Explore the Agenda
8:00 am Check-In & Coffee + Light Breakfast
8:45 am Chair’s Opening Remarks
Harnessing Biologic & Genetic Approaches to Enable Previously Undruggable Ion Channel Targets
9:00 am KnotBody® Technology: Engineering Ion Channel–Modulating Antibodies via Knottin Integration into Antibody Loops
- Overview of the KnotBody Platform: A novel approach enabling potent, selective, and durable modulation of ion channels
- Lead Optimisation Highlights: Progress towards optimised KnotBody candidates targeting autoimmune diseases, demonstrating enhanced efficacy and developability
- Platform Versatility: Evidence of broad applicability across diverse target classes, underscoring the flexibility of the KnotBody platform
9:30 am Targeting TRPV6 with Monoclonal Antibodies to Enable Selective Ion Channel Modulation for Anti-Tumor Activity in Oncology
- Demonstrating the role of TRPV6 in tumor progression to validate its potential as a novel ion channel target across oncology indications
- Defining the mechanism of action of a TRPV6-targeting monoclonal antibody to establish on-target activity and functional impact on cancer cell biology
- Generating pre-clinical in vitro and in vivo efficacy data to strengthen translational confidence and support progression toward clinical development
10:00 am Using Nucleic Acid & Gene-Based Strategies to Achieve Subtype-Selective Ion Channel Modulation
- Designing subtype-selective ion channel modulation beyond conventional small molecule targeting approaches
- Addressing key challenges in oligonucleotide delivery, tissue distribution, and neuronal uptake to enable effective modulation of excitability pathways
- Exploring where nucleic acid-based approaches offer the greatest advantage in pain and broader excitability disorders
10:30 am Morning Break & Networking
Evaluating Emerging Modality Strategies to Improve Durability, Precision & Therapeutic Fit
11:00 am NaV1.7 Targeting Antisense Oligonucleotides: Emerging Modalities for Pain & Pipeline Development
- Utilizing ASOs as a novel way to modulate NaV1.7, which can circumvent the existing issues with small molecule inhibitors (e.g. autonomic side effects)
- Exploring how Quiver’s integrated platform based on all-optical electrophysiology and machine learning has led to identifying novel candidate molecules alongside therapeutic targets
- Demonstrating how Quiver’s platform can enrich the genetic medicine drug candidates with maximized therapeutic index
11:30 am Targeting Ion Channel Expression Through Epigenetic Gene Editing to Improve Specificity & Durability
- Comparing DNA, RNA, and protein level strategies to clarify where epigenetic gene editing offers distinct advantages for ion channel modulation
- Addressing specificity, delivery, and durability challenges when regulating ion channel expression rather than channel activity
- Evaluating translational potential of DNA targeting approaches to reduce off target effects in pain related ion channel programs
12:00 pm Panel: Comparing Modality Selection Decisions to Match Biology & Indication Requirements
- Examining how biology, delivery constraints & safety risk shaped final modality choices
- Weighing durability, tissue access, reversibility & tolerability across competing strategies
- Identifying modality decision principles that remain consistent across CNS & peripheral programs
12:30 pm Networking Lunch
Demonstrating Human Proof-of-Concept to Validate Next-Generation Ion Channel Therapies
1:30 pm Selective Sodium Channel Blockade to Deliver Clinical Efficacy in Refractory Chronic Cough
- Selective local targeting of cough nociceptors to provide meaningful clinical outcomes in patients with refractory chronic cough
- Interpreting Phase 2b clinical data to understand exposure response relationships, tolerability & efficacy
- Achieving sufficient local exposure in lung tissue while avoiding systemic liability
- Human proof of concept for selective sodium channel blockade to de-risk respiratory ion channel development
2:00 pm Restoring SCN1A-Mediated Neuronal Excitability Through Cell-Selective Gene Regulation in Clinical Dravet Syndrome
- Demonstrating durable SCN1A upregulation using AAV9-based gene regulation in clinical development
- Translating cell-selective targeting of inhibitory neurons into disease-modifying potential in Dravet syndrome
- Optimizing precision gene control strategies to improve safety and efficacy in CNS ion channel disorders
2:30 pm Afternoon Break & Networking
Translating Clinical Experience to Refine Future Ion Channel Development Strategies
3:00 pm Selective Sodium Channel Modulation to Deliver Meaningful Clinical Benefits in Chronic Chemotherapy-Induced Neuropathic Pain
- A late-stage Phase 2 development program applying tetrodotoxin-based sodium channel modulation to treat chemotherapy-induced neuropathic pain
- Achieving durable pain reduction while minimizing CNS and systemic safety liabilities associated with chronic pain therapies
- Establishing clinical proof of concept for selective ion channel modulation in nonopioid pain management
3:30 pm Stabilizing Neuronal Hyperexcitability to Advance Clinically Translatable Kv7 Therapies for ALS
- Targeting selective Kv7.2/7.3 activation to reduce motor neuron hyperexcitability associated with ALS progression
- Applying electrophysiology and translational biomarkers to improve confidence in CNS ion channel clinical development
- Demonstrating how improved selectivity and brain penetration can support safer chronic dosing in neurodegenerative disease
4:00 pm Fireside Chat: Reflecting on Clinical Experience to Shape Next‑Generation Ion Channel Programs
- Discussing how programs evolved in response to clinical data and operational realities
- Sharing insights gained through adaptation, iteration, and risk management during development
- Informing future discovery and development choices with experience-based perspective