues
Synopsis
- How Dyne’s Force platform is enhancing delivery of oligos to muscle tissues
- Properties and modularity of the FORCE Platform
- Provide evidence of translation between pre-clinical models and clinical proof of concept with DYNE-101
12:30 pm CNS Delivery Frontiers, Enabling Oligonucleotide Therapeutics for Neurological Disorders
Synopsis
- Latest innovations in crossing the blood–brain barrier with oligonucleotide drugs
- Design considerations for enabling safe and effective CNS delivery
1:00 pm Lunch Break & Networking
Engineering Smarter Oligonucleotides, Unlocking Chemical Designs that Enhance Selectivity, Potency & Stability
2:00 pm Utilization of Oligonucleotides’ Half-Life to Optimize Dosing Regiment for Desirable Efficacy & Reduced CNS Toxicity
Synopsis
- Explore how chemical modifications and structural engineering can make oligos more resistant to nuclease degradation
- Understand the impact of half-life extension on dosing frequency, therapeutic window, and patient compliance
- Learn how conjugation to long-circulating carriers like antibodies can enhance PK/PD profiles and tissue retention
2:30 pm Hybridization-Mediated Off-Targets of Oligonucleotide Therapeutics: Case Studies, Current Industry Recommendations, & Future Perspectives
Synopsis
- Detection and risk assessment of hybridization-mediated off-targets
- Current strategic recommendations from an industrial perspective
- Pending challenges and the need for enhanced specificity and selectivity
3:00 pm Afternoon Break & Scientific Poster Session
Synopsis
This is an informal session to help you connect with your peers in a relaxed atmosphere to continue forging new and beneficial relationships. With an audience of biopharma experts eager to hear the latest innovations in oligonucleotide Therapeutics, you will have the opportunity to display a poster presenting your own research.
4:00 pm Assessing & Improving Specificity of Therapeutic siRNAs in Preclinical Models
Synopsis
- Review how small chemical tweaks can dramatically improve target engagement and hybridization kinetics
- Unpack the structural features that drive binding specificity and reduce unwanted interactions with non-target transcripts
- Explore screening methods and predictive tools to de-risk oligo designs before clinical translation
4:30 pm Advancing Oligonucleotide Therapeutics Through Innovative Chemistry, Novel siRNA designs for Improved Delivery & Biodistribution
Synopsis
- Engineering next-generation oligonucleotide platforms to overcome delivery barriers, enhance intracellular uptake, and achieve durable therapeutic knockdown
- Developing innovative chemical modifications to enhance stability, potency, safety and improve the overall therapeutic index of oligonucleotide therapeutics